Neil Kumar, BridgeBio
March 20, 2021 11:00 AM EDTUpdated March 22, 04:11 PM
R&D

Bridge­Bio team clears a key hur­dle with their proof-of-con­cept da­ta for a rare ge­net­ic dis­ease. And they can thank the NIH for a quick suc­cess

John Carroll

Editor & Founder

BridgeBio’s Calcilytix team has several good reasons to offer some special thanks to the NIH today.

Mike Collins, an NIH researcher and division chief who specializes in bone and mineral metabolism, suggested the idea that the right calcium sensing receptor (CaSR) antagonist could provide a direct way to treat a rare disease called ADH1, a genetic ailment that triggers a dangerous imbalance in calcium in a small patient population.

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SPONSORED
March 20, 2023 06:00 AM EDT

Has the mo­ment fi­nal­ly ar­rived for val­ue-based health­care?

Sean Dodge

Healthcare Technology Analyst, RBC Capital Markets

RBC Capital Markets’ Healthcare Technology Analyst, Sean Dodge, spotlights a new breed of tech-enabled providers who are rapidly transforming the way clinicians deliver healthcare, and explores the key question: can this accelerating revolution overturn the US healthcare system?

Key points

Tech-enabled healthcare providers are poised to help the US transition to value, not volume, as the basis for reward.
The move to value-based care has policy momentum, but is risky and complex for clinicians.
Outsourced tech specialists are emerging to provide the required expertise, while healthcare and tech are also converging through M&A.
Value-based care remains in its early stages, but the transition is accelerating and represents a huge addressable market.

Alaa Halawa, executive director at Mubadala’s US venture group
March 22, 2023 08:00 AM EDTUpdated 02:30 PM
Bioregnum
Discovery

The ven­ture crew at Mubadala are up­ping their biotech cre­ation game, tak­ing care­ful aim at a new fron­tier in drug de­vel­op­ment

It started with a cup of coffee and a slow burning desire to go early and long in the biotech creation business.

Wrapping up a 15-year discovery stint at Genentech back in the summer of 2021, Rami Hannoush was treated to a caffeine-fueled review of the latest work UCSF’s Jim Wells had been doing on protein degradation — one of the hottest fields in drug development.

“Jim and I have known each other for the past 15 years through Genentech collaborations. We met over coffee, and he was telling me about this concept of the company that he was thinking of,” says Hannoush. “And I got immediately intrigued by it because I knew that this could open up a big space in terms of adding a new modality in drug discovery that is desperately needed in pharma.”

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March 22, 2023 06:09 PM EDT
Pharma
FDA+

FDA ad­vi­sors unan­i­mous­ly rec­om­mend ac­cel­er­at­ed ap­proval for Bio­gen's ALS drug

Max Gelman

Senior Editor

A panel of outside advisors to the FDA unanimously recommended that the agency grant accelerated approval to Biogen’s ALS drug tofersen despite the drug failing the primary goal of its Phase III study, an endorsement that could pave a path forward for the treatment.

By a 9-0 vote, members of the Peripheral and Central Nervous System Drugs Advisory Committee said there was sufficient evidence that tofersen’s effect on a certain protein associated with ALS is reasonably likely to predict a benefit for patients. But panelists stopped short of advocating for a full approval, voting 3-5 against (with one abstention) and largely citing the failed pivotal study.

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March 23, 2023 03:31 AM EDTUpdated 04:45 AM
R&D
Pharma

Sanofi, Re­gen­eron boast PhI­II win with Dupix­ent in COPD, clear­ing first bar for ex­pan­sion

Amber Tong

Senior Editor

Dupixent, the blockbuster anti-inflammatory drug from Sanofi and Regeneron, has cleared a high-stakes Phase III study in chronic obstructive pulmonary disease, the companies announced Thursday morning.

If they hold up in a second, identical trial, the data pave the way for Dupixent to become the first biologic to treat patients whose COPD remains uncontrolled despite being on maximal standard-of-care inhaled therapy — the patient population studied in the pivotal program. The companies had spotlighted this as a key readout as they look to expand the Dupixent franchise and explore its full potential.

March 21, 2023 12:30 PM EDT
AI
Pharma
Marketing

Chat­G­PT with phar­ma da­ta de­buts for med­ical meet­ings, be­gin­ning with AACR

Beth Snyder Bulik

Senior Editor

What do you get when you combine ChatGPT generative AI technology with specific pharma and clinical datasets? A time-saving tool that can answer questions about medical conference abstracts and clinical findings in seconds in one new application from ZoomRx called FermaGPT.

ZoomRx is debuting a public version of its generative AI product specifically for medical conferences beginning this week for the upcoming American Association for Cancer Research (AACR) annual meeting that runs April 14-19.

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Vas Narasimhan, Novartis CEO (Gian Ehrenzeller/Keystone via AP)
March 22, 2023 03:00 PM EDT
R&D

No­var­tis pulls the plug on UK-based car­dio­vas­cu­lar study

Nicole DeFeudis

Editor

Novartis is calling off a UK-based trial for Leqvio in the primary prevention of cardiovascular events in patients with high cholesterol, the company confirmed on Wednesday.

The Swiss pharma giant made the decision after “careful evaluation,” a spokesperson told Endpoints News via email. The trial, dubbed ORION-17, was planned in partnership with England’s National Health Service (NHS) and was part of the company’s strategy to establish Leqvio as a standard of care in cardiovascular disease management.

March 22, 2023 11:22 AM EDTUpdated 11:37 AM
R&D
Pharma
FDA+

FDA re­jects Ab­b­Vie's in­fu­sion ther­a­py for Parkin­son's, re­quests more in­fo on pump de­vice

Kyle LaHucik

Associate Editor

The FDA rejected AbbVie’s 24-hour infusion therapy for Parkinson’s, saying it needs more information on a device used to administer the treatment before it can clear it.

The Chicago-area drugmaker said in a press release that the complete response letter from the agency didn’t include any requests for more efficacy or safety trials related to the drug, known as ABBV-951. The company said it aims to “resubmit the NDA as soon as possible.”

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March 22, 2023 11:19 AM EDT
Financing
R&D

Quince Ther­a­peu­tics faces takeover bid from share­hold­er Echo Lake Cap­i­tal

Tyler Patchen

News Reporter

A bid to take over the biotech Quince Therapeutics has been put forward by one of its shareholders.

On Tuesday, Echo Lake Capital sent a letter to Quince’s board of directors putting forth a proposal to acquire all the biotech’s stock for $1.60 per share, which would value a takeover at around $58 million.

In the letter, Echo Lake said that it believes Quince’s stock is severely undervalued and that no drugs are being actively marketed or developed that require cash expenditures. It’s trading below the value of its assets, Echo Lake said.

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March 22, 2023 10:49 AM EDT
FDA+

FDA+ roundup: Leg­isla­tive asks for 2024 fo­cus on more au­thor­i­ties, gener­ic drug com­pe­ti­tion

Zachary Brennan

Senior Editor

The FDA’s legislative priorities for the next year highlight the agency’s focus on expanding generic drug competition, backstopping the supply chain and growing its current authorities.

On the new authorities front, FDA is seeking to expand its mandatory recall authority for all drugs, as the agency has been embroiled in a long process to remove some from the market. Covis Pharma refused to pull its preterm birth drug Makena, which won accelerated approval, for almost five years after failing its confirmatory trial. The company has since reversed course after a negative adcomm.

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Bridge­Bio team clears a key hur­dle with their proof-of-con­cept da­ta for a rare ge­net­ic dis­ease. And they can thank the NIH for a quick suc­cess

Has the mo­ment fi­nal­ly ar­rived for val­ue-based health­care?

The ven­ture crew at Mubadala are up­ping their biotech cre­ation game, tak­ing care­ful aim at a new fron­tier in drug de­vel­op­ment

FDA ad­vi­sors unan­i­mous­ly rec­om­mend ac­cel­er­at­ed ap­proval for Bio­gen's ALS drug

Sanofi, Re­gen­eron boast PhI­II win with Dupix­ent in COPD, clear­ing first bar for ex­pan­sion

Chat­G­PT with phar­ma da­ta de­buts for med­ical meet­ings, be­gin­ning with AACR

No­var­tis pulls the plug on UK-based car­dio­vas­cu­lar study

FDA re­jects Ab­b­Vie's in­fu­sion ther­a­py for Parkin­son's, re­quests more in­fo on pump de­vice

Quince Ther­a­peu­tics faces takeover bid from share­hold­er Echo Lake Cap­i­tal

FDA+ roundup: Leg­isla­tive asks for 2024 fo­cus on more au­thor­i­ties, gener­ic drug com­pe­ti­tion