Graphite Bio be­gins SCD cell ther­a­py tri­al; Syn­thet­ic Bi­o­log­ic­s' fu­ture rests on VCN ac­qui­si­tion

Graphite Bio has dosed its first sick­le cell dis­ease pa­tient with its cell ther­a­py, the Bay Area biotech an­nounced this morn­ing.

In its proof-of-con­cept CEDAR tri­al, the biotech plans to en­roll 15 pa­tients and share ear­ly da­ta mid-2023.

Graphite joins late on the hunt for a long-term treat­ment and po­ten­tial cure for sick­le cell dis­ease. Ver­tex Phar­ma­ceu­ti­cals and CRISPR Ther­a­peu­tics could file for ap­proval for their ther­a­py, dubbed exa-cel, lat­er this year, and blue­bird bio said it plans to file a BLA in the first quar­ter of next year, though its pro­gram is un­der par­tial clin­i­cal hold for pe­di­atric pa­tients. Last month, Ed­i­tas al­so dosed its first pa­tient fol­low­ing a clin­i­cal hold.

Un­like Ver­tex/CRISPR and Ed­i­tas, which ac­ti­vate a fe­tal he­mo­glo­bin gene in pa­tients, Graphite hopes to cor­rect the mu­tat­ed adult he­mo­glo­bin. Graphite Bio’s stock $GRPH went up around 8% to­day, though it is down over 75% since it went pub­lic. — Lei Lei Wu

A Mary­land biotech’s pipeline gets over­hauled by Barcelona merg­er part­ner

Five months af­ter com­plet­ing its ac­qui­si­tion of VCN Bio­sciences, Syn­thet­ic Bi­o­log­ics’ clin­i­cal de­vel­op­ment path large­ly rests on the biotech it snapped up for less than $10 mil­lion up­front.

In­stead of fo­cus­ing on its mi­cro­bio­me and gas­troin­testi­nal pipeline, which in­clud­ed the mid-stage drug rib­axa­m­ase, the Mary­land biotech will move for­ward with the on­colyt­ic ade­n­ovirus plat­form from its Barcelona-based merg­er part­ner, which is cen­tered around on­col­o­gy.

Steven Shall­cross

“We have made the strate­gic de­ci­sion to ra­tio­nal­ize our spend and pri­or­i­tize our on­col­o­gy pro­gram, which is ex­pect­ed to ex­tend cash run­way in­to the first quar­ter of 2024,” Syn­thet­ic CEO Steven Shall­cross said in the com­pa­ny’s quar­ter earn­ings up­date Thurs­day morn­ing.

By the end of this year, Syn­thet­ic ex­pects to start a Phase II study of VCN-01 in com­bo with chemo as a first-line ther­a­py in new­ly di­ag­nosed metasta­t­ic PDAC pa­tients and, in the sec­ond half of next year, kick off a Phase II/III clin­i­cal tri­al of the drug as an ad­junct to chemo in kids with ad­vanced retinoblas­toma. The Uni­ver­si­ty of Leeds will al­so help study the drug in brain tu­mors this year.

For now, the com­pa­ny still ex­pects da­ta from one of its in-house pro­grams, SYN-004, to read out in the sec­ond half of this year and then ini­ti­ate a sec­ond co­hort of the drug in al­lo-HCT pa­tients.  — Kyle LaHu­cik

Af­fimed’s AML plans in US axed

Af­fimed with­drew an IND that it sub­mit­ted to the FDA just two months ago. Af­ter the agency gave feed­back on the dose es­ca­la­tion study de­sign, the Hei­del­berg, Ger­many-based biotech de­cid­ed it was bet­ter to vol­un­tar­i­ly with­draw and in­stead fo­cus the drug’s clin­i­cal de­vel­op­ment out­side the US.

The bis­pe­cif­ic, known as AFM28, tar­gets CD16A on NK cells and macrophages and CD123 on leukemic blasts and leukemic stem cells. The drug will now en­ter hu­man tri­als in the first half of 2023, based on the US with­draw­al, the biotech said in its quar­ter­ly earn­ings up­date Thurs­day.

The orig­i­nal plan was to start a Phase I tri­al of the drug, test­ed as a monother­a­py, in pa­tients with re­lapsed/re­frac­to­ry acute myeloid leukemia, the biotech said in June. The goal was to al­so test it in com­bi­na­tion with al­lo­gene­ic NK cell ther­a­py once a start­ing dose had been de­ter­mined. — Kyle LaHu­cik

Pi­o­neer­ing Click Chem­istry in Hu­mans

Reimagining cancer treatments

Cancer is a leading cause of death worldwide, accounting for nearly 10 million deaths in 2020, which is nearly one in six deaths. Recently, we have seen incredible advances in novel cancer therapies such as immune checkpoint inhibitors, cell therapies, and antibody-drug conjugates that have revamped cancer care and improved survival rates for patients.

Despite this significant progress in therapeutic targeting, why are we still seeing such a high mortality rate? The reason is that promising therapies are often limited by their therapeutic index, which is a measure of the effective dose of a drug, relative to its safety. If we could broaden the therapeutic indices of currently available medicines, it would revolutionize cancer treatments. We are still on the quest to find the ultimate cancer medicine – highly effective in several cancer types, safe, and precisely targeted to the tumor site.

Justin Klee (L) and Joshua Cohen, Amylyx co-CEOs (Cody O'Loughlin/The New York Times; courtesy Amylyx)

Ad­vo­cates, ex­perts cry foul over Amy­lyx's new ALS drug, cit­ing is­sues with price, PhI­II com­mit­ment

Not 24 hours after earning the first ALS drug approval in five years, Amylyx Pharmaceuticals’ Relyvrio is already drawing scrutiny. And it’s coming from multiple fronts.

In an investor call Friday morning, Amylyx revealed that it would charge about $158,000 per year, a price point that immediately drew backlash from ALS advocates and some outside observers. The cost reveal had been highly anticipated in the immediate hours after Thursday evening’s approval, though Amylyx only teased Relyvrio would cost less than previously approved drugs.

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Land­mark Amy­lyx OK spurs de­bate; Some... pos­i­tive? Alzheimer's da­ta; Can­cer tri­al bot­tle­neck; Sanofi's CRISPR bet; 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.

After brief stops in Paris and Boston, John Carroll and the Endpoints crew are staying on the road in October with their return for a live/streaming EUBIO22 in London. The hybrid event fireside chats and panels on mRNA, oncology and the crazy public market. We hope you can join him there.

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Joshua Cohen (L) and Justin Klee, Amylyx co-CEOs

Up­dat­ed: Af­ter long and wind­ing road, FDA ap­proves Amy­lyx's ALS drug in vic­to­ry for pa­tients and ad­vo­ca­cy groups

For just the third time in its 116-year history, the FDA has approved a new treatment for Lou Gehrig’s disease, or ALS.

US regulators gave the thumbs-up to the drug, known as Relyvrio, in a massive win for patients and their families. The approval, given to Boston-area biotech Amylyx Pharmaceuticals, comes after two years of long and contentious debates over the drug’s effectiveness between advocacy groups and FDA scientists, following the readout of a mid-stage clinical trial in September 2020.

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Nooman Haque, head of life sciences and healthcare at Silicon Valley Bank, and John Carroll

I’m head­ed to Lon­don soon for #EU­BIO22. Care to join me?

It was great getting back to a live ESMO conference/webinar in Paris followed by a live pop-up event for the Endpoints 11 in Boston. We’re staying on the road in October with our return for a live/streaming EUBIO22 in London.

Silicon Valley Bank’s Nooman Haque and I are once again jumping back into the thick of it with a slate of virtual and live events on October 12. I’ll get the ball rolling with a virtual fireside chat with Novo Nordisk R&D chief Marcus Schindler, covering their pipeline plans and BD work.

#AAO22: J&J’s first look at com­mon eye dis­ease port­fo­lio pads the case for PhII of gene ther­a­py

CHICAGO — While the later-stage drug developers in the geographic atrophy field are near the finish line, Johnson & Johnson’s Janssen is taking a more deliberate route, with a treatment that it hopes to be a one-time fix.

The Big Pharma will take its Hemera Biosciences-acquired gene therapy into a Phase II study later this year in patients with GA, a common form of age-related macular degeneration that impacts about five million people worldwide. To get there, Janssen touted early-stage safety data at the American Academy of Ophthalmology annual conference Saturday morning, half a day after competitors Apellis and Iveric Bio revealed their own more-detailed Phase III analyses.

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Up­dat­ed: Al­ny­lam re­in­forces APOL­LO-B patisir­an da­ta be­fore head­ing to the FDA

Weeks after uncorking some mostly positive data for patisiran in transthyretin-mediated (ATTR) amyloidosis with cardiomyopathy, Alnylam is bolstering its package with new exploratory and subgroup data before shipping it off to regulators.

The RNAi drug maintained “generally consistent” benefits in efficacy and quality of life across several prespecified subgroups at month 12, Alnylam announced on Friday afternoon, including age, baseline tafamidis use, ATTR amyloidosis type, baseline six-minute walk test score and others.

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Jerome Durso, Intercept Pharmaceuticals CEO

In­ter­cep­t's OCA fails a PhI­II NASH tri­al, rais­ing fresh doubts about its years­long quest for an OK

Intercept Pharmaceuticals has run into another big setback in its yearslong quest to win an approval for OCA in NASH. The biotech put out word Friday morning that its Phase III REVERSE study failed the primary endpoint for the liver disease, sending its share price into a tailspin.

There was no significant improvement in fibrosis among the patients suffering from cirrhosis who were treated with obeticholic acid, with investigators hunting for a minimum 1-stage histological improvement in the disease after 18 months of therapy.

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Bo Cumbo, new Solid Bio CEO

Sol­id Bio gets a new CEO, $75M cash and drops lead drug as big in­vestors ju­ry-rig a merg­er deal

Three months after Endpoints News broke the story that gene therapy outfit AavantiBio had gutted its CMC group in a reorganization, the biotech’s CEO has helped engineer a merger with struggling penny stock player Solid Bio. And he’s going to remain in charge of the combined operation, as Solid founder Ilan Ganot steps aside.

The merger news this morning features some high-profile investors.

Perceptive Advisors, RA Capital Management and Bain Capital Life Sciences are leading a $75 million raise to add to the pool of cash Solid will have after the tie-up. That will leave Solid $215 million in cash as Bain’s Adam Koppel jumps on the board — enough to pay for ops and get through some key data milestones on their way into 2025.

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