Verona Pharma launches second part of ensifentrine study in lung disease

Verona Pharma (NASDAQ:VRNA) jumps 10% premarket after initiating the second, multiple dose, part of a Phase 2 trial to evaluate the pressurized metered-dose inhaler (“pMDI”) formulation of ensifentrine in patients with moderate to severe chronic obstructive pulmonary disease (COPD), a type of progressive lung disease characterized by long-term breathing problems and poor airflow.

Results from the study (Part B) are expected in H1 2021.

Positive efficacy and safety data from Part A of the study in 40 patients were announced on March 31, 2020.

Details of Part B Crossover trial are as follows:

Patient Population: Approx. 30 patients who participated in Part A are planned to continue to Part B at two sites in UK.

Dose/Duration: Patients will be randomized to receive 3 dose levels (300 µg, 1000 µg, 3000 µg) of pMDI ensifentrine or placebo, twice-daily over one week.

Primary Endpoint: Improvement in lung function as measured by peak FEV₁ with ensifentrine compared to placebo after 7 days of treatment.

“Data from the single dose part of this pMDI study are very encouraging and consistent with data from Phase 2 clinical trials with our nebulized and dry powder inhaler formulations of ensifentrine,” said David Zaccardelli, Pharm. D., President and CEO.

https://seekingalpha.com/news/3606934-verona-pharma-launches-second-part-of-ensifentrine-study-in-lung-disease

Dr. Reddy’s launches Avigan for Covid in India

Dr. Reddy’s Laboratories (NYSE:RDY) announces the launch of Avigan (Favipiravir) 200 mg tablets in India. The launch is part of the global licensing agreement with Fujifilm Toyama Chemical Co. Ltd.

Avigan has been approved by the Drugs Controller General of India (DCGI) for the treatment of patients with mild-to-moderate COVID-19 disease.

Dr. Reddy’s Avigan comes in a complete therapy pack of 122 tablets with a two-year shelf life.

https://seekingalpha.com/news/3606943-dr-reddys-launches-avigan-in-india

Galapagos down on arthritis drug filgotinib stumble

Galapagos NV (NASDAQ:GLPG) and licensee Gilead Sciences (NASDAQ:GILD) are down 29% and 7%, respectively, premarket on the heels of a CRL from the FDA regarding GILD’s marketing application for filgotinib for rheumatoid arthritis (RA). The agency’s request for more data will extend the review period well into 2021.

The European Commission should approve filgotinib for RA next month. In late July, the advisory group CHMP adopted a positive opinion backing approval.

The companies inked a 10-year collaboration agreement a year ago that included the Janus kinase inhibitor, an expected blockbuster. GLPG received a $3.95B upfront payment in the deal.

In May, they announced positive results from a Phase 2b/3 study in ulcerative colitis.

https://seekingalpha.com/news/3606954-galapagos-down-29-on-arthritis-drug-filgotinib-stumble

Merck’s Keytruda extends survival in first-line esophageal cancer

Merck (NYSE:MRK) announces positive results from a Phase 3 clinical trial, KEYNOTE-590, evaluating Keytruda (pembrolizumab), combined with chemo, for the treatment of locally advanced or metastatic esophageal cancer in a first-line setting.

An interim analysis by the independent Data Monitoring Committee showed that the study met the primary endpoints of overall survival (OS) and progression-free survival (PFS) compared to chemo plus placebo.

A key secondary endpoint, objective response rate (ORR), was also met.

No new safety signals were reported.

The company plans to share the data with global regulatory authorities and submit the results for presentation at ESMO in late September.

Keytruda is currently approved in the U.S. and China for second-line squamous cell carcinoma of the esophagus whose tumors express PD-L1.

https://seekingalpha.com/news/3606973-mercks-keytruda-extends-survival-in-first-line-esophageal-cancer

J&J takeout of Momenta lifts argenx SE

Thinly traded argenx SE (NASDAQ:ARGX) perks up 1% premarket on light volume on the heels of Johnson & Johnson’s (NYSE:JNJ) $6.5B takeout of Momenta Pharmaceuticals (NASDAQ:MNTA).

The Belgian biotech’s antibody engineering technology is similar to Momenta’s, both involving the Fc region where antibodies modulate other cells in the immune system.

In May, argenx reported positive results from a Phase 3 clinical trial evaluating lead drug efgartimod in myasthenia gravis.

Momenta is developing lead asset nipocalimab for the same indication (among others).

https://seekingalpha.com/news/3606965-j-and-j-takeout-of-momenta-lifts-argenx-se

Precision Bio CAR-T therapy Fast Track’d for type of blood cancer

The FDA has designated Precision BioSciences’ (NASDAQ:DTIL) CAR-T candidate PBCAR0191 for Fast Track review for the treatment of advanced B-cell precursor acute lymphoblastic leukemia (B-ALL).

Fast Track status provides for more frequent interaction with the FDA review team and a rolling review of the marketing application.

A Phase 1/2a clinical trial is in process.

https://seekingalpha.com/news/3606978-precision-bio-car-t-therapy-fast-trackd-for-type-of-blood-cancer

BioMarin down big on FDA rejection of gene therapy for hemophilia A

BioMarin Pharmaceutical (NASDAQ:BMRN) slumps 22% premarket on increased volume in reaction to its announcement that it received a Complete Response Letter (CRL) from the FDA regarding its marketing application seeking approval of gene therapy valoctogene roxaparvovec for the treatment of severe hemophilia A.

The CRL cited the need for additional data (two years) from the ongoing Phase 3 study, 270-301, to confirm the duration of the treatment effect based on the primary endpoint of annualized bleeding rate. The review team recommended that the company complete the study and submit the two-year results. As such, the timeline for the data filing will be late 2021/early 2022 since the last participant will complete the follow-up in November 2021.

The FDA’s action date was August 21.

It plans to meet with the agency in the coming weeks to clarify next steps.

Its marketing application in Europe is currently under EMA review.

https://seekingalpha.com/news/3607019-biomarin-down-big-on-fda-rejection-of-gene-therapy-for-hemophilia