Sanofi’s sutimlimab successful in late-stage cold agglutinin disease study

A Phase 3 clinical trial, CARDINAL, evaluating Sanofi’s (NASDAQ:SNY) sutimlimab (BIVV009) in patients with primary cold agglutinin disease (CAD) met the primary and secondary endpoints. The results were presented at ASH in Orlando.

The primary efficacy endpoint was the response rate as measured by a composite of an increase in hemoglobin from baseline or reaching a certain hemoglobin level at week 26 and the absence of transfusions from week 5 to 26.

54% (n=13/24) of subjects met the composite criteria and 63% (n=15/24) achieved hemoglobin levels of at least 12 g/dL (normal range for men and women: 13.5 – 17.5 g/dL and 12.0 – 15.5 g/dL, respectively). 83% (n=20/24) achieved clinically significant improvements in hemoglobin levels.

On the safety front, 29% (n=7/24) of patients experienced at least one serious adverse event, none considered related to the study drug.

Sutimlimab is a humanized monoclonal antibody that binds to (inhibits) a complement-specific serine protease enzyme called C1s that plays a key role in the mechanism of hemolysis in CAD, a rare autoimmune disorder in which the body’s immune system destroys red blood cells.

The company plans to file marketing applications in the near future. The indication has Breakthrough Therapy status in the U.S.

#ASH19

https://seekingalpha.com/news/3525094-sanofis-sutimlimab-successful-in-late-stage-cold-agglutinin-disease-study

Amneal to acquire majority stake in AvKARE

Amneal Pharmaceuticals (NYSE:AMRX) and AvKARE Inc. have entered into a definitive agreement under which Amneal will acquire a 65.1% majority interest in AvKARE and its related affiliate doing business as R&S Northeast for an implied enterprise value of $340M.

Amneal will acquire its majority interest through an unrestricted subsidiary, which will finance the purchase with a new $180M senior secured term loan facility, ~$75M cash and an ~$44M Seller Note, with the balance value contributed through the selling shareholders’ rollover interest in the newly formed subsidiary.

The transaction is expected to be completed in early 2020.

https://seekingalpha.com/news/3525100-amneal-to-acquire-majority-stake-in-avkare

Supernus Pharma up on updated late-stage SPN-810 results

Supernus Pharmaceuticals (NASDAQ:SUPN) is up 10% premarket on light volume in reaction to updated data from a Phase 3 clinical trial, P301, evaluating SPN-810 for the treatment of impulsive aggression (IA) in patients (ages 6 – 11 years) with attention deficit hyperactivity disorder (ADHD).

Topline results, first reported last month, showed a median 58.6% reduction in average weekly frequency of IA episodes from baseline compared to placebo. The separation, however, was not statistically significant (p=0.092).

The issue was an increase in variability in the 36 mg treatment arm due to six participants (out of 135) with mild IA conditions (seven with same were in the control arm). Excluding these, the separation was statistically valid (p=0.017).

Results from a second Phase 3, P302, based on a statistical plan excluding patients with mild IA, should be available next quarter.

https://seekingalpha.com/news/3525134-supernus-pharma-up-10-premarket-on-updated-late-stage-spnminus-810-results

Bristol-Myers’ CC-486 extends survival in late-stage AML study

Bristol-Myers Squibb (NYSE:BMY) announces positive results from a Phase 3 clinical trial, QUAZAR AML-001, evaluating CC-486 as maintenance therapy in newly diagnosed patients with acute myeloid leukemia (AML) who have achieved remission with intensive induction chemo. The data were presented as ASH in Orlando.

The primary endpoint was overall survival (OS) at month 60. At a median follow-up of 41.2 months, median OS was 24.7 months compared to 14.8 months for placebo (p=0.0009).

Median relapse-free survival, a secondary endpoint, was 10.2 months versus 4.8 months for control (p=0.0001).

Median duration of treatment was 12 cycles.

On the safety front, the most common adverse events (AEs) were nausea (65%), vomiting (60%) and diarrhea (50%). The most common serious/life-threatening AEs were neutropenia (41%), thrombocytopenia (23%) and anemia (14%). Serious AEs occurred in 34% of treated patients. The discontinuation rate was 13% versus 4% for placebo.

The company plans to submit regulatory filings in H1 2020.

CC-486 is an orally administered cytidine nucleoside analogue that incorporates into DNA and RNA and is believed to kill rapidly dividing cancer cells via DNA hypomethylation (loss of a methyl group in the cytosine base).

#ASH19

https://seekingalpha.com/news/3525079-bristol-myers-ccminus-486-extends-survival-in-late-stage-aml-study

Sanofi and Regeneron to restructure Kevzara/Praluent collaboration

Sanofi (NASDAQ:SNY) and Regeneron Pharmaceuticals (NASDAQ:REGN) have mutually agreed to revamp their antibody collaboration for Kevzara (sarilumab) and Praluent (alirocumab) into a royalty-based deal.

Under the terms of the new agreement, Sanofi will gain exclusive global rights to Kevzara and exclusive ex-U.S. rights to Praluent (Regeneron will own exclusive U.S. rights). Each party will be solely responsible for development and commercialization costs in their respective territories.

The changes should be finalized next quarter.

The terms of their Dupixent collaboration will remain as is.

https://seekingalpha.com/news/3525109-sanofi-and-regeneron-to-restructure-kevzara-praluent-collaboration

Sanofi to exit diabetes research; shares up 4% premarket

Citing its lack of success in bringing a new blockbuster to market and the expense of trying to do so, Sanofi (NASDAQ:SNY) has decided to stop further research in diabetes in favor of more specialized disease areas like cancer.

The diabetes business accounted for 13.3% of its Q3 sales (€1,261M/9,499M), down 9.9% from a year ago. Lantus was the top seller at €751M (-17.5%).

It will also stop research in cardiovascular diseases in light of development headwinds and disappointing sales of Praluent (alirocumab).

Key focus areas going forward will be specialty diseases, including hemophilia, breast cancer and multiple sclerosis and vaccines.

The company believes IL-4 receptor alpha antagonist Dupixent (dupilumab) will be a big winner, expected to generate €10B ($11B) in peak annual sales (from €2B this year).

It also plans to separate its OTC business and cut €2B in operating costs by 2022.

Shares up 4% premarket on light volume.

Insulin competitors: Eli Lilly (NYSE:LLY), Novo Nordisk (NYSE:NVO)

https://seekingalpha.com/news/3525050-sanofi-to-exit-diabetes-research-shares-up-4-premarket

UCB Results from Phase II Thrombocytopenia Trial at 2019 ASH

• Phase II data demonstrate that rozanolixizumab was well tolerated by patients with primary ITP across all dose groups
• Clinically relevant improvements in platelet count and decrease in immunoglobin G (IgG) levels were observed in all dose groups
• Safety, tolerability and efficacy data support Phase III development of rozanolixizumab for primary ITP
• Rozanolixizumab’s subcutaneous route of administration could provide a new treatment option for patients with primary ITP

https://www.biospace.com/article/releases/ucb-presents-final-results-from-phase-ii-study-of-rozanolixizumab-in-primary-immune-thrombocytopenia-itp-at-2019-ash-annual-meeting/