David Southwell (L) and Christoph Westphal

Har­vard spin­out kicks off 2021 with a crossover round and sights set on the clin­ic

Sev­er­al months af­ter strik­ing an al­liance with No­var­tis, TCR ther­a­py-fo­cused TScan Ther­a­peu­tics has reeled in a crossover round that should hold it over for the next two years as it eyes a pub­lic de­but.

The Christoph West­phal port­fo­lio com­pa­ny had been ar­rang­ing the crossover for the last few months, CEO David South­well said. Just be­fore Christ­mas, they nailed down what he called a “re­al­ly blue-chip” syn­di­cate of four new in­vestors, in­clud­ing Black­Rock, RA Cap­i­tal Man­age­ment and two undis­closed funds. They closed on the $100 mil­lion Se­ries C just over a week ago, and wait­ed un­til Mon­day morn­ing to an­nounce it.

“I think it’s like­ly that we’re go­ing to go pub­lic at some point,” South­well told End­points News. But for now, the Se­ries C cash gives them flex­i­bil­i­ty through 2022.

The Har­vard Uni­ver­si­ty spin­out is build­ing a repos­i­to­ry of clin­i­cal­ly-ac­tive TCRs us­ing its high-through­put whole genome dis­cov­ery plat­form. The com­pa­ny had com­pared it to a vend­ing ma­chine or a li­brary of sorts, be­fore fi­nal­ly set­tling on the term “bank,” South­well said.

The process be­gins with T cells from pa­tients’ tu­mors. Re­searchers use the dis­cov­ery plat­form to find out ex­act­ly what tar­gets the T cell is hit­ting, then clin­i­cal­ly val­i­date the TCR to see if it has any off-tar­get ef­fects. When the TCR is val­i­dat­ed, it gets added to the bank. Know­ing a pa­tient’s HLA type and tu­mor tar­get, re­searchers can then take TCRs out of the bank, “grow them up, and put them in­to the pa­tient,”  South­well said.

“Those T cell re­cep­tors are of­ten there in the pa­tient, but the prob­lem is that they’re not there in suf­fi­cient abun­dance to re­al­ly at­tack the tu­mor,” he added.

The ap­proach comes from the lab of Har­vard pro­fes­sor Stephen Elledge, who set out years ago to screen anti­gen-TCR match­es in a faster, more sys­tem­at­ic way. He spent 7 years putting to­geth­er the tech for a plat­form that could run mul­ti­ple TCRs against anti­gen epi­topes and pin­point the ex­act pairs that ap­pear to in­ter­act. Now, what be­gan as 96 plates in Elledge’s lab has trans­formed in­to a com­pa­ny that has raised $180 mil­lion to date and at­tract­ed the likes of No­var­tis.

Back in April, the No­var­tis In­sti­tutes for Bio­Med­ical Re­search put down $30 mil­lion to kick off a new TCR im­muno-on­col­o­gy pro­gram with TScan. The part­ners are work­ing on dis­cov­er­ing tar­gets in a “se­lect sol­id tu­mor in­di­ca­tion,” TScan re­vealed. NI­BR pitched in­to TScan’s Se­ries B round, along­side the phar­ma’s ven­ture fund.

“There’s a lot with this dis­cov­ery plat­form that we can do, that we’re not go­ing to de­vel­op on our own,” South­well said, in­clud­ing Covid-19 work.

Com­ing up in 2021, TScan plans on fil­ing INDs for two liq­uid tu­mor TCR T cell ther­a­pies — TSC-100 and TSC-101. It has an­oth­er three sol­id tu­mor can­di­dates ex­pect­ed to hit the clin­ic in 2022.

While the com­pa­ny cur­rent­ly has 60 staffers, South­well pre­dicts they’ll have well over 100 in the next six to nine months as they build out their man­u­fac­tur­ing and cell pro­cess­ing units.

The feel­ing at TScan? “We’re re­al­ly ex­cit­ed,” South­well said.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 151,400+ biopharma pros reading Endpoints daily — and it's free.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 151,400+ biopharma pros reading Endpoints daily — and it's free.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 151,400+ biopharma pros reading Endpoints daily — and it's free.

#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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 151,400+ biopharma pros reading Endpoints daily — and it's free.

George Church (courtesy EnPlusOne BioSciences)

George Church, Wyss sci­en­tists and North­pond chal­lenge con­ven­tion­al RNA man­u­fac­tur­ing with new biotech

RNA medicine has been at the forefront for the past few years, with the first RNA silencing therapy approved in 2018, and mRNA Covid vaccines following after. But flying under the radar has been the process of actually making RNA for these treatments.

That’s what Daniel Wiegand and Jonathan Rittichier have been working on in George Church’s lab for the past six years.

Friday morning, they unveiled EnPlusOne Biosciences, a biotech built on their RNA synthesis platform. Wiegand will serve as the Watertown, MA-based biotech’s CEO, and Rittichier will be CSO. And no different from his other startups, Church will be acting as scientific advisor. Its fourth co-founder, Dan Ahlstedt, joined through a Harvard Business School program, and will be COO.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 151,400+ biopharma pros reading Endpoints daily — and it's free.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 151,400+ biopharma pros reading Endpoints daily — and it's free.

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.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 151,400+ biopharma pros reading Endpoints daily — and it's free.