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.”

Biotech Half­time Re­port: Af­ter a bumpy year, is biotech ready to re­bound?

The biotech sector has come down firmly from the highs of February as negative sentiment takes hold. The sector had a major boost of optimism from the success of the COVID-19 vaccines, making investors keenly aware of the potential of biopharma R&D engines. But from early this year, clinical trial, regulatory and access setbacks have reminded investors of the sector’s inherent risks.

RBC Capital Markets recently surveyed investors to take the temperature of the market, a mix of specialists/generalists and long-only/ long-short investment strategies. Heading into the second half of the year, investors mostly see the sector as undervalued (49%), a large change from the first half of the year when only 20% rated it as undervalued. Around 41% of investors now believe that biotech will underperform the S&P500 in the second half of 2021. Despite that view, 54% plan to maintain their position in the market and 41% still plan to increase their holdings.

David Livingston (Credit: Michael Sazel for CeMM)

Renowned Dana-Far­ber sci­en­tist, men­tor and bio­phar­ma ad­vi­sor David Liv­ingston has died

David Livingston, the Dana-Farber/Harvard Med scientist who helped shine a light on some of the key molecular drivers of breast and ovarian cancer, died unexpectedly last Sunday.

One of the senior leaders at Dana-Farber during his nearly half century of work there, Livingston was credited with shedding light on the genes that regulate cell growth, with insights into inherited BRCA1 and BRCA2 mutations that helped lay the scientific foundation for targeted therapies and earlier detection that have transformed the field.

No­vo CEO Lars Fruer­gaard Jør­gensen on R&D risk, the deal strat­e­gy and tar­gets for gen­der di­ver­si­ty

 

I kicked off our European R&D summit last week with a conversation involving Novo Nordisk CEO Lars Fruergaard Jørgensen. Novo is aiming to launch a new era of obesity management with a new approval for semaglutide. And Jørgensen had a lot to say about what comes next in R&D, how they manage risk and gender diversity targets at the trendsetting European pharma giant.

John Carroll: I’m here with Lars Jørgensen, the CEO of Novo Nordisk. Lars, it’s been a really interesting year so far with Novo Nordisk, right? You’ve projected a new era of growing sales. You’ve been able to expand on the GLP-1 franchise that was already well established in diabetes now going into obesity. And I think a tremendous number of people are really interested in how that’s working out. You have forecast a growing amount of sales. We don’t know specifically how that might play out. I know a lot of the analysts have different ideas, how those numbers might play out, but that we are in fact embarking on a new era for Novo Nordisk in terms of what the company’s capable of doing and what it’s able to do and what it wants to do. And I wanted to start off by asking you about obesity in particular. Semaglutide has been approved in the United States for obesity. It’s an area of R&D that’s been very troubled for decades. There have been weight loss drugs that have come along. They’ve attracted a lot of attention, but they haven’t actually ever gained traction in the market. My first question is what’s different this time about obesity? What is different about this drug and why do you expect it to work now whereas previous drugs haven’t?

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Man­u­fac­tur­ing woes for No­vavax’s Covid jab bad­ly dis­rupt plans for roll­out to the poor — re­port

Production problems at a Novavax facility in Maryland have led to delays in the Covax vaccine sharing program. Now, a shortage of 1 billion doses is expected, as the supplier tries to navigate producing a shot up to regulators’ standards, Politico reported Tuesday.

The company has run into trouble with the purity of the vaccine. Novavax has had trouble proving it can produce a shot consistently up to standards, and it has caused significant delays in the rollout to low- and middle-income countries. This follows several delays at Novavax that has put the executive crew on the defensive.

Sur­geons suc­cess­ful­ly at­tach pig kid­ney to a hu­man for the first time, us­ing tech from Unit­ed's Re­vivi­cor

In a first, researchers reportedly successfully transplanted a pig kidney into a human without triggering an immediate immune response this week. And the technology came from the biotech United Therapeutics.

Surgeons spent three days attaching the kidney to the patient’s blood vessels, but when all was said and done, the kidney appeared to be functioning normally in early testing, Reuters and the New York Times were among those to report. The kidney came from a genetically altered pig developed through United’s Revivicor unit.

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Michel Vounatsos, Biogen CEO (Credit: World Economic Forum/Valeriano Di Domenico)

Up­dat­ed: Bio­gen sells just $300K worth of Aduhelm in Q3, as ques­tions on long-term vi­a­bil­i­ty re­main

Barely anyone is accessing Biogen’s controversial Alzheimer’s treatment, with the company reporting just $0.3 million in Aduhelm sales in the third quarter. Although investors will be looking to the longer term, when CMS may decide to cover the drug and open the floodgates for more reimbursement, use of the drug is currently stalled.

Since June, when the FDA first signed off on the drug under its accelerated pathway, Biogen said Wednesday that it’s sold a total of $2 million worth of Aduhelm. That’s a far cry from the peak Wall Street sales estimate of about $9 billion in annual sales.

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With hun­dreds of mil­lions spent on failed ac­cel­er­at­ed ap­provals, re­searchers call for faster FDA with­drawals

Between 2017 and 2019, Medicare spent more than $220 million on cancer drugs for which the indications were either voluntarily pulled by their applicants or FDA’s oncology adcomm had recommended their withdrawal.

That kind of massive spending on cancer drugs lacking overall survival benefit is wasteful and risks harming people’s health, a research letter published in JAMA Internal Medicine on Monday said. The researchers from Harvard and the London School of Economics called on the FDA to move faster in both requiring timely postmarketing trials and accelerating the speed in pulling these dangling approvals when the confirmatory studies fail.

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Bill Gates at the Global Investment Summit in London, Oct. 19, 2021 (Leon Neal/Pool via AP Images)

Gates Foun­da­tion pledges $120M to ramp up gener­ic sup­ply of Mer­ck­'s Covid-19 pill while ac­tivists blast Pfiz­er's dis­pro­por­tion­ate pow­er

Merck’s molnupiravir may not be officially authorized anywhere in the world yet, but who will get access to it has shaped up to be a huge issue. The Bill & Melinda Gates Foundation is now stepping up to ensure lower-income countries won’t be left behind — and calling on others to follow its lead.

The oral antiviral pill, which was shown to dramatically cut the risk of severe Covid-19 disease and death in a Phase III study, is the latest rallying symbol in the battle against not just the coronavirus but the inequality it’s exposed.

Raymond Stevens, ShouTi Pharma CEO

A new Schrödinger-backed start­up emerges from the sci­en­tist who mapped the first hu­man GPCR

One of the most popular targets in drug development, representing about a third of existing drugs, are G-protein coupled receptors — the tiny but integral membrane proteins responsible for recognizing things like light, taste, smell, hormones and pain.

But due to challenges in mapping their structure, the protein family remains largely unexplored.

A slate of companies has emerged over the last few years to change that. If one can figure out the structure of these elusive membrane receptors, it might be possible to create small molecule drugs that overcome the limitations of, say, biologic and peptide therapies. That promise is what gets serial entrepreneur Raymond Stevens out of bed in the morning.