Friday Five – the pharma week in review

The US government takes a preliminary step towards allowing Medicare to negotiate prices for Part B drugs, Regeneron looks to expand its footprint in oncology; it may be a case of too much of a good thing with priority review vouchers; the busy week that was for the FDA; and the steady news flow continues from the world of gene therapy, some announcements more encouraging than others…

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The US Centers for Medicare and Medicaid Services (CMS) finally revealed a much-anticipated rule change on August 7 that removes certain restrictions on Medicare Advantage plans, allowing them to hold a preference for some drugs over others, bringing the programmes one step closer to being able to engage in unrestrained price negotiations.

Among the companies hit hard by the new rule was Regeneron Pharmaceuticals, which earns a fair chunk of change from Part B sales of flagship eye drug Eylea (aflibercept), along with Amgen and Roche, which also have a sizable slice of revenues that could be affected. On the flipside, analysts expect that the change could be good news for the makers of biosimilars, which could see a resulting boost in adoption. More here

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Speaking of Regeneron, the long-time mAb specialist may be counting down the days until FDA gives it the green light to begin marketing anti-PD-1 mAb cemiplimab, but in the meantime the company delved deeper into the cell therapy space by hitching its wagon to one of the industry leaders in bluebird bio.

Regeneron will make a $100-million equity investment in bluebird, and the partners will co-develop cell immunotherapies, including fully humanised antibodies and TCR candidates, against six targets that are all currently in preclinical testing. More here

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Ultragenyx quietly announced the sale of a priority review voucher (PRV) that was co-owned with partner Kyowa Hakko Kirin to an undisclosed buyer, though the agreed price of $80 million – a far cry from the $350 million paid by AbbVie for one a couple years ago – suggests that regulators or better yet politicians may need to step in and tweak the programme before all of its strength is sapped away, according to an academic who first envisioned the use of vouchers to stimulate drug development for neglected indications.

The PRV was obtained earlier this year in parallel with the US approval of Crysvita (burosumab) to treat X-linked hypophosphatemia.  More here

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It turned out to a relatively busy week for the FDA, which gave some thumbs up and down while also hosting multiple AdCom meetings.

Kyowa Hakko Kirin scored its second approval of the year as Poteligeo (mogamulizumab) was given the green light to treat two rare forms of non-Hodgkin’s lymphoma: mycosis fungoides and Sézary syndrome. The news comes several months after Crysvita was allowed on the market (see above).

In meetings on back-to-back days this week, the agency’s Antimicrobial Drugs Advisory Committee recommended in favour of approving Insmed’s ALIS (amikacin liposome inhalation suspension) to treat nontuberculous mycobacterial lung disease and Paratek Pharmaceuticalsantibiotic omadacycline to treat both acute bacterial skin and skin structure infections and community-acquired bacterial pneumonia.

However, not every company was popping bottles of champagne this week. Pain Therapeutics received a fourth rejection letter from the FDA for Remoxy, an abuse-deterrent formulation of oxycodone. What’s more, the agency also caused shares of Ampio Pharmaceuticals to crater almost 70 percent on August 8 after determining that the company would need to obtain more data for osteoarthritis pain candidate Ampion before submitting a marketing application.

On the less innovative front, the FDA made use for the first time of a new pathway for generic drugs in cases where no competition exists, as the agency gave Apotex the go ahead to begin marketing an oral solution of potassium chloride for patients with low potassium levels. The new approval route was established by legislation passed last year and is meant to speed alternatives to market to help increase competition and thereby reduce prices.

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It was not exactly a banner week for gene therapy players as several clinical setbacks were reported, though it was certainly not all doom and gloom.

Spark Therapeutics was punished by investors after revealing on August 7 that two patients with haemophilia A receiving the highest dose of SPK-8011 in a Phase I/II trial experienced immune reactions causing their Factor VIII expression levels to fall well below the threshold needed to prevent spontaneous bleeds. This was good news for BioMarin Pharmaceuticals, though, as it boosts the competitive profile of its valoctogene roxaparvovec (formerly known as BMN 270), which is in Phase III testing. More here

The following day Sangamo Therapeutics released preliminary top-line findings from a Phase I/II trial of its SB-525, showing that the first patient with haemophilia A in the third dose group had achieved therapeutic Factor VIII levels, while an absence of safety concerns pleased the Street. More here

In addition, updated results from a pair of Phase I/II trials of gene therapies from Regenxbio appeared to be a bit of a mixed, as RGX-501 had not meaningfully lowered cholesterol in patients with homozygous familial hypercholesterolaemia, but RGX-314 for wet age-related macular degeneration showed a dose-dependent increase in protein expression with a clean safety profile.

On a more positive note, Boehringer Ingelheim launched a collaborative effort to develop a nebulised gene therapy for cystic fibrosis that will involve a handful of industry, non-profit and academic partners. More here

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