‘The headlines are the headlines, but’: BioMarin talks up potential savings as hemophilia gene therapy launch looms

February 27, 2020 07:22 AM EST

'The headlines are the headlines, but': BioMarin talks up potential savings as hemophilia gene therapy launch looms

Amber Tong

Editor

BioMarin execs are still staying tight-lipped about their pricing plans for what is poised to be the world’s first hemophilia gene therapy. But as the company enters the final regulatory stretch and approaches a potential launch this summer, they are also dropping more hints to get investors ready.

First thing to know: They really, really don’t expect an advisory committee to be convened for valrox, which is under priority review, to pop up before its PDUFA date on August 21.

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Minoryx and Sperogenix ink an exclusive license agreement to develop and commercialize leriglitazone in China

Pharmcube

September 23, 2020 – Hong Kong, Beijing, Shanghai (China) and Mataró, Barcelona (Spain)

Minoryx will receive an upfront and milestone payments of up to $78 million, as well as double digit royalties on annual net sales

Sperogenix will receive exclusive rights to develop and commercialize leriglitazone for the treatment of X-linked adrenoleukodystrophy (X-ALD), a rare life-threatening neurological condition

Vas Narasimhan (AP Images)

September 23, 2020 07:35 AM EDTUpdated 07:56 AM

Cell/Gene Tx

Regulatory

UPDATED: Still held down by clinical hold, Novartis' Zolgensma falls further behind Biogen and Roche as FDA asks for a new pivotal study

Amber Tong

Editor

Last October, the FDA slowed down Novartis’ quest to extend its gene therapy to older spinal muscular atrophy patients by slapping a partial hold on intrathecal administration. Almost a year later, the hold is still there, and regulators are adding another hurdle required for regulatory submission: a new pivotal confirmatory study.

The new requirement — which departs significantly from Novartis’ prior expectations — will likely stretch the path to registration beyond 2021, when analysts were expecting a BLA submission. That could mean more time for Biogen to reap Spinraza revenues and Roche to ramp up sales of Evrysdi in the absence of a rival.

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David Berry (Flagship)

September 24, 2020 06:03 AM EDT

Venture

Startups

Flagship's next big technological bet? The cloud

Jason Mast

Associate Editor

Earlier this month, Flagship announced their big bet on the software half the industry is talking about, launching the AI and machine learning startup. Now, they and a couple other investors are gambling $100 million on a software that much of the public generally thinks of as a cool, IT afterthought: cloud computing.

The idea, says founder and Flagship partner David Berry, is one of scale: The sheer magnitude of biological data that you can store on cloud technology is unprecedented. And that size, when leveraged properly, can allow you to ask questions and form insights that are similarly unprecedented.

ENDPOINTS CAREERS

Clinical Operations Lead

Vigeo Therapeutics

Cambridge, MA

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FDA commissioner Stephen Hahn at the White House (AP Images)

September 22, 2020 04:47 PM EDTUpdated 22 hours ago

Regulatory

Coronavirus

Under fire, FDA to issue stricter guidance for Covid-19 vaccine EUA this week — report

Jason Mast

Associate Editor

The FDA has been insisting for months that a Covid-19 vaccine had to be at least 50% effective – a measure of transparency meant to shore public trust in the agency and in a vaccine that had been brought forward at record speed and record political pressure. But now, with concerns of a Trump-driven authorization arriving before the election, the agency may be raising the bar.

The FDA is set to release new guidance that would raise safety and efficacy requirements for a vaccine EUA above earlier guidance and above the criteria used for convalescent plasma or hydroxychloroquine, The Washington Post reported. Experts say this significantly lowers the odds of an approval before the election on November 3, which Trump has promised despite vocal concerns from public health officials, and could help shore up public trust in the agency and any eventual vaccine.

September 23, 2020 07:00 AM EDTUpdated 07:30 AM

Cell/Gene Tx

Manufacturing

Scoop: ARCH’s Bob Nelsen is backing an mRNA upstart that promises to upend the entire manufacturing side of the global business

John Carroll

Editor & Founder

For the past 2 years, serial entrepreneur Igor Khandros relied on a small network of friends and close insiders to supply the first millions he needed to fund a secretive project to master a new approach to manufacturing mRNA therapies.

Right now, he says, he has a working “GMP-in-a-box” prototype for a new company he’s building — after launching 3 public companies — which plans to spread this contained, precise manufacturing tech around the world with a set of partners. He’s raised $60 million, recruited some prominent experts. And not coincidentally, he’s going semi-public with this just as a small group of pioneers appears to be on the threshold of ushering in the world’s first mRNA vaccines to fight a worldwide pandemic.

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Samit Hirawat (Bristol Myers Squibb)

September 22, 2020 08:14 AM EDT

R&D

Cell/Gene Tx

After bruising rejection, bluebird and Bristol Myers Squibb land ide-cel priority review. But will it matter for the CVR?

Jason Mast

Associate Editor

With the clock all but up, the FDA accepted and handed priority review to Bristol Myers Squibb and bluebird bio’s BCMA CAR-T, keeping a narrow window open for Celgene investors to still cash in on the $9 CVR from the $63 billion Celgene merger.

The acceptance comes five months after the two companies weres slammed with a surprise refuse-to-file that threatened to foreclose the CVR entirely. Today’s acceptance sets the FDA decision date for March 27, 2021 – or precisely 4 days before the CVR deadline of March 31. Given the breakthrough designation and strong pivotal data — 81.5% response rate, 35.2% complete response rate — priority review was largely expected.

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September 23, 2020 10:59 AM EDTUpdated 11:18 AM

R&D

PhII Alzheimer's failure deals new blow to Roche, AC Immune — but the tau hypothesis is far from dead

Amber Tong

Editor

The leading anti-tau antibody has failed its first Phase II testing, casting a shadow on a popular target (just trailing amyloid beta) for Alzheimer’s disease.

Roche and AC Immune are quick to acknowledge disappointment in the topline readout, which suggested that semorinemab did not reduce cognitive decline among patients with early Alzheimer’s disease, who are either just starting to have symptoms or have mild manifestations.

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ENDPOINTS CAREERS

Director of Regulatory Affairs

Recursion Pharmaceuticals

Salt Lake City, UT

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September 23, 2020 09:42 AM EDTUpdated 3 hours ago

Coronavirus

Covid-19 roundup: J&J begins pivotal Phase III trial for vaccine; Controversial human challenge trials to begin in London — report

Nicole DeFeudis

Associate Editor

Amber Tong

Editor

Johnson & Johnson announced it’s beginning a pivotal Phase III trial for its Covid-19 candidate, JNJ-78436735 — the first single-dose vaccine in this stage.

The Phase III trial, dubbed ENSEMBLE, will enroll 60,000 patients worldwide, making it the largest Phase III study of a Covid-19 vaccine to date. J&J said the candidate achieved positive interim results in a Phase I/IIa study, which will be published “imminently.” There’s a possibility that the first batches will be ready for potential emergency use in early 2021, according to the company.

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Patrick Enright, Longitude co-founder (Longitude)

September 23, 2020 07:31 AM EDTUpdated 09:43 AM

Venture

As its biotechs hit the pandemic exit, Longitude raises $585M for new neuro, cancer, aging and orphan-focused fund

Jason Mast

Associate Editor

The years have been kind to Longitude Capital. This year, too.

A 2006 spinout of Pequot Capital, its founders started their new firm just four years before the parent company would go under amid insider trading allegations. Their first life sciences fund raised $325 million amid the financial crisis, they added a second for $385 million and then in, 2016, a third for $525 million. In the last few months, the pandemic biotech IPO boom netted several high-value exits from those funds, as Checkmate, Vaxcyte, Inozyme and Poseida all went public.

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