BPMC’s VOYAGER Fails, CLSD’s NDA Resubmission Faces Delay, SNDX Hits New High

Today’s Daily Dose brings you news about Blueprint Medicines’ disappointing results from gastrointestinal stromal tumor trial; revised timeline of Clearside Biomedical’s resubmission of XIPERE NDA; encouraging trial results of INOVIO’s MERS coronavirus vaccine and initial data from Syndax Pharma’s acute leukemia trial.

Read on…

Shares of Blueprint Medicines Corporation (BPMC) lost over 17 percent of its value on Tuesday, following disappointing results from the Company’s VOYAGER clinical trial of Avapritinib.

The VOYAGER trial is a phase III study evaluating the Company’s investigational drug Avapritinib against Bayer’s Stivarga (regorafenib) in patients with locally advanced unresectable or metastatic gastrointestinal stromal tumor, say in the third-or-fourth line settings.

The VOYAGER trial did not meet the primary endpoint of an improvement in progression-free survival (PFS) for Avapritinib versus Regorafenib.

Avapritinib, under brand name Ayvakit, received FDA approval in January of this year for the treatment of PDGFRa exon 18 mutant gastrointestinal stromal tumors.

Gastrointestinal stromal tumor (GIST) is a rare cancer that occurs in the digestive tract or nearby structures within the abdomen, say stomach or small intestine.

Avapritinib for the treatment of adults with fourth-line gastrointestinal stromal tumors is under FDA review, with a decision expected on May 14, 2020. At the time of submission of Avapritinib NDA, the FDA had indicated that the VOYAGER trial data would be informative in its review of the proposed fourth-line indication.

BPMC closed Tuesday’s trading at $58.63, down 17.31%.

Clearside Biomedical Inc. (CLSD) now expects the resubmission of its NDA for XIPERE to occur in the fourth quarter of 2020 and not by the end of August 2020 as previously projected.

XIPERE is being developed for the treatment of macular edema associated with uveitis.

The FDA had refused to approve XIPERE last October, requiring the Company to present additional stability data and reinspection of the drug product manufacturer as well as additional data on the clinical use of the final to-be-marketed SCS Microinjector delivery system.

Bausch Health (BHC) licensed Clearside Biomedical’s XIPERE last October.

CLSD closed Tuesday’s trading at $1.90, down 12.44%.

ERYTECH Pharma’s (ERYP) investigational drug Eryaspase has been granted Fast Track Designation by the FDA for the development of second-line treatment of patients with metastatic pancreatic cancer.

Eryaspase is being evaluated in a phase III trial in second-line metastatic pancreatic cancer, dubbed TRYbeCA-1, in 11 countries in Europe and the United States. An interim superiority analysis, to be conducted by an independent data monitoring committee (IDMC) when two-thirds of the events have occurred, is currently expected to take place around year-end 2020 and the final analysis in the second half of 2021.

ERYP touched a new high of $9.77 in intraday trading on Tuesday, before closing at $8.29, up 10.53%.

INOVIO’s (INO) phase I/IIa trial of DNA vaccine INO-4700 for MERS coronavirus has yielded encouraging results.

The vaccine recipients demonstrated strong antibody and T cell immune responses after 2 or 3 doses with 0.6 mg of INO-4700, a DNA vaccine that targets the MERS-CoV Spike (S) glycoprotein, delivered with intradermal CELLECTRA device, noted the Company.

INO-4700 DNA vaccine, being co-developed by Inovio and South Korea’s GeneOne Life Science, demonstrated 100% binding and 92% neutralizing antibody responses against MERS-CoV. The vaccination regimen was well-tolerated with no vaccine-associated severe adverse events (SAEs).

The researchers at the Wistar Institute, Seoul National University Hospital, and the International Vaccine Institute (IVI) collaborated on this study with funding from IVI.

INOVIO is planning to advance INO-4700 into a Phase 2 clinical trial for MERS coronavirus in the Middle East with a previously announced funding of $56 million by the Coalition for Epidemic Preparedness Innovations (CEPI).

In other news, the Company announced that its phase I U.S. clinical trial for COVID-19 DNA vaccine INO-4800 is fully enrolled with all 40 healthy volunteers receiving their first dose. The interim immune responses and safety results are expected in late June.

INOVIO is planning to advance the COVID-19 DNA vaccine INO-4800 to a Phase 2/3 efficacy trial this summer.

INO closed Tuesday’s trading at $13.75, up 0.36%. In after-hours, the stock was up 8.15% to $14.87.

Syndax Pharmaceuticals Inc. (SNDX) surged as much as 80 percent in intraday trading on Tuesday to touch a new high of $20.73, following initial phase I data, which demonstrated clinical activity of its drug candidate SNDX-5613 in adults with relapsed/refractory acute leukemias.

The initial data were from the phase I portion of the phase I/II trial, dubbed AUGMENT-101, evaluating orally administered SNDX-5613 in adult patients with relapsed/refractory acute leukemias including MLL-r and nucleophosmin (NPM1) mutant acute leukemias.

According to the Company, as of the April 17th data cutoff date, a total of six patients have been treated in the phase I portion of the ongoing open-label AUGMENT-101 trial at increasing dose levels of SNDX-5613. Responses were observed in two of three patients harboring an MLL rearrangement.

One patient had a complete response with incomplete blood count recovery (CRi) after 28 days of therapy and subsequently improved to a complete response (CR). The second patient achieved a partial response with incomplete blood count recovery (PRi) after 28 days of therapy, added the Company.

The Company expects to present additional results from AUGMENT-101 at a medical conference in the fourth quarter of 2020.

The final overall survival analysis from a phase III registration trial of Entinostat, a Class I selective HDAC inhibitor, plus exemestane in advanced hormone receptor positive, human epidermal growth factor receptor 2 negative (HR+, HER2-) breast cancer is expected this quarter. A positive overall survival assessment would allow the Company to file for full regulatory approval in the U.S.

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Will 2020 Be An Exciting And Transformative Year For Syndax Pharma (SNDX)?

SNDX closed Tuesday’s trading $19.50, up 69.42%.

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