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STAT+: Pharmalittle: We’re reading about Trump boosting psychedelic treatment, the future for weight-loss drugs, and more
Good morning, everyone, and welcome to another working week. We hope the weekend respite was relaxing and rewarding, because that oh-so-familiar routine of meetings, online calls, and deadlines has predictably returned. But what can you do? The world keeps spinning no matter how much we push back. So time to get on with it. Please join us for a cup of stimulation — hot buttered rum is our choice today — as we cast about for items of interest. Speaking of which, here are some tidbits. Hope your day goes well…
Weight-loss drugs may eventually reach only about 50% of the overweight and obese people who could benefit from them due to complexities of health care systems and financial constraints, Reuters writes, citing comments made by Eli Lilly chief executive officer David Ricks said at a conference. Lilly and Novo Nordisk are competing for share in the global market for GLP-1 drugs, which analysts see topping $100 billion per year in the next decade. But just one in 10 people who are overweight or obese are using GLP-1s. “It’s never going to be a hundred,” Ricks said. “For institutional reasons in health care and some other complexities in managing health, it’s never going to be that high.” He pointed to low-cost statins, the most commonly prescribed cholesterol drugs, as a comparable example. “Between 40 and 50% of people who should be on them, are on them. I think of that as maybe a ceiling,” he said.
The Trump administration’s desire to pry open the black box of prescription drug prices is facing stiff opposition from the phalanx of lobbyists representing pharmacy benefit managers and health insurers, STAT notes. In January, the U.S. Department of Labor proposed a rule that would mandate PBMs disclose a wide range of drug pricing information to employers and make it easier to be audited. The public had until last week to submit comments. A review of the more than 500 letters reflects varying interests: predictable resistance from PBMs and health insurers; enthusiasm from Mark Cuban’s pharmacy and others in the business community who want middlemen to face more accountability; and drug companies cheered PBMs being in the regulatory crosshairs but wanted pullback on disclosure of drug pricing data.
Good morning, everyone, and welcome to another working week. We hope the weekend respite was relaxing and rewarding, because that oh-so-familiar routine of meetings, online calls, and deadlines has predictably returned. But what can you do? The world keeps spinning no matter how much we push back. So time to get on with it. Please join us for a cup of stimulation — hot buttered rum is our choice today — as we cast about for items of interest. Speaking of which, here are some tidbits. Hope your day goes well…
Weight-loss drugs may eventually reach only about 50% of the overweight and obese people who could benefit from them due to complexities of health care systems and financial constraints, Reuters writes, citing comments made by Eli Lilly chief executive officer David Ricks said at a conference. Lilly and Novo Nordisk are competing for share in the global market for GLP-1 drugs, which analysts see topping $100 billion per year in the next decade. But just one in 10 people who are overweight or obese are using GLP-1s. “It’s never going to be a hundred,” Ricks said. “For institutional reasons in health care and some other complexities in managing health, it’s never going to be that high.” He pointed to low-cost statins, the most commonly prescribed cholesterol drugs, as a comparable example. “Between 40 and 50% of people who should be on them, are on them. I think of that as maybe a ceiling,” he said.
The Trump administration’s desire to pry open the black box of prescription drug prices is facing stiff opposition from the phalanx of lobbyists representing pharmacy benefit managers and health insurers, STAT notes. In January, the U.S. Department of Labor proposed a rule that would mandate PBMs disclose a wide range of drug pricing information to employers and make it easier to be audited. The public had until last week to submit comments. A review of the more than 500 letters reflects varying interests: predictable resistance from PBMs and health insurers; enthusiasm from Mark Cuban’s pharmacy and others in the business community who want middlemen to face more accountability; and drug companies cheered PBMs being in the regulatory crosshairs but wanted pullback on disclosure of drug pricing data.
Biotechnology company SonoThera has raised $125 million in an oversubscribed Series B financing round. The financing was led by Vida Ventures, with participation from ARK Invest, CureDuchenne Ventures, Leaps by Bayer, Otsuka Pharmaceutical, SymBiosis, UCB Ventures SA, Vivo Capital, and existing investors ARCH Venture Partners, Alexandria Venture Investments, Duquesne Family Office, Illumina Ventures, Johnson & Johnson Innovation – JJDC, Medical Excellence Capital, RA Capital, and Vertex Ventures HC.
SonoThera will use the funds to advance its lead programs in Duchenne muscular dystrophy (DMD) and autosomal dominant polycystic kidney disease (ADPKD) in the clinic. The funds will also support efforts to expand its pipeline of targeted redosable genetic medicines across multiple organ systems and scale its proprietary platform technologies for safe, targeted therapy delivery.
The company’s platform combines a proprietary ultrasound-mediated delivery technology dubbed RIPPLE
, with a payload engineering platform dubbed PORE. The platforms are designed to support the development of DNA and RNA therapeutics, gene editing, and gene silencing approaches. SonoThera is using its tech to develop genetic medicines that it claims will address key limitations of conventional gene therapies including delivery challenges, payload size constraints, immune responses, safety events, and difficulties with redosing.
As Kenneth Greenberd, PhD, SonoThera’s co-founder and CEO, stated “we founded SonoThera to take a fundamentally different approach, with a platform designed to broaden the therapeutic possibilities of the field. We believe our technology has the potential to expand the range of diseases addressable by genetic medicines while enabling more precise, durable, safer, and repeatable therapies for patients.”
SonoThera has already demonstrated the targeted delivery and expression capabilities of its platform across multiple tissues, including skeletal muscle, heart, liver, kidney, adipose, and brain. It has also shown that it can deliver large payloads such as full-length dystrophin for DMD and RNA-based payloads for gene silencing applications in preclinical studies.
The company expects to initiate its first clinical trial in DMD in 2027.
Commenting on the financing, Rajul Jain, MD, managing director at Vida Ventures, said “we believe SonoThera, with its RIPPLE delivery and PORE payload engineering technologies, has the potential to unlock opportunities in diseases with significant unmet need that have been previously inaccessible to other genetic medicine approaches.”
In connection with the financing, Jain and Rakhshita Dhar, MS, vice president & head of Healthcare Venture Investments at Leaps by Bayer, have joined SonoThera’s Board of Directors.
The post SonoThera Raises $125M to Develop Ultrasound-Mediated Genetic Medicines appeared first on GEN – Genetic Engineering and Biotechnology News.
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STAT+: Up and down the ladder: The latest comings and goings
Hired someone new and exciting? Promoted a rising star? Finally solved that hard-to-fill spot? Share the news with us, and we’ll share it with others. That’s right. Send us your changes, and we’ll find a home for them. Don’t be shy. Everyone wants to know who is coming and going.
And here is our regular feature in which we highlight a different person each week. This time around, we note that AstronauTx hired Michelle Mellion as chief medical officer. Previously, she held the same role at PepGen and EveryONE Medicines.
But all work and no play can make for a dull chief medical officer.
Hired someone new and exciting? Promoted a rising star? Finally solved that hard-to-fill spot? Share the news with us, and we’ll share it with others. That’s right. Send us your changes, and we’ll find a home for them. Don’t be shy. Everyone wants to know who is coming and going.
And here is our regular feature in which we highlight a different person each week. This time around, we note that AstronauTx hired Michelle Mellion as chief medical officer. Previously, she held the same role at PepGen and EveryONE Medicines.
But all work and no play can make for a dull chief medical officer.
Officials sanctioned Dabur India months after FDA inspectors found bird droppings and data integrity deficiencies during an inspection of the plant.
Illinois’ financial crisis could bring the state to a halt
The final 6 ‘Game of Thrones’ episodes might feel like a full season
New Season 8 Walking Dead trailer flashes forward in time
Mod turns ‘Counter-Strike’ into a ‘Tekken’ clone with fighting chickens
Meet Superman’s grandfather in new trailer for Krypton
Disney’s live-action Aladdin finally finds its stars
SonoThera Raises $125M to Develop Ultrasound-Mediated Genetic Medicines
STAT+: Up and down the ladder: The latest comings and goings
FDA imposes import alert on Indian plant after inspectors flag GMP failings
STAT+: Pharmalittle: We’re reading about a discontinued cancer drug, a Novo security breach, and more
Amgen shores up Tavneos’ FDA defense with Duke data analysis
Chile offers new data on food warning label efficacy
Illinois’ financial crisis could bring the state to a halt
The final 6 ‘Game of Thrones’ episodes might feel like a full season
New Season 8 Walking Dead trailer flashes forward in time
Mod turns ‘Counter-Strike’ into a ‘Tekken’ clone with fighting chickens
Meet Superman’s grandfather in new trailer for Krypton
Disney’s live-action Aladdin finally finds its stars
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