Aducanumab has its approval — now, let citizen-scientists come together to see if it works

July 14, 2021 05:35 PM EDTUpdated July 16, 10:36 AM

Aducanumab has its approval — now, let citizen-scientists come together to see if it works

Madhav Thambisetty

Adjunct professor of neurology at Johns Hopkins University School of Medicine

Biotech Voices is a collection of exclusive opinion editorials from some of the leading voices in biopharma on the biggest industry questions today. Think you have a voice that should be heard? Reach out to senior editors Kyle Blankenship and Amber Tong.

Editor’s Note: Madhav Thambisetty was an advisor on the FDA’s Peripheral and Central Nervous System Drugs advisory committee that voted overwhelmingly not to recommend Alzheimer’s drug aducanumab’s approval by the FDA. Thambisetty’s views in this op-ed are not reflections of the positions of the FDA or the NIH’s National Institute on Aging, where he is a section chief of the clinical and translational neuroscience section in the laboratory of behavioral neuroscience.

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Adaptive Design Methods Offer Rapid, Seamless Transition Between Study Phases in Rare Cancer Trials

Rupa Doshi

Executive Director, Oncology Program Strategy

Rare cancers account for 22 percent of cancer diagnoses worldwide, yet there is no universally accepted definition for a “rare” cancer. Moreover, with the evolution of genomics and associated changes in categorizing tumors, some common cancers are now characterized into groups of rare cancers, each with a unique implication for patient management and therapy.

Adaptive designs, which allow for prospectively planned modifications to study design based on accumulating data from subjects in the trial, can be used to optimize rare oncology trials (see Figure 1). Adaptive design studies may include multiple cohorts and multiple tumor types. In addition, numerous adaptation methods may be used in a single trial and may facilitate a more rapid, seamless transition between study phases.

Marianne De Backer (L) and Jeff Hatfield

August 5, 2021 01:30 AM EDT

Deals

Bayer nabs star biotech Vividion with a $2B buyout and an ‘arms-length’ pact, pulling a partner out of the IPO conga line

John Carroll

Editor & Founder

Vividion is canceling that IPO it filed. Instead of following the industry-wide migration to Nasdaq, the biotech that has captured considerable attention for its still-preclinical work finding cryptic pockets to bind to on proteins is going to work for Bayer now.

The pharma giant is putting out word today that it has bought out Vividion for $1.5 billion in cash and another half-billion dollars in milestones.

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Tadataka Yamada (Photographer: Kiyoshi Ota/Bloomberg via Getty Images)

August 4, 2021 06:32 PM EDTUpdated 35 minutes ago

People

Science pioneer, pharma research chief, global health advocate and biotech entrepreneur Tadataka ‘Tachi’ Yamada has died

Jason Mast

Editor

Tadataka Yamada, a towering physician-scientist who made his name in academia before transforming drug development at GlaxoSmithKline and developing vaccines for malaria and meningitis at the Gates Foundation, died unexpectedly of natural causes at his home in Seattle Wednesday morning.

He was 76. Frazier Healthcare Partners’ David Socks confirmed his death.

Known widely by the mononym “Tachi,” Yamada had a globetrotting career and arrived in industry relatively late in life. A 2004 Independent article noted GSK had asked Yamada to stay on beyond his approaching 60th birthday, the company’s usual retirement age. Yamada would continue working for the next 17 years, steering the Gates Foundation’s global health division for 6 years, funding Jim Wilson’s gene therapy work when few would touch it, launching Takeda Vaccines and co-founding a series of high-profile biotechs.

ENDPOINTS CAREERS

Director, Clinical Program Management

Pliant Therapeutics

South San Francisco, CA, USA

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August 5, 2021 06:55 AM EDT

R&D

UK re-investigates Pfizer's eye-popping price gouging on an epilepsy drug

Zachary Brennan

Senior Editor

When a drugmaker raises the price of a drug in the US by more than 2,000% overnight, and without any particular reason for that increase, nothing typically happens to the company. No fines, no court orders, just business as usual.

Martin Shkreli’s decades-old anti-parasitic drug Daraprim was the perfect example — massive price spike on an old drug, lots of media attention, public outcry, Congressional committees dragging his former company through multiple hearings, and at the end of it? Nothing happened to the price or the company (until generic competition came).

Thomas Lingelbach, Valneva CEO

August 5, 2021 06:47 AM EDT

R&D

A small vaccine developer favored by the UK government in Covid-19 touts a PhIII first in chikungunya

Amber Tong

Senior Editor

Before Valneva garnered the favor of the UK government as a potential supplier of Covid-19 vaccines, the French biotech prided itself on being the first company to bring a chikungunya vaccine into Phase III.

It now has positive pivotal results to back up the breakthrough therapy designation the FDA granted just weeks ago.

There are currently no approved jabs to prevent chikungunya virus infection despite decades of R&D efforts, a fact that underscores just how arduous traditional vaccine development can be, particularly for neglected tropical disease. In a absence of a major commercial market, the US government and NGOs such as CEPI have deployed various grants and incentives to spur on a small crew of academics and industry players, with Merck, via its acquisition of Themis, claiming a spot in that race.

Josh Hoffman, outgoing Zymergen CEO (Zymergen)

August 3, 2021 04:50 PM EDTUpdated August 3, 11:57 PM

People

R&D

UPDATED: Synbio unicorn Zymergen jettisons founding CEO, cuts guidance as customers report lead product doesn't work

Kyle Blankenship

Managing Editor

Zymergen, just months off a $500 million IPO that put the synthetic bio firm in rarified air, has now ejected its founding CEO and downgraded its revenue forecasts after customers reported its lead film product doesn’t work as advertised, the company said Tuesday afternoon.

CEO Josh Hoffman will leave his role and sacrifice his board seat immediately in favor of Jay Flatley, the former CEO of Illumina who will take the lead role on an interim basis as the company conducts a search for its next leader.

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Zymergen co-founders Zach Serber, Josh Hoffman, and Jed Dean (Zymergen via website)

August 4, 2021 04:02 PM EDTUpdated 3 hours ago

Financing

Zymergen's sudden implosion shocked biotech. A lingering loan could make things even worse

Kyle Blankenship

Managing Editor

As former synbio unicorn Zymergen picks up the pieces from its spectacular implosion Tuesday, an outstanding loan from Perceptive Advisors — the only blue-chip biotech crossover investor to touch Zymergen’s fundraising efforts — could make the situation worse, according to public documents.

In December 2019, more than a year before Zymergen filed for what would eventually become a $500 million IPO, the “biofacturing” firm signed a $100 million credit facility with Perceptive to help supplement the nearly $700 million the company had raised across four VC rounds.

ENDPOINTS CAREERS

Director-Regulatory

Icosavax, Inc.

Seattle, WA, USA

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August 4, 2021 09:46 AM EDTUpdated 11:00 AM

FDA+

Biogen, Eisai are pushing for another accelerated Alzheimer's OK — this time for BAN2401

John Carroll

Editor & Founder

Now that the door at the FDA has been opened wide for Alzheimer’s drugs that can demonstrate a reduction in amyloid, Biogen and its partners at Eisai are pushing for a quick OK on the next drug to follow in the controversial path of aducanumab.

In a presentation to analysts, Eisai neurology chief Ivan Cheung outlined some bullish expectations for their newly-approved treatment and set the stage for what he believes will be a fast follow for BAN2401 (lecanemab) — after a dry spell in new drug development that’s lasted close to 20 years.

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Paul Hudson, Sanofi CEO (Eric Piermont/AFP via Getty Images)

August 2, 2021 06:08 PM EDTUpdated August 3, 04:33 PM

Deals

UPDATED: Sanofi buys mRNA player Translate Bio for $3.2B. And the price fits a popular range for biotech M&A

John Carroll

Editor & Founder

Sanofi CEO Paul Hudson is dead serious about his intention to vault directly into contention for the future of mRNA vaccines.

A year after paying Translate Bio $TBIO a whopping $425 million in an upfront and equity payment to help guide the pharma giant to the promised land of mRNA vaccines, Sanofi closed the deal with a buyout early Tuesday, spending $38 a share in a $3.2 billion buyout.

Translate’s stock $TBIO soared after the market closed Monday when Reuters reported the first word of the acquisition just hours ahead of the formal announcement. The wire service, though, didn’t have a price to report in its scoop, and investors chased the stock up 78% in the wild ride that followed. Once the price was announced, gains shriveled to 29% ahead of the bell.

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