Pfizer gene therapy ups levels of dystrophin expression in Duchenne trial — but another safety setback mars results

May 15, 2020 08:02 AM EDTUpdated 09:47 AM

Pfizer gene therapy ups levels of dystrophin expression in Duchenne trial — but another safety setback mars results

John Carroll

Editor & Founder

Pfizer’s gene therapy group is back up at bat with a fresh update of data from its Phase Ib study for a gene therapy intended as a once-and-done treatment for Duchenne muscular dystrophy. And they had some good news, and some bad news, to report.

The good news was primarily in the dystrophin expression they tracked among the now 9 evaluable patients in their first clinical foray for PF-06939926.

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David Chang, Allogene CEO (Jeff Rumans)

May 29, 2020 08:00 AM EDT

R&D

Headed to PhII: Allogene CEO David Chang completes a positive early snapshot of their off-the-shelf CAR-T pioneer

John Carroll

Editor & Founder

Allogene CEO David Chang has completed the upbeat first portrait of the biotech’s off-the-shelf CAR-T contender ALLO-501 at virtual ASCO today, keeping all eyes on a drug that will now try to go on to replace the first-wave personalized pioneers he helped create.

The overall response rate outlined in Allogene’s abstract for treatment-resistant patients with non-Hodgkin lymphoma slipped a little from the leadup, but if you narrow the patient profile to treatment-naïve patients — removing the 3 who had previous CAR-T therapy who didn’t respond, leaving 16 — the ORR lands at 75% with a 44% complete response rate. And 9 of the 12 responders remained in response at the data cutoff, offering a glimpse on durability that still has a long way to go before it can be completely nailed down.

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May 28, 2020 05:00 PM EDTUpdated 1 hour ago

R&D

AstraZeneca trumpets the good data they found for Tagrisso in an adjuvant setting for NSCLC — but many of the experts aren’t cheering along

John Carroll

Editor & Founder

AstraZeneca is rolling out the big guns this evening to provide a salute to their ADAURA data on Tagrisso at ASCO.

Cancer R&D chief José Baselga calls the disease-free survival data for their drug in an adjuvant setting of early stage, epidermal growth factor receptor-mutated NSCLC patients following surgery “momentous.” Roy Herbst, the principal investigator out of Yale, calls it “transformative.”

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Paul Hudson, Sanofi CEO (Getty Images)

May 27, 2020 07:03 AM EDT

Bioregnum

R&D

Sanofi CEO Paul Hudson has $23B burning a hole in his pocket. And here are some hints on how he plans to spend that

Sanofi has reaped $11.1 billion after selling off a big chunk of its Regeneron stock at $515 a share. And now everyone on the M&A side of the business is focused on how CEO Paul Hudson plans to spend it.

After getting stung in France for some awkward politicking — suggesting the US was in the front of the line for Sanofi’s vaccines given American financial support for their work, versus little help from European powers — Hudson now has the much more popular task of managing a major cash cache to pull off something in the order of a big bolt-on. Or two.

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ENDPOINTS CAREERS

Process Development Scientist, Compound Management

Recursion Pharmaceuticals

Salt Lake City, UT

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The Avance Clinical leadership team: CEO Yvonne Lungershausen, Sandrien Louwaars - Director Business Development Operations, Gabriel Kremmidiotis - Chief Scientific Officer, Ben Edwards - Chief Strategy Officer

How Australia Delivers Rapid Start-up and 43.5% Rebate for Early Phase Oncology Trials

Ben Edwards

Chief Strategy Officer

About Avance Clinical

Avance Clinical is an Australian owned Contract Research Organisation that has been providing high-quality clinical research services to the local and international drug development industry for 20 years. They specialise in working with biotech companies to execute Phase 1 and Phase 2 clinical trials to deliver high-quality outcomes fit for global regulatory standards.

As oncology sponsors look internationally to speed-up trials after unprecedented COVID-19 suspensions and delays, Australia, which has led the world in minimizing the pandemic’s impact, stands out as an attractive destination for early phase trials. This in combination with the streamlined regulatory system and the financial benefits including a very favourable exchange rate and the R & D cash rebate makes Australia the perfect location for accelerating biotech clinical programs.

Pablo Legorreta, founder and CEO of Royalty Pharma AG, speaks at the annual Milken Institute Global Conference in Beverly Hills, California (Patrick T. Fallon/Bloomberg via Getty Images)

May 28, 2020 09:28 AM EDTUpdated 09:38 AM

People

Pharma

Capitalizing Pablo: The world’s biggest drug royalty buyer is going public. And the low-key CEO divulges a few secrets along the way

John Carroll

Editor & Founder

Pablo Legorreta is one of the most influential players in biopharma you likely never heard of.

Over the last 24 years, Legorreta’s Royalty Pharma group has become, by its own reckoning, the biggest buyer of drug royalties in the world. The CEO and founder has bought up a stake in a lengthy list of the world’s biggest drug franchises, spending $18 billion in the process — $2.2 billion last year alone. And he’s become one of the best-paid execs in the industry, reaping $28 million from the cash flow last year while reserving 20% of the cash flow, less expenses, for himself.

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May 29, 2020 07:28 AM EDT

R&D

Pharma

Pfizer, Merck KGaA cement Bavencio bladder cancer win with OS data — while carving another niche in rare cancer

Amber Tong

Editor

Pfizer and Merck KGaA have detailed the Phase III data that inspired FDA regulators to designate Bavencio a “breakthrough” for first-line advanced bladder cancer and offered an early glance at how the PD-L1 can help patients with a rare gynecological cancer — carving out niches in the checkpoint space for itself after being shut out of numerous others.

In JAVELIN Bladder 100, Bavencio led to a 31% reduction in risk of death compared to standard care alone. It also extended median survival by more than seven months — a historic feat in this setting, according to investigators at Queen Mary University of London.

May 29, 2020 07:22 AM EDTUpdated 07:39 AM

People

Peer Review

Sanofi brings in 4 new executives in continued shake-up, as vaccines and consumer health chief head out the door

Jason Mast

Associate Editor

In the middle of Sanofi’s multi-pronged race to develop a Covid-19 vaccine, David Loew, the head of their sprawling vaccines unit, is leaving – part of the final flurry of moves in the French giant’ months-long corporate shuffle that will give them new-look leadership under new CEO Paul Hudson.

The company also said today that Alan Main, the head of their consumer healthcare unit, is out, and they named 4 executives to fill new or newly vacated positions, 3 of whom come from both outside both Sanofi and from Pharma.

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ENDPOINTS CAREERS

Senior Drug Discovery Scientist

New York, NY

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May 29, 2020 06:30 AM EDTUpdated 07:36 AM

R&D

Ironwood, AbbVie kick delayed-release Linzess formulation to the curb after trial failure

Natalie Grover

Reporter

The delayed-release formulation of Ironwood and Allergan’s bowel drug Linzess will not see the light of day.

The experimental drug, MD-7246, failed to help patients with abdominal pain associated with irritable bowel syndrome with diarrhea (IBS-D) in a mid-stage study, prompting the partners to abandon the therapy.

First approved in 2012, Linzess (known chemically as linaclotide) enhances the activity of the intestinal enzyme guanylate cyclase-C to increase the secretion of intestinal fluid and then transit through the intestinal tract, as well as reduce visceral pain, to relieve pain and constipation associated with IBS.

May 29, 2020 07:14 AM EDTUpdated 07:30 AM

People

Peer Review

Looking to move past an R&D fiasco, Ipsen poaches their new CEO from Sanofi

John Carroll

Editor & Founder

Ipsen has turned to another Paris-based biopharma company for its next CEO.

Sanofi Pasteur chief David Loew — who’s been leading one of the most advanced efforts to develop vaccines for Covid-19 — is making the journey to Ipsen, 5 months after David Meek jumped ship to run a startup in late-stage development.

Loew arrives as Ipsen works to get back on track with their rare bone disease drug palovarotene, picked up in the $1.3 billion Clementia buyout, which was slammed with a partial hold after researchers observed cases of “early growth plate closure” in patients under the age of 14. But they are pushing ahead with the over-14 crowd after writing down slightly more than half of its initial development.