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For years, paper-based processes and individual point solutions dominated the clinical research landscape, and patient participation in clinical trials was largely an in-person engagement. But when the COVID-19 pandemic took a stronghold, traditional clinical trial methods emerged as inadequate, putting clinical trials and the life sciences industry at a crossroads. Practically overnight, the industry had to rapidly shift to decentralized clinical trial methods, while maintaining data quality and regulatory compliance.
In a surprise setback, Merck has slammed the brakes on the development of an experimental HIV drug — including a Phase II trial — after investigators flagged a drop in immune cell counts that an external committee determined was related to treatment.
The Phase II study that first sounded the alarm, dubbed IMAGINE-DR, was testing the once-weekly combination of MK-8507 (a non-nucleoside reverse transcriptase inhibitor) and islatravir, or ISL, a nucleoside reverse transcriptase translocation inhibitor.
Jean-Jacques Bienaimé (BioMarin via Youtube)
The FDA on Friday signed off on an accelerated approval for BioMarin’s Voxzogo (vosoritide) injection, the first treatment to target the underlying genetics of dwarfism, which can increase the height of children five years of age and older with the condition.
The injection, first approved in Europe with a $300,000 annual price tag, works by binding to a specific receptor called natriuretic peptide receptor-B, which reduces the growth regulation gene’s activity and stimulates bone growth in children.
The European Medicines Agency on Friday said that Merck’s antiviral molnupiravir can now be used to treat adults with Covid-19 who do not require supplemental oxygen and who are at increased risk of developing severe disease.
The pill, known commercially in Europe as Lagevrio, has not yet been authorized in the US and has to be taken twice a day for 5 days. The EMA said it should be administered as soon as possible after diagnosis of Covid, and within 5 days of the start of symptoms. The quick authorization is based on data showing the drug reduced the chance that a newly diagnosed Covid-19 patient would be hospitalized or die by 50%, according to data presented by the company in October.
Peter Marks (Jim Lo Scalzo/Pool via AP Images)
All US adults who received two doses of an mRNA Covid-19 vaccine are now eligible for a booster shot, the FDA announced Friday.
The moves will amend the existing EUAs for the Pfizer/BioNTech and Moderna boosters, which had previously been limited to immunocompromised individuals, those older than 65 and adults at high risk for severe disease through certain comorbidities or occupational exposure. For Moderna in particular, the booster shots come in a half-dose of the original vaccine series — 50 µg instead of 100 µg — while Pfizer’s remains at 30 µg for each shot.
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Douglas Fambrough, Dicerna CEO (Dicerna via YouTube)
Early this year researchers at Novo Nordisk were beaming as they announced the first drug identified in their RNAi alliance with Dicerna was headed into the clinic. And now they’re coming back for the whole thing.
This morning the Copenhagen-based pharma giant put out word that it is buying Dicerna $DRNA — an RNAi pioneer that has had its up and downs over the years — for $3.3 billion. Novo is paying $38.25 a share — an 80% premium.
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Gilead is going all in — hook, line and sinker — on its oncology alliance with Arcus. And they are going for broke.
The big biotech unveiled a deal that now delivers $725 million in opt-in payments covering the clinical development programs for Arcus, ranging from their closely watched anti-TIGIT programs for domvanalimab and AB308 to etrumadenant (the A2a/A2b adenosine receptor antagonist) and quemliclustat, the small molecule CD73 inhibitor. Gilead will also cover half of the development costs, handing Terry Rosen’s biotech a deal that gives them a clear cash runway to achieving all its goals in oncology.
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Mathai Mammen, head of R&D for J&J’s Janssen unit (Rob Tannenbaum)
Last week, J&J took a step familiar to other pharma conglomerates in spinning out its consumer business to focus on R&D, but offered few details on what that might look like. But on Thursday, the company followed up with the scoop, and it’s making some bold predictions.
Over the course of a two-plus hour presentation on its pharmaceutical business, execs outlined their strategy for the new, slimmer J&J, promising investors it will file about 14 drugs for approval through 2025. Across all these drugs, J&J said it expects $4 billion average peak annual sales, and five could top the $5 billion mark.
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Jay Luly, Enanta CEO (via YouTube)
A Massachusetts biotech will discontinue the development of its oral drug intended to treat patients suffering from chronic hepatitis B infections, the company said Thursday.
Enanta Pharmaceuticals will no longer develop EDP-721. The news comes after safety signals were seen in healthy participants in a Phase I trial after they were administered the drug, and despite a clean safety profile demonstrated in preclinical trials.
https://endpts.com/biogens-no-good-week-inside-gene-editings-open-secret-novo-nordisk-absorbs-rnai-partner-boosters-for-all-and-more/
