3 Things In Biotech, June 15: Keep Rolling, Pembro

Includes: MRK, NITE, NVS
by: Zach Hartman


Merck nets a second niche approval in so many days.

Nightstar hits the gas in gene therapy.

Novartis outlines a possible new direction for its blockbuster IL-1beta drug.

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Welcome to another edition of "3 Things In Biotech," a daily digest dedicated to helping you keep pace with the fast-moving world of pharmaceutical and biotechnology research.

Merck nets a second niche approval in so many days

Company: Merck (MRK)

Therapy: Pembrolizumab

Disease: Primary mediastinal large B-cell lymphoma (PMBCL)

News: MRK announced that the FDA has approved its PD-1 antibody pembrolizumab for the treatment of PMBCL that has relapsed after two or more prior lines of therapy. PMBCL is an entity of diffuse large B-cell lymphoma, one that primarily affects younger women. This approval marks just the second for MRK in the hematologic malignancies, and it adds further evidence for the use of immune checkpoint inhibitors in non-Hodgkin lymphoma.

Looking forward: Although it's a relatively small population of patients, this is an important step forward for MRK. All along, the immune checkpoint inhibitors have been primarily limited to solid tumors, with notable failure for the company in the realm of multiple myeloma. It has made me wonder...when do these agents start impacting on the hematologic malignancies? And it looks as though the gap may start to close. So this is very interesting news, indeed!

Nightstar hits the gas in gene therapy

Company: Nightstar Therapeutics (NITE)

Therapy: NSR-REP1

Disease: Choroideremia

News: NITE announced that the US FDA has granted its gene therapy NSR-REP1 Regenerative Medicine Advanced Therapy designation for the treatment of choroideremia, an inherited disorder that leads to night vision loss and eventual blindness. This designation grants similar advantages to both Fast Track and Breakthrough Therapy designations, qualifying the therapy for potential accelerated approval and the advantage of rolling NDA submission, in addition to increased attention from the FDA when dealing with inquiries.

Looking forward: This designation could lead to a serious drop in the time it takes to get this therapy through clinical trials. In addition to Breakthrough Therapy designation benefits, NITE can now convene with the FDA to lay out a specific study that would yield results sufficient for accelerated approval. Considering it's in phase 3 study, it has sufficient cash to get through whatever the FDA should require and get its drug to market at this pace. That kind of strength makes me want to give it a deeper dive.

Novartis outlines a possible new direction for its blockbuster IL-1beta drug

Company: Novartis (NVS)

Therapy: Canakinumab

Disease: Gout

News: Researchers presented findings at the Annual European Congress of Rheumatology demonstrating impressive reduction in gout flareups with NVS's anti-interleukin 1-beta antibody canakinumab. Compared with placebo, canakinumab treatment reduced the risk of flare-ups in a pooled analysis of patients by 52%.

Looking forward: This adds to a growing collection of surprising results for canakinumab, which is currently approved for a number of rheumatologic conditions. More recently, NVS has been showing the ability to protect against other adverse outcomes, including heart failure and, now, gout. So it would seem as though we're seeing the beginning of something very interesting: a drug that has the potential to impact numerous indications positively.

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