Edited Transcript of CRBP earnings conference call or presentation 9-May-19 12:30pm GMT

In addition, we're very excited that Rachelle Jacques joined the company's board of directors. Rachelle has more than 25 years of experience in the U.S. in global commercial leadership and marketing companies such as Alexion, Shire and Baxalta. Her appointment complements and enhances our board's expertise, and we are pleased to welcome her to the board.

During the quarter, we strengthened our balance sheet with the completion of a $40-million public offering and receipt of $27 million in the form of the upfront payment from Kaken Pharmaceuticals, our Japanese partners. We ended the quarter with $89.9 million in cash.

Lenabasum, our first endocannabinoid-system-modulating compound, is in a potentially registrational Phase III study in people with systemic sclerosis. This RESOLVE-1 study completed subject enrollment in the first quarter and the study's primary endpoint is now the ACR CRISS score in the U.S.A. Data from RESOLVE-1 Phase III study are expected in the summer of 2020.

Lenabasum is also in Phase III testing in people with dermatomyositis and in Phase IIb testing in people with cystic fibrosis. In addition, a first-in-patient Phase II study of lenabasum in people with lupus is being conducted by the NIH.

As we approach the completion of RESOLVE-1 study over the next year, we will focus on developing commercialization strategies for lenabasum globally. Importantly, our unencumbered global patent rights for lenabasum increase the opportunities for strategic partnership -- partnering for commercialization of lenabasum.

As a reminder, we see significant commercial potential for lenabasum, targeting about 350,000 patients in the major markets. Our first major expansion for lenabasum outside the U.S. remains on track. Our partnership in Japan with Kaken Pharmaceuticals presents an important opportunity for Corbus, as Japan itself represents a market of about 28,000 systemic sclerosis patients and 9,000 dermatomyositis patients who have limited therapeutic options. Our Japan deal with -- the Japan deal we announced in the first quarter has already resulted in a $27-million upfront payment with additional milestone payments due of up to $173 million, as well as royalties.

We continue to evaluate other partnership strategies in international markets and, as I have noted previously, we view the Kaken agreement as a model for pursuing similar licensing deals in geographies less conducive to development by a U.S. company of our size, while providing near- and longer-term capital to fund our growth.

Looking ahead, we anticipate that CRB-4001 will enter Phase I safety testing at the end of 2019. Upon a successful completion of this Phase I study, the National Institutes of Health have committed to sponsor and conduct a follow-on early Phase II study to test CRB-4001 for safety and effects on metabolism and other biomarkers in patients with metabolic syndrome or NASH. It is our intention to develop CRB-4001 for the treatment of NAFLD or NASH.

We are on track to select our next candidates for pre-IND development. We are excited about the potential of the endocannabinoid-system-targeting compounds in our library and will describe some of these compounds to you in the near future. Our clinical development and commercialization opportunities continue to be supported by the strength of our balance sheet, which will fund our operations through the release of our pivotal Phase III data for lenabasum next summer and beyond, to the end of 2020.

We'd like to provide a more detailed update on our clinical pipeline. And with that, let me turn the call over to Dr. Barbara White, our Chief Medical Officer. Barbara?

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Barbara White, Corbus Pharmaceuticals Holdings, Inc. - Chief Medical Officer [4]

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Thank you, Yuval. Starting with our lead clinical asset. Lenabasum is currently being evaluated in several late-stage studies. The RESOLVE-1 Phase III study of lenabasum for treatment of systemic sclerosis completed enrollment on May 1. This was an important milestone to support potential filing of a new drug application in the U.S. and marketing authorization applications elsewhere around the world. The RESOLVE-1 Phase III study has 12 months of active dosing with lenabasum or placebo, plus 1-month follow-up. Database locks and then unblinding of treatment assignment in this study will occur in mid-2020 and we anticipate top line data will be available soon thereafter.

Following a Type C meeting with the U.S. Food and Drug Administration, we announced that the ACR CRISS score at Week 52 will be the primary efficacy endpoint in the RESOLVE-1 Phase III study in the U.S., and the change in modified Rodnan Skin Score, or mRSS, will become a secondary efficacy outcome rather than the primary. ACR CRISS stands for American College of Rheumatology Combined Response Index in diffuse cutaneous systemic sclerosis. ACR CRISS was the primary endpoint in the previous Phase II study. No changes to size or length for the Phase III study are required. As a reminder, systemic sclerosis affects approximately 200,000 patients in the U.S., Europe and Japan.

DETERMINE is our second Phase III program evaluating the efficacy and safety of lenabasum for the treatment of dermatomyositis. This study is ongoing. The Phase III study design is consistent with guidance from the FDA at an end-of-Phase-II meeting, formal consultation with Japanese regulatory authorities and scientific advice from European regulatory authorities. Dermatomyositis affects approximately 80,000 people in the U.S., Europe and Japan.

As for our one genetic disease indication, enrollment remains on track for our ongoing Phase IIb study of lenabasum for the treatment of cystic fibrosis, with top line data expected in 2020. As a reminder, our Phase IIb study in CF has rate of pulmonary exacerbations as the primary endpoint. Pulmonary exacerbations are responsible for about half of long-term decline in lung function in cystic fibrosis. Despite major advances in the treatment of CF, there remains a need for safe and effective treatment to reduce number and severity of pulmonary exacerbations in people with CF. In our Phase IIb study, we are testing the opportunity for lenabasum to address this unmet need. We are grateful to the Cystic Fibrosis Foundation, who, to date, has provided Corbus with the opportunity to receive up to $30 million in awards to fund our CF program. Cystic fibrosis affects approximately 70,000 people in the U.S. and Europe.

With that, I'll turn the call back over to Yuval.

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Yuval Cohen, Corbus Pharmaceuticals Holdings, Inc. - CEO & Director [5]

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Thank you, Barbara. Now let me briefly comment on our financial position, and as I mentioned earlier, we closed the first quarter of 2019 with a strong balance sheet, with $89.9 million in cash that funds our operations through lenabasum's pivotal systemic sclerosis Phase III data. We believe that the company is well funded into the fourth quarter of 2020.

Before I turn the call over to questions and answers, let me reiterate the progress that we are making against our clinical programs and in laying a solid foundation towards commercialization. During the quarter, to summarize, we bolstered our leadership team and board with key additions that bring commercial expertise. We continued advancing our commercial expansion outside of the U.S. -- for example, in Japan -- and we made progress with our late-stage clinical programs.

With our financial strength, strategic optionality through unencumbered global commercial rights and initial commercial planning, we are confident that Corbus is well positioned as we approach potential FDA approval for lenabasum and commercial launch. We look forward to continuing to update you on our progress and we plan to take CRB-4001 into Phase I testing, advance select compounds from our portfolio of novel drug candidates and develop our commercialization strategy.

On that note, I'm very pleased to announce that the company will be hosting a Research and Development Analyst Day in New York City in June. We will be releasing the date shortly, and we are looking forward to seeing you there and sharing with you our roadmap to becoming the leader in the treatment of inflammatory and fibrotic diseases using novel drugs that target the endocannabinoid system.