Bionest

A Look at Duchenne Muscular Dystrophy: Part 1 – Marathon Pharmaceuticals, PTC Therapeutics, and Drug Pricing

  In February of this year, Marathon Pharmaceuticals received approval for Emflaza (deflazacort) tablets and oral suspension for the treatment of patients over the age of five years with Duchenne muscular dystrophy (DMD), regardless of the particular genetic mutation associated with their condition. The drug — an older corticosteroid with no patent protection which has...

Bionest

Reflecting on AACR

The American Association for Cancer Research annual meeting is always a source of major news and discussion on the oncology research front, and the 2017 meeting held earlier this month was no exception.   One area of particular interest was the encouraging data on long-term survival that is emerging for some patients treated with immuno-oncology...

Bionest

Alzheimer’s Disease Part Three: Meeting 2025 Goals

  In early 2011, the National Alzheimer’s Project was formed — a strategic plan aimed at addressing the looming health care crisis posed by the disease and the aging population. Its aim: to coordinate research efforts across the U.S. federal government with the goal of preventing or effectively treating Alzheimer’s Disease by 2025. Since then,...

Bionest

Communicating Value

— Robert J. Easton, Co-Chairman, Bionest Partners   During the annual January gathering of the biopharma world in San Francisco this year, in-coming President Donald J. Trump put drug developers on notice and sent pharmaceutical and biotechnology stock prices plunging by accusing the industry of “getting away with murder” with respect to drug pricing.  ...

Bionest

FDA Grants Priority Review for KEYTRUDA® IN MSI-H Cancers

On November 28, the U.S. Food and Drug Administration granted Priority Review to Merck’s Supplemental Biologics License Application (sBLA) for KEYTRUDA® (pembrolizumab) in the new indication of microsatellite instability-high (MSI-H) cancer. MSI-H is a biomarker caused by a deficiency in a cell’s ability to repair errors in DNA sequences that occur during cell division. The...

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No Matter What You Think of the U.S. Election Outcome…

By Alain J. Gilbert, Co-Chair, Bionest Partners   The U.S. Presidential election is over. No matter how you personally feel about the outcome, the fact of Donald Trump’s election is likely to be positive for the healthcare industry and biopharma. The reason: the issue of new drug pricing is not going to be a top...

Bionest

Mapping the Brain: Dawn of a New Era for Neuroscience Research?

On July 20, a team of neuroscience researchers from Washington University in St. Louis, Missouri and other centers published a precise new brain map in Nature — an outcome of the White House BRAIN (Brain Research through Advancing Innovative Neurotechnologies) initiative, first announced in 2013. The new map draws in part on the brain’s internal...

Bionest

PRIME Launched by EMA to Speed Review and Approval of Innovative, “Breakthrough” Drugs

Four years ago, the US Food and Drug Administration launched its “breakthrough designation” program, designed to speed the review and potential approval of new therapeutics deemed to offer the possibility of substantial improvement over existing options for patients with life-threatening or serious diseases. Since then, the FDA has approved more than 30 drugs in this category, many...