Roche and Genen­tech re­searchers plot $53M dis­cov­ery quest aimed at spark­ing a 'Holy moly' piv­ot in neu­ro R&D

Roche and Genen­tech have com­mit­ted $53 mil­lion to back a 10-year quest aimed at go­ing back to the draw­ing board to use new tech­nol­o­gy and fresh sci­en­tif­ic in­sights to gen­er­ate a pipeline of drugs for neu­ro­log­i­cal dis­eases.

Re­searchers from both Roche and its big South San Fran­cis­co hub — mix­ing teams from gRED and pRED this time — will mix it up with the sci­en­tists drawn to­geth­er for the Weill Neu­ro­hub — formed in 2019 as a joint re­search part­ner­ship in­volv­ing UCSF, Berke­ley and the Uni­ver­si­ty of Wash­ing­ton — in an ex­plo­ration of the field to de­vel­op new ther­a­pies for some of the tough­est dis­eases in drug R&D: Alzheimer’s, Parkin­son’s, Hunt­ing­ton’s, ALS and autism.

Roche has had plen­ty of set­backs to re­port on that front over the years, like every oth­er big play­er that’s com­mit­ted large sums to dis­eases that af­flict mil­lions of peo­ple around the world. And the crew here ap­pears com­mit­ted to find­ing drugs that can do more than in­flu­ence symp­toms or slight­ly bend the curve of dis­ease pro­gres­sion. They’ve all vot­ed to back a long-await­ed rev­o­lu­tion.

“This part­ner­ship is not about pick­ing off the low-hang­ing fruit — it’s about trans­form­ing the treat­ment of neu­ro­log­i­cal and psy­chi­atric dis­ease, which will re­quire long-term in­vest­ment and the flex­i­bil­i­ty to go where the sci­ence guides us,” says neu­ro ex­pert Stephen Hauser from UCSF.

“While all these dis­eases of the brain or CNS are in­cred­i­bly chal­leng­ing,” said Ehud Isacoff, pro­fes­sor of Neu­ro­bi­ol­o­gy at UC Berke­ley, “it is re­al­ly hard to imag­ine that sig­nif­i­cant progress will not be made in each of these ar­eas, be­cause the sci­en­tif­ic ex­per­tise and pas­sion across the teams are so strong. What I am hop­ing for is that some of these things lead to a break­through where, even­tu­al­ly, we are look­ing back a decade or two from now and say­ing, ‘Holy moly! That is when we turned the cor­ner on neu­ro­log­i­cal dis­ease.’ That’s the dream.”

BY­OD Best Prac­tices: How Mo­bile De­vice Strat­e­gy Leads to More Pa­tient-Cen­tric Clin­i­cal Tri­als

Some of the most time- and cost-consuming components of clinical research center on gathering, analyzing, and reporting data. To improve efficiency, many clinical trial sponsors have shifted to electronic clinical outcome assessments (eCOA), including electronic patient-reported outcome (ePRO) tools.

In most cases, patients enter data using apps installed on provisioned devices. At a time when 81% of Americans own a smartphone, why not use the device they rely on every day?

Pascal Soriot (AstraZeneca via YouTube)

Af­ter be­ing goad­ed to sell the com­pa­ny, Alex­ion's CEO set some am­bi­tious new goals for in­vestors. Then Pas­cal So­ri­ot came call­ing

Back in the spring of 2020, Alexion $ALXN CEO Ludwig Hantson was under considerable pressure to perform and had been for months. Elliott Advisers had been applying some high public heat on the biotech’s numbers. And in reaching out to some major stockholders, one thread of advice came through loud and clear: Sell the company or do something dramatic to change the narrative.

In the words of the rather dry SEC filing that offers a detailed backgrounder on the buyout deal, Alexion stated: ‘During the summer and fall of 2020, Alexion also continued to engage with its stockholders, and in these interactions, several stockholders encouraged the company to explore strategic alternatives.’

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Near­ly a year af­ter Au­den­tes' gene ther­a­py deaths, the tri­al con­tin­ues. What hap­pened re­mains a mys­tery

Natalie Holles was five months into her tenure as Audentes CEO and working to smooth out a $3 billion merger when the world crashed in.

Holles and her team received word on the morning of May 5 that, hours before, a patient died in a trial for their lead gene therapy. They went into triage mode, alerting the FDA, calling trial investigators to begin to understand what happened, and, the next day, writing a letter to alert the patient community so they would be the first to know. “We wanted to be as forthright and transparent as possible,” Holles told me late last month.

The brief letter noted two other patients also suffered severe reactions after receiving a high dose of the therapy and were undergoing treatment. One died a month and a half later, at which point news of the deaths became public, jolting an emergent gene therapy field and raising questions about the safety of the high doses Audentes and others were now using. The third patient died in August.

“It was deeply saddening,” Holles said. “But I was — we were — resolute and determined to understand what happened and learn from it and get back on track.”

Eleven months have now passed since the first death and the therapy, a potential cure for a rare and fatal muscle-wasting disease called X-linked myotubular myopathy, is back on track, the FDA having cleared the company to resume dosing at a lower level. Audentes itself is no more; last month, Japanese pharma giant Astellas announced it had completed working out the kinks of the $3 billion merger and had restructured and rebranded the subsidiary as Astellas Gene Therapies. Holles, having successfully steered both efforts, departed.

Still, questions about precisely what led to the deaths of the 3 boys still linger. Trial investigators released key details about the case last August and December, pointing to a biological landmine that Audentes could not have seen coming — a moment of profound medical misfortune. In an emerging field that’s promised cures for devastating diseases but also seen its share of safety setbacks, the cases provided a cautionary tale.

Audentes “contributed in a positive way by giving a painful but important example for others to look at and learn from,” Terry Flotte, dean of the UMass School of Medicine and editor of the journal Human Gene Therapy, told me. “I can’t see anything they did wrong.”

Yet some researchers say they’re still waiting on Astellas to release more data. The company has yet to publish a full paper detailing what happened, nor have they indicated that they will. In the meantime, it remains unclear what triggered the events and how to prevent them in the future.

“Since Audentes was the first one and we don’t have additional information, we’re kind of in a holding pattern, flying around, waiting to figure out how to land our vehicles,” said Jude Samulski, professor of pharmacology at UNC’s Gene Therapy Center and CSO of the gene therapy biotech AskBio, now a subsidiary of Bayer.

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Kristin Fortney, BioAge Labs CEO

An­ti-ag­ing biotech up­start plucks a drug from Am­gen's dis­card pile, piv­ot­ing from heart fail­ure to mus­cle con­di­tions

Back in April 2019, Amgen quietly shut down a Phase I trial for a drug named AMG 986. There was no safety concern; the molecule just didn’t hit the mark on helping the small band of heart failure patients who received it.

A small biotech, though, believes it would stand a chance in the burgeoning anti-aging field.

BioAge Labs has licensed AMG 986 — now renamed BGE-105 — with plans to parlay the existing IND into a quick Phase I trial teasing out the pharmacodynamic effects and set the stage for mid-stage tests focused on acute muscle indications.

Anand Shah (FDA)

For­mer head of FDA’s med­ical and sci­en­tif­ic af­fairs on Covid: ‘FDA has nev­er been test­ed like this’

Anand Shah has served the American public in a unique way, crisscrossing over the last two administrations between serving as an attending radiation oncologist focused on prostate cancer at NIH, serving as CMO at the Center for Medicare and Medicaid Innovation, and most recently, leading the FDA’s operations on medical and scientific affairs from within the commissioner’s office.

Shah, who stepped down from the FDA in January, caught up with Endpoints News in a phone interview on Tuesday afternoon, offering his thoughts on the agency’s latest decision to pause the J&J vaccinations in the US, and reflecting on his time at an agency during this once-in-a-lifetime pandemic.

Launched by MIT grads, a small start­up gets $20M to back a ro­bot­ics rev­o­lu­tion in cell ther­a­py man­u­fac­tur­ing

As co-director of an experimental cellular therapy process development and manufacturing group at UCSF specializing in T cell therapies for autoimmune conditions, Jonathan Esensten has learned a lot about the challenges involved when his group hand-fashions a cell therapy. Esensten — who was a postdoc in Wendell Lim’s lab and counts the legendary Jeffrey Bluestone as a mentor — gives them all high marks at being great at what they do, but time and again there are variations in the treatments they construct.

Barbara Weber, Tango Therapeutics CEO (Tango)

It takes two to Tan­go: The biotech us­ing CRISPR to dis­cov­er new can­cer gene tar­gets rides a $353M SPAC deal to Nas­daq

Editor’s note: Interested in following biopharma’s fast-paced IPO market? You can bookmark our IPO Tracker here.

The latest biotech-SPAC deal has arrived, and it’s dancing its way to Nasdaq to the tune of several hundred million dollars.

Tango Therapeutics and its CRISPR-focused search for new cancer genes is reverse merging with Boxer Capital’s blank-check company, the biotech announced Wednesday morning. With a spotlight on three lead programs, Tango expects total proceeds to equal about $353 million in the deal, which includes the roughly $167 million held in the SPAC and an additional $186 million in PIPE financing.

Min Li, SciNeuro CEO

Eli Lil­ly seeds a well-con­nect­ed Chi­na neu­ro start­up with drugs aim­ing at where Bio­gen, Voy­ager missed

When Min Li took the wraps off a $100 million round for his Bob Nelsen-backed startup last December — as with most biotech CEOs when they launch — he left a couple cards unturned.

Keen to build a first-of-its-kind neuro play in China, the GlaxoSmithKline R&D vet already had a good idea of which assets he wanted to in-license for SciNeuro, conducting Zoom negotiations from the East Coast office that he’s been somewhat stuck in since JP Morgan early in the year. Now, he’s finally ready to shine a spotlight on the first program: a slate of alpha-synuclein targeted antibody therapies from Eli Lilly.

Stéphane Bancel, Moderna CEO (Jeff Rumans)

'Learned a lot last year': Af­ter Covid-19 suc­cess, Mod­er­na's Stéphane Ban­cel plans to give rest of pipeline a big push

A year ago, Stéphane Bancel would have described Moderna as cautious — walking step-by-step to investigate whether mRNA vaccines could prevent a host of viruses. Then the pandemic hit, and the Cambridge, MA-based biotech got a multibillion-dollar windfall to produce the world’s second-ever authorized mRNA vaccine in a matter of months.

What’s next? Bancel is planning a big acceleration and expansion of the rest of the pipeline, including the company’s Phase III-ready candidate for cytomegalovirus (CMV), which was the lead program before Covid-19 came around.