Prescribed Reading: CRISPR, Trump and FOURIER. Oh my!

A weekly guide to the goings-on in the biopharma industry.

Biopharma is a complex, rapidly evolving industry that is highly regulated and closely watched — and that means there is constant news. Here's a closer look at the clinical trials, M&A, cool science and regulations that are driving the industry this week.

In case you missed it

Mergers & analysis

CRISPR technology has been the talk of the town for the last couple years, from the promise of its gene editing technology to the epic patent battle that has been ongoing between two of the most prestigious scientific institutions.

Allergan is giving people one more reason to keep a close eye on CRISPR. The specialty pharma has teamed up with Editas Medicine to develop its eye-disease candidates, including the Cambridge biotech's lead program in Leber Congenital Amaurosis (LCA10). Allergan is paying $90 million upfront for the license, further adding to its eye care portfolio that its been building out over the last year.

Another deal had tongues wagging this week, but mostly for its idiocy (or is it genius?): Beleaguered PTC Therapeutics, which has been battling with the Food and Drug Administration to get its Duchenne muscular dystrophy (DMD) drug Translarna (ataluren) approved, decided to pick up another questionable DMD drug.

PTC is paying Marathon Pharmaceuticals $140 million for the steroid that it just got FDA-approved for DMD. Marathon is happy to wave goodbye to the drug, which has gotten the company in hot water with just about everyone from Congress to the public to industry groups.

Marathon got the old steroid approved and decided it was going to charge $89,000 for a course of treatment. This very poor decision now has industry group PhRMA questioning the company’s membership in the group and has Senators calling for the company to answer for its bad behavior.

Now it’s PTC’s problem.

Clinically relevant

AstraZeneca shored up its position in the closely watched market of PARP inhibitors this week. Its drug Lynparza (olaparib) was the first of this class to be approved and has shown strong results so far, but recent data in an ovarian cancer trial shows that the drug could be best-in-class as well.

Two biotechs – Clovis Oncology and Tesaro – have been battling with the British pharma to show that their respective PARP inhibitors are top of the heap. Tesaro, which has not yet gotten its PARP inhibitor approved, took a hit to its stock on the news.

In another closely watched space, Novartis got the greenlight for its CDK 4/6 inhibitor Kisqali (ribociclib), which will set it up to battle Pfizer’s Ibrance (palbociclib). Ibrance got a strong lead ahead of CDK 4/6 inhibitors from Novartis and Eli Lilly, and recent data has further bolstered its position in the breast cancer market.

Novartis is looking to head off Ibrance and is placing a lot of hope into Kisqali as it tried to build out its oncology portfolio.

Meanwhile, Amgen finally released the results from its long-awaited cardiovascular outcomes trial FOURIER. While the results were largely positive – showing the PCSK9 inhibitor Repatha (evolocumab) had a cardiovascular benefit – the magnitude of effect was not as robust as some had hoped and Repatha failed to prevent cardiovascular death. This likely means that the struggling cholesterol drug will continue to be just that – struggling.

Highly regulated

President Trump and his potential deregulation of the industry took another turn this week when his administration released its proposal for the 2018 discretionary budget.

Aside from all of the deregulating the President has promised to do at the FDA – something the industry has pushed back on – the budget, if passed in its current form, could derail early research.

The proposed plan cuts funding to the Department of Health and Human Services by $5.8 billion, or about 18%. This includes cuts to the National Institutes of Health, which handle early research, award grants for research at academia, and fund initiatives to work with foreign scientific institutions. The budget also proposed increasing the fees industry pays to have their drug’s reviewed – by more than $1 billion.

The Bill and Melinda Gates Foundation responded negatively to the proposal. “We are deeply troubled that the budget proposal disproportionately affects the poorest people, abroad and at home. In just 25 years, American investments overseas have helped prevent the deaths of 122 million children, cut extreme poverty in half, and put the world on the verge of eradicating polio. . .The proposed cuts will ultimately make America, and the world, less prosperous and less safe,” said Sue Desmond-Hellmann, CEO of the Foundation.

Bill Gates is set to meet with the President on Monday. This should be interesting. Stay tuned. 

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Filed Under: Clinical Trials Mergers and Acquisitions / Deals Regulatory / Compliance