James Peyer, Cambrian

UP­DAT­ED: Can a cell ther­a­py treat mus­cu­lar dy­s­tro­phy? A Ger­man bil­lion­aire's an­ti-ag­ing start­up is try­ing to find out

Gene ther­a­py com­pa­nies have faced huge hur­dles try­ing to de­liv­er healthy genes in­to mus­cu­lar dy­s­tro­phy pa­tients’ mus­cle cells, so here’s an idea: Why don’t we just re­place the mus­cle cells them­selves?

Over the last two years, Vi­ta Ther­a­peu­tics has been ex­plor­ing that pos­si­bil­i­ty, build­ing on ear­ly stem cell work from Johns Hop­kins pro­fes­sors Gab­sang Lee and Kathryn Wag­n­er. And on Tues­day they an­nounced a $32 mil­lion Se­ries A to be­gin to move their first ther­a­py in­to the clin­ic, where they hope it will help re­build mus­cle in pa­tients with a type of dy­s­tro­phy that af­flicts the arms and legs.

Chris­t­ian Anger­may­er

The start­up is the sec­ond port­fo­lio com­pa­ny un­veiled by Cam­bri­an Bio­phar­ma, the an­ti-ag­ing port­fo­lio com­pa­ny launched last year by ATAI co-founder Chris­t­ian Anger­may­er. (The first was the now pub­lic can­cer start­up, Sen­sei Bio). Cam­bri­an is tak­ing the ap­proach ATAI took to psy­che­del­ic ther­a­pies, bring­ing a broad range of re­lat­ed com­pa­nies un­der a sin­gle roof, and ap­ply­ing it an­ti-ag­ing ther­a­pies, said CEO and co-founder James Pey­er.

They’ve in­vest­ed in sev­er­al tech­nolo­gies that could slow or re­verse the ef­fects of ag­ing, such as boost­ing mi­to­chon­dria or re­gen­er­at­ing tis­sue. But they’re ap­ply­ing them first in dis­eases where they could have a more im­me­di­ate im­pact.

For mus­cu­lar dy­s­tro­phies, Vi­ta will try to take the same strat­e­gy that CRISPR Ther­a­peu­tics and oth­er gene edit­ing com­pa­nies have used for ge­net­ic blood dis­or­ders such as sick­le cell dis­ease. This is not a sim­ple task: Doc­tors have decades of ex­pe­ri­ence re­mov­ing and re­plac­ing a pa­tient’s blood cells — and over a decade of ex­pe­ri­ence en­gi­neer­ing those cells — but it’s not as if you can just ex­tract all of a pa­tient’s mus­cle cells and then put them back.

Ac­cord­ing­ly, com­pa­nies such as AskBio and Sarep­ta de­vised ways of cor­rect­ing mus­cle cells in vi­vo, strap­ping healthy genes on­to (most­ly) harm­less virus­es and ad­min­is­ter­ing them to pa­tients as gene ther­a­py. That process, though, has faced a num­ber of hur­dles: The genes in­volved don’t fit well on­to the virus­es used for gene ther­a­py and the virus­es don’t reach all the mus­cle. Ear­ly clin­i­cal tri­als yield­ed mixed re­sults.

Pey­er said Vi­ta doesn’t need to re­place all a pa­tient’s mus­cle cells. In­stead, they’ll take cells from a pa­tient’s blood, turn them in­to stem cells in the lab, use CRISPR to cor­rect the dis­ease-caus­ing mu­ta­tions and fi­nal­ly ma­nip­u­late them in­to a class of cells called myosatel­lite cells. These cells are re­spon­si­ble for pro­duc­ing ma­ture mus­cle cells.

Vi­ta, led by first time CEO Doug Falk and Hop­kins pro­fes­sor Pe­ter An­der­son as CSO, be­lieves that they can in­ject these cells in­to dif­fer­ent parts of the body, where they will pro­duce healthy mus­cle cells that can re­place the dam­aged ones.

“We have this amaz­ing da­ta in mice that they rein­te­grate in­to the mus­cles,” Pey­er told End­points News. “And in the mice can im­prove time on a tread­mill or grip strength… It’s re­al­ly awe­some func­tion­al out­comes.”

Vi­ta will look to start a clin­i­cal tri­al next year for limb gir­dle dy­s­tro­phy, where they will com­pete with a gene ther­a­py from Sarep­ta. But even if the ther­a­py works, Pey­er ac­knowl­edged there will be steep lim­i­ta­tions.

For one, it’s not clear how long the cells will last. And al­though the ap­proach might work to re­plen­ish mus­cle cells in the limbs, there are no myosatel­lite cells in the heart. For many pa­tients with mus­cu­lar dy­s­tro­phy, in­clud­ing those with Duchenne, it’s de­gen­er­a­tion in these vi­tal mus­cles that ul­ti­mate­ly brings the worst symp­toms and death.

“Yeah,” Pey­er said, “That’s why we’re not start­ing im­me­di­ate­ly DMD.”

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

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.

Matt Gline (L) and Pete Salzmann

UP­DAT­ED: Roivant bumps stake in Im­muno­vant with a $200M deal. But with M&A off the ta­ble, shares crater

Roivant has worked out a deal to pick up a chunk of stock in its majority-owned sub Immunovant $IMVT, but the stock buy falls far short of its much-discussed thoughts about buying out all of the 43% of shares it doesn’t already own.

Roivant, which recently inked a SPAC move to the market at a $7 billion-plus valuation, has forged a deal to boost its ownership in Immunovant by 6.3 points, ending with 63.8% of the biotech’s stock following a $200 million injection. That cash will bolster Immunovant’s cash reserves, giving it a $600 million war chest to fund a slate of late-stage studies for its big drug: the anti-FcRn antibody IMVT-1401.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 113,300+ biopharma pros reading Endpoints daily — and it's free.

Sanofi preps a multi­bil­lion-dol­lar buy­out of an mR­NA pi­o­neer af­ter falling be­hind in the race for a Covid-19 jab — re­port

It looks like 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 a whopping $425 million in an upfront and equity payment to help guide the pharma giant to the promised land of mRNA vaccines for Covid-19, Sanofi is reportedly ready to close the deal with a buyout.

Translate’s stock $TBIO soared 78% after the market closed Monday. A spokesperson for Sanofi declined to comment on the report, telling Endpoints News that the company doesn’t comment on market rumors.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 113,300+ biopharma pros reading Endpoints daily — and it's free.

UP­DAT­ED: Watch out Glax­o­SmithK­line: As­traZeneca's once-failed lu­pus drug is now ap­proved

Capping a roller coaster journey, AstraZeneca has steered its lupus drug anifrolumab across the finish line.

Saphnelo, as the antibody will be marketed, is the only treatment that’s been approved for systemic lupus erythematosus since GlaxoSmithKline’s Benlysta clinched an OK in 2011. The British drugmaker notes it’s also the first to target the type I interferon receptor.

Mirroring the population that the drug was tested on in late-stage trials, regulators sanctioned it for patients with moderate to severe cases who are already receiving standard therapy — setting up a launch planned for the end of August, according to Ruud Dobber, who’s in charge of AstraZeneca’s biopharmaceuticals business unit.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 113,300+ biopharma pros reading Endpoints daily — and it's free.

Not all mR­NA vac­cines are cre­at­ed equal. Does it mat­ter?; Neu­ro is back; Pri­vate M&A af­fair; 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.

As part of our broader and deeper drive, Endpoints has been pairing webinars with our special reports to cover more angles on a given topic. In conjunction with Max Gelman’s neuroscience feature, Kyle Blankenship moderated an insightful panel to discuss where the field is headed. You can register to watch it on demand here.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 113,300+ biopharma pros reading Endpoints daily — and it's free.

Bris­tol My­ers pulls lym­phoma in­di­ca­tion for Is­to­dax af­ter con­fir­ma­to­ry tri­al falls flat

Amid an industrywide review of cancer drugs with accelerated approval, Bristol Myers Squibb had to make the tough call last month to yank an approval for leading I/O drug Opdivo after flopping a confirmatory study. Now, a second Bristol Myers drug is on the chopping block.

Bristol Myers has pulled aging HDAC inhibitor Istodax’s indication in peripheral T cell lymphoma after a Phase III confirmatory study for the drug flopped on its progression-free survival endpoint, the drugmaker said Monday.

Rick Pazdur (via AACR)

FDA's on­col­o­gy head Rick Paz­dur de­fends the ac­cel­er­at­ed ap­proval path­way, claim­ing it is 'un­der at­tack'

The FDA is sounding the alarm over its accelerated approval pathway as backlash continues over the recent nod in favor of Biogen’s Alzheimer’s drug Aduhelm, and an ODAC meeting on six such approvals that could potentially be pulled from the market — two of which already have.

“Do you think accelerated approval is under attack? I do,” Rick Pazdur, head of FDA’s Oncology Center of Excellence, said at a Friends of Cancer Research webinar on Thursday.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

FTC pulls re­main­ing case against Ab­b­Vie; New EU clin­i­cal tri­als sys­tem com­ing in 2022; Abing­worth bets big on CymaBay

The Federal Trade Commission on Friday withdrew its remaining case against AbbVie after the Supreme Court declined to review a lower court’s ruling.

The punt by SCOTUS means that while the Illinois pharma company illegally blocked patients’ access to lower-cost alternatives to its testosterone drug AndroGel, the FTC will no longer be able to return about $500 million directly to AndroGel consumers.

Alan Hirzel, Abcam

Drug sup­pli­er Ab­cam brings a long­time col­lab­o­ra­tor in house as part of $340M buy­out pact

BioVision has supplied Abcam with research tools since 1999, and now the two are making it official as part of a merger unveiled Monday.

Abcam will buyout BioVision as part of a $340 million acquisition deal to bring aboard the supplier’s biochemical and cell-based assays for biological research, as well as recombinant proteins, antibodies and enzymes.

The deal will give Abcam control of BioVision’s portfolio and allow for both the expansion of research existing areas of focus such as oncology, neuroscience and epigenetics and preparation to expand into new products. As a part of the deal, Abcam will develop and supply products and services to NKY, the previous owner of BioVision and receive support for ongoing development and commercialization of in vitro diagnostic products.