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BioStock Investor Pitch: Prolevi Bio

Prolevi Bios vd Sahil Gupta på Medicon Village

Privately-owned Prolevi Bio’s goal is to improve the treatment of hypothyroidism, a common condition involving a lack of thyroid hormone. The company has developed a method that releases medicine based on the patient’s circadian rhythm. According to Prolevi Bio, this should improve the effect of the drug, something that CEO Sahil Gupta explains more about in a company presentation in BioStock’s studio.

Watch Prolevi Bio’s CEO Sahil Gupta present the company below.

Stayble approaches study results and other milestones

Stayble

Stayble Therapeutics is facing an eventful 2023 with expected data reporting from the phase IIb study with STA363. Patient recruitment was completed in 2022 and the six-month follow-up is predicted to be completed in the spring. In addition, the partnering discussions and preparations for phase III are now intensified. This includes a pre-IND meeting with the FDA, according to CEO Andreas Gerward.

Stayble Therapeutics develops the injectable treatment STA363 for patients with chronic disc-related back pain. STA363 transforms the disc into connective tissue, which potentially can reduce the patient’s pain – for life. The treatment is mainly aimed at those patients who do not respond to analgesics or physiotherapy, and who do not want to undergo spine surgery.

Results from the phase IIb study are approaching

Safety and effect on pain are currently being evaluated in a clinical phase IIb study that was initiated in 2020 and which was fully recruited in 2022. The study includes 110 patients who have received a dose of STA363 or a placebo. A follow-up period of 12 months is now underway and then the data will be compiled and analyzed. The goal is to present top-line results in Q4 2023.

After that, the next step is to initiate a phase III study together with one or more partners. In the event of positive study results in phase II, the next clinical phase could begin as early as the second half of 2024, according to an interview with Sara Richardson, Stayble’s VP CMC & Regulatory Affairs.

Upcoming milestones in 2023

Stayble
Andreas Gerward, CEO Stayble Therapeutics

For the next step in the development, Stayble Therapeutics will intensify the partnering activities and regulatory preparations, in parallel with the completion of the phase IIb study with STA363. BioStock talked to CEO Andreas Gerward about the expectations for 2023.

First of all, how would you describe Stayble Therapeutics’ development in 2022?

– 2022 has been a transformative but very positive year for Stayble. We managed to reverse a trend of slow patient recruitment to reach the highest screening as well as recruitment levels, which resulted in a fully recruited study in August. In parallel, we have attended several scientific and business conferences with great interest from potential partners and experts in the field. With a fully recruited study, we notice that partners’ interest is concretized and the discussions become more in-depth.

We managed to reverse a trend of slow patient recruitment to reach the highest screening as well as recruitment levels, which resulted in a fully recruited study in August […] With a fully recruited study, we notice that partners’ interest is concretized and the discussions become more in-depth«

– We also see positive trends linked to back pain with the FDA classifying back pain as a priority development area. In addition, more companies are taking an interest in hospital-based products.

What is the timetable until the reporting of top-line results from the phase IIb study in the fourth quarter of 2023?

– The last patient was recruited in August and then the patients are followed for 12 months. This means that the last patient will have their last follow-up in early September this year. After that, the patient follow-up is completed and all the clinics’ data is checked by our local partners, after which the database is locked. Then the code is broken regarding which patients have received active treatment or placebo. After that, top-line data is presented to Stayble. A month or so later, the final report will be published.

Are you planning to present any kind of study data before the full study results?

– We intend to provide the market with updates on the development of the study before top-line data. However, since the study is double-blinded, no data linked to STA363’s effect will be presented before Q4 2023.

A pre-IND meeting with the FDA is also on the agenda for 2023. Why is it important for your development plans in the US?

– The US is the most important market for us. Discussing our existing data and plans going forward with their regulatory authority will give us important insights into how we should design the upcoming clinical study and an overview of the approval path for a new drug.

– In addition to the FDA, we will also meet with the Swedish Medical Products Agency during the first half of 2023 for scientific advice for the European market. The Swedish Medical Products Agency has a very good reputation in Europe. Our discussions with them will provide a good understanding of how authorities in Europe reason in general.

»In addition to the FDA, we will also meet with the Swedish Medical Products Agency during the first half of 2023 for scientific advice for the European market. The Swedish Medical Products Agency has a very good reputation in Europe«

Last but not least, what can we expect in terms of partnering agreements for STA363 in 2023?

– During the year, Stayble will continue detailed discussions with several existing and new contacts to have as many actors as possible with interest in the results from our phase IIb study. However, it can take time to reach this type of comprehensive agreement and we will thus not have a final partnering agreement in place in 2023. But we will see which stakeholders we will prioritize for further negotiations, with the goal of concluding a partnering agreement in 2024.

Saniona and Cephagenix comment on their joint migraine programme

Saniona Cephagenix Migraine Collaboration

Last week, Danish biotech company Saniona announced the successful preclinical in vivo validation for a migraine treatment in the Cephagenix joint venture programme. BioStock reached out to Professor Jes Olesen, CEO of Cephagenix and Palle Christophersen, Executive Vice President, Research, Saniona, to learn more about the collaboration and the recent development milstone.

Copenhagen-based Saniona has research and development partnerships with Boehringer Ingelheim GmbH, Productos Medix, and in May 2021, Saniona also became a minority shareholder (27.6 per cent) of Cephagenix ApS, a private Denmark-based company formed to explore ion channel modulators for the treatment of migraine.

Migraine – a debilitating neurological condition

Approximately one billion people worldwide suffer from migraine, a chronic neurological disorder, thus making it the most prevalent neurological disorder, particularly affecting young adults and females. Individuals suffering from migraine have recurrent episodes of severe headache that can last for hours to several days, often accompanied by other symptoms such as nausea, vomiting, and sensitivity to light and sound. Comorbidities associated with migraine include stress, depression, stroke and anxiety.

Common treatments targeting migraine are CGRP antagonists, botulinum toxin and triptans. But, since only a small percentage of patients use current migraine treatments due to side effects or lack of effectiveness, there is a strong need for new and more tolerable migraine treatment options.

Saniona´s joint migraine project with Cephagenix

The Cephagenix and Saniona collaboration is aimed at identifying subtype-selective ATP-sensitive potassium channel (KATP) inhibitors for the treatment of migraine. Cephagenix has produced highly selective novel inhibitors of the specific KATP channel subtype expressed in the intracranial arteries and first-generation compounds from this series have now shown efficacy in a relevant rodent migraine model.

The drug candidates from the Cephagenix programme are initially intended for acute migraine treatment with potential for preventive treatment in chronic migraine patients. Cephagenix’s inhibitors were successfully developed using Saniona’s Ionbase technology platform. Most recently selected compounds from the series have demonstrated promising in vitro and in vivo preclinical results, thus validating the concept and the initial tool compounds from the series.

Cephagenix and Saniona comment on the collaboration

To learn more about Saniona´s and Cephagenix’s joint venture programme in migraine, BioStock reached out to Professor Jes Olesen, Danish Headache Center, University of Copenhagen and CEO of Cephagenix, and Palle Christophersen, Executive Vice President, Research at Saniona.

Jes Olesen, Cephagenix and Saniona aim to develop first-in-class KATP channel inhibitors as an efficacious and safe treatment of migraine. What is the medical need within migraine?

– There is a significant medical need for new effective and better tolerated migraine treatment options, and we believe that our subtype selective KATP inhibitors are well positioned to become the next generation effective migraine medicine. Current migraine treatments are only used by a limited fraction of patients partly because of side effects and/or insufficient response rates. We know that in Denmark only one out of seven migraine patients use triptans although these generally show the highest response rates of the medicines used. The new CGRP antibodies and antagonists appear to be better tolerated but their response rates generally seem to be lower.

Jes Olesen vd för Cephagenix
Jes Olesen, vd för Cephagenix

What are the potential benefits of KATP channel inhibitors?

– We believe the KATP channel is a common pathway for several migraine mechanisms and therefore expect efficacy in a broad segment of patients. Furthermore, we do not expect that this type of medicines will have CNS side effects.

What have your preclinical studies shown that makes you convinced that these findings could lead to new, more effective treatment options?

– We have demonstrated potent effects of our subtype-selective KATP inhibitors on the relevant arteries and shown efficacy in rodent pain models predictive for picking up anti-migraine drugs. Openers of the channel provoke migraine attacks in patients with migraine and blocking the channel is effective in our mouse and rat models of migraine.

– Based on both preclinical and clinical evidence generated by our group during the past two decades we have concluded that activation of KATP channels in intracranial arteries is the most effective means of inducing migraine attacks. This may be explained by the notion that most other migraine provoking principles e.g., CGRP, PACAP and NO seem to be ultimately mediated by opening of the KATP channels in intracranial arteries. Thus, our research shows that the KATP channel is a final common pathway for known triggers of migraine. Therefore, blocking this channel is likely to be highly efficacious in migraine. Moreover, the KATP channel targeted is located in blood vessels outside of the brain and not expressed by neurons. Therefore, no side effects from the brain are expected.

Palle Christophersen, in 2021 Saniona became a minority shareholder of Cephagenix. What made you interested in the migraine programme?

– Saniona has a long-time experience with the pharmacology of different potassium channels. When Jes Olesen approached us with this scientifically very well-validated project for migraine, we saw immediately that we were in an ideal position to quickly establish the necessary technology for driving this project. Furthermore – and very importantly – Jes Olesen convinced us that migraine is still an underserved disease, with a great opportunity to be first movers.

Palle Christophersen Executive Vice President, Research, Saniona
Palle Christophersen, Executive Vice President, Research, Saniona

Cephagenix’s novel subtype-selective inhibitors of KATP channels were developed using Saniona’s Ionbase technology platform. Can you elaborate on this procedure?

– IonBase is our compound collection and information database comprising more than 120.000 chemical structures. This knowledge base has been developed via many years of ion channel drug discovery and can be considered as a “library” enriched with chemical structures interacting with ion channels. Therefore, IonBase represents a unique starting point for establishing a new ion channel project.

How far have you come during the first two years of Cephagenix activities?

– During the first year we established the biological technology and screened the entire IonBase and ended up with a number of interesting “compound families.” These were characterised in detail for general off-target effects and basic properties, a process that led to the selection of a few chemical series to proceed further with. During the second year we established the necessary chemical technology and started what we call the hit-to-lead phase. In this phase we synthesised analogues and tested whether potency and selectivity as well as other compound properties could be improved. We also made the first tests on isolated cerebral blood vessels to be sure that the new molecules possessed the ability to counteract dilation of cerebral vessels, which is a prerequisite for an efficient anti-migraine compound with this special mechanism of action.

Jes Olesen, what do you see in the future of your collaboration to bring new migraine treatment options to the patients?

– We already have good contacts to pharma companies in the migraine space and at some point, we see a pharma partnership as the most effective way of developing the program through clinical studies and to the patients. We have seen that introduction of new migraine treatment principles have dramatically increased the migraine market, and there is significant big pharma interest in the field. It is worth noticing that Pfizer recently acquired the CGRP migraine company Biohaven for a total price of more than 11.6 billion USD.

CMO gives insight on new BioInvent collaboration

BioInvent LLS TAP

Having achieved a strong shareholder base, several important partnerships and a very successful capital raise last year, biotech company BioInvent has received yet another quality stamp. This one comes in the form of a collaboration agreement with the Leukemia & Lymphoma Society – whose blood cancer acceleration programme invests 3 MUSD in the company through a directed new issue. Going forward, BioInvent will get access to important knowledge and guidance from the patient organisation. BioStock contacted CMO Andres McAllister to get his comment.

BioInvent is a biotech company that develops first-in-class drugs for cancer therapy. The focus is to identify new antibodies that can significantly improve the efficacy of current checkpoint inhibitors and/or achieve immune activation in patients who no longer respond to treatment.

The company’s most advanced candidate is BI-1206, and also in the pipeline are BI-1607, BI-1808, BT-001 and BI-1910. BI-1206 is being developed in two separate phase I/IIa studies – one in non-Hodgkin’s lymphoma (NHL), a type of blood cancer, and one in solid tumours. An important part of the company’s technology is the screening tool F.I.R.S.T, which identifies both target structures and matching antibodies.

BioInvent attracts investors as well as partners

BioInvent has attracted great interest in recent years. This is particularly clear looking at the company’s shareholder base, where the largest shareholders consist of strong names such as Redmile, van Herk Investments, HBM, Forbion, Omega, Avanza Pension, Handelsbanken Fonder and Nordnet Pensionsförsäkring (as of December 31, 2022). BioInvent has also been at the forefront of signing agreements. Until today, the company has several ongoing partnerships, with names including Exelixis, MSD, Transgene, and CASI. Also, BioInvent has out-licensed drug candidates currently in clinical development by companies such as Daiichi Sankyo, Bayer Healthcare, Mitsubishi Tanabe, and Takeda.

Last week, it was announced that BioInvent has been selected as a partner for the Leukemia & Lymphoma Society’s Therapy Acceleration Program (LLS TAP), which will support the company’s clinical development of BI-1206 in non-Hodgkin’s lymphoma and BI-1808 in cutaneous T-cell lymphoma.

LLS is an American non-profit organisation active in research, patient support, and education in blood cancer. The goal is to prevent and cure leukaemia, lymphoma, myeloma, and Hodgkin’s disease, and to improve the quality of life for those affected. Therefore, LLS finances research in blood cancer and supports drug development activities in the process towards market approval.

LLS TAP makes a strategic investment of 3 MUSD

As part of the collaboration, LLS TAP is making an investment in BioInvent of 3 million USD in newly issued shares. This is backed by the board’s decision on a directed issue of 836,478 new shares, with a price tag of 37.40 SEK per share. The shares were expected to begin trading on Nasdaq Stockholm around January 23, 2023, and after the new issue, LLS TAP will own approximately 1.27 percent of the shares in BioInvent.
Commenting on the recent collaboration, Lore Gruenbaum, Vice President of LLS TAP, praised BioInvent:
”This partnership demonstrates our commitment to support and accelerate the development of the most promising and innovative blood cancer therapeutics worldwide. BioInvent has brought together a strong discovery platform, deep experience with therapeutic antibodies and a thorough understanding of immune oncology to create a rich and unique pipeline of promising therapeutics. We look forward to working with BioInvent to explore how these clinical candidates can benefit blood cancer patients.”

BioInvent’s CMO comments

BioStock spoke with CMO Andres McAllister about the new collaboration.

Andres McAllister, CMO BioInvent
Andres McAllister, CMO BioInvent

Could you tell us a bit about the unmet medical need within the indications that BI-1206 and BI-1808 are being developed for?
– Despite recent advances in the treatment of some forms of Non-Hodgkin Lymphomas (NHL), the great majority of the patients relapse after treatment, and relapses become ever more frequent and aggressive with time. Patients develop resistance to currently available treatments and few or no options remain available as the disease progresses. In that context, BioInvent is developing two important alternatives for the treatment of B and T cell lymphomas. The first, BI-1206, targets an important mechanism of resistance to rituximab, a great drug that revolutionized the treatment of B cell lymphomas and the backbone of current treatment regimens. Resistance to rituximab is developed with time and unfortunately sensitivity to the drug is progressively lost. BI-1206 has the potential of enhancing and recovering the activity of rituximab, by targeting an important mechanism of resistance to the drug, the overexpression by the tumor of FcgR2B, a receptor the modulates the activity of antibodies.

– The second drug, BI-1808 targets TNFR2, an important potential target present on tumor cells of T cell origin. In the case of T-cell lymphomas, the medical need is perhaps even greater since few treatment options exist, and the efficacy of the existing drugs is moderate, at best. The potential synergism with anti-PD1/PDL1 targeting agents could be a breakthrough for the treatment of these patients.

Having reached this agreement with LLS TAP – how will it affect BioInvent’s ability to advance BI-1206 and BI-1808 through the different stages of clinical development and into the market?
– LLS has a demonstrated ability and impressive track record recognizing and supporting the best treatment candidates under development. LLS has supported the development of 70% of the drugs that have been approved in recent years for the treatment of these diseases. Their support has been instrumental in improving recruitment rates and motivating clinical investigators to participate in the clinical trials implemented to develop the new drug candidates. We expect this endorsement will be key to enhance the recruitment of patients in our clinical studies. This is particularly important in the US, but it will also very likely have an impact in Europe. It is important to recognize that these diseases meet the qualification of orphan diseases, and in that regard, recruiting patients in these indications remains challenging.

How does BioInvent plan to leverage the expertise and resources of the LLS to advance its research in the field of blood cancer?
– We are pursuing the discussions with the LLS leadership, and they are making their network available to BioInvent. It is a network of very highly accomplished investigators and key opinion leaders. Our aim is to develop a very active partnership with LLS.

How will this collaboration position BioInvent in the blood cancer treatment landscape?
– It will increase BioInvent’s visibility in the field of Lymphoma and Leukemia, and LLS endorsement will help introduce the company and our approach to treating clinicians and investigators in the field.

12 Q&As about the stock market

12 Q&A about the stock market

Stock market activity is the object of intense scrutiny, especially in Sweden. Here, direct ownership in stocks as a share of total financial assets is among the highest in Europe. The Swedish environment for equities benefits not only large companies, but also innovation companies in, for example, the life science sector. What is a stock, why does a company want to list its stocks and does it matter which marketplace the company’s stocks are listed on? BioStock sought answers to some common questions on the subject.

Interest in the stock market is particularly high in Sweden. According to a report from 2020, the proportion of Swedes’ financial assets accounted for by direct ownership in stocks amounts to 11 per cent. Only Finland, with 19 per cent, reports a higher figure in Europe.

What is a stock, what marketplaces are available in Sweden and why do the companies want to list their stocks? These are some questions addressed in this article.

1. What is a stock?

A stock is an ownership interest in a company. When you buy a stock, you become a stockholder in the company. As a stockholder, you get to take part of the company’s assets and earnings. If the company is profitable, the board can decide on dividends, i.e. that the stockholders receive a part of the company’s profits. As a stockholder, you are also allowed to participate in the General Meeting. In these meetings, decisions are made on how the company should be governed and financed.

2. What is common stock?

When talking about stocks in a company, you usually mean common stock –  simply, the most common class of stock. In Sweden, so-called A and B shares are common, a type of ordinary share where the difference lies in how strong each share is in terms of votes at the general meeting. An A share gives the holder more votes at a general meeting than a class B share. For a private investor, the most relevant aspect is usually which share has the highest turnover, i.e. which share is traded most actively.

3. What is a preferred stock?

Preferred stocks are a class of stocks in which the owners are entitled to dividends before the ordinary stockholders. In many cases, the dividend is determined in advance – something that does not usually apply to common stocks. Therefore, preferred stocks can be seen as a middle ground between a common stock and a corporate bond.

In the event of bankruptcy or liquidation of a corporation, the preferred stockholders may prevail over the common stockholders, implying a higher price of the preferred stocks compared to the common stock.

4. What is a depositary receipt?

A depository receipt is a security that represents a specified number of stocks in a foreign company. For example, AstraZeneca stocks can be traded on the US Nasdaq. However, this does not involve buying and selling the actual stock, rather, a depositary receipt issued by a US custodian bank – AstraZeneca ADR (American Depositary Receipt).

Similarly, there are foreign companies whose depositary receipts are traded on the Stockholm Stock Exchange. For example, the engineering company Autoliv’s main listing is on the New York Stock Exchange, but it is also listed in Stockholm with Autoliv SDB (Swedish Depositary Receipt).

Owners of a depository receipt receive the same development and rights as a stockholder of the company’s ordinary stocks.

5. What is an MTF?

Stock trading is also available on other marketplaces, so-called MTFs. A Multilateral Trading Facility is a European term for trading in stocks or other financial assets. It is not a regulated exchange, but falls under EU’s MiFID II regulations, which are there to protect retail investors. Examples of MTFs in Sweden are Nasdaq First North Growth Market, Spotlight Spot Market and Nordic SME.

In the US, the equivalent is called ATS – Alternative Trading Systems – and although these are less regulated than the official exchanges, they are under the supervision of the SEC, the American equivalent of the Swedish FI.

7. What is a stock index?

A stock index is a way of categorising listed stocks according to various criteria, such as size, field of activity, region, etc.

OMXS30 is an index with the 30 most traded stocks on Nasdaq Stockholm. The index is the leading stock index on the Stockholm Stock Exchange thanks to the good liquidity of the underlying stocks. Trading in the stocks on the stock exchange is measured continuously and every six months an evaluation determines the 30 most traded stocks. The stocks included in the OMXS30 are weighted by market value, with the largest having the heaviest weight.

The broader Stockholm All Share is also a value-weighted stock index that includes all companies listed on Nasdaq Stockholm.

8. What is a sector index?

The companies on the stock exchange can also be divided according to their area of activity – industrial companies, finance, healthcare, etc. Morgan Stanley Capital International (MSCI) and Standard and Poor’s have developed a system for this type of categorisation that has become a financial industry standard. The Global Industry Classification Standard (GICS) divides all companies into 11 main categories:

Energy, materials, industrial goods, durable goods, consumables, healthcare, finance, information technology, telecom operators, power supply and real estate.

Each sector is further divided into subgroups – under the healthcare sector we find categories such as pharmaceuticals, biotechnology, medical technology and healthcare companies.

In the United States, which is the leading country in the world when it comes to stock trading, there are, for example, sector indices such as the Nasdaq Biotechnology Index (NBI). This index consists of approximately 275 companies listed on Nasdaq under the categories of biotechnology or pharmaceutical companies. Well-known American companies are included, such as Amgen and Gilead Sciences, but also large foreign companies such as AstraZeneca and Sanofi.

9. Where is the threshold for a large cap company?

The companies on Nasdaq Stockholm are divided into segments sorted by size – large cap, mid cap and small cap. A large cap on Nasdaq Stockholm has a market value of over 1 billion EUR. The mid-cap segment includes companies with market values in the range of 150 million EUR to 1 billion EUR. The small cap list lists companies with a market value of up to 150 million EUR.

10. How do you trade stocks?

A retail investor can access the stock market through banks and brokers. Many of these traders offer online services that make it possible to trade stocks via computer and mobile phone.

The order depth also has a practical significance for trading. How many buyers and sellers have placed their bids on the market? For larger, more liquid companies, the order depth is large and there is often enough buying and selling interest to handle most orders.

For smaller companies, the lack of order depth may mean that you are not guaranteed to buy or sell all the shares at the desired price, as the matching interest is insufficient.

11. What is the difference between a listed and unlisted company?

A listed company has opened its stocks for trading on a public marketplace. In the marketplace, investors buy and sell orders are matched at the current market price.

An unlisted company is privately-owned, and there is no established trading of the company’s shares. Often, unlisted companies are small organisations, sometimes in the development phase. However, there are examples of very large unlisted companies, such as IKEA and Tetra Pak.

12. What determines the stock price?

The demand and supply of the stock in question controls the market price. A positive view of the company’s future growth and profitability will drive up the stock price. Conversely,  a negative view of the future will put pressure on the price.

News that affects the conditions for the company often has an immediate impact on the share price. In life science, such news can be about achieved milestones in the development process, results in clinical studies, licensing or partnering agreements with major players, etc.

In this article, we have addressed a few common questions and answers about the stock market. Of course, there are many more, and if you would like to learn more about investing in the biotechnology sector, read the first part of BioStock’s article series on the matter here.

Evaxion receives Fast Track for cancer vaccine candidate

Evaxion_FastTrack

2023 is off to a good start for Evaxion Biotech. After receiving the go-ahead from the FDA to initiate a phase IIb trial their personalised cancer therapy EVX-01, which builds on the company’s proprietary AI platform, PIONEER. The Danish company has announced receiving fast track designation for the drug candidate in combination with Keytruda – a form of validation of its AI-driven immunotherapy development platform.

Melanoma is the deadliest form of skin cancer killing about 60 000 people each year world-wide. Immunotherapies like checkpoint inhibitors have improved survival chances for melanoma patients, however, if melanomas reach a more advanced stage and become metastatic, prognosis is extremely poor. As many as one third to one half of metastatic patients do not respond to treatment at all. This results in early death even after introduction of immunotherapies. Thus, the need for improved therapies is still significant.

Copenhagen-based Evaxion Biotech is addressing that need through its proprietary AI drug discovery platform, which is designed to develop personalised cancer immunotherapies structured to boost a patient’s immune system. By computing large amounts of data about the human immune system and cross-referencing them with a genetic analysis of each patient’s tumour, Evaxion’s technology can generate a unique drug for each patient.

Positive FDA feedback

The company’s most advanced candidate EVX-01 was discovered through the AI platform PIONEER, and it is being developed as a patient-specific cancer vaccine for the treatment of metastatic melanoma.

Promising results were generated with EVX-01 in a phase I/IIa study, and the drug candidate is currently being evaluated in a phase IIb trial in combination with Merck’s blockbuster PD1 checkpoint inhibitor Keytruda, with patients currently being enrolled in Australia. To expand the trial to the US, in November, the company submitted an Investigational New Drug application to the FDA. The application was sent together with a Fast Track designation application.

At the turn of 2022, Evaxion received the approval to initiate the study in the US, and last week the company also got the Fast Track nod. This designation is aimed at facilitating frequent communication with the FDA, related guidance on clinical trial design, and can result in expedited review timelines to ensure that novel therapies are available sooner for patients with serious illnesses.

Read more about Fast Track and other regulatory mechanisms aimed at streamlining drug development in our Article Series on Drug Development.

Evaxion’s CEO Per Norlén commented on the importance of the designation in a press release:

”We are extremely pleased that our cancer vaccine candidate EVX-01 has received the FDA fast track designation, as it enables a potentially faster approval of the vaccine. This is first and foremost to the benefit of the patients. And it is a great validation of our AI platform, PIONEER, and our drug development candidate.”

RhoVac becomes Chosa Oncology after acquisition

RhoVac acquires Chosa Oncology

After last year´s disappointing phase IIb-readout, a lot has happened for Lund based biotech RhoVac. With the acquisition of Chosa ApS, shareholders are in the position they had hoped to be last spring, with a cancer drug candidate churning out promising phase II results, preparing for a licensing deal. BioStock has taken a closer look to learn more about the new company.

Chosa Oncology is a Danish biotech company focused on the development of cancer treatments. The company´s iCIP concept revolves around two components, LiPlaCis and its Drug Response Predictor (DRP) companion diagnostic. The treatment core of the concept, LiPlaCis, is a liposome formulation of Cisplatin, a chemotherapy that is commonly used in the treatment of many forms of cancer today, primarily testicular, lung and ovarian cancer.

Cisplatin works by damaging the DNA of cancer cells, preventing their growth and ability to reproduce. It is considered one of the corner stones in many cancer treatment regimes, often just as important as surgery and radiotherapy. Cisplatin has been around in medicine since the late 1970s, and today sales of the popular chemotherapy amount to about 1.3 BUSD annually.

Responder prediction to help the right patients

For patients responding the drug can work wonders but, depending on the cancer indication, only between 20 and 60 per cent of patients actually respond to Cisplatin. If the wrong medication is given you lose valuable time, while exposing the patient to the side effects in vain.

Chosa Oncology address this problem with its DRP companion diagnostic tool – an algorithm that uses the expression state of 205 tumour genes to predict whether the use of Cisplatin will work for the specific patient or not. So far, the company has seen promising results, where patients with high DRP score have seen tumour shrinkage when treated with LiPlaCis. On the other side, treatment of patients with low DRP scores have not had any meaningful impact on the tumour.

Enhancing efficacy while limiting side effects

Just as with other chemotherapies, Cisplatin is associated with a set of serious side effects, including bone marrow suppression, hearing problems and kidney damage. This is exactly what Chosa tries to get around with the LiPlaCis formulation. With the new formulation, Cisplatin is encapsuled in liposomes that target and release their payload at the tumour site, sparing the healthy cells, with the aim of limiting the side effects.

So far, clinical studies have shown that LiPlaCis preferentially targets the tumour and it appears that some of the side effects associated with Cisplatin can be avoided with this new formulation. On top of that, in-vivo studies have shown that the LiPlaCis formulation is more effective than Cisplatin in preventing tumour growth. Further evidence of the drug´s potential is expected from a focused phase IIb-study where results is to be published in june2023.

Targeting metastatic breast cancer

Right now, Chosa Oncology pursues its iCIP concept for the treatment of metastatic breast cancer and for the treatment of solid tumours in children, a group of patients especially vulnerable to the hearing loss side effect of Cisplatin. With these two indications, the company sees sales potential for iCIP of about 560 MUSD on a yearly basis, in the US alone.

Given the broad use of Cisplatin today, the company also believes that the concept has good potential in several other types of cancer. Specifically, the company points out neoadjuvant treatment of early breast and lung cancer as two cases where iCIP could have the potential to increase the share of cured patients. Neoadjuvant use, where chemotherapy is used before surgery, is not possible with Cisplatin today due to its side effects. In this use case, the DRP is especially helpful with its targeting capabilities.

Deal to merge with RhoVac completed

As of Wednesday, the potential mentioned above is being pursued in a listed setting, with Lund-based RhoVac completing the deal to acquire Chosa ApS. In the deal, RhoVac will issue approximately 43.7 million shares at a subscription price of 1.05 SEK, directed to the Chosa’s owners. They will thereby hold approximately 70 per cent of the fused company, which is currently in the process of changing its name to Chosa Oncology.

Commenting on the completion of the deal, the company´s new CEO Peter Buhl Jensen, states: “We at Chosa would like to thank the RhoVac board of directors and RhoVac’s investors for supporting our project. I believe we are in a situation very similar to where RhoVac would have been if the RhoVac prostate cancer vaccine had been effective. With the good phase II data, Chosa’s project has demonstrated its promise. Chosa is now aiming to find partners to continue development for approval or for acquiring Chosa or the iCIP project.”

SynAct Pharma’s acquisition of TXP Pharma concluded

SynAct

Just before the end of 2022, SynAct Pharma announced its plans to acquire the Swiss biotech company TXP Pharma. The acquisition was approved on the Extraordinary General Meeting on January 12 and then completed on January 16. The addition of TXP’s platform puts SynAct Pharma in the forefront of resolution treatment through selective melanocortin stimulation and provides development opportunities in a broad range of autoimmune and inflammatory diseases.

SynAct Pharma is harnessing melanocortin biology to trigger the immune system’s natural inflammatory resolution and healing mechanisms. The company’s lead asset, AP1189, promotes resolution of inflammation through selective activation of melanocortin receptors 1 and 3. AP1189 is currently in three phase II studies: A phase IIb study, EXPAND, in early severe rheumatoid arthritis (RA), a phase IIa/b study, RESOLVE, in RA patients with inadequate response to DMARDs, and a phase IIa study in membranous nephropathy.

TXP Pharma’s portfolio – a valuable addition

SynAct Pharma’s leading position in inflammation resolution is now being strengthened by the acquisition of TXP Pharma, a Swiss privately held biotech company founded in 2013.

Similar to SynAct Pharma, TXP Pharma is focusing on research and development of pharmaceutical drugs for the treatment of autoimmune and inflammatory diseases by stimulation of melanocortin receptors. The company has created a platform of more than 70 peptide melanocortin agonists with a range of receptor selectivity profiles, as well as high receptor selectivity and stability.

Lead asset approaching clinical phase

TXP Pharma’s lead asset TXP-11 is being developed for the prevention of acute organ failure and damage in connection with complicated surgeries. Preclinical toxicology studies have been completed, and phase I studies is expected to be initiated in 2023. TXP-11 is intravenously administered, compared to AP1189 which is orally administered.

Two other promising assets are TXP-35 and TXP-59 that can provide subcutaneous administration and slow-release mechanism of action.

A related party transaction – independently executed

SynAct Pharma acquires all shares in TXP Pharma from a number of sellers, including Torbjørn Bjerke, Chairman of the Board in SynAct Pharma, CSO Thomas Jonassen, CEO Jeppe Øvlesen and COO Thomas Boesen. The purchase price is composed of an upfront fee of 136 MSEK and a potential earn-out of 55 MSEK upon positive phase II results of a TXP asset. The fixed purchase price is paid through 2.17 million newly issued shares in SynAct Pharma.

The acquisition has been handled by an independent committee of four members, chaired by Uli Hacksell. Watch BioStocks interview with him and Patrik Renblad here. Uli Hacksell also explains in a press release why he believes the acquisition is beneficial:

»By combining the pipeline and scientific capabilities of SynAct and TXP, we strengthen SynAct’s leading position within therapies for resolution treatment through melanocortin biology, and boosts its growing development portfolio. Also, we can now tackle the full range of inflammatory and autoimmune diseases with two complementary platforms«

Strengthens leading position in inflammation resolution

As a result of the acquisition, SynAct Pharma has two platforms that complement each other well. This means that they will shortly have two projects in clinical development, both AP1189 and TXP-11, and another two in advanced preclinical phase, TXP-35 and TXP-59.

AP1189 itself has great opportunities in several diseases, but the addition of TXP’s melanocortin agonists provides further possibilities in terms of for example indications, receptor selectivity and route of administration. The combined technologies will provide SynAct Pharma with drug development opportunities in a wide range of autoimmune and inflammatory disease. Some therapeutic areas that the company has mentioned are critical care, rheumatology and ophthalmology.

In summary, the acquisition of TXP Pharma expands SynAct Pharma’s potential to become a gamechanger in how inflammatory diseases are being treated.

Lipum’s clinical development journey continues in 2023

Lipum
Lipum

2022 was the year when the biopharmaceutical company Lipum entered the clinical phase with the drug candidate SOL-116 for the treatment of chronic inflammatory diseases. The phase I study is progressing according to plan and healthy subjects and patients with rheumatoid arthritis will be included in the study. But what will happen in 2023? The company’s CEO Einar Pontén provides answers in an interview with BioStock.

Umeå-based Lipum is developing a biological drug with a unique mechanism of action for the treatment of chronic inflammatory diseases. The company’s drug candidate SOL-116 is a humanised monoclonal antibody that blocks Bile Salt-Stimulated Lipase (BSSL), a previously overlooked target molecule in the immune system. BSSL binds to monocytes that in turn are active in the inflammation.

SOL-116 is being developed primarily for the treatment of rheumatoid arthritis (RA), but Lipum also sees opportunities to treat other inflammatory diseases such as Crohn’s, Ulcerative Colitis and Systemic Lupus Erythematosus.

Important achievements already at the beginning of the year

2022 was an eventful year for Lipum with many important milestones in the development of SOL-116, not least the step from preclinical to clinical development. The year began with Lipum’s partner Abzena successfully completing the GMP production of SOL-116. The drug formulation was shipped from San Diego to Umeå where APL (Apoteket Laboratorier Produktion) carried out aseptic fill into small bottles, so-called vials.

Shortly thereafter, the company initiated a collaboration with the Dutch CRO QPS Netherlands for the clinical phase I study with SOL-116.

Before the study could begin, the toxicology and safety program needed to be completed, which the company carried out at the beginning of the summer. The results showed that SOL-116 is safe and tolerable, and no side effects were observed.

First-in-Human study underway at full speed

In August, Lipum submitted a clinical trial application to start the phase I study with SOL-116 at QPS’ facility in Groningen, the Netherlands. The application was approved by the ethics committee and the regulatory authority in September and the study could thus start a few weeks later.

The purpose of the study is to ensure safety and tolerability, but also to evaluate the pharmacokinetics of the drug candidate. The study is conducted as a dose escalation study where a low dose is given to the first group and then the dose is increased for each group. By November, the patients in the first patient group had been dosed.

The study includes up to six groups of healthy volunteers and a group of eight patients with rheumatoid arthritis which provide an opportunity to study possible differences between healthy volunteers and RA patients.

The company aims to report study results by the end of 2023.

Funded for the next steps

To secure capital for the study, Lipum carried out a directed share issue of 39 MSEK in October. The majority of the issue was subscribed by Flerie Invest and other existing investors, but also new owners such as Torgny Stigbrand and Lars Stenlund.

BioStock had the opportunity to interview Torgny Stigbrand regarding his Lipum investment and his expertise in monoclonal antibodies – read the interview here.

External events support the development of novel RA drugs

In 2022, a few things happened in the RA area with impact on Lipum. In October, the European Medicines Agency (EMA) issued restrictions on JAK inhibitors, a relatively new class of drugs that, has been shown to cause a number of serious side effects. The FDA issued similar safety warnings back in 2021 when an increased risk of heart-related events, blood clots and cancer was shown in a large safety study with a JAK inhibitor.

This is a clear sign that there is still a need for new, safe RA drugs – read more about this here. Today, there is no cure for the disease, but the disease activity can be suppressed with antirheumatic drugs, so-called disease modifying anti-rheumatic drugs (DMARDs). The drugs that dominate this market are TNF inhibitors (biological DMARDs), as well as the aforementioned JAK inhibitors (targeted synthetic DMARDs) and methotrexate (synthetic DMARDs).

Blockbuster potential for SOL-116

Lipum sees an opportunity to meet the need for new RA treatments with SOL-116, which has the potential to become a “first-in-class and blockbuster” drug according to CEO Einar Pontén’s presentation at the BioStock Life Science Fall Summit. At the event, he also mentioned, with a twinkle in his eye, that the informal name of the phase I study with SOL-116 is “Sunshine”.

In 2022, Lipum also participated in a number of partnering events where they noticed an increased interest from potential partners after the Sunshine study started. BioStock had the opportunity to interview COO Pernilla Abrahamsson at the partnering conference Nordic Life Science Days in Malmö – watch the interview here.

Einar Pontén on the plans for 2023

Einar Pontén. vd Lipum
Einar Pontén, CEO Lipum

To find out more about the progress in 2022 and the plans for 2023, BioStock contacted Lipum’s CEO Einar Pontén.

Einar, what have you learned from 2022?

­– We managed to follow our plan despite a turbulent world and confirmed that it is important to have a competent team. With commitment and foresight from all employees, we were able to avoid delays, which is absolutely not easy as there are a lot of puzzle pieces that need to be put in place before you can start a clinical trial.

The most important milestone 2022 was, of course, the start of the clinical development program. What will be the most important milestone in 2023?

– As a company in clinical phase, we have definitely entered a new stage. The clearest and also most important milestone will of course be to be able to report the results from the current clinical study. Of course, it is good if we get results quickly, but the most important thing is that we get as much new knowledge as possible. Hopefully, we will be able to take advantage of this study in the design of future studies so that the total time until clinical verification of effect is as short as possible.

»The clearest and also most important milestone will of course be to be able to report the results from the current clinical study«

The start of the study also means that you have more time to explore additional chronic inflammatory diseases and evaluate the mechanism of action in preclinical studies. Do you expect to be able to present any new preclinical results in 2023?

– Our continued and intensified preclinical development has several sub-goals that will increase our knowledge and probably also our value. In general, we are restrictive in communicating preclinical results, but with more time and resources, I expect that we will present some scientific publications during the year.

Finally, what are your plans for out-licensing and/or partnerships?

– There is a clear interest in our work and this is expressed through both questions and benevolent advice. We try to take advantage of this to be efficient and provide information that is in demand, while breaking new ground around a unique target molecule. With SOL-116 in clinical phase, exploring a new target molecule, in a very large market with a significant medical need, it would actually be strange if we did not attract interest. I think the interest will increase when we can present clinical results.

Iconovo starts 2023 with negotiations for ICOpre

Iconovo

Iconovo enters the new year with confidence after a rewarding and eventful 2022. During the year, the company moved into new facilities at Ideon Science Park, strengthened the patent portfolio, entered into collaboration with Galenica and initiated a pandemic project together with the Bill & Melinda Gates Foundation. In addition, Iconovo recently entered negotiations with a potential licensing partner for the company’s inhaler ICOpre. CEO Johan Wäborg talks more about the negotiations and possibilities in 2023 in an interview with BioStock.

Iconovo develops inhalation drugs in collaboration with international pharmaceutical companies. It can be about completely new types of drugs as well as generic products. The company has developed several inhalers: ICOres, ICOcap, ICOpre, ICOone and ICOone Nasal.

In Iconovo’s new facilities in Ideon Science Park in Lund, there is a specially equipped laboratory for developing inhalers and dry powder formulation, enabling the development of complete inhalation products.

Progress in ICOres budesonid/formoterol project

In 2022, Iconovo made progress on several fronts, not least in the customer projects. The company’s most advanced project is ICOres budesonide/formoterol, a generic version of the blockbuster Symbicort Turbuhaler for the treatment of asthma and COPD. In early 2022, Iconovo’s partner Amneal Pharmaceuticals completed a clinical study that established the pharmacokinetic correlation between the products. Annual sales of Symbicort are estimated at USD 3.3 billion, of which USD 1 billion is in Europe (November 2022).

In 2022, a patent application for ICOres was also approved by the European Patent Office EPO. Iconovo has several approved patents for ICOres in the US, Europe, China, and Japan.

The clinical development of ICOres budesonide/formoterol contains a series of pilot pharmacokinetic studies that are carried out sequentially to optimize the product before a pivotal study to obtain market approval. The plan is to market the product in the Nordic region through the subsidiary Iconovo Pharma, while Amneal owns the rights in other markets. Read more about the establishment of Iconovo Pharma in BioStock’s interview with Fredrik Radencrantz, Director Business Development.

Potential licensing agreement for ICOpre with global generics company

In 2022, Iconovo also continued the development of ICOpre – a multidose pre-metered inhaler that has a dose counter and can be loaded with three different drug substances. ICOpre provides the same functions as GSK‘s well-known Ellipta inhaler but is based on its technical solutions, which minimizes the risk of patent infringement.

Iconovo intends to develop generic versions of all five products in GSK’s Ellipta portfolio (Relvar, Anoro, Trelegy, Incruse and Arnuity) which together generated sales of USD 4.2 billion in 2021. In 2025, the first patent for Relvar expires and Iconovo is far ahead in taking advantage of the business opportunity, through ICOpre.

Iconovo recently announced that they have entered into negotiations with a global generic company with the aim of a licensing agreement for one or more products in the ICOpre portfolio. According to Iconovo, the negotiating partner is a fast-growing and resourceful company in a group of companies that focuses on complex generic products and is expected to have a turnover of approximately USD 500-1000 million in 2022. The goal is to enter a licensing partnership within six months. However, Iconovo underlines that there are no guarantees that the ongoing discussions will result in an actual agreement.

Seamless client projects with Galenica collaboration

In 2023, Iconovo will continue to work on customer projects and aim to bring in more customers, as the negotiations on ICOpre testify.

To facilitate the process towards the customers’ clinical studies, Iconovo entered a collaboration with Malmö-based Galenica in 2022. The agreement covers GMP manufacturing of clinical trial material, primarily for Iconovo’s disposable inhaler ICOone.

The company currently has several collaboration agreements for ICOone, one of which is with Monash University for the development of a preventive oxytocin treatment for postpartum hemorrhage. In addition, ICOone will be used in Arcede‘s clinical study with RCD405, a new drug for COPD and asthma. The third project is the development of a nasally administered covid-19 vaccine in ICOone Nasal together with ISR.

Both ICOone and ICOone Nasal have low manufacturing costs and are easy to use. ICOone Nasal’s user-friendliness was demonstrated in a user study in 2022.

Collaboration with the Bill & Melinda Gates Foundation continues

ICOone is also used in the ongoing collaboration project with the Bill & Melinda Gates Foundation for the development of new inhalation treatments for the next pandemic. Iconovo has received a grant of 9 MSEK to ensure that there is both a technology platform and manufacturing equipment in place so that it can quickly develop a vaccine or prophylactic drug in the event of a new pandemic. The project runs until the summer of 2023.

CEO comments on the negotiations and goals for 2023

Johan Wäborg, vd Iconovo
Johan Wäborg, CEO Iconovo

BioStock contacted Iconovos CEO Johan Wäborg to find out more about the progress in 2022 and his hopes for 2023.

The market for generic drugs is characterized by extremely low prices and thin profit margins. Why are generic companies still an attractive customer group for Iconovo?

– We help customers with both the development of original drugs and generic products, so we have one foot in both segments. And as far as generics are concerned, one must be aware that the market for inhaled drugs has a completely different dynamic than that of, for example, tablets and capsules.

– To be able to launch a generic inhalation drug, a new inhaler is required that does not infringe the originator’s patent, and the drug substances are formulated so that they work together with the new inhaler. All these challenges mean that few companies dare, and even fewer succeed, to develop a generic inhalation product – especially those that must meet the requirements to be directly interchangeable with the original drug. Therefore, when patents expire for the original products, the price level is usually reduced by only 20–50 percent for inhalation treatments, compared to 80 per cent for drugs given in tablet form. It makes a huge difference to profit margins.

You have now entered into negotiations with a generic company for a potential licensing agreement for ICOpre. What opportunities does ICOpre offer to you and the potential partner?

– ICOpre offers the user the same convenience as GSK’s inhaler Ellipta. The drugs administered with Elllipta lose their patent protection over the next few years and sales of them amount to over four billion dollars, so there is of course a huge potential for generic versions. But few companies can handle the complex interaction between an inhaler and active drug substances. Since we can offer both a first-class inhaler and world-leading development expertise, we are a sought-after partner for generic companies. The fact that we have come a long way in the negotiations with a fast-growing global company is a clear sign of just this. There is enormous value here for both us and a future licensee.

»Since we can offer both a first-class inhaler and world-leading development expertise, we are a sought-after partner for generic companies. The fact that we have come a long way in the negotiations with a fast-growing global company is a clear sign of just this«

What are your main goals for 2023?

– We would like to establish a partnership for ICOpre; whether it will then be with the party we are now negotiating with or with another company remains to be seen, of course. At the same time, we continue to build up Iconovo Pharma, our Nordic pharmaceutical company, ahead of our first regional product launches under our own auspices. The basis of Iconovo Pharma’s product portfolio is future drugs based on ICOres, which are being developed by our partner Amneal, and in 2023 we will continue to evaluate opportunities to expand the portfolio through in-licensing, collaborations, or smaller acquisitions.

– Then we want to continue to give all our current partners the best possible support and look forward to more access and milestone payments. The collaborative projects are usually profitable for us throughout the development period, but the big financial leverage is the sales-based royalties after a fully developed product has been launched. The operations of Iconovo Pharma also have the potential to generate significant revenues with high profit margins, and all in all, we aim to deliver a profit margin of approximately 50 per cent in the slightly longer term.