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 to 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.”

A new era of treat­ment: How bio­mark­ers are chang­ing the way we think about can­cer

AJ Patel was recovering from a complicated brain surgery when his oncologist burst into the hospital room yelling, “I’ve got some really great news for you!”

For two years, Patel had been going from doctor to doctor trying to diagnose his wheezing, only to be dealt the devastating news that he had stage IV lung cancer and only six months to live. And then they found the brain tumors.

“What are you talking about?” Patel asked. He had never seen an oncologist so happy.

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Mihael Polymeropoulos, Vanda Pharmaceuticals CEO

Phar­ma com­pa­ny con­tin­ues its FDA law­suit spree, this time af­ter agency de­nies fast-track des­ig­na­tion

Vanda Pharmaceuticals is making a name for itself, at least in terms of suing the FDA.

The DC-headquartered firm on Monday filed its latest suit against the agency, with the company raising concerns over the FDA’s failure to grant a fast track designation for Vanda’s potential chronic digestive disorder drug tradipitant, which is a neurokinin 1 receptor antagonist.

Specifically, Vanda said FDA’s “essential point” in its one-page denial letter on the designation pointed to “the lack of necessary safety data,” which was “inconsistent with the criteria for … Fast Track designation.”

Mod­er­na seeks to dis­miss Al­ny­lam suit over Covid-19 vac­cine com­po­nent, claim­ing wrong venue

RNAi therapeutics juggernaut Alnylam Pharmaceuticals made a splash in March when it sued and sought money from both Pfizer and Moderna regarding their use of Alnylam’s biodegradable lipids, which Alnylam claims have been integral to the way both companies’ mRNA-based Covid-19 vaccines work.

But now, Moderna lawyers are firing back, telling the same Delaware district court that Alnylam’s claims can only proceed against the US government in the Court of Federal Claims because of the way the company’s contract is set up with the US government. The US has spent almost $10 billion on Moderna’s Covid-19 vaccine so far.

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Cracks in the fa­cade: Is phar­ma's pan­dem­ic ‘feel good fac­tor’ wan­ing?

The discordant effects of the Covid-19 pandemic on pharma reputation continues. While the overall industry still retains a respectable halo from its Covid-19 quick response and leadership, a new patient group study reveals a different story emerging in the details.

On one hand, US patient advocacy groups rated the industry higher-than-ever overall. More than two-thirds (67%) of groups gave the industry a thumbs up for 2021, a whopping 10 percentage point increase over the year before, according to the PatientView annual study, now in its 9th year.

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Alan Wise (L) and Peter Trill (Duke Street Bio)

They sold their last biotech to Mer­ck. Now they're back with a PARP out­fit named af­ter a Lon­don street

In 2016, Peter Trill and Alan Wise sold IOmet Pharma (an I/O outfit as the name suggests) to Merck for $400 million.

Now, some six years later, the duo has returned with another cancer biotech, Duke Street Bio, that emerged from stealth Tuesday. Duke Street Bio, named for the street where it’s located in London, is making its public debut as a next-gen PARP player, hoping to break into a field that already has a number of Big Pharma competitors.

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Michael Corbo, Pfizer CDO of inflammation & immunology

UP­DAT­ED: Plan­ning ahead for crowd­ed ul­cer­a­tive col­i­tis mar­ket, Pfiz­er spells out PhI­II da­ta on $6.7B Are­na drug

Pfizer has laid out the detailed results behind its boast that etrasimod — the S1P receptor modulator at the center of its $6.7 billion buyout of Arena Pharma — is the winner of the class, potentially leapfrogging an earlier entrant from Bristol Myers Squibb.

Pivotal data from the ELEVATE program in ulcerative colitis — which consists of two Phase III trials, one lasting 52 weeks and the other just 12 weeks — illustrate an “encouraging balance of efficacy and safety,” according to Michael Corbo, chief development officer of inflammation & immunology at Pfizer. The company is presenting the results as a late breaker at Digestive Disease Week.

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Robert Califf (Michael Brochstein/Sipa USA via AP Images)

House Re­pub­li­cans at­tack Chi­na-on­ly da­ta in FDA sub­mis­sions, seek new in­ves­ti­ga­tion in­to re­search in­spec­tions

Three Republican representatives are calling on the FDA to take a closer look at the applications including only clinical data from China.

The letter to FDA commissioner Rob Califf late last week comes as the agency recently rejected Eli Lilly’s anti-PD-1 antibody, which attempted to bring China-only data but ran into a bruising adcomm that may crush the hopes of any other companies looking to bring cheaper follow-ons based only on Chinese data.

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Amid mon­key­pox fears, biotechs spring to ac­tion; Mod­er­na’s CFO trou­ble; Cuts, cuts every­where; Craft­ing the right pro­teins; 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.

It’s always a bittersweet moment saying goodbye, but as Josh Sullivan goes off to new adventures we are grateful for the way he’s built up the Endpoints Manufacturing section — which the rest of the team will now carry forward. If you’re not already, this may be a good time to sign up for your weekly dose of drug manufacturing news. Thank you for reading and wish you a restful weekend.

Co­pay coupons gone wrong, again: Pfiz­er pays al­most $300K to set­tle com­plaints in four states

Pfizer has agreed to pay $290,000 to settle allegations of questionable copay coupon practices in Arizona, Colorado, Kansas, and Vermont from 2014 to 2018.

While the company has not admitted any wrongdoing as part of the settlement, Pfizer has agreed to issue restitution checks to about 5,000 consumers.

A Pfizer spokesperson said the company has “enhanced its co-pay coupons to alleviate the concerns raised by states and agreed to a $30,000 payment to each.”