Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.
This week, we highlight patient voices in our coverage — first in Jared Whitlock’s feature story following a family at the forefront of a nascent personalized medicine initiative, and also in Lei Lei Wu’s piece about a new use of KRAS drugs.
A rare disease first
For 14 years of his life, teenager Connor Dalby faced constant seizures due to an ultra-rare genetic mutation. Last March, he became one of the first patients to be treated by the n-Lorem Foundation, a nonprofit set up to develop medicines for diseases affecting 30 or fewer patients. Endpoints’ Jared Whitlock followed Connor’s family on this journey for more than three years. Here’s their story — and the broader story of personalized medicine.
Lilly’s Alzheimer’s push
Facing a tepid Alzheimer’s market, Eli Lilly is looking to build “a sense of urgency” to jumpstart sales of its newly approved drug donanemab, or Kisunla. It’s up against a medical system that is in many ways unprepared for the new Alzheimer’s therapies, despite decades of efforts by companies to bring them out of the lab and to patients.
Argenx’s next act
After a handful of stumbles, argenx is doubling down and making the case to investors that its pipeline-in-a-product model is just getting started. In addition to unveiling new data for its sole commercial product, Vyvgart, the biotech also discussed hopes to replicate the strategy with new drugs. In an interview with Ryan Cross, its CEO also spoke about how the IRA is influencing argenx’s strategy and the company’s push to find its next blockbuster medicine.
What Novartis won’t do in obesity
Vas Narasimhan made clear that Novartis doesn’t want to play the me-too game in obesity. Trying to compete in the multibillion-dollar obesity market with an injectable or oral GLP-1 or GIP drug, he said on an earnings call, “is not a prudent approach for us as a company.” His comments come as Novartis settles into a new normal with a simplified structure, even as it continues to fine-tune.
SPOTLIGHT
Lilly upped its offer for Morphic by $11 per share, documents show
R&D
- Revolution Medicines’ experimental drug staved off cancer progression for seven and a half months in an early-stage trial with second-line metastatic pancreatic cancer patients, marking a potential breakthrough in tackling cancers driven by RAS mutations.
- Roche showcased data from not one, but two drugs from its acquisition of Carmot Therapeutics. The first update came in the form of a conference abstract, showing that in a Phase 1b study, its GLP-1/GIP agonist had not plateaued in weight reduction at the 24-week mark. The second was a topline announcement of Phase 1 results around its oral GLP-1 receptor agonist, which it said could help with weight loss maintenance after using injectable GLP-1s.
- FDA thwarts Agenus’ accelerated approval plans for CTLA-4/PD-1 combo in ‘disappointing’ move
- Roivant recently sold one spinout for billions. Is Priovant next?
- Roche touts Phase 3 Susvimo data in diabetes-related ophthalmic diseases
- Aveo Oncology’s Fotivda combo fails in Phase 3 kidney cancer test
PHARMA
- Johnson & Johnson has the final price offers under the IRA in hand. And while it won’t disclose the offers just yet, it said during an earnings call that those prices won’t affect the long-term sales growth projection it gave last year.
- EMA warns patients to tell doctors about GLP-1 usage before surgery
- China approves Eli Lilly’s weight loss drug as country launches obesity awareness campaign
- GSK HQ officially returns to central London after decades in city’s suburbs
- European Commission president’s second-term pledge: simpler regulations, better funding and tackling shortages
- GoodRx launches discount for Boehringer Ingelheim’s Humira biosimilar
- US inks $113M deal with Siga to bolster smallpox, mpox reserves
- Vermont attorney general sues two major PBMs for allegedly driving up costs
- Big three PBMs will testify before Congress next week, following FTC report
FINANCING
- Artiva completed its second attempt at IPO with a $167 million raise. While the clinical-stage cell therapy developer priced below the range spelled out earlier in the week, it upsized the number of shares offered for its Nasdaq debut.
- Biotech industry flirts with XBI milestone as it surpasses $100
- Alzheimer’s biotech Asceneuron raises $100M to see if it can compete with Lilly, Biogen on tau
- Scorpion raises $150M with sights on the next wave of targeted therapies
- Three months after delisting, NGM Bio is back with $122M for slimmed-down pipeline
- Bain-backed Cardurion raises $260M to fund ex-Imara drug, buy more cardio assets
- German biotech CatalYm raises $150M to repurpose obesity target for cancer
- AI drug development company gets $80M backing from Pfizer, Nvidia and Thermo Fisher
- George Church spinout raises $60M for protein therapies from genomically recoded organisms
- Parker Institute for Cancer Immunotherapy invests another $125M into research
- OrbiMed forms biotech with assets from China-based Keymed, enlists former Biohaven exec as CEO
- Renalys raises $37.8M to take Travere’s kidney disease drug into Phase 3 trial in Japan
DEALS
- Roche’s Genentech became the latest pharmaceutical company to renege on an SHP2 partnership in oncology, scrapping a collaboration and license agreement with Relay Therapeutics surrounding the biotech’s clinical-stage oral SHP2 inhibitor.
- Sionna licenses clinical-stage cystic fibrosis assets from AbbVie shortly after large Series C
- After last year’s ImmunoGen pact, Vertex finds another conditioning agent partner in Orum
- J&J ditches epilepsy development with Addex, blindsiding 20-year partner
- ‘Search mode’: Human challenge trial company looks to buy small CROs
PEOPLE
- Merdad Parsey, Gilead’s chief medical officer, will leave the company at a time when its pipeline has stalled and it’s faced several major stumbles in cancer drug development.
- Cassava founder and CEO resigns as federal probes progress
- Nadir Mahmood returns to Nkarta a year after leaving; BeiGene picks longtime Merck exec for CFO post
CELL/GENE TX
- Caribou Biosciences is cutting its CAR-NK research and laying off 21 employees, or 12% of its staff. The move will extend its cash runway by around six months.
- Updated: Lexeo Therapeutics’ gene therapy shows potential for Friedreich’s ataxia cardiomyopathy with early data
- 4DMT builds out case for wet AMD gene therapy with new Phase 2 data
- Adverum prepares pivotal study for wet AMD gene therapy, touts Phase 2 data
LAW
- Vertex sued the federal government in an effort to expand its fertility support program to certain patients taking its new gene therapy, Casgevy, for sickle cell disease and transfusion-dependent beta thalassemia.
- Merck asks DC federal court for IRA decision ‘as soon as practicable’
- AstraZeneca criticizes CMS guidance in appeal over price negotiations
- J&J calls proposed class action talc suit ‘meritless’ as settlement vote deadline nears
- Ardelyx sues Medicare over attempt to change coverage for dialysis drug
- Large group of state attorneys general backs Oregon drug price transparency law
- Federal judge dismisses Hawaii’s lawsuit against top three PBMs
FDA+
- The FDA will launch a rare disease center to unify its policies and find new ways to evaluate drugs that can pose a regulatory dilemma. The new hub looks to connect various parts of the agency and will be led by CBER Director Peter Marks and CDER Director Patrizia Cavazzoni.
- FDA warns Chinese drugmaker for serious GMP record violations
- Phathom wins label expansion on heartburn drug to include non-erosive patients
- FDA again rejects Orexo’s nasal spray for opioid overdose rescue
- EMA reasserts differences with FDA for Covid vaccine strain selection
MANUFACTURING
- In its biggest investment so far, Swiss CDMO CordenPharma is earmarking €900 million ($982 million) over the next three years to expand its peptide platform capacity in the US and Europe to meet rising GLP-1 manufacturing demand.
- Sanofi seeks deeper roots in India with €400M hub expansion
- Investment firm launches with $100M for bioprocessing entrepreneurs
HEALTH TECH
- Is anyone in retail health going to be successful? After Walgreens’ retreat from primary care, Endpoints’ Shelby Livingston caught up with Brian Tanquilut, equity analyst at Jefferies, about the state of the market and why retail health seems to be failing across the board.
- Exclusive: TMRW Life Sciences raises $28M Series D to upgrade storage for egg freezing
- Thyme Care scores $480M valuation in new funding round
- Exclusive: Kins raises $7M to expand in-home and virtual physical therapy