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BIO Innovation Zone Company Snapshots: ONL Therapeutics and Lipella Pharmaceuticals

May 19, 2015
Next month in Philadelphia at the BIO International Convention, BIO will be partnering with the National Institutes of Health (NIH) and the National Science foundation (NSF) to host the 2nd annual, newly-expanded BIO Innovation Zone. The Zone will feature Small Business Innovation Research (SBIR) funded early-stage biotech companies.

The SBIR/STTR program provides U.S. federal funding to small businesses engaged in research with the potential for commercialization. Each of the companies has been rigorously vetted through the SBIR/STTR review process prior to receiving the non-dilutive funding to engage in R&D that has the potential for commercialization. The NIH and NSF invest a combined $940 million annually in the programs.

Today, we spoke with John Freshley, the CEO of Ann Arbor, Michigan-based ONL Therapeutics, and Jonathan Kaufman, the CEO of Pittsburgh, Pennsylvania-based Lipella Pharmaceuticals. Both biotechnology companies are supported by the NIH’s SBIR program.

What is your company’s lead product or technology?

John Freshley, ONL Therapeutics: ONL Therapeutics is a private biotechnology company committed to protecting and improving the vision of patients with a range of retinal diseases and conditions, including retinal detachment and both the wet and dry forms of age-related macular degeneration (AMD).  We are the first and only company focused on preventing the death of photoreceptors - the root cause of vision loss and leading cause of blindness.

The company is currently advancing a novel, first-in-class, small molecule peptide that has demonstrated the ability to inhibit the Fas receptor (a TNF family member), block the activation of the Fas apoptosis pathway, and prevent photoreceptor death.  Unlike other programs under development, our therapeutic is a complement to current treatment regimens, fitting seamlessly into existing clinical practice and offering the potential for significantly improved outcomes.

We are pursuing retinal detachment as our initial indication although our therapeutic candidate has also demonstrated feasibility of photoreceptor protection in models of both wet and dry AMD.  We also believe that there are potential applications in several other retinal diseases.

We have focused our initial development efforts on retinal detachment, as we believe it offers a rapid and capital-efficient development and regulatory path.  We believe that we can advance our lead program to a new drug application (NDA) in as little as five years with ~$20-25 million of investment.  Furthermore, the design of our early clinical program allows the company to de-risk the program by demonstrating clinical utility of the pathway with a single injection in an acute setting.

The company has been granted orphan drug designation for the retinal detachment application, and we've completed our pre-IND meeting with FDA for this indication and the agency has deemed the company’s IND and clinical development plans appropriate.

Finally, ONL possesses a strong and wide-ranging intellectual property portfolio providing protection of the company’s drug candidate, and strong management and expert advisory teams with decades of experience in drug development and retinal disease.

Jonathan Kaufman, Lipella Pharmaceuticals: Our lead product, LP-08, is a clinical-stage drug candidate for Interstitial Cystitis, a painful inflammatory condition of the urinary bladder. We have recently published the results of an open-label clinical trial, which found a significant treatment-related reduction in both pain and urgency. Although Interstitial Cystitis was once considered rare, the National Institutes of Health currently estimates the U.S. prevalence to range between 700,000 to 1 million, 90 percent of whom are women. Just last month, we began recruitment for a new SBIR funded, Phase-2, double-blind, placebo-controlled clinical trial.

How has the NIH SBIR program helped your company grow?  

John Freshley, ONL Therapeutics: The NIH has been a strong supporter of ONL Therapeutics and our development of novel small molecule Fas inhibitors for the prevention of photoreceptor apoptosis.  SBIR funding received from NIH has allowed us to establish initial pre-clinical proof-of-concept and complete critical IND-enabling studies for the retinal detachment indication.  These efforts have included GLP toxicology and CMC development, as well as formulation development.  The results of this work have been essential to our advancement of our lead therapeutic candidate, and we are grateful to NIH for its role in our successes to-date.

Jonathan Kaufman, Lipella Pharmaceuticals: The NIH SBIR program has not only funded the development of our lead product, but it has also been instrumental to the development of our candidates for the treatment of Refractory Overactive Bladder and Hemorrhagic Cystitis. When we first launched our programs ten years ago, few venture capital firms made investments in pre-clinical assets. Fortunately, there are more options today, but at the time, the NIH SBIR program was almost the only willing funder for early-stage programs. It was a lifeline for us, and I am sure for many other companies as well.

What are the upcoming milestones and long-term priorities for your company?

John Freshley, ONL Therapeutics: ONL is currently in the process of raising a $15 million Series A round of financing.  Once complete, we plan to file an investigational new drug (IND) application and initiate our Phase I/II clinical trials in retinal detachment.

With respect to AMD, we expect to initiate pre-clinical development work, including multi-dose formulation and toxicology studies, in early 2016.  This work will be conducted concurrently with our clinical development in retinal detachment.

Jonathan Kaufman, Lipella Pharmaceuticals: Our next milestone is to obtain IND (Investigational New Drug) approval for our planned Hemorrhagic Cystitis clinical trial, although we may initially obtain approval to treat a few patients on a compassionate use basis. Hemorrhagic Cystitis can be devastating, ultimately leading to cystectomy or even death. Our long-term priority is to bring all of our product candidates to the market. To do so, we’ll seek partnership opportunities that can add strong clinical development and marketing resources.

What do you hope to gain out of your participation at the 2015 BIO International Convention?

John Freshley, ONL Therapeutics: It is our general hope to introduce ONL Therapeutics and our first-in-class therapeutics for the protection of photoreceptors to the broader biotechnology and healthcare community.  Specifically, we hope to engage with investors, pharmaceutical companies, strategic partners, and other biotechnology companies that share our mission and wish to align with ONL Therapeutics to advance our novel treatments for the benefit of patients worldwide.

Jonathan Kaufman, Lipella Pharmaceuticals: For us, the BIO International Convention is a nexus of potential partners, a meeting place to discover where some good fits may be. This year’s meeting comes at a perfect time for us, since we seek to expand the size of one clinical trial and to initiate a second clinical trial, both via partnership or financing.

Tell us something about your company that investors might not know.

John Freshley, ONL Therapeutics: ONL's management team is comprised of executives and scientists with drug development experience and ophthalmic expertise that is commonly reserved for the largest pharmaceutical companies.  Further our advisory team is comprised of key opinion leaders who are widely recognized as the world's leading experts in ophthalmic drug development.  Combined, ONL's leadership is well-suited to successfully advance our lead therapeutic candidate through NDA approval.

Jonathan Kaufman, Lipella Pharmaceuticals: They may be interested to know that Lipella is located in an economically depressed urban area of Pittsburgh. Apart from benefitting from a low cost of doing business, this location provides us with the opportunity to participate in the economic development of a most deserving neighborhood.