R&D channel feed

Pascal Soriot, AstraZeneca CEO (via Getty images)

UP­DAT­ED: FDA slaps As­traZeneca's MCL-1 can­cer drug with a hold af­ter safe­ty is­sue — 2 years af­ter Am­gen axed a trou­bled ri­val

There are new questions being posed about a class of cancer drugs in the wake of the second FDA-enforced clinical hold in the field.

Two years after the FDA hit Amgen with a clinical hold on its MCL-1 inhibitor AMG 397 following signs of cardiac toxicity, AstraZeneca says that regulators hit them with a hold on their rival therapy of the same class.

The pharma giant noted on clinicaltrials.gov that its Phase I/II study for the MCL-1 drug AZD5991 “has been put on hold to allow further evaluation of safety related information.”

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Kelly Martin, Radius Health CEO

Ra­dius rock­ets high­er as Kel­ly Mar­tin boasts of a big PhI­II suc­cess — but the spoils be­long to Menar­i­ni

Radius Health stuck with some fuzzy top-line Phase III results for its oral SERD therapy elacestrant, but investors saw enough to push a rally that sparked a big surge in its share price.

According to researchers, the drug hit both primary endpoints among ER+/HER2- breast cancer patients: beating standard of care on progression-free survival in the overall population and PFS with tumors harboring estrogen receptor 1 mutations — a key factor in developing resistance. And while some analysts were left wondering about specific data, most of the crowd seemed happy to hear that the drug is now being steered to the FDA.

Sur­geons suc­cess­ful­ly at­tach pig kid­ney to a hu­man for the first time, us­ing tech from Unit­ed's Re­vivi­cor

In a first, researchers reportedly successfully transplanted a pig kidney into a human without triggering an immediate immune response this week. And the technology came from the biotech United Therapeutics.

Surgeons spent three days attaching the kidney to the patient’s blood vessels, but when all was said and done, the kidney appeared to be functioning normally in early testing, Reuters and the New York Times were among those to report. The kidney came from a genetically altered pig developed through United’s Revivicor unit.

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No­vo CEO Lars Fruer­gaard Jør­gensen on R&D risk, the deal strat­e­gy and tar­gets for gen­der di­ver­si­ty

 

I kicked off our European R&D summit last week with a conversation involving Novo Nordisk CEO Lars Fruergaard Jørgensen. Novo is aiming to launch a new era of obesity management with a new approval for semaglutide. And Jørgensen had a lot to say about what comes next in R&D, how they manage risk and gender diversity targets at the trendsetting European pharma giant.

John Carroll: I’m here with Lars Jørgensen, the CEO of Novo Nordisk. Lars, it’s been a really interesting year so far with Novo Nordisk, right? You’ve projected a new era of growing sales. You’ve been able to expand on the GLP-1 franchise that was already well established in diabetes now going into obesity. And I think a tremendous number of people are really interested in how that’s working out. You have forecast a growing amount of sales. We don’t know specifically how that might play out. I know a lot of the analysts have different ideas, how those numbers might play out, but that we are in fact embarking on a new era for Novo Nordisk in terms of what the company’s capable of doing and what it’s able to do and what it wants to do. And I wanted to start off by asking you about obesity in particular. Semaglutide has been approved in the United States for obesity. It’s an area of R&D that’s been very troubled for decades. There have been weight loss drugs that have come along. They’ve attracted a lot of attention, but they haven’t actually ever gained traction in the market. My first question is what’s different this time about obesity? What is different about this drug and why do you expect it to work now whereas previous drugs haven’t?

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Manos Perros, Entasis CEO

As­traZeneca's for­mer an­tibi­otics unit claims a PhI­II win in drug-re­sis­tant bac­te­ria af­fect­ing sick­ly pa­tients

The management team at Entasis, AstraZeneca’s old antibiotics unit spun out six years ago, has a new dataset it’s simply delighted to share.

Entasis revealed Phase III data outlining how an antibiotic for infections caused by Acinetobacter baumannii reached its primary endpoint of non-inferiority. Researchers compared the program — known as sulbactam-durlobactam or SUL-DUR — to the antibiotic colistin, saying their drug saw lower levels of all-cause mortality after four weeks in treatment-resistant patients.

UP­DAT­ED: Galera shares take a nose­dive in the wake of un­ex­pect­ed PhI­II flop

Galera was sure it had a winner when it launched its pivotal severe oral mucositis (SOM) trial back in 2018 — and so were investors. Fresh off a $150 million raise, the company plunged headfirst into Phase III, even making commercial plans.

But those plans went splat Tuesday, as an unexpected failure sent shares spiraling into penny stock territory.

Galera’s lead candidate avasopasem (also known as GC4419) failed to significantly reduce the incidence of SOM in patients with locally advanced head and neck cancer compared to placebo, the company announced. While those in the treatment group saw a slight reduction, 54% compared to 64% in the placebo group, the endpoint failed to achieve statistical significance (p=0.113).

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Paul Bolno, Wave Life Sciences CEO

Take­da, Wave Life Sci­ences re­vise their $230M R&D col­lab­o­ra­tion, drop­ping dis­cov­ery work

Three years after Takeda paid a hefty down payment on a research collab with Wave Life Sciences, the two partners have revised the pact.

The Cambridge biotech announced a revision to its deal with Takeda yesterday — ending their alliance on discovery work. As part of the amendment, Takeda will pay Wave more than $22 million for research and preclinical expenses from the collaboration.

Back in 2018, Cambridge, MA-based Wave entered a mega-deal with Takeda. The deal was two-fold: Wave gave Takeda the option to co-develop and co-commercialize programs in Huntington’s disease, amyotrophic lateral sclerosis (ALS), frontotemporal dementia, and spinocerebellar ataxia type 3. Takeda also had the right to license several of Wave’s preclinical programs targeting CNS disorders, including Alzheimer’s and Parkinson’s.

Jean-Pierre Sommadossi, Atea CEO

Roche-Atea’s Covid-19 pill fails mid-stage tri­al, cloud­ing fu­ture for close­ly watched pro­gram

When Merck announced this month that its Covid-19 pill cut newly diagnosed patients’ risk of being hospitalized by 50%, researchers and analysts hoped that other pills would soon prove similarly effective. They paid particular attention to two drugs from Pfizer and Roche.

But on Tuesday, Roche and its biotech partner, Atea, announced that their antiviral had failed a key interim test.

In a Phase II trial, AT-527 didn’t reduce the amount of virus in mild to moderate Covid-19 patients any more than a placebo pill did. That was true for both the patients who received a high dose and those who received the low dose.

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Terrie Curran, Phathom CEO (Credit: Arcutis)

Phath­om's old Take­da drug bests Pre­vacid in a PhI­II GI tri­al. Next stop? The FDA

There’s no time for rest in biopharma — at least not at Phathom Pharmaceuticals. Just over a month after submitting two NDAs for its lead acid-fighter vonoprazan, the biotech is already lining up a third, and collecting an extra $50 million to push things along.

Vonoprazan met its primary non-inferiority endpoints in a Phase III study comparing it to standard-of-care Prevacid in a type of gastroesophageal reflux disease (GERD) called erosive esophagitis (EE). It also proved superior to the popular heartburn drug by multiple measures, including healing rate and maintenance of healing.

Reshma Kewalramani, Vertex CEO (YouTube)

Ver­tex gets much-need­ed win with ‘ex­tra­or­di­nary’ first pa­tient re­sults on po­ten­tial di­a­betes cure

Vertex said Monday that the first patient dosed with its cell therapy for type 1 diabetes saw their need for insulin injections vanish almost entirely, a key early step in the decades-long effort to develop a curative treatment for the chronic disease.

The patient, who had suffered five potentially life-threatening hypoglycemic — or low blood sugar — episodes in the year before the therapy, was injected with synthetic insulin-producing cells. After 90 days, the patient’s new cells produced insulin steadily and ramped up their insulin production after a meal like normal cells do, as measured by a standard biomarker for insulin production.

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