Blueprint Medicines: FDA Accepts Application for GI Tumor Treatment

Blueprint Medicines Corporation (BPMC), a precision therapy company focused on genomically defined cancers, rare diseases and cancer immunotherapy, today announced that the U.S. Food and Drug Administration (FDA) has accepted the company’s New Drug Application (NDA) for avapritinib for the treatment of adult patients with PDGFRA Exon 18 mutant gastrointestinal stromal tumors (GIST), regardless of prior therapy, and fourth-line GIST. The FDA granted Priority Review and set an action date of February 14, 2020 under the Prescription Drug User Fee Act (PDUFA). At this time, the FDA is not planning to hold an advisory committee meeting to discuss this application. Avapritinib, an investigational therapy, is a potent and highly selective KIT and PDGFRA inhibitor for patients with advanced GIST.

“Patients with PDGFRA Exon 18 mutant GIST and fourth-line GIST are in need of new treatment options that address the underlying drivers of the disease,” said Andy Boral, M.D., Ph.D., Chief Medical Officer at Blueprint Medicines. “The FDA’s acceptance of our application for Priority Review brings us closer to our goal of delivering avapritinib to patients with GIST, and we look forward to working closely with the FDA during the review process.”

The FDA’s acceptance of the NDA indicates the application is sufficiently complete to permit a substantive review. A Priority Review designation accelerates the FDA’s review time from 12 months to a goal of eight months from the NDA submission date, and is granted to drugs that may offer a significant improvement in the safety or effectiveness of the treatment, prevention or diagnosis of a serious condition. Previously, the FDA granted avapritinib Breakthrough Therapy Designation for the treatment of patients with unresectable or metastatic GIST harboring the PDGFRα D842V mutation.

In July 2019, the European Medicines Agency validated Blueprint Medicines’ Marketing Authorization Application for avapritinib in adult patients with PDGFRα D842V mutant GIST, regardless of prior therapy, and fourth-line GIST.

https://finance.yahoo.com/news/blueprint-medicines-announces-fda-acceptance-120000827.html

Bayer: Next glyphosate lawsuit likely to be postponed

 Bayer said the next U.S. lawsuit scheduled to be heard over claims that its glyphosate-based weedkiller Roundup causes cancer would likely be postponed.

Bayer, which acquired the weedkiller as part of its purchase of Monsanto last year, was initially scheduled to face its first trial outside California in St. Louis, Missouri, on Aug. 19, brought by Illinois resident Sharlean Gordon, who blames her non-Hodgkin’s lymphoma on using Roundup at home.

“We anticipate that this court case will be postponed but we have not received any written decision,” a Bayer spokesman said. He would not comment on how Bayer was informed of the delay or on the reason for it.

German weekly Wirtschaftswoche earlier reported the likely delay.

Bayer has seen its market value slashed by about 30 billion euros since August last year, when a California jury in the first such lawsuit found that Monsanto should have warned of the alleged cancer risks.

The company, which says regulators and extensive research have found glyphosate to be safe, is banking on U.S. appeals courts to reverse or tone down the first three court rulings that have so far awarded tens of millions of dollars to each plaintiff.

Bayer has previously flagged two more St. Louis cases due to start in September and October, respectively, but the spokesman on Wednesday said he could not comment on their timing.

https://www.marketscreener.com/BAYER-AG-436063/news/Bayer-says-next-glyphosate-lawsuit-likely-to-be-postponed-29026708/

Lilly Senior Vice Presidents Carroll, Garraway Departing

Eli Lilly & Co. (LLY) on Wednesday unveiled a series of management changes spurred by the departures of a pair of executives.

The Indianapolis drug maker said Darren Carroll, senior vice president of corporate business development, will retire at the end of the month. Mr. Carroll, who has led the corporate business development organization for nearly a decade, oversaw the recent $8.1 billion acquisition of Loxo Oncology–the largest acquisition in Lilly’s history.

Eli Lilly said Phil Johnson, currently senior vice president and treasurer, will assume additional oversight of merger-and-acquisition activity. The company said it named Heather Wasserman vice president of corporate business development, with responsibility for business development transactions, emerging technologies and innovation, and venture capital.

Eli Lilly also said Levi Garraway has resigned as senior vice president of oncology research and early-phase development to pursue other opportunities. The company said Josh Bilenker, chief executive of Loxo, will lead oncology research and early-phase development on an interim basis.

https://www.marketscreener.com/ELI-LILLY-AND-COMPANY-13401/news/Eli-Lilly-and-Senior-Vice-Presidents-Carroll-Garraway-Departing-29027482/

Inogen EPS misses by $0.04, misses on revenue

Inogen (NASDAQ:INGN): Q2 GAAP EPS of $0.45 misses by $0.04.

Revenue of $101.06M (+3.9% Y/Y) misses by $5.82M.

Press Release

https://seekingalpha.com/news/3488396-inogen-eps-misses-0_04-misses-revenue

MeiraGTx reports Q2 results

MeiraGTx (NASDAQ:MGTX): Q2 GAAP EPS of -$0.63.

License Revenue of $1.98M

Cash and cash equivalents of $204.3M

Press Release

https://seekingalpha.com/news/3488398-meiragtx-reports-q2-results

Intra-Cellular Therapies EPS beats by $0.17

Intra-Cellular Therapies (NASDAQ:ITCI): Q2 GAAP EPS of -$0.68 beats by $0.17.

Cash and equivalents of $91.76M

Press Release

https://seekingalpha.com/news/3488400-intra-cellular-therapies-eps-beats-0_17

Rhythm Pharma up 38% premarket on positive late-stage setmelanotide data

Thinly traded Rhythm Pharmaceuticals (NASDAQ:RYTM) is up 38% premarket on increased volume following its announcement of positive results from two Phase 3 clinical trials evaluating setmelanotide for the treatment of pro-opiomelanocortin (POMC) and leptin receptor (LEPR) deficiency obesities.

Both studies met the primary and key secondary endpoints, demonstrating statistically significant and clinically meaningful reductions in weight loss and insatiable hunger.

Specifically, 80% (n=8/10) of patients with POMC deficiency obesity achieved more than a 10% reduction in weight over ~one year (p<0.0001). Mean weight reduction from baseline was -25.4%. Mean reduction from baseline in most hunger rating was -27.8%.

45% (n=5/11) of patients with LEPR deficiency obesity achieved at least a 10% reduction in weight (p=0.0001). Mean weight loss from baseline was -12.5% and mean reduction from baseline in most hunger rating was -41.9%.

No new safety signals were observed.

Complete data will be submitted for presentation at a future medical conference and publication.

89% (n=17/19) of eligible patients have elected to participate in the extension study.

The company expects to complete its rolling U.S. marketing application next quarter and will request Priority Review status. An application in Europe will follow.

Setmelanotide activates a protein called melanocortin 4 receptor (MC4R) that plays a key role in controlling appetite.

https://seekingalpha.com/news/3488410-rhythm-pharma-38-percent-premarket-positive-late-stage-setmelanotide-data