Chimerix, Inc.

Good morning, ladies and gentlemen, and welcome to the Kimerit's fourth quarter and year-end 2020 earnings conference call. I would now like to introduce you to your host for today's call, Michelle Laspaluto, Vice President of Strategic Planning and Investor Relations at Kimerit. Please proceed.

Thank you. Good morning, everyone, and welcome to the Kimerit's fourth quarter and year-end 2020 financial and operating results conference call. This morning, we issued two press releases. on our fourth quarter operating update and a second one on the ongoing COVID-19 trial of D-STAT. You can access these press releases in our investor section of the website. With me on today's call are President and Chief Executive Officer, Mike Sherman, Chief Medical Officer, Alan Melamed, Chief Financial and Business Officer, Mike Andriel, Chief Science Officer, Randall Lanier, and our newest member of the team, Chief Inferno Technology Officer, Josh Allen. Before we begin, I would like to remind you that the statements made on today's call include forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 and are subject to risks and uncertainties and other factors. These risks and uncertainties and other factors could actually cause results to differ materially from those referred to in the forward-looking statements. Please refer to our filings with the SEC for more complete disclosure of these risks and uncertainties. At this time, I would like now to return the call over to President and Chief Executive Officer, Mike Sherman.

Thanks, Michelle, and good morning, everyone. Thanks for joining us. I'm pleased to provide some additional color behind today's press releases. You'll note we've positioned ourselves to deliver on multiple value driving milestones in 2021 across all of our programs. That's a credit, of course, to the way this team is executing in every functional area. I'm happy to say that team is now even more capable, as we've completed ahead of schedule, the integration of the Oncocutics staff into the Chimerics organization structure. We've quickly pivoted from organizing ourselves to the process of accelerating the Oncocutics pipeline, particularly Onc201. Let me start my comments with the update we received in the last few days from the FDA on the extension of the PDUFA date to July. The extension is intended to allow FDA additional time to review new dose modeling we provided related to infants, newborns up to three months of age. The results of that modeling that we've already submitted supported the same weight-based suspension formulation dosing regimen we had already recommended for older pediatric patients. The fact that the FDA asked for this additional information highlights a key aspect of our program that satisfies an important unmet need, a formulation easily administered in a broad pediatric setting. We continue to feel very good about the NDA review process and look forward to the final steps. We confirmed with BARDA their process remains on track and will not be impacted by the review timing we still expect to see in RFP next month. The fact that we hadn't planned on shipping product into the stockpile until the second half of the year anyway means there's no financial impact to the updated review timing. We're still in a position to ship up to $100 million of product into the stockpile in the second half of this year. Let me move now to our D-STAT program for acute lung injury in patients with COVID-19. I have to say I was not expecting to see such a differentiation in outcomes between the 0.25 milligram dose of D-STAT and the control arm in this trial. The fact that all six D-STAT patients achieved the targeted 2.9 ad ordinal scale improvement and only two placebo control arm patients did the fact that there were two deaths on the control arm and none on D-STAT, the fact that these clinical results were supported by biomarker analysis relevant to D-STAT's mechanisms of action, and the fact that there were no treatment discontinuations due to adverse events on D-STAT compared to two on placebo. All of those things are very promising. Of course, this is a small cohort. and we know there were demographic imbalances which favored the D-STAT arm, so we take a dose of caution with that optimism. That having been said, I've seen a lot of excitement from other trials based on single-arm data. In our case, I'm glad we stuck to the discipline of randomization and blinding. I'll let Alan go into more detail on these findings, but it's also important to note we've completed enrollment of the second cohort, so we'll be able to supplement this data with another analysis next quarter. Recall that positive signals here are not just promising signals in the context of COVID, but also relevant to acute lung injury or acute respiratory syndrome from other causes. There may indeed be a longer-term need to treat a COVID population, particularly if we continue to see mutant variants of the virus. But even if the COVID infections decline dramatically, as we hope they do, we know these other indications are quite large opportunities. For the more advanced D-STAT program, the phase three trial in frontline AML, we've recently opened the first clinical sites and are ready to begin screening patients. You'll hear us refer to that as the DASH AML trial. Recall this multi-center, randomized, double-blind, placebo-controlled study We'll evaluate the efficacy and safety of D-STAT in combination with standard intensive induction and consolidation chemotherapy for the treatment of newly diagnosed AML patients. Our trial design includes an early assessment of the first 80 randomized evaluable patients, and in particular, we'll have a robust assessment of comparative complete response and MRD rates, minimal or measurable residual disease. A recent publication in the Journal of the American Medical Association detailed a meta-analysis of over 11,000 patients linking MRD negativity with disease-free survival and overall survival. As a result, this important data readout will give us a strong signal on the likelihood of success for the full Phase III trial. We expect that analysis will occur in 2022. Last and certainly not least, our newly combined teams are detailing our plans to advance OG201 toward registration. Recall this is the program that caught our attention and drove our acquisition of Oncosudix. We were excited by several aspects of the program. It's targeting among the most challenging diseases in an already challenging field, the H3K27M mutant form of glioma, which was recently classified by the WHO as a grade 4 glioma, regardless of histology. There are no good options for these patients today, and no drugs approved for them in the last 15 years. ONC201 has already demonstrated compelling and particularly durable responses from single-agent treatment using the most challenging of metrics to define responses in gliomas. is RANO-HGG, which stands for Response Assessment in Neuro-Oncology High-Grade Glioma. RANO criteria have both imaging and clinical hurdles required for bona fide responses, so it was indeed a high hurdle for ALK-201 to clear in achieving these responses. These tumor responses have been accompanied by meaningful clinical benefit, including performance status and neurological improvements. ALK-201 is also an easily delivered oral drug with a very attractive safety profile. Alka-Sootic management had previously completed robust dialogue with the FDA to agree on very detailed inclusion criteria to define the potential registration cohort of patients. Our research also supports the notion that there are low barriers to commercial success. The neuro-oncology field is already testing for this mutation and has a very high unaided awareness of the ONC-201 program. As the data matures for the last couple of patients treated, which we expect in the next few months, we will prepare to trigger a blinded independent central review to confirm response rate and duration of response. Recall much of the data from this 50 patient cohort has already been reviewed in a blinded fashion. We also had our own imaging expert review patient data as part of our diligence. This new blinded assessment will be performed in the second half of this year using two readers with a third for adjudication as necessary. We do plan to announce those results. It's also worth mentioning the other imipridones in the pipeline that are continuing to progress in development, ALK206 and ALK212. We're currently enrolling patients to be treated with ALK206 under an NIH-funded first in human dose escalation trial. I should note the first cohort has been completed without a dose-limiting toxicity, so we've moved on to the second cohort. In the meantime, we're continuing the preclinical work on ALK212. with support from Brown University. Let me hand the call over now to Alan, Dr. Melmed, to give a little more color on the data from our first cohort in the COVID-19 trial.