Chimerix, Inc.

Good morning, ladies and gentlemen, and welcome to the Chimerics Third Quarter 2021 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 Chimerics. Please proceed.

Thank you. Good morning, everyone, and welcome to the Chimerics Third Quarter 2021 Financial and Operating Results Conference Call. This morning, we issued our third quarter financial results press release and a separate release reporting positive top line results from the Onc201 recurrent H3K27M mutant glioma. You can access those press releases in our investor relations section of the website. With me on today's call are President and Chief Executive Officer Mike Sherman, Chief Financial and Business Officer Mike Andriel, Ellen Malamed, our Chief Medical Officer, and Josh Allen, our Chief Technology Officer of Inifrido. 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 Litigation Reform Act of 1995 and are subject to risks and uncertainties and other factors. These risks and uncertainties and other factors could cause actual results to differ materially from those referred to in the forward-looking statements. Please refer to our filings with the SEC for a more complete disclosure of these risks and uncertainties. At this time, I would like to turn the call over to Mike Sherman.

Thanks, Michelle, and good morning, everyone. Thank you for joining us. We're happy to share with you today the positive top-line results from the Blinded Independent Central Review, or BICR, of ALK-201 in the treatment of recurrent H3K27M mutant glioma. This is really just a preview of the analysis that will be presented in a couple of weeks at the Society for Neuro-Oncology annual meeting in Boston. You'll recall that we had worked with the FDA to define a cohort of patients that have the potential to form the basis of an NDA filing. These were the first 50 patients that met a very specific set of criteria across three different studies and two expanded access programs. The criteria were designed with two objectives in mind. The first, to define a homogeneous population of patients, and second, to select a group of patients in which tumor response could be assessed objectively with assurance that those responses were the result of ONC201 single-agent treatment alone. On top of that, perhaps the most stringent response criteria was utilized at the FDA's request, the response assessment in neuro-oncology criteria for high-grade gliomas, or renal HDG, as it's commonly referred to. For those accustomed to resist response criteria, renal HDG represents a higher bar, requires a tumor reduction of at least 50%, in addition to clearing non-imaging hurdles, including stable or improving performance status, and stable or declining use of steroids. So when you subject this analysis to a blinded independent central review, you're faced with a laundry list of reasons a response might not be confirmed. As we described in the past, the first 30 patients were evaluated previously with a single reader blinded review. And the last 20 had been investigator assessed. From those assessments, we had a 22% response rate with a few more patients maturing. This blinded review was a fresh assessment of all 50 patients and included a two-reader assessment with a third-reader adjudication when it was required. So you can imagine we're pleased to report a 20% overall response rate with that criteria. Alan will expand a little bit more on the approach for this protocol on this fresh assessment, which is really meeting a regulatory standard. It may be worth noting that the response rate in the first 25 patients enrolled for this data set recorded 20%, and the second group of 25 also had a 20% response rate. While achieving any Raynaud HGG response in this population of patients is not expected, we know the durability of response is also important. And these data are certainly compelling from a durability standpoint. Responses emerge gradually as tumors tend to shrink over a period of months in response to Onc201. And among responders, the Raynaud HGG hurdle of 50% reduction was achieved at a median rate of 8.3 months. On top of that, the duration of response was an additional 11.2 months. That translates to a median progression-free survival in responders of over 18 months, which is probably a better perspective on durability given the gradual onset of initial response. By the way, that's superior to the PFS among responders we previously reported of just over 15 months. There's several details we look forward to seeing presented at a plenary session during the upcoming snow conference, which will build on this data. That includes the disease control rate, which includes patients with shrinking tumors who fell short of the 50% threshold, and a few patients were indeed close. When you look at that disease control rate, roughly double the size of the response rate. We'll also report other forms of clinical benefit, which, as you might expect, were largely concentrated in those patients who achieved a renal response or disease control. The presentation will also contain results of renal low-grade glioma assessment, referred to as renal LGG, which are quite consistent with renal HGG, further confirming the robustness of these findings. The presentation will also include both PFS and OS analyses. A regulatory strategy has been to remain closely engaged with the FDA, and we will share this data with them as we continue our efforts on the ongoing clinical pharmacology studies and safety package covering a broader population of treated patients beyond the 50 in this analysis and CMC work. We don't see any of this as high risk per se, but it is work that's necessary to advance to an NDA. Lately, there's been a series of complete response letters from the FDA on several other companies' NDAs, and some of those CRLs are related to deficiencies in these areas, and we intend not to repeat those situations. At the FDA's request, we're also gathering natural disease history data, which we expect to support the notion that the data we're seeing in this patient cohort is truly differentiated. At this point, my apologies to Dr. Melamed, as I've probably already stolen the punchline of the data highlights, but I think he can provide some important context for these results. So let me turn it over to Alan.