Vivek Ra­maswamy ramps up biotech start­up #14 — this time work­ing on a gene ther­a­py for sick­le cell dis­ease and β-tha­lassemia

Vivek Ra­maswamy’s Roivant Sci­ences has nabbed its lat­est clin­i­cal-stage pro­gram for biotech start­up #14 — Aru­vant — and this time they’re go­ing up against a cou­ple of the lead­ers in the gene ther­a­py world.

Their new drug is now dubbed RVT-1801, and it has a fa­mil­iar pro­file, in­sert­ing a mod­i­fied fe­tal he­mo­glo­bin gene in­to au­tol­o­gous stem cells through a lentivi­ral ve­hi­cle in or­der to treat sick­le cell dis­ease and β-tha­lassemia.

Ac­cord­ing to the wheel­ing and deal­ing ex­ecs at Roivant, their pro­gram is “the on­ly known clin­i­cal-stage gene ther­a­py to de­liv­er the gene en­cod­ing fe­tal he­mo­glo­bin, which has been mod­i­fied to op­ti­mize oxy­gen car­ry­ing ca­pac­i­ty and an­ti-sick­ling prop­er­ties.”

The drug is com­ing from the lab of Punam Ma­lik, di­rec­tor of the Cincin­nati Com­pre­hen­sive Sick­le Cell Cen­ter at Cincin­nati Chil­dren’s, which re­ceived an undis­closed up­front pay­ment and a mile­stone sched­ule. Ma­lik is sched­uled to re­veal more about their work at the up­com­ing ASH con­fer­ence next Mon­day. 

And Ma­lik, who trained as a physi­cian in In­dia, was sold on Roivant’s strat­e­gy for de­vel­op­ing new treat­ments for pa­tients around the world.

Roivant is al­so es­tab­lish­ing a non-prof­it group to im­prove ac­cess to drugs for pa­tients in the de­vel­op­ing world.

“A crit­i­cal rea­son why we chose to work with Roivant on this pro­gram was their au­then­tic com­mit­ment to pa­tients glob­al­ly,” said the sci­en­tist.

But they won’t be op­er­at­ing alone. At least 3 oth­er biotechs are al­so spear­head­ing gene ther­a­pies for these two ail­ments.

Just weeks ago CRISPR Ther­a­peu­tics $CR­SP and its part­ners at Ver­tex got a green light to start Phase I/II clin­i­cal work on CTX001, al­so de­signed to spur the pro­duc­tion of fe­tal he­mo­glo­bin in pa­tients’ stem cells. Blue­bird bio $BLUE has al­so been work­ing on a gene ther­a­py for these con­di­tions, while Sanofi’s Biover­a­tiv $BI­IV and Sang­amo $SG­MO had their IND ap­proved for a ri­val gene ther­a­py a few months ago. Glob­al Blood Ther­a­peu­tics, mean­while, is look­ing for an OK on their new sick­le cell dis­ease drug vox­elo­tor while No­var­tis and oth­ers have their own new meds in the pipeline.

Ra­maswamy has raised more than $3 bil­lion to back his grow­ing sta­ble of com­pa­nies, which now em­ploy more than 750 sci­en­tists and ex­ecs and sup­port staff around the world. 

Im­age: Vivek Ra­maswamy at BI­IS18 in Shang­hai End­points News

Has the mo­ment fi­nal­ly ar­rived for val­ue-based health­care?

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.

Lat­est on ul­tra-rare dis­ease ap­proval; Pos­i­tive, if mixed, signs for Bio­gen's ALS drug; Clay Sie­gall finds a new job; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

Over the last four years, we’ve honored 80 women whose extraordinary accomplishments have changed the game in biopharma R&D. You can now nominate someone to be highlighted in this year’s special report. Details are here.

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FDA spells out how can­cer drug de­vel­op­ers can use one tri­al for both ac­cel­er­at­ed and full ap­provals

The FDA’s Oncology Center of Excellence has been a bright spot within the agency in terms of speeding new treatments to patients. That flexibility was on full display this morning as FDA released new draft guidance spelling out exactly how oncology drug developers can fulfill both the accelerated and full approval’s requirements with just a single randomized controlled trial.

While Congress recently passed legislation that will allow FDA to require confirmatory trials to be recruiting and ongoing prior to granting an accelerated approval, the agency is now making clear that the initial trial used to win the AA, if designed appropriately, can also serve as the trial for converting the accelerated approval into a full approval.

Clay Siegall, Morphimmune CEO

Up­dat­ed: Ex-Seagen chief Clay Sie­gall emerges as CEO of pri­vate biotech

Clay Siegall will be back in the CEO seat, taking the helm of a private startup working on targeted cancer therapies.

It’s been almost a year since Siegall resigned from Seagen, the biotech he co-founded and led for more than 20 years, in the wake of domestic violence allegations by his then-wife. His eventual successor, David Epstein, sold the company to Pfizer in a $43 billion deal unveiled last week.

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

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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No­vo Nordisk oral semaglu­tide tri­al shows re­duc­tion in blood sug­ar, plus weight loss

Novo Nordisk is testing higher levels of its oral version of its GLP-1, semaglutide, and its type 2 diabetes trial results released today show reductions in blood sugar as well as weight loss.

In the Phase IIIb trial, Novo compared its oral semaglutide in 25 mg and 50 mg doses with the 14 mg version that’s currently the maximum approved dose. The trial looked at how the doses compared when added to a stable dose of one to three oral antidiabetic medicines in people with type 2 diabetes who were in need of an intensified treatment.

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Ly­me vac­cine test com­ple­tion is pushed back by a year as Pfiz­er, Val­ne­va say they'll ad­just tri­al

Valneva and Pfizer have adjusted the end date for the Phase III study of their investigational Lyme disease vaccine, pushing it back by a year after issues at a contract researcher led to thousands of US patients being dropped from the test.

In a March 20 update to, Valneva and Pfizer moved the primary completion date on the trial, called VALOR, from the end of 2024 to the end of 2025.

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Stuart Peltz, former PTC Therapeutics CEO

Stu­art Peltz re­signs as PTC Ther­a­peu­tics CEO af­ter 25 years

Stuart Peltz, the longtime CEO of PTC Therapeutics who’s led the rare disease drug developer since its founding 25 years ago, is stepping down.

Succeeding him in the top job is Matthew Klein, who joined PTC in 2019 and was promoted to chief operating officer in 2022. In a call with analysts, he said the CEO transition has been planned for “quite some time” — in fact, as part of it, he gave the company’s presentation at the JP Morgan healthcare conference earlier this year.

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Bet­ter Ther­a­peu­tics cuts 35% of staff while await­ing dig­i­tal ther­a­peu­tic ap­proval

Digital therapeutics company Better Therapeutics announced on Thursday that it’s cutting 35% of its staff as it awaits FDA clearance for its first product.

The company, which launched eight years ago, is one of a growing group of companies seeking a digital alternative to traditional medicine. The space saw a record $7.5 billion in investments in 2021, according to Chris Dokomajilar at DealForma, with uses spanning ADHD, PTSD and other indications. However, private insurers have been slow to hop on board.