ALETH / LIFESCI-BRIEF / 2026-05-01 / FIRST IN VIVO CRISPR SUCCESS IN PHASE 3The Aleth Briefs link to the sources behind the stories and show how the week unfolded.
The week in five lines:
Intellia’s in vivo CRISPR therapy cut angioedema attacks by 87% in phase 3.
Sun Pharma agreed to buy Organon at an $11.8bn enterprise value.
The FDA moved to withdraw Amgen’s Tavneos, citing manipulated trial results.
Health Canada authorised the G7’s first generic semaglutide, a copy of Ozempic.
AstraZeneca restarted its frozen £300m ($405m) UK investment programme.
Browse by day:
Weekend catch-up: Cenrifki · psychedelic vouchers
Monday: Intellia · Sun-Organon · Lilly-Ajax
Tuesday: Tavneos · real-time trials · survodutide · semaglutide · Purdue
Wednesday: AZ UK · Germany · KalVista · Emalex · Hikma v Amarin · Biohub
Thursday: camizestrant · Krazati · Auvelity · Summit · CBER
Weekend catch-up
The EMA’s medicines committee backed Sanofi’s Cenrifki (tolebrutinib) for EU approval in non-relapsing secondary progressive multiple sclerosis.
Sanofi said on Friday that the opinion rests on the phase 3 HERCULES trial, in which the BTK inhibitor delayed disability progression. Drug-induced liver injury is an identified risk. The European Commission decides in the coming months.
Cenrifki recommended for EU approval by the CHMP · Sanofi
The FDA gave national priority vouchers to three psychedelic drug programmes, including London-based Compass Pathways’ psilocybin treatment COMP360.
The vouchers cover psilocybin in treatment-resistant depression, psilocybin in major depressive disorder and methylone in post-traumatic stress disorder (PTSD). Fierce reported that the Usona Institute and Transcend Therapeutics confirmed the other two. Compass said the FDA also granted it a rolling New Drug Application (NDA) submission. The voucher brings a 1-2 month review after filing. The FDA also cleared the first US study of an ibogaine derivative.
FDA accelerates action on treatments for serious mental illness · FDA
Compass awarded Commissioner’s National Priority Voucher · Compass Pathways
Compass, Usona and Transcend score FDA national priority vouchers · Fierce Biotech
Monday 27 April
Intellia’s CRISPR therapy lonvo-z cut hereditary angioedema attacks by 87% in phase 3.
In the 80-patient trial, a single dose cut attacks by 87% against placebo over six months, and 62% of patients were free of attacks and other treatment, against 11% on placebo. Intellia has started a rolling submission to the FDA.
Lonvo-z edits DNA inside the body. If approved, it would be the second CRISPR medicine after Vertex’s Casgevy and the first to edit genes in vivo.
Intellia reports positive phase 3 results in hereditary angioedema · Intellia
Sun Pharma agreed to buy Organon at an enterprise value of $11.8bn.
Sun pays $14.00 a share in cash. Organon, spun out of Merck in 2021, had $6.2bn of revenue and $8.6bn of debt in 2025. Sun says the deal makes it a top-three company in global women’s health and a top-10 biosimilar player. It should close in early 2027.
Sun Pharma signs definitive agreement to acquire Organon · Organon
Eli Lilly agreed to buy Ajax Therapeutics for up to $2.3bn in cash.
Ajax’s AJ1-11095 is an oral Type II JAK2 inhibitor in phase 1 for myelofibrosis that has been treated with a Type I JAK2 inhibitor. Lilly was a founding investor and expects proof-of-concept data later in 2026.
Lilly to acquire Ajax Therapeutics · Eli Lilly
Tuesday 28 April
The FDA proposed withdrawing Amgen’s Tavneos, saying trial results were manipulated.
The Center for Drug Evaluation and Research (CDER) said new information shows unblinded staff manipulated the pivotal trial results to make Tavneos (avacopan) look effective. It said Amgen’s ChemoCentryx unit did not disclose the original analysis. CDER also cited fatal drug-induced liver injury as a new safety concern. Tavneos stays on sale unless Amgen withdraws it or the FDA Commissioner orders removal.
CDER proposes to withdraw approval of Tavneos · FDA
Proposal to withdraw Tavneos NDA approval, with a hearing offer · Federal Register
The FDA unveiled two trials, run by AstraZeneca and Amgen, that will report data to the agency in real time, and asked for input on a wider pilot.
The agency has received and validated signals from AstraZeneca’s phase 2 TRAVERSE trial in mantle cell lymphoma. Amgen’s phase 1b STREAM-SCLC trial in small cell lung cancer is still selecting sites. The pilot is due to launch this summer.
FDA announces major steps to implement real-time clinical trials · FDA
Boehringer Ingelheim’s glucagon/GLP-1 drug survodutide cut weight by up to 16.6% at 76 weeks in phase 3, against 3.2% on placebo.
The trial, in adults with obesity or overweight but not type 2 diabetes, met both co-primary endpoints. Up to 85.1% of patients lost at least 5% of body weight. Both results use an efficacy analysis that assumes patients stayed on treatment for the whole study. Full data go to the American Diabetes Association meeting in June.
Results from phase III SYNCHRONIZE-1 obesity trial · Boehringer Ingelheim
Health Canada authorised Dr. Reddy’s generic Ozempic, the first generic semaglutide in the G7.
It is approved for type 2 diabetes. Health Canada is reviewing eight more generic semaglutide submissions.
Canada first G7 country to approve a generic semaglutide · Health Canada
A federal court in Newark sentenced Purdue Pharma to pay >$5bn in criminal penalties for its role in the opioid epidemic.
The penalties are a $3.5bn fine and $2bn in forfeiture. The Justice Department will credit up to $1.7bn of the forfeiture if Purdue emerges from bankruptcy as a public benefit company. Purdue pleaded guilty in 2020.
Purdue sentenced for fraud and kickback conspiracies · US Department of Justice
Wednesday 29 April
AstraZeneca restarted its frozen £300m UK investment programme.
AstraZeneca will resume a £300m ($405m) UK investment seven months after freezing it over drug-pricing talks, Fierce reported. It covers a £200m ($270m) Rosalind Franklin building in Cambridge and a “lab of the future” in Macclesfield, which the local combined authority puts at £100m ($135m).
Merck & Co scrapped plans for a £1bn ($1.35bn) London R&D centre during the same dispute. It told Fierce it has “no updates to our previously announced plans”.
AZ restarts £300M UK investment after govt boosts med spending · Fierce Pharma
AZ’s £100m Macclesfield investment · Cheshire and Warrington Combined Authority
Germany’s cabinet approved a draft bill that would add a variable component to the 7% rebate drugmakers pay statutory insurers.
The change is aimed mainly at high-priced patented drugs. The bill would also let insurers pick preferred drugs where several comparable patented ones compete.
Kabinett beschließt GKV-Beitragssatzstabilisierungsgesetz · German Health Ministry
Chiesi agreed to buy KalVista Pharmaceuticals for $27.00 a share in cash, an equity value of about $1.9bn.
The deal brings Chiesi Ekterly (sebetralstat), the first oral on-demand treatment for hereditary angioedema. The deal should close in Q3 2026.
Chiesi to acquire KalVista Pharmaceuticals · Chiesi
Teva agreed to buy Emalex for $700m, plus up to $200m in milestones and royalties, to get the paediatric Tourette syndrome drug ecopipam.
Ecopipam is ready for filing and blocks dopamine D1 receptors, while current Tourette drugs act mainly on D2. The deal should close by Q3 2026.
Teva to acquire Emalex Biosciences · Teva
The US Supreme Court heard Hikma v Amarin, on whether a generic sold with a skinny label can infringe patents if its maker encourages patented uses.
Amarin says Hikma’s promotion of its generic of Amarin’s fish-oil heart drug went beyond the label’s unpatented uses. PhRMA and the Biotechnology Innovation Organization filed a brief backing Amarin.
Supreme Court takes up generic drug labeling fight · Axios
Hikma v Amarin oral argument transcript · Supreme Court
Biohub committed $500m over five years to a Virtual Biology Initiative to build the data for AI models that predict how human cells behave.
$100m goes to a coordinated worldwide data-generation effort and $400m to Biohub’s own data and measurement technologies. Nvidia is a technology partner.
Virtual Biology Initiative to create open data foundation for AI-biology · Biohub
Thursday 30 April
FDA advisers voted 6 to 3 against AstraZeneca’s camizestrant switch strategy.
The Oncologic Drugs Advisory Committee (ODAC) did not back first-line camizestrant with a CDK4/6 inhibitor in HR-positive, HER2-negative advanced breast cancer once an ESR1 mutation emerges. In the SERENA-6 trial, switching to camizestrant cut the risk of progression or death by 56% against staying on an aromatase inhibitor.
ODAC voted 7 to 1 for AstraZeneca’s Truqap (capivasertib) with Zytiga in PTEN-deficient metastatic hormone-sensitive prostate cancer. Neither vote binds the FDA.
Update on FDA advisory committee vote on camizestrant · AstraZeneca
Truqap recommended by FDA advisory committee · AstraZeneca
Bristol Myers Squibb’s Krazati (adagrasib) failed its confirmatory trial in second-line colorectal cancer, a company spokesperson told Endpoints News.
Endpoints reported that the result puts Krazati’s colorectal cancer approval at risk.
Krazati fails confirmatory trial in colorectal cancer · Endpoints News
The FDA approved Axsome Therapeutics’ Auvelity (dextromethorphan-bupropion) for agitation associated with Alzheimer’s dementia.
Auvelity acts on NMDA and sigma-1 receptors. Alzheimer’s agitation is its second approved neuropsychiatric indication.
Axsome announces FDA approval of Auvelity in Alzheimer’s agitation · Axsome
Summit said the squamous cohort of its phase 3 HARMONi-3 lung cancer trial of ivonescimab will continue after an added interim analysis.
The independent data monitoring committee reviewed the interim and recommended no change. Summit said significance at this look needed a meaningfully higher bar than the final progression-free survival analysis, still due in H2 2026. It did not say whether the bar was met.
Summit Therapeutics reports Q1 2026 results and operational progress · Summit Therapeutics
The FDA named Katherine Szarama acting head of CBER, the centre that regulates vaccines, gene therapies and the blood supply.
Szarama was deputy to Vinay Prasad, who left the agency on Thursday.
FDA names Katherine Szarama acting director of CBER · STAT
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