James Peyer, Cambrian

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.”

Late Fri­day ap­proval; Trio of biotechs wind down; Stem cell pi­o­neer finds new fron­tier; Biotech icon to re­tire; 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.

I hope your weekend is off to a nice start, wherever you are reading this email. As for me, I’m trying to catch the tail of the Lunar New Year festivities.

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Am­gen lays off about 300 work­ers, cit­ing 'in­dus­try head­wind­s'

Amgen has laid off about 300 employees, a company spokesperson confirmed to Endpoints News via email Sunday night.

Employees posted to LinkedIn in recent days about layoffs hitting Amgen last week. The Thousand Oaks, CA-based biopharma, which employs about 24,000 people, said the reduction “mainly” impacted US-based workers on its commercial team.

Drug developers of all sizes, including small upstarts and pharma giants, have let employees go in recent months as the biopharma market drags through a quarters-long winter doldrum.

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Boehringer In­gel­heim touts pre­ven­tion re­sults in rarest form of pso­ri­a­sis

Boehringer Ingelheim uncorked some positive results suggesting that Spevigo can help prevent flare-ups in patients with a severe form of psoriasis, months after the drug was approved to treat existing flares.

Spevigo, an IL-36R antibody also known as spesolimab, met its primary and a key secondary endpoint in the Phase IIb EFFISAYIL 2 trial in patients with generalized pustular psoriasis (GPP), Boehringer announced on Monday. While the company is keeping the hard numbers under wraps until later this year, it said in a news release that it anticipates sharing the results with regulators.

As­traZeneca, No­vo Nordisk and Sanofi score 340B-re­lat­ed ap­peals court win over HHS

AstraZeneca, Novo Nordisk, and Sanofi won an appeals court win on Monday, as the US Court of Appeals for the Third Circuit found that the companies cannot be forced to provide 340B-discounted drugs purchased by hospitals from an unlimited number of community and specialty pharmacies.

“Legal duties do not spring from silence,” the decision says as the court makes clear that the federal government’s interpretation of the “supposed requirement” that the 340B program compels drugmakers to supply their discounted drugs to an unlimited number of contract pharmacies is not correct, noting:

Ap­peals court toss­es J&J's con­tro­ver­sial 'Texas two-step' bank­rupt­cy case

A US appeals court has ruled against Johnson & Johnson’s use of bankruptcy to deal with mounting talc lawsuits, deciding that doing so would “create a legal blind spot.”

The Third Circuit Court of Appeals reversed a previous bankruptcy court decision on Monday, calling for the dismissal of a Chapter 11 filing by J&J’s subsidiary LTL Management.

Faced with more than 38,000 lawsuits alleging its talc-based products caused cancer, J&J spun its talc liabilities into a separate company called LTL Management back in October 2021 and filed for bankruptcy, a controversial move colloquially referred to as a “Texas two-step” bankruptcy. Claimants argued that the strategy is a misuse of the US bankruptcy code — and on Monday, a panel of judges agreed.

Chad Mirkin, Flashpoint co-founder

‘The field is at a flash­point’: New Chad Mirkin-found­ed biotech hopes to make more ef­fec­tive can­cer vac­cines

Following the success of the mRNA Covid vaccines, cancer vaccines are seeing renewed interest after years of middling results. But a group of researchers suggests that more attention needs to be paid not to what goes into those vaccines, but how the parts are put together.

In a recent paper published in Nature Biomedical Engineering, researchers led by Northwestern University’s Chad Mirkin describe how the placement of different antigens in a cancer vaccine impacts its efficacy. The paper builds on past work done by Mirkin’s lab that suggests the structure, or how the parts of a vaccine are arranged, impact a vaccine’s efficacy, not just its components.

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#JPM23: Reg­u­la­to­ry un­cer­tain­ty? What about M&A? Da­ta rule? Alessan­dro Masel­li and John Car­roll take out their crys­tal balls

Endpoints editor and founder John Carroll sat down the Catalent CEO Alessandro Maselli to talk about what’s ahead in 2023. Right or wrong, this covers all the big issues faced by biopharma. This transcript has been edited for brevity and clarity.

John Carroll:

I think 2022 had to be one of the worst years ever for crystal balls. You went into 2022 thinking all sorts of nice things about what was ahead, not thinking about a European land war, maybe not thinking that the Federal Reserve was going to be jacking up interest rates as fast as they could to get ahead of inflation. Just a tremendous number of macroeconomic issues that were out there. The sudden and complete collapse of support on the markets in Nasdaq for biotech. A lot of darlings in the industry that had been out there for a while suddenly found themselves moving from a really hot market to a really cold market all of a sudden and had to make a lot of different changes in terms of strategizing.

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Richard Gonzalez, AbbVie CEO (Chris Kleponis/picture-alliance/dpa/AP Images)

Up­dat­ed: $100B+ in sav­ings? Why the in­com­ing Hu­mi­ra biosim­i­lars will take time to catch on

The 20-year reign of AbbVie’s best-selling biologic of all time — the autoimmune disease biologic Humira (adalimumab) that has brought in upwards of $200 billion during its monopoly — is coming to an end tomorrow with the launch of Amgen’s biosimilar Amjevita.

The launch comes more than four years after Europe saw the exact same competition, leading to steep discounts in price, higher uptake, and big cost savings across the board.

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A new Genentech 'MS Visibility' campaign video features Black women living with or connected to MS talking about their experiences. (Genentech)

Roche’s Genen­tech de­buts next it­er­a­tion of MS cam­paign, high­lights ex­pe­ri­ences in Black com­mu­ni­ty

Roche’s Genentech is tackling diversity in multiple sclerosis again, this time with a focus on the Black community. Its “MS Visibility” effort, debuted in 2021, is now adding to the awareness campaign with new work that includes a set of videos featuring discussions among Black women and healthcare professionals.

“They’re incredibly inspiring Black women living with or connected to MS and they’re having just honest conversation about their experience and the unique barriers that their community faces,” said Jennifer Kim, head of neuroimmunology at Genentech marketing.

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