#ASH20: Bain ex­ecs step in to po­si­tion a some­what bat­tered and bruised Sy­ros with a new clin­i­cal can­di­date and enough mon­ey to charge ahead

Nan­cy Si­mon­ian Sy­ros

Sy­ros $SYRS CEO Nan­cy Si­mon­ian had more than da­ta to dis­cuss dur­ing the vir­tu­al ASH ses­sions this week­end.

On top of a new cut of the da­ta for their lead can­cer drug, SY-1425, a trio of Bain ex­ecs has stepped in to set up a ma­jor new in­fu­sion of funds along with a new prod­uct ac­qui­si­tion aimed at beef­ing up the pipeline as they shoot for the prover­bial “ful­ly in­te­grat­ed” sta­tus of a com­mer­cial play­er.

“We had been talk­ing to Bain for the last cou­ple of years,” says Si­mon­ian, who’s been in­ter­est­ed in see­ing how they could work to­geth­er based on a sim­ple premise: “We’ve got this great team, great pipeline, if there’s an in­ter­est­ing as­set you’ve been see­ing, let’s talk.”

It took some time, but Bain’s Ricky Sun, An­drew Hack and Adam Kop­pel came up with an in­ter­est­ing prospect.

They point­ed to ORH-2014 from a biotech called Ors­enix as the kind of as­set that could make a ma­jor dif­fer­ence for a com­pa­ny like Sy­ros, which has had its share of ups and downs in re­cent years.

A lit­tle more than a year ago a de­ci­sion to switch out an IV CDK7 in­hibitor in fa­vor of an oral mod­el — SY-5609 — was greet­ed with a rout of the share price. Three years ago it was trou­ble with the lead, their RARα ag­o­nist, that trig­gered a plunge. And the biotech — which al­ways main­tains a sun­ny at­ti­tude on events — has con­tin­ued to have a rocky re­la­tion­ship with the mar­ket; since ear­ly Sep­tem­ber their shares have bled away close to half of their val­ue, with the stock trad­ing at about a third of what they fetched be­fore trou­ble came knock­ing in 2017.

In Bain’s world, though, biotechs that stand some­what bat­tered and bruised of­fer an in­ter­est­ing op­por­tu­ni­ty, pro­vid­ed they can per­form on their pipeline plans. So the trio of Bain ex­ecs came through with a new as­set as well as a syn­di­cate to pro­vide a $90.5 mil­lion fi­nanc­ing in­jec­tion, which was made at a slight­ly el­e­vat­ed price of $8 a share.

Sy­ros gets a clin­i­cal-stage drug — now dubbed SY-2101 — that could pro­vide an oral al­ter­na­tive to the IV form of ar­senic tri­ox­ide used to treat pa­tients with acute promye­lo­cyt­ic leukemia. It’s been through a Phase I, of­fer­ing some sig­nif­i­cant de-risk­ing, and is slat­ed for a dose con­fir­ma­tion study in 2021. If that all goes well, Si­mon­ian and her team are plan­ning a 2022 start for a reg­is­tra­tion study with an eye to get­ting to the FDA in 2024 with their NDA.

The drug comes at a rel­a­tive­ly mod­est cost: $12 mil­lion up­front, with $6 mil­lion in reg­u­la­to­ry mile­stones and $10 mil­lion on the com­mer­cial end, along with sin­gle-dig­it roy­al­ties.

One of the rea­sons why they’ve had trou­ble main­tain­ing en­thu­si­asm for SY-1425 is the weak da­ta they’ve re­port­ed on it as a monother­a­py. Si­mon­ian switched fo­cus to a com­bi­na­tion of the drug, us­ing it with azac­i­ti­dine. At ASH they up­dat­ed the on­col­o­gy world with the lat­est from Phase II, which fo­cused on new­ly di­ag­nosed AML pa­tients “who are not suit­able can­di­dates for stan­dard chemother­a­py, as well as hard to treat RARA-pos­i­tive AML pa­tients.”

Some of the high­lights in­clude a snap­shot of da­ta from 51 new­ly di­ag­nosed un­fit AML pa­tients, with:

An over­all re­sponse rate (ORR) was 67% (12/18), with a com­pos­ite CR rate of 61%, (11/18) in­clud­ing nine pa­tients (50%) achiev­ing a com­plete re­sponse (CR), two pa­tients (11%) achiev­ing a com­plete re­sponse with in­com­plete blood count re­cov­ery. Me­di­an du­ra­tion of re­sponse was 10.8 months, and me­di­an over­all sur­vival among pa­tients who achieved a CR or CRi was 18 months.

Among re­lapsed and re­frac­to­ry pa­tients, there was a more mod­est im­pact:

ORR was 19% (4/21), con­sist­ing of one CRc, two CRi and one MLFS (mor­pho­log­ic leukemia-free state). Me­di­an OS was 5.9 months.

At the In­flec­tion Point for the Next Gen­er­a­tion of Can­cer Im­munother­a­py

While oncology researchers have long pursued the potential of cellular immunotherapies for the treatment of cancer, it was unclear whether these therapies would ever reach patients due to the complexity of manufacturing and costs of development. Fortunately, the recent successful development and regulatory approval of chimeric antigen receptor-engineered T (CAR-T) cells have demonstrated the significant benefit of these therapies to patients.

Stéphane Bancel, Moderna CEO

'This is not go­ing to be good': Mod­er­na CEO Ban­cel warns of a 'ma­te­r­i­al drop' in vac­cine ef­fi­ca­cy as Omi­cron spreads

Even as public health officials remain guarded about their comments on the likelihood Omicron will escape the reach of the currently approved Covid-19 vaccines, there’s growing scientific consensus that we’re facing a variant that threatens to overwhelm the vaccine barricades that have been erected.

Stéphane Bancel, the CEO of Moderna, one of the leading mRNA players whose quick vault into the markets with a highly effective vaccine created an instant multibillion-dollar market, added his voice to the rising chorus early Tuesday. According to Bancel, there will be a significant drop in efficacy when the average immune system is confronted by Omicron. The only question now is: How much?

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Philip Dormitzer, new GSK global head of vaccines R&D

Glax­o­SmithK­line poach­es Pfiz­er's vi­ral vac­cines lead in rush to cap­i­tal­ize on fu­ture of mR­NA

GlaxoSmithKline has appointed Philip Dormitzer, formerly chief scientific officer of Pfizer’s viral vaccines unit, as its newest global head of vaccines R&D, looking to leverage one of the leading minds behind Pfizer and BioNTech’s RNA collaboration that led to Covid-19 jab Comirnaty, the British drug giant said Tuesday.

Dormitzer had been with Pfizer for a little more than six years, joining up after a seven-year stint with Novartis, where he reached the role of US head of research and head of global virology for the company’s vaccines and diagnostics unit.

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In­tro­duc­ing End­points Stu­dio, a new way to ad­ver­tise with End­points-craft­ed brand­ing cam­paigns

Since our start in 2016, Endpoints has grown fast while executing our mission to cover biopharma’s most critical developments for industry pros worldwide. As readership has grown, our advertising business has too. Endpoints advertising partners support the mission and engage their desired audiences through announcements on our email and web platforms, brand recognition in our event coverage and sponsorships of Endpoints daily and weekly reports.

Reshma Kewalramani, Vertex CEO (Vertex via YouTube)

Bat­tling a line­up of skep­tics, Ver­tex claims an­oth­er ear­ly clin­i­cal win — this time in kid­ney dis­ease

Vertex claimed its second early-stage win of the fall Wednesday, announcing positive results in a small study on a genetically defined form of kidney disease.

The 16-patient, Phase II trial focused on patients with focal segmental glomerulosclerosis, a rare disease where kidneys are unable to filter blood properly. Over 13 weeks on an experimental pill, the level of protein in the patients’ urine fell by an average of 47.6%.

With on­ly burns to show in gene ther­a­py, Astel­las inks deal with AAV spe­cial­ist Dyno in push for a bet­ter cap­sid

On the hunt for a better AAV capsid for gene therapy, Eric Kelsic’s Dyno Therapeutics has set itself apart with its focus on machine learning to help speed discovery. Now, Japanese drugmaker Astellas — fresh off a slate of gene therapy burns — is taking a bet on Dyno as it looks to the future.

Astellas and Dyno will work together as part of an R&D pact to develop next-gen AAV vectors for gene therapy using Dyno’s CapsidMap platform directed at skeletal and cardiac muscle, the companies said Wednesday. Under the terms of the deal, Dyno will design AAV capsids for gene therapy, while Astellas will be responsible for conducting preclinical, clinical and commercialization activities for gene therapy product candidates using the capsids.

As first Omi­cron case in US crops up, re­searchers won­der: which an­ti­bod­ies, vac­cines will hold up?

As Covid-19 drug and vaccine developers race to figure out which of their products might be hampered by the new variant, the CDC on Wednesday afternoon announced the first confirmed case of the Omicron variant (B.1.1.529) in the US, found in San Francisco.

The unidentified individual was a traveler who returned from South Africa on Nov. 22, 2021, was fully vaccinated, and had mild symptoms that the CDC described as improving. All close contacts have been contacted and have tested negative, the centers said.

As lead drug runs in­to a wall, De­ci­phera slims down its pipeline, puts 140 jobs on the chop­ping block

Barely a month after disappointing data shattered hopes for a major label expansion for the GI tumor drug Qinlock, Deciphera is making a major pivot — scrapping development plans for that drug and discarding another while it hunkers down and focuses on two remaining drugs in the pipeline.

As a result, 140 of its staffers will be laid off.

The restructuring, which claims the equivalent of 35% of its total workforce, will take place across all departments including commercial, R&D as well as general and administrative support functions, Deciphera said, as it looks to streamline Qinlock-related commercial operations in the US while concentrating only on a “select number of key European markets.”

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How to use reg­istry da­ta to sup­port FDA de­ci­sion mak­ing: Agency ex­plains in new guid­ance

Drugmakers looking to design a new registry or use an existing one to support a regulatory decision on a drug’s effectiveness or safety will need to consult with a new draft guidance released Monday by the FDA.

The agency’s reliance on registry data for regulatory decisions dates back more than two decades, at least, as in 1998 Bayer won approval for its anticoagulant Refludan (withdrawn from the market in 2013 for commercial reasons) based in part on a historical control group pulled from a registry.