Soligenix is deeply entrenched in developing therapies for rare diseases; how did you come to choose specifically this healthcare area and how is your pipeline looking?
Having more than 30 years of experience in the rare disease setting, I always tend to bring this specific mindset into the new therapies that we are developing. A good example is synthetic hypericin - an active molecule that can be used to treat Psoriasis, which is an area of unmet medical need even though it affects millions of people worldwide. This compound is found in two of our drug candidates: SGX302, which is currently in a Phase 2 clinical trial, directed at plaque psoriasis, and HyBryte™, for which we recently submitted a new drug application (NDA) to the FDA, following a successful Phase 3 clinical study for the treatment of cutaneous T-cell lymphoma (CTCL). CTCL is a rare class of Non-Hodgkin's Lymphoma where malignant T-cells migrate to the skin surface to form patches, lesions or even tumors and there are currently no approved frontline therapies for it. Since it is a chronic disease, we are trying to keep it in its early stages, where the prognosis for survival is very good. HyBryte™, is applied topically to the cancerous skin lesions followed by activation with safe, visible fluorescent or LED light. This results in killing the cancer cells through a mitochondrial pathway that eludes the usual side effects seen with the DNA damaging treatments, such as chemotherapy.
Another development program we have in our pipeline is SGX203 (oral beclomethasone dipropionate). It is a topically active steroid that is delivered orally as a two-pill delivery of immediate and delayed release tablets to coat the gastrointestinal tract in order to reduce inflammation. The benefit of SGX203 is that it does not get as readily absorbed systemically, like Prednisone, thus managing the disease without the usual negative steroid side effects. This drug candidate is targeting pediatric Crohn's disease, which is also in the orphan sector and is Phase 3 ready.
At the end of the day, our mission is to execute on our drug development programs in order to help as many patients suffering from these diseases as possible. We are confident that if we execute properly and can demonstrate success in Phase 3 clinical trials, it has the potential to translate into financial success for the company and its shareholders.
What are the advantages of orphan designation and what challenges kept testing your resilience during development?
The orphan designation in the U.S. provides companies receiving the designation with several potential incentives that are designed to invigorate the pharmaceutical industry to develop products for the treatment of rare diseases (i.e., historically underserved patient populations). Companies that have obtained orphan designation for active pharmaceutical ingredients (e.g Hypericin in Soligenix’s case) qualifies the organization for incentives including: potential to receive grant funding for key development studies; tax credits for qualified clinical trials;Potential of 7 years of marketing exclusivity following approval and exemption from application user fees.
A similar program is maintained in the European Union where financial incentives including 10 years of marketing exclusivity following approval may be awarded. The extended exclusivity timeframe in both the U.S. and Europe is the key to incentivizing companies to commit resources to the development of drugs in rare and orphan disease areas where the revenues are not as substantial as you would typically see for broader disease indications. Moreover, due to the challenges of enrolling such rare patient populations in clinical trials when working with FDA and EMA, there is often the potential for approval to be based on a single, well-executed Phase 3 study.
How would you describe the investment climate at the present and in which ways can current difficulties be circumvented?
Over the last two years the investment climate has been difficult due, in large part, to the broader global market conditions. As you would imagine, this has not translated well for smaller, earlier stage biotech companies, where both development risk and capital need is higher. Coupled with the length of the drug development process that can take 10-15 years in order to achieve potential success, it can make for even harsher times. In such conditions, avoiding milestone delays is critical, as well as effectively managing development costs. One potential way to do this is to prioritize those programs that have the highest probability of success in the shortest time period. At Soligenix, we have done just that, by focusing our efforts on HyBryte™ in CTCL, where we have successfully completed a pivotal Phase 3 clinical trial and recently submitted a NDA for marketing authorization with the FDA. In addition, since CTCL is a highly specialized orphan disease, it will not require a large commercialization effort. For this reason, we are planning to commercialize it ourselves in the U.S. in order to begin generating revenue while looking to partner ex-U.S.
When the markets are difficult, being able to secure non-dilutive funding for your programs can also help with survivability. At Soligenix, we have been fortunate to secure a significant amount of non-dilutive capital over the years for a number of our programs. We aggressively submit grants and contracts with various government agencies. In particular, we established a Public Health Solutions business segment that has been funded almost entirely by the U.S. government to date. In this segment, we concentrate predominantly on heat stabilized vaccine candidates for Ebola, Marburg, COVID-19 and ricin poisoning that do not require cold chain storage and distribution. All are subunit vaccines, similar to the vaccines we have taken as children for many years, so the safety profile of subunit vaccines in general is well established and should provide an added level of comfort to the general public. Since 2006, we have been fortunate to have brought in approximately $60 million in non-diluted funding, which has been instrumental to not only advancing good technology, but more effectively managing cash burn during difficult market conditions that inevitably occur.
What are some future prospects for Soligenix?
First and foremost, our focus is to pursue FDA approval of HyBryte™ in CTCL and launch it ourselves in the U.S. in early 2024, which has the potential to be transformational for the company.
We will also continue to evaluate all strategic options, including the potential for partnership of HyBryte™ and our other development programs. Beyond that, our goal is continuing to expand our footprint in rare diseases and areas of unmet medical need, building value for our shareholders, and most importantly, developing as many important and life-saving drug therapies as possible for patients that so desperately need them.
