Drag­on­fly gets Mer­ck on sec­ond TriN­KET can­di­date since 2018 col­lab; Chi­nese eye gene ther­a­py de­vel­op­er nabs $60M

Drag­on­fly Ther­a­peu­tics is do­ing bet­ter and bet­ter in get­ting Big Phar­ma on its TriN­KET plat­form. First Cel­gene, then BMS, and now Mer­ck — again.

Af­ter Mer­ck li­censed its first can­di­date from the plat­form back in No­vem­ber 2020, the phar­ma gi­ant is back for sec­onds as part of a 2018 col­lab­o­ra­tion be­tween the two com­pa­nies, which was ini­tial­ly fo­cused on a num­ber of sol­id tu­mor tar­gets.

The com­pa­nies ex­pand­ed their col­lab­o­ra­tion last year with an agree­ment to de­vel­op and com­mer­cial­ize NK cell en­gag­ing im­munother­a­pies for mul­ti­ple tar­gets in on­col­o­gy, in­fec­tious dis­eases and im­mune dis­or­ders.

“We are de­light­ed that Mer­ck has now ex­er­cised its op­tion for this sec­ond im­munother­a­py can­di­date from our ini­tial col­lab­o­ra­tion, and ex­cit­ed by the progress we are mak­ing to­geth­er on bring­ing Drag­on­fly’s TriN­KET tech­nol­o­gy to tar­gets across a broad­er set of dis­eases,” said Drag­on­fly’s CEO Bill Haney.

Mer­ck ex­er­cised its op­tion to li­cense ex­clu­sive world­wide in­tel­lec­tu­al prop­er­ty rights on its sec­ond im­munother­a­py can­di­date de­vel­oped us­ing the TriN­KET plat­form, un­der a pre-ex­ist­ing agree­ment. As for Drag­on­fly, it re­ceived an pay­ment from Mer­ck at an undis­closed amount.

Chi­nese eye dis­ease biotech Neu­roph­th rais­es $60M in Se­ries C to progress pipeline

Chi­nese eye dis­ease biotech Neu­roph­th Ther­a­peu­tics closed $60 mil­lion in a Se­ries C round, the com­pa­ny an­nounced to­day.

The round, co-led by CMG-SDIC Cap­i­tal and Se­quoia Cap­i­tal Chi­na, in­clud­ed new in­vest­ing funds and ac­counts man­aged by Sun­shine In­sur­ance and Chi­na Mer­chant Bank In­ter­na­tion­al Cap­i­tal.

Ac­cord­ing to Neu­roph­th, this round of fi­nanc­ing will be used to progress its lead clin­i­cal pro­gram in LHON (Leber Hered­i­tary Op­tic Neu­ropa­thy) out­side of Chi­na, im­prove its R&D pipeline and pro­grams through busi­ness de­vel­op­ment and ex­pand Neu­roph­th’s gene ther­a­py man­u­fac­tur­ing plat­form.

Neu­roph­th’s lead can­di­date NR082 was the first AAV2 gene ther­a­py to get IND ap­pli­ca­tion ap­proval in Chi­na, with the first pa­tient be­ing dosed in June.

“We are very pleased to have lead­ing Chi­na and ex-Chi­na in­vestors to sup­port the com­pa­ny mis­sion and vi­sion,” said Neu­roph­th chair­man and founder Bin Li.

Ieso rais­es $53M in Se­ries B to de­vel­op dig­i­tal ther­a­py sys­tems for men­tal health

UK-based dig­i­tal ther­a­peu­tics de­vel­op­er ieso has more mon­ey, thanks to lead in­vestor Morn­ing­side.

The com­pa­ny an­nounced to­day that it has fin­ished its Se­ries B round — with a to­tal of $53 mil­lion raised. Oth­er in­vestors that tagged along in­clude Sony In­no­va­tion Fund, IP Group and Molten Ven­tures.

The funds raised will help ieso de­vel­op au­tonomous dig­i­tal ther­a­py sys­tems to de­liv­er a high­er stan­dard of care — ones “that match the high stan­dards of hu­man care de­liv­ery,” ac­cord­ing to the com­pa­ny — at sig­nif­i­cant­ly low­er cost to health sys­tems.

Ieso plans to de­vel­op these AI-en­abled dig­i­tal as­sess­ment tools and ther­a­peu­tics on their out­comes-linked ther­a­py dataset, which in­cludes at least 460,000 hours of in­for­ma­tion.

Ieso in­tends to seek reg­u­la­to­ry ap­proval for these dig­i­tal tools in the US and UK ahead of bring­ing them to mar­ket. And as part of the raise, ieso will ex­pand its com­mer­cial teams to the US and tar­get both co-de­vel­op­ment and dis­tri­b­u­tion part­ner­ships.

“Through the sup­port of our in­vestors, who share our pur­pose, we’ll con­tin­ue on our mis­sion of com­bin­ing clin­i­cal ex­per­tise, da­ta sci­ence and tech­nol­o­gy to en­able bet­ter and more ac­ces­si­ble men­tal health­care,” said ieso chair­man Andy Richards in a state­ment.

FDA ex­tends re­view for Am­ryt Phar­ma’s EB drug

Am­ryt Phar­ma an­nounced this morn­ing that the FDA has ex­tend­ed the re­view pe­ri­od for the NDA for its epi­der­mol­y­sis bul­losa (EB) treat­ment Oleogel-S10. EB, a rare ge­net­ic skin dis­or­der, has no ap­proved treat­ment.

The FDA ex­tend­ed the PDU­FA by three months to Feb­ru­ary 28 of next year. And ad­di­tion­al­ly, the FDA al­so is­sued a new In­for­ma­tion Re­quest re­gard­ing ex­ist­ing study da­ta in or­der to con­tin­ue the agency’s eval­u­a­tion of Am­ryt’s NDA.

Mean­while, across the At­lantic, the EMA’s re­view process for Oleogel-S10 in EB is on­go­ing — and Am­ryt is in the process of re­spond­ing to the re­main­ing Ma­jor Ob­jec­tions in the List of Out­stand­ing Is­sues sent by the EMA. Those ob­jec­tions re­main undis­closed. The CHMP opin­ion is ex­pect­ed in Jan­u­ary.

“We are well po­si­tioned to ad­dress these reg­u­la­to­ry re­quests from our ex­ist­ing da­ta with­in the time pe­ri­ods re­quired and we look for­ward to our con­tin­ued pro­duc­tive dis­cus­sions as the reg­u­la­to­ry agen­cies com­plete their re­view,” said Am­ryt CEO Joe Wi­ley.

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.

Ivan Cheung, Eisai US chairman and CEO

Bio­gen, Ei­sai re­fresh amy­loid hy­poth­e­sis with PhI­II show­ing Alzheimer's med slows cog­ni­tive de­cline

In the first look at Phase III data for lecanemab, Eisai and Biogen’s follow-up Alzheimer’s drug to the embattled Aduhelm launch, results show the drug passed with flying colors on a test looking at memory, problem solving and other dementia metrics.

One of the most-watched Alzheimer’s therapies in the clinic, lecanemab met the study’s primary goal on the CDR-SB — Clinical Dementia Rating-Sum of Boxes — giving the biotech the confidence to ask for full approval in the US, EU and Japan by next March 31. The experimental drug reduced clinical decline on the scale by 27% compared to placebo at 18 months, the companies said Tuesday night Eastern time and Wednesday morning in Japan.

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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.

Aim­ing for fourth nod, Sarep­ta files an­oth­er DMD gene ther­a­py to FDA; Ax­some head­ed to­ward mi­graine re­sub­mis­sion

Sarepta Therapeutics has filed the data needed for an FDA accelerated approval, which would be the biotech’s fourth if granted by the agency.

The biotech has yet to complete confirmatory trials for those first three conditional nods. The filing for its fourth Duchenne muscular dystrophy treatment, disclosed Thursday, is not a surprise. Sarepta said in late-July it would do so after releasing positive results for the Roche-partnered gene therapy.

Vlad Coric, Biohaven CEO (Photo Credit: Andrew Venditti)

As Amy­lyx de­ci­sion waits in the wings, Bio­haven’s ALS drug sinks (again) in plat­form tri­al

The FDA’s decision on Amylyx’s ALS drug is set to come out sometime Thursday. In a space with few drugs, any approval would be a major landmark.

But elsewhere in the ALS field, things are a bit more tepid.

Thursday morning, Biohaven announced that its drug verdiperstat failed its arm of an ALS platform trial led by Massachusetts General Hospital. According to a press release, the drug did not meet its primary endpoint — improvement on an ALS functional status test — or any key secondary endpoints at 24 weeks. The trial had enrolled 167 patients, giving them either verdiperstat or placebo twice a day.

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Work taking place in the clean rooms at Vor (Credit: Vor)

Vor Bio opts to keep man­u­fac­tur­ing op­er­a­tions in-house for de­vel­op­ing stem cell, CAR-T ther­a­pies

While it is not uncommon for a biotech to go down the route of having the product manufactured by a contract organization, one small biotech is looking to keep its card close to its chest.

Vor Biopharma has started manufacturing operations at an in-house facility at its HQ in Cambridge, MA after beginning construction last summer.

According to the biotech, the facility aims to develop Vor’s hematopoietic stem cells (eHSCs) and CAR-T therapies for patients with blood cancers. The site will initially manufacture a clinical supply of its candidate VCAR33allo to support its IND, which is slated to be submitted in the first half of next year. It also plans to transfer the production of VOR33 to the facility. Vor is getting to work quickly as engineering runs for VCAR33allo has started this week.

Phillip Gomez, Siga Technologies CEO

Siga nabs $10.7M from the US gov­ern­ment in deal for its mon­key­pox an­tivi­ral

The US government is all set to buy $10.7 million worth of Siga Technologies’ monkeypox oral antiviral, the company announced Thursday.

Of the total doses, $5.1 million worth of oral antivirals called Tpoxx (tecovirimat) will be delivered this year, with the US Department of Defense having the option of buying the rest at a later point.

The new contract follows an earlier one in which the government had purchased $7.4 million worth of Tpoxx from the company.

Renhong Tang, Simcere co-CEO

Almi­rall part­ners up with Sim­cere in po­ten­tial $500M+ deal — with plans to take IL-2 can­di­date glob­al

A Chinese pharma is looking to go international with one of its preclinical candidates, and it’s teaming up with a Spanish company in a new pact potentially worth half a billion dollars to do just that.

Simcere and Almirall announced Thursday that the two companies had reached a deal for Simcere’s IL-2 mutant fusion protein drug candidate, called SIM0278. According to a statement, Almirall gets an exclusive right to develop and commercialize the drug candidate in all indications and markets outside of China, Hong Kong, Taiwan and Macau.

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Tar­sus looks to raise aware­ness of eye­lid mite dis­ease in cam­paign aimed at eye­care spe­cial­ists

Eyelid mite disease may be “gross” but it’s also fairly common, affecting about 25 million people in the US.

Called demodex blepharitis, it’s a well-known condition among eyecare professionals, but they often don’t always realize how common it is. Tarsus Pharmaceuticals wants to change that with a new awareness campaign called “Look at the Lids.”

The campaign and website debut Thursday — just three weeks after Tarsus filed for FDA approval for a drug that treats the disease.

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