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

Tower Bridge in London [Shutterstock]

#UK­BIO19: Join GSK’s Hal Bar­ron and a group of top biotech ex­ecs for our 2nd an­nu­al biotech sum­mit in Lon­don

Over the past 10 years I’ve made a point of getting to know the Golden Triangle and the special role the UK biopharma industry plays there in drug development. The concentration of world class research institutes, some of the most accomplished scientists I’ve ever seen at work and a rising tide of global investment cash leaves an impression that there’s much, much more to come as biotech hubs are birthed and nurtured.

Deborah Dunsire. Lundbeck

UP­DAT­ED: Deb­o­rah Dun­sire is pay­ing $2B for a chance to leap di­rect­ly in­to a block­buster show­down with a few of the world's biggest phar­ma gi­ants

A year after taking the reins as CEO of Lundbeck, Deborah Dunsire is making a bold bid to beef up the Danish biotech’s portfolio of drugs in what will likely be a direct leap into an intense rivalry with a group of giants now carving up a growing market for new migraine drugs.

Bright and early European time Monday morning the company announced that it will pay up to about $2 billion to buy Alder, a little biotech that is far along the path in developing a quarterly IV formulation of a CGRP drug aimed at cutting back the number of crippling migraines patients experience each month. In a followup call, Dunsire also noted that the company will likely need 200 to 250 reps for this marketing task on both sides of the Atlantic. And analysts were quick to note that the dealmaking at Lundbeck isn’t done, with another $2 billion to $3 billion available for more deals to beef up the pipeline.

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It’s fi­nal­ly over: Bio­gen, Ei­sai scrap big Alzheimer’s PhI­I­Is af­ter a pre­dictable BACE cat­a­stro­phe rais­es safe­ty fears

Months after analysts and investors called on Biogen and Eisai to scrap their BACE drug for Alzheimer’s and move on in the wake of a string of late-stage failures and rising safety fears, the partners have called it quits. And they said they were dropping the drug — elenbecestat — after the independent monitoring board raised concerns about…safety.

We don’t know exactly what researchers found in this latest catastrophe, but the companies noted in their release that investigators had determined that the drug was flunking the risk/benefit analysis.

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Scott Gottlieb, AP Images

Scott Got­tlieb has a new board po­si­tion to add to the re­sume — and this one is fo­cused on a fa­vorite sub­ject

Scott Gottlieb has another position to add to his lengthy roster of boards and advisory roles in the wake of his departure from the helm of the FDA.

He’ll be joining the advisory board of FasterCures, a think tank which former junk bond king Michael Milken set up to help drive more drugs to the market, looking to accelerate drug R&D. That’s a subject close to the heart of Gottlieb, who blazed a trail at the FDA focused on hustling up the process. That helped endear him to the industry, making him one of the most popular commissioners in FDA history.

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Karyopharm lines up $150 mil­lion cash in­jec­tion to back con­tro­ver­sial drug launch

Karyopharm has entered into a royalty agreement worth up to $150 million to back the launch of their multiple myeloma drug — recently approved by the FDA over the objections of a majority of the agency’s outside experts.

The deal with HealthCare Royalty Partners, worth $75 million now and $75 million once certain regulatory and commercial milestones have been reached, will fund the commercialization of Karyopharm’s oral SINE compound Xpovio (selinexor) for patients with multiple myeloma who have already had at least four prior therapies. The money will help Karyopharm as it markets its newly approved drug and pushes through clinical trials testing the drug on refractory multiple myeloma patients with one to three therapies and patients with treatment-resistant diffuse large B-cell lymphoma. It will give Karyopharm a cushion through mid-2021.

Af­ter a run of CT­LA-4 com­bo fail­ures, sci­en­tists spot­light a way to make it work — in se­lect pa­tients

CTLA-4/PD-(L)1 combinations have been one of the El Dorados of oncology, its promise forever behind that next hill but apparently unattainable after a series of pivotal clinical failures. But researchers at New York’s Memorial Sloan Kettering Cancer Center and the Technical University of Munich think they may know how to fix what’s wrong and boost the drive to next-gen cancer combos.

In a preclinical animal research program, researchers found that within a cell, checkpoints rely on a specific molecule — RNA-sensing molecule RIG-I — to work. If that sounds familiar, it’s because it has already been identified as a target for boosting immune responses and was subject to at least one Phase I/II trial. Pfizer in December allied itself with Kineta with $15 million upfront and $505 million in potential milestones to develop RIG-I immunotherapies, and three years ago Merck purchased German upstart Rigontec for $137 million upfront and over $400 million in potential milestones for the same purpose.

Pur­due Phar­ma files for bank­rupt­cy as first step in $10B opi­oid set­tle­ment

It’s settled. Purdue Pharma has filed for bankruptcy as part of a deal that would see the OxyContin maker hand over $10 billion in cash and other contributions to mitigate the opioid crisis — without acknowledging any wrongdoing in the protracted epidemic that’s resulted in hundreds of thousands of deaths.

The announcement came two weeks after news of a proposed settlement surfaced and largely confirm what’s already been reported.

Lisa M. DeAngelis, MSKCC

MSK picks brain can­cer ex­pert Lisa DeAn­ge­lis as its next CMO — fol­low­ing José Basel­ga’s con­tro­ver­sial ex­it

It’s official. Memorial Sloan Kettering has picked a brain cancer expert as its new physician-in-chief and CMO, replacing José Baselga, who left under a cloud after being singled out by The New York Times and ProPublica for failing to properly air his lucrative industry ties.

His replacement, who now will be in charge of MSK’s cutting-edge research work as well as the cancer care delivered by hundreds of practitioners, is Lisa M. DeAngelis. DeAngelis had been chair of the neurology department and co-founder of MSK’s brain tumor center and was moved in to the acting CMO role in the wake of Baselga’s departure.

Penn team adapts CAR-T tech, reengi­neer­ing mouse cells to treat car­diac fi­bro­sis

After establishing itself as one of the pioneer research centers in the world for CAR-T cancer therapies, creating new attack vehicles to eradicate cancer cells, a team at Penn Medicine has begun the tricky transition of using the basic technology for heart repair work.

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