Adam Rosenthal, Star Therapeutics CEO

A new start­up sets course against a con­stel­la­tion of rare dis­eases with a first shot re­pur­pos­ing the SIRP path­way

De­spite some big ad­vances in re­cent years, rare dis­ease re­mains a most­ly un­chart­ed wilder­ness with many pa­tients left un­der­served. A start­up with galac­tic am­bi­tions now hopes to bust open a wide range of such dis­eases, and its first ef­fort has eyes on a fa­mil­iar can­cer tar­get.

Star Ther­a­peu­tics emerged from stealth Wednes­day fo­cused on crack­ing the code in a con­stel­la­tion of rare dis­eases with a first spin­out com­pa­ny tar­get­ing the SIRP path­way for im­munol­o­gy and can­cer.

Star, the brain­child of the R&D team be­hind Sanofi’s re­cent­ly ap­proved rare dis­ease drug En­jay­mo, came to­geth­er af­ter the ac­qui­si­tion of True North Ther­a­peu­tics by Biover­a­tiv in 2017, which lat­er fell in­to Sanofi’s hands.

Af­ter hand­ing off the reins to En­jay­mo, a com­ple­ment sys­tem ther­a­py for the rare blood con­di­tion cold ag­glu­tinin dis­ease (CAD), Star CEO and founder Adam Rosen­thal said his ex­pe­ri­ence with that drug helped set him off on a mis­sion to ex­plore the un­tapped po­ten­tial in rare dis­ease, where a lack of in­for­ma­tion of­ten leaves pa­tients un­der­served.

“To me, it was just eye-open­ing that this in­for­ma­tion about CAD was un­known, and there were pa­tients liv­ing in the shad­ows with vir­tu­al­ly noth­ing be­ing done by the in­dus­try,” he told End­points News. “So when I was found­ing Star, I looked back on that ex­pe­ri­ence and said, ‘How can we repli­cate this to find these (high un­met) dis­eases that no one’s fo­cused on?'”

Look­ing to make the most im­pact with the fewest pos­si­ble drugs, Rosen­thal’s team in se­cret be­gan look­ing for shared path­ways among sev­er­al dif­fer­ent dis­eases, set­tling first on SIRP, a fam­i­ly of sur­face re­cep­tors on im­mune cells. The path­way has been a pop­u­lar one in can­cer re­search, no­tably the CD47-SIRP al­pha path­way, which you might re­mem­ber as the fo­cal point of Pfiz­er’s $2.3 bil­lion takeover of Tril­li­um back in Au­gust.

But Star reck­oned that SIRP could al­so be a use­ful tar­get in rare im­munol­o­gy and set to de­vel­op a lead drug that could serve as a “pipeline-in-a-prod­uct” for a range of con­di­tions.

That work cul­mi­nat­ed in the launch of Elec­tra Ther­a­peu­tics, Star’s first spin­out that al­so hit the air­waves Wednes­day with $84 mil­lion in fi­nanc­ing from some big-name biotech blue-chip­pers led by West­lake Vil­lage BioPart­ners and Or­biMed.

The start­up’s lead drug is ELA026, which tar­gets the SIRP path­way to treat a rare in­flam­ma­to­ry dis­ease known as sec­ondary he­mo­phago­cyt­ic lym­pho­his­ti­o­cy­to­sis (sHLH), which is be­lieved to be fa­tal in rough­ly 60% of adult pa­tients. The drug is al­ready in Phase I tests there, and Elec­tra is look­ing for ad­di­tion­al in­di­ca­tions.

Mean­while, Elec­tra al­so has two oth­er pre­clin­i­cal pro­grams in the works, one tar­get­ing an­oth­er rare im­munol­o­gy dis­or­der and the oth­er a po­ten­tial im­muno-on­col­o­gy plat­form. That’s an un­der­stand­able ef­fort giv­en the amount of fo­cus on the SIRP path­way in can­cer.

Ac­cord­ing to Rosen­thal, Star’s goal of spin­ning out com­pa­nies around its re­search rather than keep­ing every­thing un­der one roof stems from the de­sire to ded­i­cate re­sources and ex­per­tise to nar­row­er ar­eas of fo­cus. Keep in mind, there are some 7,000 known rare dis­eases and on­ly 650 or so ap­proved drugs so the op­por­tu­ni­ties are vast.

“I think it’s that fo­cus that re­al­ly al­lows us to ex­plore the ther­a­peu­tic po­ten­tial,” Rosen­thal said. Mean­while, a siloed-off com­pa­ny al­so makes it eas­i­er to court fi­nanc­ing and pur­sue busi­ness de­vel­op­ment op­por­tu­ni­ties, he said.

Mean­while, Elec­tra will serve as proof of con­cept for Star’s grand am­bi­tions, which could get even grander. Rosen­thal said Star al­ready has a sec­ond spin­out queued up with a lead drug in tow. De­tails on that ini­tia­tive are slim for now, but will like­ly look much like Elec­tra in terms of mul­ti­ple po­ten­tial in­di­ca­tions.

Has the mo­ment fi­nal­ly ar­rived for val­ue-based health­care?

RBC Capital Markets’ Healthcare Technology Analyst, Sean Dodge, spotlights a new breed of tech-enabled providers who are rapidly transforming the way clinicians deliver healthcare, and explores the key question: can this accelerating revolution overturn the US healthcare system?

Key points

Tech-enabled healthcare providers are poised to help the US transition to value, not volume, as the basis for reward.
The move to value-based care has policy momentum, but is risky and complex for clinicians.
Outsourced tech specialists are emerging to provide the required expertise, while healthcare and tech are also converging through M&A.
Value-based care remains in its early stages, but the transition is accelerating and represents a huge addressable market.

FDA ad­vi­sors unan­i­mous­ly rec­om­mend ac­cel­er­at­ed ap­proval for Bio­gen's ALS drug

A panel of outside advisors to the FDA unanimously recommended that the agency grant accelerated approval to Biogen’s ALS drug tofersen despite the drug failing the primary goal of its Phase III study, an endorsement that could pave a path forward for the treatment.

By a 9-0 vote, members of the Peripheral and Central Nervous System Drugs Advisory Committee said there was sufficient evidence that tofersen’s effect on a certain protein associated with ALS is reasonably likely to predict a benefit for patients. But panelists stopped short of advocating for a full approval, voting 3-5 against (with one abstention) and largely citing the failed pivotal study.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 163,500+ biopharma pros reading Endpoints daily — and it's free.

Alaa Halawa, executive director at Mubadala’s US venture group

The ven­ture crew at Mubadala are up­ping their biotech cre­ation game, tak­ing care­ful aim at a new fron­tier in drug de­vel­op­ment

It started with a cup of coffee and a slow burning desire to go early and long in the biotech creation business.

Wrapping up a 15-year discovery stint at Genentech back in the summer of 2021, Rami Hannoush was treated to a caffeine-fueled review of the latest work UCSF’s Jim Wells had been doing on protein degradation — one of the hottest fields in drug development.

“Jim and I have known each other for the past 15 years through Genentech collaborations. We met over coffee, and he was telling me about this concept of the company that he was thinking of,” says Hannoush. “And I got immediately intrigued by it because I knew that this could open up a big space in terms of adding a new modality in drug discovery that is desperately needed in pharma.”

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

Flare Therapeutics biochemists Yong Li (L) and Valerie Vivat

A $123M Flare will get Third Rock on­col­o­gy biotech in­to the clin­ic this year

Flare Therapeutics will start its first human trial this year with an investigational urothelial cancer drug after pulling together a $123 million Series B from Big Pharmas, VCs and its incubator, Third Rock Ventures.

Launched in 2021 on the idea that a biotech could finally succeed at drugging the much-sought-after but stubborn transcription factor, Flare Therapeutics said Wednesday it is now primed for the clinic after closing its large financing haul earlier this year. The raise is a relatively stark figure in a tough startup financing environment but further buoys the upbeat signals coming out of other Third Rock biotechs in recent weeks, including the $200 million CARGO Therapeutics and $100 million Rapport Therapeutics rounds.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 163,500+ biopharma pros reading Endpoints daily — and it's free.

Sanofi, Re­gen­eron boast PhI­II win with Dupix­ent in COPD, clear­ing first bar for ex­pan­sion

Dupixent, the blockbuster anti-inflammatory drug from Sanofi and Regeneron, has cleared a high-stakes Phase III study in chronic obstructive pulmonary disease, the companies announced Thursday morning.

If they hold up in a second, identical trial, the data pave the way for Dupixent to become the first biologic to treat patients whose COPD remains uncontrolled despite being on maximal standard-of-care inhaled therapy — the patient population studied in the pivotal program. The companies had spotlighted this as a key readout as they look to expand the Dupixent franchise and explore its full potential.

Chat­G­PT with phar­ma da­ta de­buts for med­ical meet­ings, be­gin­ning with AACR

What do you get when you combine ChatGPT generative AI technology with specific pharma and clinical datasets? A time-saving tool that can answer questions about medical conference abstracts and clinical findings in seconds in one new application from ZoomRx called FermaGPT.

ZoomRx is debuting a public version of its generative AI product specifically for medical conferences beginning this week for the upcoming American Association for Cancer Research (AACR) annual meeting that runs April 14-19.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 163,500+ biopharma pros reading Endpoints daily — and it's free.

Quince Ther­a­peu­tics faces takeover bid from share­hold­er Echo Lake Cap­i­tal

A bid to take over the biotech Quince Therapeutics has been put forward by one of its shareholders.

On Tuesday, Echo Lake Capital sent a letter to Quince’s board of directors putting forth a proposal to acquire all the biotech’s stock for $1.60 per share, which would value a takeover at around $58 million.

In the letter, Echo Lake said that it believes Quince’s stock is severely undervalued and that no drugs are being actively marketed or developed that require cash expenditures. It’s trading below the value of its assets, Echo Lake said.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 163,500+ biopharma pros reading Endpoints daily — and it's free.

Genen­tech to stop com­mer­cial man­u­fac­tur­ing at Cal­i­for­nia head­quar­ters

Genentech is halting commercial manufacturing at its California headquarters — and laying off several hundred employees.

The move is the result of a decision Genentech made in 2007 to relocate manufacturing operations from its South San Francisco headquarters location to other facilities or move the work to CDMOs, said Andi Goddard, Genentech’s SVP of quality and compliance for pharmaceutical technical operations, in an interview with Endpoints News. Genentech has made changes in capabilities and invested more in technology, so it doesn’t need as many large-scale manufacturing facilities as it did in the past, she said.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 163,500+ biopharma pros reading Endpoints daily — and it's free.

Francesco Marincola, newly-appointed Sonata Therapeutics CSO

Kite's head of re­search leaves for Flag­ship start­up Sonata

Another leader is departing Kite Pharma, and will to spend the “last part” of his career exploring how cancer evades the immune system.

Kite’s senior VP and global head of cell therapy research Francesco Marincola left the Gilead CAR-T unit last week for Sonata Therapeutics. Flagship last May unveiled the startup, which was pieced together from two fledgling biotechs Inzen and Cygnal Therapeutics. As CSO, Marincola will lead Sonata’s push to reprogram cancer cells to make them more immunogenic.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 163,500+ biopharma pros reading Endpoints daily — and it's free.