Daiichi Sankyo was on the receiving end of an FDA slapdown more than two years ago for FLT3 drug quizartinib and some flimsy data in acute myeloid leukemia. Looking to salvage a bad situation, Daiichi is now touting results in earlier AML patients with hopes for a better outcome with regulators.
Quizartinib added to a chemo regimen and then continued solo significantly extended the lives of patients with newly diagnosed acute myeloid leukemia with the FLT3-ITD mutation over chemo alone, according to topline results from the Phase III QuANTUM-FIRST study released Friday.
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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.
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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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.
Last week, Bristol Myers Squibb marched out long-term data for its heart drug mavacamten ahead of what execs had hoped would be a positive FDA decision in January. But regulators are saying they need a bit more time to think.
The FDA has extended mavacamten’s PDUFA date three months, from Jan. 28 to April 28, Bristol Myers announced on Friday. The news came just a few days after independent drug pricing watchdog ICER raised concerns about the candidate’s long-term safety in its final evidence report.
Zheng Wei, Connect Biopharma CEO
A suite of drugmakers is looking to topple Dupixent in the eczema market — and on Thursday, China-based Connect Biopharma uncorked some mid-stage results it says will pave the way to a pivotal trial. But investors want hard numbers, and without any to share, the company’s stock $CNTB plunged more than 50% on Friday.
Connect’s monoclonal antibody CBP-201 met the primary endpoint in a Phase II trial, showing significant improvements in Eczema Area and Severity Index (EASI) scores in all three dose groups, according to the biotech. The treatment arms — 300 mg every two weeks, 150 mg every two weeks, or 300 mg every four weeks — were all statistically superior to placebo at Week 16, and all showed significant improvements in the proportion of patients achieving at least a 50% or 75% reduction in EASI score (EASI-50 or EASI-75, respectively).
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.
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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https://endpts.com/daiichi-sankyo-looks-to-bring-flt3-drug-back-from-the-dead-with-late-stage-win-in-early-aml-patients/
