CASI Pharmaceuticals, Inc. (CASIF) Earnings Call Transcript & Summary

Greetings. Welcome to CASI Pharmaceuticals Business and Clinical Update Conference Call. Joining us today are CASI's Chairman and CEO, Dr. Wei-Wu He; and CASI's Chief Medical Officer and Executive Vice President, Dr. Alexander Zukiwski. [Operator Instructions] I'll pause briefly so you could take a moment to review the disclaimer and forward-looking statement on the screen. We will begin with opening remarks and a business update from Dr. Wei-Wu He. Following that, Dr. Alex Zukiwski will provide an update on the pipeline. After the presentations, we will open up the floor for a Q&A session. At this time, I am pleased to turn the call over to the Chairman and CEO of CASI Dr. Wei-Wu He.

Thank you for joining CASI Pharmaceuticals update call. We, as management felt the company is going through a major transformation, and we would like to make this an update call for our investors. First of all, we announced the completion of definitive agreement for the divestiture of assets in China. This allow us to focus U.S. CASI Pharmaceuticals becoming an innovation-driven autoimmune company. We entered into the agreement regarding the Precision Autoimmune Therapeutics. After completion of this agreement and the completion of the divestiture agreement, CASI Pharmaceuticals will own -- will hold the rights to CID-103 in Japan and non-Asian regions. And CNCT19 and EVOMELA are not transferred in agreement. Awards or settlements in Juventus and Acrotech disputes will be equally split per agreement. The company is going to be focusing on our leading compound CID-103. CID-103 represents an anti-CD38 franchise in a product with multiple near-term milestones to deliver potential novel treatment options. We are pivoting towards a U.S. biotech company focused on autoimmune and organ transplant rejection continues to evaluate multiple in-licensing opportunities in the autoimmune therapeutic area. With that introduction, I will give it to Alex, our CMO, to give everybody an update on CID-103. Next slide.

Good morning. I'm Alex Zukiwski, the CASI CMO. Today, I will be providing a brief clinical development update on our CID-103, CASI's IgG1 human anti-CD38 monoclonal antibody. As noted in this pipeline slide, CASI is evaluating 4 potential indications for CID-103, including ITP in an ongoing Phase I dose escalation study, an antibody-mediated rejection of renal allografts, which I will discuss later in the presentation, aplastic anemia and post-transplant lymphoproliferative disorders, which are of considerable interest to CASI. Next slide, please. This slide outlines some of the observed preclinical and clinical findings, which may differentiate CID-103 from other molecules in the class. This includes a different binding epitope enabling intellectual property coverage. There is substantially diminished red blood cell binding and decreased interference with blood bank retransfusion testing. In addition, we have observed decreased infusion-related reactions relative to the reported IRRs in other molecules in the class. Our data indicates that there is a potential increased potency relative to other molecules in the class. Next slide, please. This slide outlines the ITP dosing schedule for the Phase I study. We start with the priming dose on week 1 to avoid or diminish potential infusion-related reactions, followed by a weekly target dosing for weeks 2 through 6, after which we move to a q2-week dosing schedule administered on week 8, 10, 12, ultimately completing the 6 months of treatment with an every 4-week dosing schedule administered on week 16, 20 and 24. Concomitantly with the CID-103 administration, all patients receive a pre and post dose of IV or oral steroids to prevent or diminish infusion-related reactions. Next slide, please. This slide outlines the health authority approved dose escalation study design with incremental increased doses in sequential cohorts. As noted in the prior slide, we start with the priming dose on week 1. The target dose starts at 30 milligrams, escalating up to the fifth cohort at a 900-milligram target dose. As noted, the protocol allows for testing of lower and intermediate doses. Next slide, please. Please note that the first 3 cohorts utilize a 1 plus 3 study design and thus it is entirely possible if no grade 2 or greater toxicity is observed that these 3 cohorts may only include one patient per cohort, thus enabling the protocol to reach an efficacious dose quickly. Additional study subjects may be enrolled into the previously completed dose level to obtain additional pharmacokinetic, pharmacodynamic and safety data. Next slide, please. This slide outlines the randomized Part B of the study intended to evaluate CID at a low, intermediate and high dose. Based on the totality of the data, safety monitoring committee may choose to only study 2 dose levels or may consider studying an intermediate dose of CID-103, which may not have been studied in Part A. The maximum tolerated dose from Part A will not be exceeded in Part B. Next slide, please. Let me provide a brief update on the ongoing Phase I ITP study. Three patients have been enrolled to date. As no grade 2 or greater toxicity during the DLT period was observed in the first 2 cohorts, only one patient has been enrolled in each of these cohorts. Cohort 3 is currently open for accrual with one patient enrolled at present. All decisions on dose escalation were made by the Safety Monitoring Committee. Now let me provide you with some preliminary data on the platelet levels and safety for the 3 patients entered into the study. It is important to understand the data, which I will present is preliminary and no conclusions on the potential safety or platelet levels should be drawn or inferred. Next slide, please. This slide depicts the preliminary platelet test results for the patient treated at the 30-milligram target dose. This patient's medical history included a transient platelet response sensitivity to steroids. The marked variability of the platelet counts may be due to the pre and post-steroid administration utilized to prevent or diminish potential infusion-related reactions. Next slide, please. This slide depicts the preliminary platelet test results for the patient treated at the 150-milligram target dose. This patient had been hospitalized prior to the start of study drug treatment due to the critically low baseline platelet count. Next slide, please. This slide depicts the preliminary platelet test results for the patient treated at the 300-milligram target dose. This patient is early in the study course, having received only 3 doses of study drug treatment, but is included for the sake of completeness. The day 14 platelet count was drawn before study drug administration. Next slide. This slide depicts the preliminary safety data for the first 3 patients enrolled in the study. As noted in this preliminary safety summary, no infusion-related reactions, dose-limiting toxicities or severe adverse events have been reported. The Grade 3 iron deficiency anemia was treated with intravenous iron and resolved within 1 week. This patient also had a history of severe menorrhagia. Next slide. As noted in the CASI public communications, the FDA has issued CASI a full clinical hold notification regarding the renal allograft development protocol and has requested a conservative approach to the dose escalation study. Next slide. In response to a recent FDA CASI dialogue, the preliminary assessment by the FDA review team to our e-mail submission of technical documents was your submission seem acceptable, except for the response to the clinical hold comment 3. This FDA feedback via e-mail provided clear guidance and requires CASI to submit an acceptable written response via e-mail to the last outstanding item. Our response to the clinical hold comment 3 has been submitted, and we are awaiting FDA feedback. If our response is found to be acceptable by the division to Transplant Medicine, the revised IND will be submitted as soon as possible. Next slide, please. The division of Transplant Medicine has requested that we separate the original proposed Phase I, Phase II study into 2 separate studies. This slide illustrates the proposed Phase I study design with 4 dose escalation cohorts. Thank you. This concludes my presentation.

Now I will give this back to the operator for Q&A.

[Operator Instructions] This concludes today's conference, and you may disconnect at this time. Thank you for your participation.