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.

Big Phar­ma's Twit­ter ex­o­dus; Mer­ck wa­gers $1.35B on buy­out; $3.5M gene ther­a­py; and more

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Paul Perreault, CSL Behring CEO

CSL lands FDA ap­proval for he­mo­phil­ia B gene ther­a­py, sets $3.5M list price

The FDA has approved the world’s first gene therapy for hemophilia B, ushering into the market a treatment that’s historic in both what it promises to do and how much it will cost.

CSL will be marketing the drug, Hemgenix, at a list price of $3.5 million — which sets a new record for the most expensive single-use gene therapy in the US.

In a statement provided to Endpoints News, the Australian company noted that the current costs of treating people with moderate to severe hemophilia B can be significant over a lifetime. By some estimates, healthcare systems could spend more than $20 million per person.

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Image: Shutterstock

MIT re­searchers re­veal DNA "Paste" tech be­hind lat­est gene edit­ing start­up

MIT scientists have developed a tool that they say can insert large gene sequences where they want in the genome.

In a paper published Thursday in Nature Biotechnology, MIT fellows Omar Abudayyeh, Jonathan Gootenberg and colleagues detail a technology they call PASTE, which they say can potentially be used to insert long strands of DNA and treat genetic diseases caused by many different mutations, such as cystic fibrosis and Leber congenital amaurosis, a rare eye disorder that causes blindness.

Elon Musk (GDA via AP Images)

Biggest drug com­pa­nies halt­ed Twit­ter ad buys af­ter Lil­ly in­sulin spoof

Almost all of the drug industry’s biggest advertisers cut their spending on Twitter to zero or near-zero over the last two weeks amid worries about impersonation of their brands by pranksters and the future of the social media company.

Among 18 of the biggest pharmaceutical advertisers in the US market, 12 cut their Twitter ad spending to nothing for the week beginning Nov. 14, according to Pathmatics, which tracks data on prescription drug ad spending as well as general corporate advertising. The list of drugmakers cutting spending to zero includes Merck, AstraZeneca, Eli Lilly, Novartis, Pfizer and others.

Andrew Phillips, Nexo Therapeutics CEO

Scoop: Ver­sant, NEA launch new biotech helmed by ex-CEO of pro­tein de­grad­er C4 Ther­a­peu­tics

Long-time biotech venture firms Versant and New Enterprise Associates are backing a new startup run by former C4 Therapeutics chief executive Andrew Phillips.

The fledgling biotech has raised at least $30 million so far, according to paperwork filed with the SEC this week. The round could balloon to $60 million.

Phillips, who left protein degradation startup C4 in 2020 to be a managing director at Cormorant Asset Management, is running the show of the new venture as president, the SEC filing outlines. He also served as interim CEO of Cormorant-backed and Hansoh Pharmaceutical-partnered Blossom Bioscience last year.

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Rob Davis, Merck CEO

Up­dat­ed: No Seagen here: 'Do more' means a small $1.35B pur­chase of Ima­go for Mer­ck

Merck is making an acquisition, the Big Pharma announced before Monday’s opening bell. No, Seagen is not entering the fold, as had been speculated for quarters.

Folding under Merck’s wings will be Pfizer-backed Imago BioSciences. For nearly a year, Merck CEO Rob Davis has been saying the pharma giant needs to “do more” on the business development front after its 2021 $11.5 billion acquisition of Acceleron.

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Phil L’Huillier, CatalYm CEO

Ger­man biotech CatalYm rais­es $50M to flip weight loss tar­get for can­cer

GDF15 might sound familiar. It’s a protein that Amgen, Merck and Eli Lilly built analogs for in attempts to make new weight loss drugs. But those drugs largely failed — and Amgen, the last standing of the three — quietly pulled the plug on its GDF15 program in January.

But GDF15 is not dead. The science behind the weight loss drugs goes back to the observation that some cancer patients have high levels of GDF15 and lose a lot of weight, so cancer researchers have been making antibodies that inhibit the protein instead of mimicking it.

J&J's Spra­va­to pulls a PhI­II win against Sero­quel XR in treat­ment-re­sis­tant de­pres­sion

A day before Thanksgiving, J&J’s Janssen has a new cut of Phase III Spravato data to be grateful for.

The pharma giant announced on Wednesday that its nasal spray, also known as esketamine, beat extended-release quetiapine, previously sold by AstraZeneca as Seroquel XR, in treatment-resistant depression (TRD). Of 676 adults, a significantly higher number of patients on Spravato were able to achieve remission and avoid relapse after 32 weeks, according to J&J.

Dermavant Sciences' first consumer TV ad for its Vtama psoriasis med shows people ready for a new topical treatment.

Roivant’s Der­ma­vant de­buts first-ever TV com­mer­cial for pso­ri­a­sis cream Vta­ma

Dermavant Sciences has been marketing its first product, psoriasis med Vtama, to dermatologists for months, but on Tuesday it rolled out its first consumer campaign. The debut DTC effort including a streaming TV commercial encourages patients to a “Topical Uprising” in a nod to Vtama being a topical cream.

In the new commercial, a swell of people discards scarves and jacket coverings, gathering in the street to converge on a pharmacy to demand a steroid-free prescription. A moment of levity follows when a pharmacist says, “You know you can just talk to your doctor, right?” The gathered crowds collectively says, “Oh.”

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