Corbus Pharmaceuticals(US:CRBP)2020-03-16 21:16Clinical Development - Lenabasum is currently in Phase 3 studies for systemic sclerosis (SSc) with 365 patients enrolled and topline data expected in summer 2020[25]. - The Phase 2b study for cystic fibrosis (CF) has completed enrollment of 426 patients, with topline data also expected in summer 2020[42]. - The ongoing Phase 3 study for dermatomyositis (DM) is expected to enroll approximately 150 subjects, with a primary efficacy outcome based on the Total Improvement Score[48]. - The Phase 2 clinical trial for systemic lupus erythematosus (SLE) is expected to enroll 100 adult patients and report top line data in 2020[56][57]. - Lenabasum treatment showed a mean improvement of 9.3 points in the CDASI activity score compared to a 3.7 points reduction for placebo, with a p-value of 0.04[52]. - Lenabasum has demonstrated potent anti-inflammatory effects in a human model, enhancing bacterial clearance and inhibiting neutrophil infiltration[69][75]. - Lenabasum is designed to transition the innate immune response from the activation phase to the resolution phase, potentially addressing chronic inflammatory diseases[62]. - Lenabasum is proposed as a novel drug that resolves inflammation and halts fibrosis without immunosuppression, potentially addressing a significant unmet medical need in chronic inflammatory diseases[110]. - Lenabasum is being developed for autoimmune disorders with a unique mode of action, potentially resolving inflammation and halting fibrosis without immunosuppression[117]. Market Opportunity - The market opportunity for lenabasum includes serious inflammatory and fibrotic diseases such as systemic sclerosis, systemic lupus erythematosus, and nonalcoholic steatohepatitis[78]. - Approximately 200,000 people in the U.S., Europe, and Japan are affected by systemic sclerosis (SSc), with a median disease duration of 7.1 years from symptom onset to death[80][82]. - Approximately 80,000 individuals in the U.S., Europe, and Japan suffer from dermatomyositis (DM), which typically affects adults aged 50-60 years[85]. - The current median life expectancy for cystic fibrosis (CF) patients is about 40 years, with 30,000 Americans and 70,000 people in total suffering from the disease[96]. - An effective anti-inflammatory and anti-fibrotic drug for systemic sclerosis (SSc) is believed to address a significant unmet medical need, particularly if it is orally administered and not immunosuppressive[84]. - An effective drug that controls inflammation in DM is believed to address a significant unmet medical need, especially if it is orally administered and not immunosuppressive[89]. Financials and Funding - The company incurred approximately $88.6 million and $48.6 million in research and development expenses for the years ended December 31, 2019, and 2018, respectively[121]. - The company expects to continue incurring substantial losses for the foreseeable future due to ongoing research and development expenses related to its drug candidates[183]. - The company held cash and cash equivalents of approximately $31.7 million as of December 31, 2019, and anticipates that combined with expected proceeds from offerings, it will meet operating and capital requirements into the fourth quarter of 2020[191]. - The company has received a development award of up to $25 million from the Cystic Fibrosis Foundation to support a Phase 2b clinical trial of lenabasum, of which $17.5 million has been received to date[188]. - The company completed an underwritten public offering in February 2020, raising gross proceeds of $46 million[190]. - The company has not generated any revenue from its drug candidates to date and relies heavily on the successful development and commercialization of lenabasum[196]. Regulatory and Approval Challenges - Lenabasum has received Orphan Drug Designation and Fast Track Status from the FDA for SSc and CF, indicating its potential for addressing unmet medical needs[26]. - The regulatory approval process for lenabasum requires substantial time and financial resources, with compliance to various regulations being critical[134][135]. - The FDA may impose significant difficulties or costs in obtaining approvals, including the requirement for risk evaluation and mitigation strategies (REMS) for drug safety[145]. - The company must obtain separate state registrations and licenses for handling controlled substances, which could further delay clinical studies and product distribution[163]. - The company relies on third-party contract research organizations for regulatory approval processes, indicating limited in-house experience in this area[209]. - Regulatory approval is subject to various risks, including disagreements with regulatory authorities on trial design and insufficient evidence of safety and efficacy[207]. Strategic Collaborations and Partnerships - The company entered into a strategic collaboration with Kaken Pharmaceutical Co., Ltd., receiving an upfront payment of $27 million and potential additional payments of up to $173 million upon achieving certain milestones[189]. - The company plans to commercialize lenabasum in the U.S. independently while exploring partnerships in Europe[120]. Risks and Uncertainties - The company faces significant risks related to its limited operating history and the speculative nature of pharmaceutical product development[178]. - The ongoing impact of COVID-19 in regions like China and Italy could disrupt the company's business operations and clinical trials, potentially delaying regulatory approvals[199]. - Delays in preclinical and clinical trials could adversely affect the timeline for data readouts and regulatory filings, impacting the ability to commercialize product candidates[202]. - Even with regulatory approval, the commercial success of drug candidates depends on acceptance by the medical community and may be limited by factors such as pricing and competition[215]. - The company anticipates pricing pressures in the pharmaceutical industry due to managed healthcare trends and legislative proposals, which could adversely affect operations[166].