Biotech company Annexin Pharmaceuticals earlier announced encouraging first results in one of two studies evaluating the candidate ANXV in retinal vein occlusion. In the imaging study SIGHT, data from the first patient show that the candidate binds to the desired area and target molecule. The results provide support for the continued development of ANXV as a treatment for the disease. CMO/CSO and Co-founder Anna Frostegård tells BioStock more about the results and the ARVO congress that the company recently participated in.
Annexin Pharmaceuticals is focusing on the development of the biological drug candidate ANXV. The candidate is being evaluated in a phase II clinical study in the USA in the eye disease retinal vein occlusion (RVO), as well as in an imaging study in the same indication in the Netherlands. Since the turn of the year, the company has also made investments in the cancer area. Read more here. In both RVO and cancer, the candidate has the potential to be First-in-Class, i.e., a treatment with a completely new mechanism of action.
Positive initial data
In mid-April, Annexin reported encouraging first data from the Dutch imaging study SIGHT, to which the company is providing ANXV for evaluation in patients with RVO. In the study, ANXV is marked with a fluorescent marker, and thus can be detected by special eye cameras and tracked over time. The results show an accumulation of ANXV in the area of the retinal blood vessels, which is the area of damage in RVO. This provides an important indication that the drug candidate reaches its target molecule. In addition, the observation showed an overexpression of phosphatidylserine (PS) on the surface of the patient’s red blood cells, which provides further support for PS as an underlying disease mechanism in RVO. Read more about RVO here.
The data justify the continuation of the study through dose escalations and may guide Annexin regarding future selection in studies of patients with RVO. Recently, the Dutch regulatory authorities approved the inclusion of patients with diabetes-related retinal damage, so-called diabetic retinopathy. The study can thus provide support for ANXV’s potential in this disease as well. In total, up to 16 patients will be included, and SIGHT is expected to be completed by the end of 2023.
CMO gives insights on results
BioStock got in touch with Annexin’s CMO/CFO and Co-founder Anna Frostegård, who told us more about the first results and the ARVO congress that the company participated in.
Anna, you recently presented at the ARVO congress in New Orleans in the USA. Could tell us a little about this?
— The Association for Research in Vision and Ophthalmology (ARVO) has over 10,000 members in nearly 80 countries and holds annual gatherings for ophthalmologists, researchers and industry to share the latest research findings and to find innovative solutions in ophthalmology. This year, the congress was held in New Orleans in the US and was the first meeting after the pandemic with several thousand participants from all over the world in attendance. It is an honour to be selected to speak about the ANXV programme in retinal vein occlusion at such a premier meeting. We presented the general overview of ANXV’s development in RVO, data from our phase I study, and touched on our ongoing phase II study.
Looking at the initial data from the SIGHT study, how important are these for Annexin and ANXV?
— The preliminary results from the SIGHT study through which we collaborate with partners in the Netherlands are significant for Annexin and the development of our drug candidate ANXV for several reasons.
First, the results are important evidence of the technical feasibility of this project. Our ANXV was linked to a new fluorescent molecule, CW800, which was then injected intravenously into a patient with RVO and followed in the back of the eye in real-time with a sophisticated eye camera. This has not been attempted before, and it is therefore not surprising that we had to optimise our approach slightly and use e.g., a more sensitive camera to detect the fluorescent signal.
Second, it is important to view these data as potential evidence that ANXV, when injected intravenously into an RVO patient, actually behaves as a targeting protein, reaching the back of the eye and accumulating where we want it to be — namely at the blood vessels affected by RVO. It is encouraging and supports our ongoing phase II study in patients with RVO.
How do the initial results compare to your expectations?
– Although fluorescently marked Annexin A5 has been investigated in eye diseases such as glaucoma in the past, using a so-called DARC technique, there has never been an attempt to study RVO patients. We had expectations that we would be able to visualise ANXV at occlusion and we are pleased that this was possible even with the lowest ANXV dose investigated to date.
– It is also gratifying that overexpression of phosphatidylserine (PS) on the surface of the red blood cells in the patient in this study is consistent with data from previous independent studies, providing support for PS as an underlying disease mechanism in RVO.
How can the study guide Annexin for future clinical trials?
– The use of marked ANXV in the SIGHT study is expected to provide us with knowledge and images of ANXV distribution at the back of the eye. Such knowledge may be important for the design of future studies and cannot be generated from our ongoing phase II study, in which ANXV is administered without a fluorescent marker.
Do you plan to provide more updates from SIGHT for the rest of the year?
– The SIGHT study is a so-called investigator-sponsored study (ISS), which means that the investigators in the Netherlands take sponsor responsibility for the study, while Annexin supports the study by providing ANXV and sharing our knowledge with researchers. It is the sponsor who makes decisions about how and when study results are communicated. We look forward to the continuation of this study, as well as data from more RVO patients and at higher ANXV doses. But also that this study will include patients with diabetic retinopathy, which is an important chronic eye disease, and where ANXV may be of interest in the future. It is important for us to build knowledge and expand the data regarding ANXV in patients, which is crucial to confirm the potential of ANXV. The SIGHT study is expected to contribute to this.The content of BioStock’s news and analyses is independent but the work of BioStock is to a certain degree financed by life science companies. The above article concerns a company from which BioStock has received financing.