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Comments from Orphazyme on the investment in CombiGene

Comments from Orphazyme on the investment in CombiGene

Orphazyme has acquired 10 per cent of the shares in CombiGene, making the Danish investment company one of the largest shareholders. BioStock reached out to Orphazyme’s Chairman of the Board Michael Hove and CombiGene’s CEO Peter Ekolind to learn more about the reasons for the investment.

CombiGene made the headlines in 2021 when it out-licensed its epilepsy project CG01 to US-based gene therapy company Spark Therapeutics, following a series of convincing preclinical data.

According to the agreement, CombiGene was eligible to receive up to USD 328.5 million excluding royalties, with 8.5 million USD upon signing, as well as up to USD 50 million at several preclinical and clinical milestones. In January 2024, CombiGene regained the global rights to CG01 after the collaboration agreement was terminated due to a strategic audit of Spark’s pipeline. CombiGene is now scouting for a new licensee for the project.

CG01 is not CombiGene’s sole asset. In early 2023, CombiGene entered into a collaboration with the Danish biotech company Zyneyro to develop more effective treatments for severe chronic pain conditions. The joint pipeline includes the most advanced project – the peptide treatment COZY01 – as well as the gene therapy treatment COZY02.

Comments from Orphazyme on the investment

Recently, CombiGene announced that the Danish investment company Orphazyme has acquired more than 10 per cent of the outstanding shares in CombiGene. According to Holdings, this makes Orphazyme the biggest shareholder in the company.

BioStock reached out to Michael Hove, Chairman of the Board of Orphazyme, and Peter Ekolind, CEO of CombiGene, to get a background for the investment.

Michael Hove, Chairman of the Board of Orphazyme
Michael Hove, Chairman of the Board of Orphazyme

Michael, eight years ago, Orphazyme was listed on Nasdaq Copenhagen, and in May 2022, all biotechnology assets were sold in favour to focus on biopharmaceutical investments. Can you briefly discuss this transition?

– Orphazyme was founded in Denmark in 2009 on a scientific discovery regarding the role of heat shock proteins (HSPs), which was described in a well-cited article published in the journal Nature.

– In 2017, the company was listed at Nasdaq Copenhagen where over the coming years was able to attract various industrial investors (IE. Novo Holding as majority shareholder) and due to promising clinical data able to raise more than EUR 350 million to go in full scale on finalizing their main product Arimoclomol in all clinical stages internally and ready to launch also on the US market. In February 2022 the clinical data failed in phase III and that initiated a 180 degree turnaround of the company which ended in May 2022 when the company sold all assets and business activities, including those relating to the development and approval of arimoclomol to KemPharm Denmark A/S, a wholly owned subsidiary of KemPharm, Inc. for approximately USD 12.8 million in cash and then it was in the process of redefining its strategy.

– I took over the chairmanship in May 2023 with a new board and accelerated the clean-up of the company, recapitalizing and in January we presented our new strategy which would be to buy strategic positions in early-stage biotech companies and add value to the partnership through our competencies in the board. CombiGene is the first investment in the new strategy from January 2024.

Why are Orphazyme investing in CombiGene, and why right now?

–  When we monitored the Nordic market CombiGene was unique both regarding its long history in gene therapy with CG01 as the front runner, and also because they succeeded in signing a strategic partnership with Spark, which we consider as proof of concept. We have high expectations that the company will succeed in finding a new partner that will take the promising product all the way to product launch, and also look forward to following the two projects which are launched in cooperation with the Danish biotech company Zyneyro to develop more effective treatments for severe chronic pain conditions.

Peter Ekolind, CEO CombiGene
Peter Ekolind, CEO CombiGene

Peter, what are your comments on the investment made by Orphazyme?

– We are pleased to welcome Orphazyme as a lead investor with the ambition to join the growth journey that CombiGene strive for by developing existing assets and in-licensing new ones to develop further. I believe that Orphazyme can contribute with valuable knowledge and experience to the company.

According to your recent press release, CombiGene’s Board of Directors have begun a dialogue with Orphazyme. Can you discuss the specifics of these discussions?

– It is a bit early to elaborate at this point as the dialogue is just beginning. I will certainly have more to disclose later once the dialogue has matured further.

CLS sees opportunity to increase revenue

CLS

In the year-end report for 2023, CLS summarizes an eventful year with increased revenues, primarily driven by the urology segment and the new Mobile Service Provider model. Moreover, the company has identified a way to increase revenues within the neurosurgery segment.

Clinical Laserthermia Systems (CLS) recently published its year-end report, reporting a full-year net sales of SEK 8.27 million which is 32 per cent higher compared to 2022. With increased sales and improved margins, the operating result has improved somewhat compared to the previous year, from SEK –67.4 million to SEK –65.9 million.

Like previous years, the US market was the primary source of revenue. Nevertheless,  the company also sees an opportunity to sell its TRANBERG Thermal Therapy products globally, driven by new distributor deal  in Europe alongside market approval in Singapore, enabling expansion into the Asia-Pacific region.

Focus on MSP

During 2023, CLS established a Mobile Service Provider (MSP) model, allowing clinics to test and evaluate TRANBERG for the treatment of prostate cancer. In the fourth quarter, CLS entered into two new MSP agreements in the USA, with Kearney Urology Center PC and  KASRAEIAN Urology respectively. These collaborations are in the startup phase and expected to generate revenue in the first quarter of 2024. Moreover, CLS already has an MSP agreement with AVANT Concierge Urology Clinic.

According to the report, CLS is set to enter additional MSP agreements in 2024, with several potential agreements already in the pipeline.

Identified revenue opportunities within neurosurgery

In 2023, CLS also introduced aneurosurgery segment for its US market endeavours. They saw  orders from the  neurosurgery partner ClearPoint Neuro and received positive feedback from users. Yet, revenues from the segment remained at the same level as in 2022.

However, CLS has identified improvement potentials in their offer which could result in increased revenues from the segment, by providing access to a larger portion of the neurosurgery market. Albeit, such improvements would require investment and thus increase costs  in the coming years – while bringing significant benefits in the long term, according to the company.

Strategic investments and milestones in 2024

In light of the upcoming strategic investments in neurosurgery, as well as the later-than-expected market approval in Singapore, the company has postponed the goal of achieving positive operating profit (EBITDA) to 2026.

The company is now looking forward to unlocking the market potential in the Asia-Pacific region and shifting into full market launch in the US, as well as securing additional MSP and commercial agreements. In addition, the market can expect clinical trial results.

Resigning CEO on the future of WntResearch: “It will be exciting to follow!”

Pernilla Sandwall, vd WntResearch presenterar bolaget

Upon writing her last CEO comment in the company’s Q4 report, WntResearch’s resigning CEO Pernilla Sandwall talks about a “transformative” 2023 for the company. Based on the company releasing new data, updating their NeoFox trial and aquiring a new CEO, the labelling seems appropriate. BioStock reached out to Sandwall for a comment.

Malmö-based WntResearch is developing Foxy-5, a peptide designed to mimic the function of the body’s own WNT5A protein. The objective is to develop a treatment that can prevent the ability of cancer cells to spread in the body and thereby avoid metastasis – the primary cause of cancer-related deaths.

NeoFox to confirm early observations

The first target indication is colon cancer, where the aim is to reach Proof-of-Concept, before adding additional indications. Here, the phase II study NeoFox is underway, looking to confirm previous ad hoc observations where a tumour-inhibiting effect was detected after as little as three weeks of treatment.

The study was adjusted to confirm these findings as quickly and cost-effectively as possible, and patient recruitment resumed last fall. Since then, the study has evaluated two doses and WntResearch recently announced that, upon approval from the safety committee, they will proceed with the highest dose in the next part of the study. Now, the goal is to recruit another 80 colon cancer patients, who will either be treated with Foxy-5 or be included in the control group. According to the company, there is great interest in participating in the study, with a high recruitment rate so far, which underlines the communicated timelines.

First read out by end of 2024

They plan for an initial read out of efficacy data after approximately half of the patient population has received treatment, i.e. by the end of 2024. The results will provide an important indication for the continuation of the study. Topline results are then expected to be presented in 2025.

In addition to the implications for the continued clinical development, the results will also form a key component in the ongoing business development activities. The positive data generated by previous studies has attracted great interest from the industry, which WntResearch intends to build on.

Teaming up with Business Sweden

As part of the business development advances, the company recently announced a collaboration project with Business Sweden, with the aim to establish one or more commercial partnerships with Japanese and South Korean companies. The agreement states that Business Sweden will help establish contact with potential partners. According to WntResearch, the focus on Japan and South Korea was motivated by making the best use of Business Sweden’s network and deep industry knowledge in these markets.

Strengthened finances

Another recent activity with high importance was the rights issue carried out in January, which brought approximately SEK 31 million to the company. In addition, the issue included two series of warrants, TO 7 and TO 8. The warrant series are exercised at the end of 2024 and in April 2025, respectively, and if things go as planned, they will add significant capital injections to the company.

Comments from the CEO

With the year-end report, Pernilla Sandwall hands over the keys of WntResearch to incoming CEO Per Norlén. Before then, BioStock took the opportunity to talk to Sandwall about the report and her view on the company’s future prospects.

Pernilla, if you look back on your time as CEO of WntResearch, what are your key take-aways?

– The positive ad hoc observations and that we were able to change the study so that we now measure precisely these parameters in a forward-looking manner. This is what it is like in drug development; for the data to be approved by regulatory authorities and other players, it must be pre-defined and determined before initiating the study.

– Moreover, we have gained the trust of the shareholders and managed to add additional capital to the company in this difficult market situation.

– Finally, I value all the interactions with talented colleagues, physicians and initiated owners, which have been interesting, albeit sometimes difficult, but always rewarding and inspiring.

In the dosing study, recruitment went faster than expected. Does this affect your expectations of the recruitment for the next phase?

– My expectation is that patient recruitment will go faster now than it did during the pandemic. I see several reasons for this; we have the ad hoc observations from patients in place, and we increased the dose. I believe that the latter means that both doctors, other study staff and patients have an increased interest in the study.

– Furthermore, the study is easier for patients to participate in now that it only covers the time before operation, without any extended follow-up period.

– The fact that the pandemic is over also allows hospitals to devote more time to clinical trials such as ours. Combined, all these factors all but guarantee a faster recruitment rate than we have seen and set our timelines after.

If we focus on your collaboration with Business Sweden; what makes the Japanese and South Korean markets so attractive?

– There are many examples of very good collaborations of this kind. There is the possibility of both global and regional agreements, which does not close any doors. Business Sweden’s network in these markets, alongside its deep industry knowledge, is a fantastic asset for us.

What is your strategy for finding a partner in Europe or the USA?

– In these markets we have direct contact with the companies we view as potential partners. We participate in large meetings and conferences, which provides solid opportunities for dialogues with our potential partners. For example, both SwissNordicBio and BIO Europe Spring are coming up shortly.

Looking ahead, what will be the focus of WntResearch as we move into spring and summer?

– Firstly, the meetings I mentioned above are crucial. My successor Per Norlén will present the company at SwissNordicBio next week in Zurich. We will also focus on the study and patient recruitment by visiting most of the clinics during the second quarter, which I believe will be appreciated by everyone involved.

Since this will be your last interview with BioStock as CEO of WntResearch, I must ask about your vision for the company and the project in the longer term. What do you dream of Foxy-5 being able to achieve?

– Foxy-5 has the potential to develop into a completely new type of cancer treatment without troublesome side effects. The data we have from our ad hoc observations is very promising and I believe that good study results from the ongoing trial will lead to a large interest from major pharmaceutical companies, who will be able to develop Foxy-5 into a drug.

– I dream that this will happen, partly for the sake of all patients, but also for everyone who has been engaged in this project – either in the development or through funding of the research. It will be exciting to follow!

Aptahem comments on the publication in Cells

Aptahem comments on the publication in Cells

Aptahem’s collaboration with the research group at the University Health Network in Toronto, has now resulted in a publication in the scientific journal Cells. BioStock reached out to Aptahem’s CEO Mikael Lindstam to find out more about what this means for the company.

Aptahem’s lead candidate Apta-1 is being developed as an emergency drug for sepsis, with the aim of preventing the occurrence of life-threatening organ and tissue damage that can occur in connection with the condition.

Collaboration with researchers in Toronto

In 2020, thanks to a research proposal initiative of Dr Luiza Jedlina, Aptahem’s CSO and founder, the company initiated a collaboration with a research group at the University Health Network in Toronto. The study is led by Professor Mingyao Liu together with Dr Jedlina. The collaboration has included several study steps in a Covid-induced SARS-model. According to Aptahem, the virus used in the studies is about 10 times deadlier virus than the one that causes COVID-19.

In 2021, the collaboration generated encouraging data, which showed positive biological effects that, according to Aptahem. The data included evidence that Apta-1 supports and maintains systemic circulation, a very important part of protecting the organism in e.g. sepsis. Furthermore, it was found that Apta-1 significantly reduces bleeding in the lung tissue and inhibits hemolysis and inflammatory response while keeping vital organs stable.

The CEO comments on the publication in Cells

Mikael Lindstam, CEO Aptahem
Mikael Lindstam, CEO Aptahem

Aptahem has previously announced that it has been in the process of completing a manuscript describing the progress, to publish this in a scientific journal. Yesterday it was announced that the Swiss scientific journal Cells – with an impact factor of 6 – 7 – had accepted and published the manuscript.

The article can be read here: “Therapeutic effects of a novel aptamer on coronaviral infection induced lung injury and systemic inflammatory responses.”

BioStock reached out to Aptahem’s CEO Mikael Lindstam to get his view on what the publication means in the long term for the company.

Mikael, what does it mean to you to have an article published in Cells?

– Yes, it is of course a strong sign of value to have been assessed for publication through peer reviewed by renowned experts and their editorial team. Since we are at the forefront of this type of new and multi-efficient candidate, it is expected that the burden of proof and more requires significantly greater data to gain understanding so that it leads to publication. This opens up the opportunity for us to refer to ourselves in other scientific articles we are working on, and thus facilitate the acceptance of the fine and positively complex results we have generated over the years.

What are the main conclusions that can be drawn from the article?

– The fact that we can have a protective and supportive effect on virus-generated acute lung conditions and as a bonus also see that we can reduce the amount of virus was something we could not have predicted. The results also resonate with those we have from our bacterial toxin (LPS) stimulated sepsis models, which of course is a testament to Apta-1’s ability to handle severe inflammatory conditions.

Can the publication to any extent affect your ongoing discussions with potential partners?

– Yes, we really hope so, as it is an acknowledgment of what we have previously announced about our results assessed by independent experts. It is also likely to be positive for continued clinical collaborations as well as scientific collaborations.

Finally, what milestones do you see ahead for the rest of 2024?

– First and foremost, that we reach the finish line with the report of phase Ia and before that preliminary results of this study, which is in its final phase, planning of the next clinical development step and potentially more publications from the extensive harvest of results we have generated.

Abliva’s CEO comments on the proposed capital raise

Ablivas vd kommenterar den föreslagna kapitalanskaffningen

Abliva’s 2023 Year-End report focuses on the successful enrolment in December for Wave 1 of the phase II ‘FALCON’ study with lead candidate KL1333. Other key milestones include initiating dosing and receiving Fast Track Designation for the programme. BioStock contacted Abliva’s CEO Ellen Donnelly to learn more about the most impactful milestones achieved during the quarter and the recently announced rights issue that aims to support ongoing activities and provide additional runway.

In December 2022, Abliva announced the start of the global, potentially registrational phase II FALCON study with the lead candidate KL1333. KL1333 is being evaluated for the treatment of mitochondrial DNA (mtDNA)-related primary mitochondrial diseases in adult patients suffering from debilitating fatigue and muscle weakness.

The disease disturbs cellular energy conversion and causes severe symptoms such as stroke-like episodes, muscle weakness, and heart failure, but patients report that it is the impact on the quality of life, caused by the overwhelming fatigue and muscle weakness, that is most disruptive to their life. Primary mitochondrial disease has a profound effect on patients as it not only lowers the quality of life, but it also causes reduced mobility and can be fatal.

There are no approved therapies for the treatment of systemic mitochondrial disease, and the competitive landscape is limited, providing opportunities for KL1333. The company states that the potential annual sales for the candidate could be over USD 1 billion.

Progress within the FALCON programme

Q4 2023 was a quarter of significant progress for the FALCON study. Achieving the enrolment target for Wave 1 of the study was a key milestone, further demonstrating the high unmet medical need in primary mitochondrial disease. KL1333 dosing in Wave 1 patients has commenced and data collection is now underway for the interim analysis expected in mid-2024.

Reneo’s failure may set the stage for KL1333

In the interim report, Abliva’s CEO Ellen Donnelly discussed the impact that Reneo Pharmaceutical’s negative results from December might have on Abliva’s programme. Donnelly concluded that the negative readout from Reneo’s pivotal STRIDE study evaluating their drug candidate, mavodelpar, in primary mitochondrial myopathies, presents an opportunity for Abliva. The mechanisms of action of the two drugs and the clinical programmes were very different, and thus the negative outcome of their study is neither predictive of success nor failure with the FALCON study.

A potentially lucrative deal with Owl Therapeutics

In November last year, Abliva announced a licensing and collaboration deal with US-based Owl Therapeutics for the development of NeuroSTAT in Traumatic Brain Injury. The deal includes potential milestone payments of USD 43.65 million. NeuroSTAT has received orphan drug designation in both the US and Europe. In addition, the drug has both Fast Track status and IND approval to start clinical studies in the US. BioStock did an interview with Ellen Donnelly where she discusses the deal. Read it here.

Rights issue to finance the final stage of the FALCON study

Last week, Abliva resolved a fully guaranteed rights issue of SEK 46 million and a directed issue of convertible bonds of SEK 42 million to prepare for the final stage of the FALCON study. The proceeds from both are intended to further support KL1333 (clinical, non-clinical and manufacturing) and provide additional runway for the company.

Comments from the CEO

BioStock talked to Ellen Donnelly to get her thoughts in the latest development and to find out more about Abliva’s goals for 2024.

First and foremost, having released the Q4 report, how would you summarise 2023?

– I think 2023 was an extremely productive and successful year. The primary focus for the team during 2023 was obviously on execution of the FALCON study – from activating sites across the globe to focusing on patient recruitment – and the team delivered all of our communicated milestones, dramatically decreasing the risk in the study. In parallel, the team was working to increase value across the portfolio, announcing NeuroSTAT deal with Owl Therapeutics, enhancing the NV354 programme with a new patent and orphan drug designations in the US and EU, and gaining approval of Fast Track designation for KL1333.

Ablivas vd Ellen K. Donnelly på Medicon Village
Ellen K. Donnelly, CEO Abliva

Can you discuss the importance of the milestones reached thus far in the KL1333 programme and the FALCON study?

– The three major milestones achieved in the FALCON study (study initiation, screening completed, Wave 1 recruitment finished) are all important for different reasons. The initiation of a global study in a rare disease is always a challenge, preceded by years of planning and discussions with regulators, ethics boards, hospitals, and sites, and thus we were thrilled to get the study started and quickly expand the footprint across six countries and 18 sites. Once the study is underway, the next risk comes in the identification of patients, as it is very difficult to predict whether the patients you are seeking will be easy to find and willing to participate in clinical research. For this reason, we were extremely happy to see the high level of interest in the study with over 90 patients identified for potential inclusion in the study. Not only did this give us confidence in our ability to recruit the full study, but it also helped support the prevalence of the disease and the potential USD 1 Billion-plus commercial opportunity. Finally, the communication of all Wave 1 patients fully recruited in December was an important milestone as we have now closed the first phase of the programme and will shift our focus to data collection, cleaning and interim readout in the middle of 2024.

How does Abliva plan to address the unmet medical need in primary mitochondrial disease with the KL1333 programme?

– The FALCON study evaluates adult patients (with genetically confirmed mitochondrial DNA mutations) who suffer from extreme fatigue and myopathy. Data from the MitoCohort registry study, the UMDF/FDA Patient Focused Drug Discovery meeting, and peer-reviewed research suggest that not only are these the most burdensome symptoms for patients, but they are also very common, occurring in 70-80 per cent of patients with primary mitochondrial disease. We are evaluating both fatigue and myopathy in the ongoing study, so if both are positive, we would expect to receive approval to treat patients with this profile. Once the drug is approved, we will commercialise KL1333 while working, in parallel, to expand the indication by running additional clinical studies.

In light of Reneo Pharmaceutical’s setback, how do you perceive the competitive landscape and opportunities for KL1333?

– The landscape of therapies being developed for diseases of mitochondrial origin can be divided into two groups – those which get into the brain and target the neurological issues of the disease and those which impact the periphery, including myopathy. KL1333, Reneo’s mavodelpar, and Astella’s bocidelpar target the mitochondria outside of the brain, whereas the drugs like Abliva’s NV354, PTC’s vatiquinone, Khondrion’s sonlicromanol, and Tisento’s zagociguat are all expected to have neurological effects. The failure of mavodelpar, a PPARdelta agonist and the most advanced programme in this area, removed a major competitor from the area, and the lack of efficacy of this drug brings additional questions to the second most advanced program, the Astellas program, as they also have a PPARdelta modulator. With regards to the drugs targeting the brain, this field is less advanced with clinical data limited to small, often open-label, studies in epilepsies, cognition or cerebral blood flow, meaning that all are a few years (or more) away from registrational data. This is all good news for KL1333 as this drug now has the potential to be the first to market, providing a huge opportunity for Abliva and patients with mitochondrial disease. And in this area, like others, there is room for many different drugs, and thus we expect continued growth of the market with the approval of other therapies and the use of multiple treatments for mito patients.

Finally, you are looking to raise a total of 88 MSEK through a rights issue and a directed issue of convertible bonds. Why did you settle for this solution?

– In June 2022 we did a financing round comprised of a directed share issue of SEK 150 million and a rights issue of SEK50M. This financing enabled Wave 1 of the ongoing FALCON study and provided with 24 months of runway. With many of the risks of study start-up of a rare disease trial behind us, we made the decision to raise additional capital now to ensure that we can prepare for Wave 2 of the study while conducting key activities in parallel that will support KL1333 marketing approval after a positive study readout. In addition, we wanted to ensure that we have sufficient runway after the interim readout to allow time for robust discussions with investors and potential partners. The financing was structured as it is, with a preferential rights issue of SEK 46 million and a convertible of SEK 42 million (both dependent upon a favourable EGM vote in March), to both allow our current shareholders to participate in the issue and retain their share in the company, whilst also providing an additional infusion of de-risked capital with a positive, non-futile, readout of the interim analysis. We are thankful to be supported by a strong group of institutional investors and retail shareholders who are as passionate and confident as we are about the potential for KL1333 as a new medicine for patients with primary mitochondrial disease.

Dicot’s CEO: “Several prominent people want to be part of the journey”

Dicots journey

Uppsala-based Dicot recently released its Year-end report. CEO Elin Trampe notes that there is great confidence in the company’s research, both among investors and researchers in the field. BioStock contacted her in connection with the report to get a comment on the latest news and to find out more about what’s on this spring’s to-do list.

Dicot’s drug candidate LIB-01 is being developed for the treatment of erectile dysfunction and premature ejaculation. The project is originally based on a tradition of folk medicine, but LIB-01 has since been developed into a modern drug candidate. Supported by a promising preclinical data package, the company is now in the clinical development stage, where phase I studies are in full swing.

The study is being conducted by the contract research company Clinical Trial Consultants, and overall results from the single dosing phase, SAD, were recently announced. LIB-01 was found to have a very good safety profile and no serious side effects occurred. The results also show that the drug is well absorbed in the body, which gives the company a strong starting point for clinical phase II.

New phase I results in Q2

In addition to having the desired medical effect, a good safety profile is an important competitive advantage for any drug, not least for drugs that treat sexual dysfunction. Disturbing side effects are one of the problems with today’s treatments, forcing many men to discontinue their use.

The next step in Dicot’s phase I programme will be to present overall results from the second and final part of the study, where participants receive repeated and increasing doses of LIB-01. This data is expected in the second quarter of this year. After that, the clinical development will revolve around studying the drug’s effect in patients.

Great interest in the development

In Dicot’s newly released Year-end report, CEO Elin Trampe states that there is a great deal of interest in the project, both from investors and in the research community. Last year, the company signed an agreement with Dr Harin Padma-Nathan, who is a big name in the field. In addition, preclinical research results were recently presented at  the European Society for Sexual Medicine’s conference – the largest conference in sexual medicine in Europe.

Dicot also did well in its investment rounds during the past year, securing subscription rates well above the industry average, in a barren financing landscape. As a result, at the end of 2023, the company had a cash position of SEK 47.3 million, ready to take the next step in its development.

New patent for new production method

An important part of the project is to ensure large-scale production. Therefore, in 2022, Dicot started a research project together with Uppsala University and the W. Szafer Institute of Botany to investigate whether the starting material in LIB-01 could be produced using plant cell cultures – an established technology for large-scale commercial production of many pharmaceutical substances that originate from nature.

According to the company, the project has led to the successful development of a specially-designed cell culture method. The project is now moving on to testing on a larger scale. At the same time, a patent application has been filed with the Swedish Patent and Registration Office to protect this method of manufacturing for the starting material. Further patent applications in other relevant markets are expected to follow.

Comments from the CEO

BioStock contacted Elin Trampe in connection with the Q4 report to find out more about what she sees ahead of her in the spring.

Elin Trampe, CEO Dicot
Elin Trampe, CEO Dicot

To begin with Elin, in your CEO statement of the report, you write that you see a very large interest in your development from several directions. What does this mean for you?

– The great interest we see from sexual medicine researchers and physicians confirms that our results are unique, and they show that LIB-01 can make a big difference. It has also helped us to get in touch with more prominent people in the field who want to participate in this journey and to contribute with their expertise, which we of course see as very positive.

How will you work to maintain that interest in the future?

– Of course, it mainly comes down to continuing to deliver good results, then the interest will automatically increase. We will also continue to be active in our communication and visit relevant conferences and forums around the world to disseminate our results in the best possible way. Having well-known experts in the field representing us in various forums also strengthens the credibility of our project.

In terms of your finances, you have a good cash position. What opportunities does that give you?

– Yes, we had a very successful year raising capital in 2023, which means that we have money to finance the company throughout phase I and it also enables all important preparations for upcoming efficacy studies in phase II.

Can you tell us more about the research project with Uppsala University and the W. Szafer Institute of Botany and why it is important?

– The project opens up a highly interesting alternative for future commercial manufacturing of the starting material for our pharmaceutical substance. The fact that the results have been successful so far means that we can now continue to evaluate the technology to a greater extent, as well as make comparisons with existing processes – where starting material is extracted from natural plant material.

How does the new production method differ from traditional methods in your field?

– The method as such of using cell culture is an already established technology for large-scale commercial production of several pharmaceutical substances derived from nature. Without going into details, we have developed a specific approach to producing and culturing cells and where we now also have enough data to be able to apply for patents and protect this way of producing our starting material.

Finally, you are now awaiting results in part two of the phase I programme. What will you focus on in the development this spring and summer?

– Yes, the final part of the study with repeated dosing, MAD, is now in full swing and we believe that we will be able to communicate results during the second quarter. In terms of clinical development, in parallel with the phase I study, we will make important preparations for clinical phase II, i.e. efficacy studies.

– Part of it is the development of the study design, which we are doing together with KOLs and interaction with the US Food and Drug Administration (FDA). It is very valuable to have an understanding with the FDA on the development program to pave the way for future market approval in the US. We are also focusing, among other things, on continued GMP manufacturing.

Evaxion partners with MSD for vaccine development

Evaxion partners with MSD for vaccine development

Last fall, Evaxion Biotech announced entering a collaboration with a leading pharma company for the development of a vaccine against a bacterial pathogen causing severe healthcare issues. Now, we know that the partner is MSD. According to Evaxion, the initial phases of the collaboration have been successful. BioStock reached out to Evaxion’s CEO, Christian Kanstrup, to learn more.

In its pursuit to develop new therapies to save and improve lives, Evaxion Biotech is harnessing the potential of artificial intelligence (AI) to spearhead advancements in targeted immunotherapy development, including precision cancer vaccines and vaccines against infectious diseases.

At the core of Evaxion’s development strategy is its proprietary AI target discovery and development platform, AI-Immunology. Through this technology, Evaxion has already advanced two cancer AI-Immunology-designed vaccine candidates into clinical development, EVX-01 and EVX-02. Read more here.

Aiming for a refined business strategy

In September 2023, Evaxion’s Board brought in Christian Kanstrup as the new CEO to give more momentum to the company’s development and better exploit the strong foundation in AI technology. His appointment was specifically aimed at accelerating the strategic development and execution of the business opportunities at Evaxion.

According to Kanstrup, who visited the BioStock Studio in December to talk about the company’s refined business strategy, Evaxion’s new focus on value creation will revolve around a three-pronged business model, with the AI-Immunology platform at its core. The three pillars are TargetsPipeline and Responders – all of which will be suitable for partnerships. Read more here.

Partnership with MSD

Speaking of partnerships, soon after Kanstrup’s appointment, Evaxion announced two major collaborations. One is with Afrigen Biologics, which aimed at developing a prophylactic vaccine against gonorrhoea. The other was with a big pharma company, which remained undisclosed at the time, aimed at developing a new vaccine to protect against an unspecified bacterial pathogen.

Last week, we learned that the latter is a collaboration with MSD (Merck & Co.). The two companies have come together to work on a new project, called EVX-B3. The project involves using Evaxion’s AI Immunology platform for antigen discovery in the effort to develop this new bacterial vaccine.

Tarit Mukhopadhyay, Vice President, Head of Infectious Diseases and Vaccine Discovery at MSD, had this to say about the collaboration:

»At MSD, we are exploring new ways to apply AI technologies across the discovery-development continuum. We are excited to further advance our work with the Evaxion team.«

EVX-B3 is the third project in Evaxion’s pipeline dedicated to developing new bacterial vaccines. The other two candidates, EVX-B1 and EVX-B2, are in development to protect against two of the most dangerous bacteria, Staphylococcus aureus (S. aureus) and Neisseria Gonorrhoeae infections, respectively.

CEO insights

In the announcement about the MSD collaboration, Kanstrup mentioned that the first phases of the collaboration have been completed successfully. BioStock reached out to Kanstrup to learn more.

Christian, why is MSD the most suitable partner for this type of project?

– With MSD being a world leader in vaccines, it is, of course, a very strong partner given their capabilities within vaccine development and commercialisation, hence you can say it is a natural choice.

EvaxionCEO_ChristianKanstrup
Christian Kanstrup, CEO Evaxion Biotech

Specifically, what does MSD bring to the table in this collaboration?

– MSD brings invaluable insights into the pathogen and the associated disease to the collaboration; the company has a profound understanding of and expertise within clinical development of prophylactic vaccines. Their deep understanding enhances our collaborative efforts, complementing our expertise in vaccine target discovery and design.

You have announced that the first phase of this collaboration has been completed successfully. Can you disclose any more details about this part of the project?

–  We have successfully applied our validated AI-Immunology platform and identified several druggable vaccine targets that are predicted to prevent infections from the pathogen. These AI-identified targets are currently being manufactured, and the next steps of the collaboration involve preclinical testing of these vaccine targets.

When will we learn more details about project, including the indication being targeted?

– We will of course ensure updates on the project at relevant milestones. You should be looking at completion and hopefully an out-license agreement with MSD in less than 12 months.

Finally, how does this collaboration fit into Evaxion’s refined business strategy?

– The MSD partnership is fully in line with our refined business strategy. Our three-pronged business strategy is based upon a multi-partner approach along the three pillars: Targets, Pipeline and Responders. The MSD partnership is a very good example of the type of partnerships we are pursuing within the Target pillar, aiming to discover and develop novel vaccines addressing severe unmet medical needs.

Prolight Diagnostics’ CTO highlights the potential of the Psyros system

Steve Ross

BioStock talked to Steve Ross, co-founder of Psyros Diagnostics, which was acquired by Prolight Diagnostics in 2022. He and the other co-founders play a crucial role in steering the continued development of the point-of-care system Psyros. Together, as a team, they have previously navigated the full process of developing a POC system, from conceptualization to market, and completed a successful exit to Novartis in 2012.

In 2022, Prolight Diagnostics acquired UK-based Psyros Diagnostics, giving them access to a ground-breaking single-molecule-counting (digital) point-of-care (POC) technology, Psyros.

Psyros can measure biomarkers at extremely low concentrations by counting single molecules from a single drop of blood. The system is initially being developed for measuring high-sensitivity troponin in  blood to rule-in, or rule-out, myocardial infarction. Current diagnostics result in long waiting times, while Prolight’s instrument provides results in less than 10 minutes.

Adding a highly experienced team

The acquisition also strengthened Prolight Diagnostics’ organisation by welcoming four experienced team members: Steve Ross, Aileen McGettrick, Julie Richards and Paul Monaghan – all four founders of Psyros Diagnostics.

The UK company was founded in 2018 by this team, with a proven track-record of developing in vitro diagnostics (IVD), particularly POC instruments. The team developed a piezofilm-based POC system and took it all the way to CE marking in Vivacta, which was later acquired by Novartis in 2012 for USD 90 million.

Aileen McGettrick and Julie Richards, both with expertise in assay development, are now the CSO and the Head of Assay Development at Prolight Diagnostics. Paul Monaghan, appointed Head of Engineering, brings vast experience in the technical and regulatory aspects of IVD development where he works closely with CTO Steve Ross, who is also responsible for managing the product’s intellectual property (IP) portfolio.

Major shareholders after the deal

Prior to the acquisition, Psyros Diagnostics was equally owned by its four founders. The deal, with a total purchase price of SEK 65 million, had an upfront payment of SEK 19.5 million (30 per cent), paid through the issue of new shares in March 2021.

In connection to Prolight’s latest rights issue in December 2023, an additional SEK 32.5 million (50 per cent) was paid via offset. The remaining purchase price of SEK 13 million is to be paid in 2024, subject to meeting predefined milestones.

Today, the four Psyros founders hold 31,5 million shares each in Prolight Diagnostic, making them significant shareholders in the company. It is noteworthy that the founders’ issued shares are subject to a lock-up period which extends to December 31, 2026

CTO Steve Ross comments on the acquisition and potential

To gain deeper insights into the capabilities of the Psyros system, BioStock reached out to CTO and board member Steve Ross. In addition to undergraduate degrees in both chemistry and mathematics, Steve holds a PhD in synthetic chemistry from Edinburgh University.

Following postdoctoral research, his industrial career started at PiezOptic in 2001, where he developed pyroelectric sensors for monitoring exposure to toxic gases. In 2006, he co-founded Vivacta, utilizing the same pyroelectric technology but focusing on POC diagnostics. Following Novartis’ acquisition of Vivacta, Steve took on the role of Head of R&D for Novartis’ POC unit.

First of all, in your opinion, what are the highlights of your career and how do these impact  on your work today?

– Forming two separate companies and helping to create new jobs for scientists and engineers has been particularly fulfilling. As part of this process, I have had to develop resilience, get outside of my comfort zone and expand my knowledge base and skills over and above my key scientific training. I believe that this helps me be effective in my current role.

Focusing on your latest innovation – Psyros – what technological characteristics signifies its potential?

– Fundamentally, the ability to count single binding events in whole blood underpins the technology. Single-molecule counting is the most sensitive detection method possible. The potential revolves around the simplicity of how this can be achieved, giving it huge commercial value, in addition to the ground-breaking science that we are carrying out.

How do you perceive Psyros’ potential compared to the Vivacta system and other POC systems?

– Personally, I belive that the Psyros technology has greater potential than other systems beause it manages to combine the sensitivity of single-molecule counting in a simple design that allows for high-scale manufacture at low cost of goods. This trade-off between performance and cost of goods is absolutely critical and Psyros manages to hit that sweet spot that satisfies both requirements and is of key interest to the big diagnostic companies.

»I belive that the Psyros technology has greater potential than other systems beause it manages to combine the sensitivity of single-molecule counting in a simple design that allows for high-scale manufacture at low cost of goods. This trade-off between performance and cost of goods is absolutely critical and Psyros manages to hit that sweet spot«

What has the merger of Psyros Diagnostics and Prolight Diagnostics meant for the future outlook of Psyros?

– The merger has been of huge significance to Psyros Diagnostics. It has allowed us to expand at a rate that simply would not have been possible through grants and seed funding rounds. It allows the Psyros team to focus 100 per cent on delivering the product without other distractions. Prolight and Psyros work together to form a great team.

What are your expectations of, and goals for, the development of Psyros in the coming year?

– The next 12 months hold incredible potential – we will finalise the cartridge manufacture and take delivery of our first commercial prototype instruments that can be used to generate clinical data that confirms the performance of the system in fresh patient samples. This will prepare us for 2025 when we will start the full clinical evaluation study for regulatory approval.

Enzymatica’s partners await MDR certification and study results

Enzymatica

Enzymatica has published its year-end report, revealing an operating result of SEK -48.1 million and net sales of SEK 50.9 million for the full year. While sales increased in Sweden, the order intake from partners in other markets has been below expectations. To reverse this trend, MDR certification and study result will be crucial factors, according to CEO Claus Egstrand. 

Enzymatica has developed ColdZyme, a mouth spray for colds that has been launched in more than 30 markets on four continents. The product is based on the enzyme formulation Penzyme, which reduces the amount of virus in the throat. ColdZyme can prevent colds, shorten the duration of colds and relieve symptoms. 

Sweden is growing but other markets are declining

In Sweden, sales of ColdZyme were up 31 percent in 2023 as a result of active campaigning. However, the other markets have not delivered the same results. Sales for the full year amounted to SEK 50.9 million, which is lower than the company had hoped for. 

The lower than expected sales were mainly due to low order intake from Enzymatica’s international distribution partners. The company therefore has ongoing constructive dialogues with its partners and has also initiated discussions with other potential partners. 

However, several partners have indicated that they are awaiting MDR approval for ColdZyme before placing new orders, which gives Enzymatica hope that order intake can pick up once the certification is in place. 

Working with MDR certification

During 2023, Enzymatica has been actively working to obtain CE marking according to MDR (Medical Device Regulation), which is the new European regulatory framework for medical devices, replacing MDD (Medical Devices Directive). 

ColdZyme is already certified according to MDD Class III, but Enzymatica is now striving to obtain MDR certification during the spring. With the certification, the company can also strengthen and clarify the health claims used in the marketing of the product. The CE mark also affirms that ColdZyme meets the regulatory and scientific requirements. With the certification, the company can also strengthen and clarify the health claims used in the marketing of the product. 

Study results from independent research groups

Enzymatica may not have achieved the sales they wanted in 2023, but they took many significant steps, especially during the second half when independent research groups presented new research findings for ColdZyme. A research group at Medical University Innsbruck demonstrated that ColdZyme reduces the viral load in cells infected with influenza A virus. The researchers believe that the product would also have a similar effect on other respiratory viruses. 

In addition, interim results from an ongoing study at the University of Kent were presented, showing that endurance athletes who use ColdZyme for symptoms of upper respiratory tract infections get a significantly reduced amount of rhinovirus in the throat, the most common cause of colds. Those who used ColdZyme also experienced milder sore throat symptoms compared to placebo. The study has been expanded with more participants to be able to show statistical significance for more symptoms and virus types. The final results will be presented in the second quarter of 2024. 

International interest

In addition, the interim results will be presented by Professor Glen Davison at the International Olympic Committee’s Conference on Sports Health in Monaco in a couple of weeks. Davison also recently participated in the American TV show The Balancing Act, together with Enzymatica’s CEO Claus Egstrand. In the interview, they talked about ColdZyme’s study results and how the product works – see the interview here. 

This shows that ColdZyme’s research results are generating international interest, which is important for increasing sales in both existing and new markets. Enzymatica is now evaluating several possible ways to establish ColdZyme in major new markets. 

Ready for growth

In order to be ready for rapid expansion, Enzymatica has chosen to upgrade its manufacturing facility in Iceland. 

In addition, the Board of Directors has decided to carry out a rights issue of SEK 27.4 million, before issue costs, to finance the company’s activities and strengthen its position in negotiations with stakeholders. The rights issue is secured to 100 per cent through subscription commitments and underwriting commitments from the three largest shareholders, members of the Board of Directors and the CEO. 

Comments from the CEO

Claus Egstrand
Claus Egstrand, CEO Enzymatica

BioStock reached out to the company’s CEO Claus Egstrand to find out more about how the company is preparing for growth. 

First of all, what are the key messages in the year-end report? 

– The most important message is that we are poised for strong growth. We anticipate achieving significant milestones in 2024, including MDR certification and new research findings, which will garner considerable attention for our unique product. Another crucial message is that we observe international interest sparked by the outstanding research results presented last fall. This opens up additional opportunities for new international collaborations and expansion. 

»The most important message is that we are poised for strong growth. We anticipate achieving significant milestones in 2024, including MDR certification and new research findings«

You mention that you are pleased with the growth in sales in Sweden. How much of the total turnover comes from Sweden and your other home markets (UK and Iceland)?  

– Since partner sales have been so low in recent years, almost all sales come from our own markets. There is a great deal of interest in ColdZyme and we have a very high repurchase rate. Nine out of ten people who test ColdZyme say they intend to buy again. So we know we have a really good product – what has been lacking is our partners’ commitment to their local markets. 

What will you do to increase sales outside your own markets?  

– It is primarily about meeting the markets with the right partners. They need to feel the same commitment as we do to the product. Therefore, we are looking at adding additional partners for certain markets, in parallel with discussing launches in new markets together with others. The MDR certification will be important as it once again confirms our strong regulatory and scientific foundation. In addition, MDR would mean that we can sharpen our product health claims. The clinical results from the University of Kent are also important as they expand our scientific documentation and confirm our unique product. 

What steps remain before you can obtain MDR certification?  

– We have completed the entire process and are basically just waiting for information about the certification. I do not intend to claim victory in advance, but we have invested a great deal of internal and external resources in this and feel confident with the documentation we have submitted. 

Finally, why do you believe that 2024 could be a turnaround financially?  

– We never give forecasts, but as I said, we expect strategically important breakthroughs in several areas during the spring. In particular, we look forward to receiving MDR certification as well as the final report from the ongoing study at the University of Kent. These will generate further interest in our unique product from many stakeholders, which in turn lays the foundation for long-term growth.  

BiBBInstruments expands patent protection to Japan

BiBBInstruments utökar patentskyddet till Japan

BiBBInstruments’ electric-driven biopsy instrument for advanced endoscopy, EndoDrill, has been market cleared in Europe and the US. The Swedish medtech is clearly aiming to expand its horizons as it has announced receiving a Decision to Grant from the Japanese Patent Office for the EndoDrill technology. BioStock reached out to CEO Fredrik Lindblad for a comment.

BiBBInstruments (“BiBB”) is on a hot streak with the development of their endoscopic ultrasound device for cancer diagnostics, EndoDrill. Last spring, the company received FDA 510(k) clearance for its gastro-intestinal product variant, EndoDrill GI, opening the doors to the world’s largest market for endoscopy.

Successful clinical cases with EndoDrill in the US

Recently, BiBB’s founder Dr Charles Walther and BiBB’s CEO Fredrik Lindblad travelled to UC Davis Health in Sacramento, California, where the first clinical cases with EndoDrill GI in the US were being conducted. After the procedures, Dr Antonio Mendoza Ladd, who is Medical Director of Endoscopy at the hospital, was so pleased with EndoDrill’s performance that he called the device “a game changer.”

Dr Walther came by the BioStock Studio to talk more about the US experience. Watch the interview here.

Since then, the hospital in California has published an article summarising the results from the first three patient cases and describing the advantages of EndoDrill GI compared to today’s standard of care, i.e. manually stabbing needle instruments. The key takeaway being that EndoDrill enables more accurate cancer diagnoses leading to improved treatment for patients, which can contribute to better patient outcomes. In the article, Dr Mendoza Ladd said:

“We are excited to offer our patients this novel endoscopic biopsy procedure that will allow us to reach more accurate diagnoses and, therefore, offer better treatment that could result in improved outcomes.”

Market approval in Europe

In the midst of all this, BiBB also received CE-certification according to MDR for market approval in Europe. This covers all three product variants (EndoDrill GI for detecting cancers of the gastrointestinal tract, EndoDrill EBUS for lung cancer and EndoDrill URO for muscle-invasive bladder cancer), and it gives the company the go-ahead to commercialise EndoDrill in Europe. According to Fredrik Lindblad, who commented on the decision in this BioStock interview, together with last year’s FDA market clearance EndoDrill GI in the US, said: “this is our single most important milestone yet.”

Expanding commercial horizons

BiBB is not stopping at only the US and Europe with regard to EndoDrill’s commercial prospects. The company has been looking towards other regions of the world to expand this intellectual property protection. The aim is to provide global patent protection for the market’s first electric-driven endoscopic biopsy instrument.

Last week, the company announced receiving a Decision to Grant from the Japanese Patent Office (JPO) for a patent that gives broad protection the whole EndoDrill system. According to BiBB, only a few additional formal steps will follow before a patent is finally issued. Then, the granted patent will be valid until October 2039.

Comments from the CEO

Fredrik Lindblad , CEO BiBBInstruments
Fredrik Lindblad , CEO BiBBInstruments

Once this patent is granted, it will support the company’s positioning ahead of future commercialisation, and it will give BiBB expanded opportunities to increase commercial value of the company and its cancer biopsy device moving forward.

BioStock contacted BiBB’s CEO Fredrik Lindblad to learn more.

Fredrik, how significant is this Decision to Grant by the JPO for BiBB?

– This announcement is of utmost strategic importance for our future sales growth as Japan is one of the largest markets and maybe the most advanced market for our prime segment, endoscopic ultrasound. Furthermore, the global market of reusable endoscopes is dominated by Japanese companies.

Does the patent cover all three EndoDrill product variants?

– Absolutely, this broad patent in Japan provides protection for all our three EndoDrill product variants.

What are the steps towards commercialisation of EndoDrill in Japan?

– Our overall global sales strategy is to partner with one of the largest distributors of endoscopic accessories. We plan to start marketing through our own local organisation later this year in Sweden and the rest of Scandinavia. After that, we want to launch EndoDrill in Europe and the US through the selected distribution partner. Registration and launch in other important regions, such as Japan, will follow.

Do you have other patent applications pending?

– Yes, we have patent pending in several important markets, such as the USA and China. We have three patent families for the EndoDrill technology, so we expect a series of patent approvals over the coming years.

Finally, both you and the company founder Charles Walther have bolstered your shareholdings in recent weeks. What message would you hope that this sends to the market?

I hope that our owners and the market appreciate that we have achieved several value-adding milestones. We have taken important steps with a unique product in a fast-growing global market. Obtaining market clearances in the US and Europe within a year in an industry with increased regulatory requirements must be seen as a sign of strength. Through the recent first patient cases in the US, we have also received confirmation that we have developed a highly effective biopsy instrument, perhaps even a “game changer” as our first users have called the EndoDrill. In other words, we have laid a fine foundation for the coming commercialisation phase.