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Trial of New Drug for Acute Leukemia Meets Primary Endpoint
Research Report

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The biopharma behind it plans to submit a new drug application, seeking approval of the compound, by year-end, noted an H.C. Wainwright & Co. report.

Syndax Pharmaceuticals Inc. (SNDX:NASDAQ) released topline data from the pivotal AUGMENT-101 trial of revumenib in relapsed/refractory KMT2A-rearranged acute leukemia in pediatrics and adults, and they were positive, reported H.C. Wainwright & Co. analyst Edward White in an October 2 research note.

Revumenib is Syndax's selective menin inhibitor and is designated a breakthrough therapy in this indication and population, for which no U.S. Food and Drug Administration (FDA)-approved treatment exists, White explained. With the current standard of care treatment, the expected response rate among these patients is less than 10%, and the expected survival period is less than three months.

Higher Target, Compelling Return

Based on the newly reported results, H.C. Wainwright increased its estimated probability of success for revumenib to 75% from 55%, White reported, and this boosted the investment bank's target price on Syndax to US$42 per share from US$38. In comparison, the current share price of this Massachusetts-based biopharma is about US$14.52 per share.

The difference between these two prices implies a significant potential return for investors of 189%.

Syndax remains a Buy.

About the Study Patients

The data cutoff for these AUGMENT-101 results was July 24, 2023, noted White. At the time, 94 patients had been treated, but only 57 were included in the efficacy evaluable population.

Patients in the study had a median of two prior lines of treatment.

Primary Endpoint Reached

White presented the new AUGMENT-101 results. The primary endpoint of the trial was complete response (CR) or complete response with partial hematologic recovery (CRh), and this was achieved.

Specifically, of the 57 evaluable patients with acute myeloid or acute lymphoid leukemia, 13 patients, or 23%, showed a statistically significant CR/CRh rate (p-value=0.0036). The overall response rate (ORR) was 63% or 36 patients. Of these 36 responders, 14, or 39%, proceeded to transplant.

Eight patients advanced to transplant without achieving CR/CRh, some of whom, White purported, likely would have reached CR/CRh. Those patients would have improved the reported overall CR/CRh rate.

In the acute lymphoid leukemia cohort of 49 patients, 12, or 24.5%, experienced a CR/CRh, and the ORR was 65%, or 32 patients.

The median duration of CR/CRh was 6.4 months. At the data cutoff date, 6 of 13 patients remained in response.

"We believe median duration of response could elongate over time," White wrote.

The independent data monitoring committee recommended Syndax stop the KMT2A-rearranged cohorts for efficacy at the protocol-defined point of interim analysis, and the biopharma did.

Safety, Tolerability Results

White discussed the safety and tolerability findings. Trial results showed that revumenib was well tolerated, and the side effects that occurred were in line with previous data.

Of the patients who had treatment-associated adverse effects (TRAEs), 28% experienced nausea, 27% experienced differentiation syndrome, and 23% experienced QTc prolongation. However, all cases of QT prolongation were grade 3 or less. As for differentiation syndrome, there was one grade 4 case but no grade 5 cases.

TRAEs, excluding QTc prolongation and differentiation syndrome, caused 6% of patients to discontinue the trial.

"We have no concerns regarding differentiation syndrome or QTc prolongation, and we do not expect the FDA to have safety concerns for approval," White commented.

Upside in Post-Transplant Use

Because relapsed/refractory KMT2A-rearranged acute leukemia patients may use and are using revumenib for maintenance following transplant, this additional use could boost commercial sales of the inhibitor, White pointed out.

According to Syndax, 50% of transplanted patients started revumenib as a post-transplant maintenance treatment, and 21% of transplanted patients were eligible for doing the same.

"We anticipate a revumenib launch in 2024 with sales of US$20 million (US$20M) that year, growing to US$591M in 2028," indicated White.

Catalysts To Watch For

More data from AUGMENT-101 are expected at an upcoming medical meeting, likely that of the American Society of Hematology, according to White, to be held in December.

Syndax is working on a new drug application (NDA) for revumenib in adult and pediatric KMT2A-rearranged acute leukemia that encompasses all cohorts. Submission of the NDA to the FDA is expected to happen by year-end 2023.  

In addition, White wrote, "There are multiple upcoming milestones for revumenib the company expects to achieve." Among them is a data readout by year-end 2023 of the AUGMENT-102 trial, evaluating revumenib in combination with chemotherapy in patients with relapsed/refractory KMT2A-rearranged and mNPM1 acute leukemias.

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Important Disclosures:

  1. Doresa Banning wrote this article for Streetwise Reports LLC and provides services to Streetwise Reports as an independent contractor.
  2. The article does not constitute investment advice. Each reader is encouraged to consult with his or her individual financial professional and any action a reader takes as a result of information presented here is his or her own responsibility. By opening this page, each reader accepts and agrees to Streetwise Reports' terms of use and full legal disclaimer. This article is not a solicitation for investment. Streetwise Reports does not render general or specific investment advice and the information on Streetwise Reports should not be considered a recommendation to buy or sell any security. Streetwise Reports does not endorse or recommend the business, products, services or securities of any company mentioned on Streetwise Reports.
  3. This article does not constitute medical advice. Officers, employees and contributors to Streetwise Reports are not licensed medical professionals. Readers should always contact their healthcare professionals for medical advice.

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Disclosures for H.C. Wainwright & Co., Syndax Pharamaceuticals Inc., October 2, 2023

This material is confidential and intended for use by Institutional Accounts as defined in FINRA Rule 4512(c). It may also be privileged or otherwise protected by work product immunity or other legal rules. If you have received it by mistake, please let us know by e-mail reply to [email protected] and delete it from your system; you may not copy this message or disclose its contents to anyone. The integrity and security of this message cannot be guaranteed on the Internet. H.C. WAINWRIGHT & CO, LLC RATING SYSTEM: H.C. Wainwright employs a three tier rating system for evaluating both the potential return and risk associated with owning common equity shares of rated firms. The expected return of any given equity is measured on a RELATIVE basis of other companies in the same sector. The price objective is calculated to estimate the potential movements in price that a given equity could reach provided certain targets are met over a defined time horizon. Price objectives are subject to external factors including industry events and market volatility.

H.C. Wainwright & Co, LLC (the “Firm”) is a member of FINRA and SIPC and a registered U.S. Broker-Dealer. I, Edward White , certify that 1) all of the views expressed in this report accurately reflect my personal views about any and all subject securities or issuers discussed; and 2) no part of my compensation was, is, or will be directly or indirectly related to the specific recommendation or views expressed in this research report; and 3) neither myself nor any members of my household is an officer, director or advisory board member of these companies. None of the research analysts or the research analyst’s household has a financial interest in the securities of Syndax Pharmaceuticals, Inc. (including, without limitation, any option, right, warrant, future, long or short position). As of August 31, 2023 neither the Firm nor its affiliates beneficially own 1% or more of any class of common equity securities of Syndax Pharmaceuticals, Inc.. Neither the research analyst nor the Firm knows or has reason to know of any other material conflict of interest at the time of publication of this research report.

The research analyst principally responsible for preparation of the report does not receive compensation that is based upon any specific investment banking services or transaction but is compensated based on factors including total revenue and profitability of the Firm, a substantial portion of which is derived from investment banking services. The Firm or its affiliates did not receive compensation from Syndax Pharmaceuticals, Inc. for investment banking services within twelve months before, but will seek compensation from the companies mentioned in this report for investment banking services within three months following publication of the research report. The Firm does not make a market in Syndax Pharmaceuticals, Inc. as of the date of this research report. The securities of the company discussed in this report may be unsuitable for investors depending on their specific investment objectives and financial position. Past performance is no guarantee of future results. This report is offered for informational purposes only, and does not constitute an offer or solicitation to buy or sell any securities discussed herein in any jurisdiction where such would be prohibited. This research report is not intended to provide tax advice or to be used to provide tax advice to any person. Electronic versions of H.C. Wainwright & Co., LLC research reports are made available to all clients simultaneously. No part of this report may be reproduced in any form without the expressed permission of H.C. Wainwright & Co., LLC. Additional information available upon request. H.C. Wainwright & Co., LLC does not provide individually tailored investment advice in research reports. This research report is not intended to provide personal investment advice and it does not take into account the specific investment objectives, financial situation and the particular needs of any specific person. Investors should seek financial advice regarding the appropriateness of investing in financial instruments and implementing investment strategies discussed or recommended in this research report. H.C. Wainwright & Co., LLC’s and its affiliates’ salespeople, traders, and other professionals may provide oral or written market commentary or trading strategies that reflect opinions that are contrary to the opinions expressed in this research report. H.C. Wainwright & Co., LLC and its affiliates, officers, directors, and employees, excluding its analysts, will from time to time have long or short positions in, act as principal in, and buy or sell, the securities or derivatives (including options and warrants) thereof of covered companies referred to in this research report. The information contained herein is based on sources which we believe to be reliable but is not guaranteed by us as being accurate and does not purport to be a complete statement or summary of the available data on the company, industry or security discussed in the report. All opinions and estimates included in this report constitute the analyst’s judgment as of the date of this report and are subject to change without notice. Securities and other financial instruments discussed in this research report: may lose value; are not insured by the Federal Deposit Insurance Corporation; and are subject to investment risks, including possible loss of the principal amount invested.

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