Syros’ stock up after sharing early, mid-stage AML data

On the heels of a 35% workforce reduction and new CEO stepping in, Syros Pharmaceuticals is sharing an early snapshot of data from an acute myeloid leukemia (AML) trial that shows a 100% response rate among nine patients.

The Cambridge, Mass.-based biotech’s data comes from a phase 2 study assessing Syros’ tamibarotene—an oral retinoic acid receptor alpha (RARα) agonist—in combination with approved treatments venetoclax and azacitidine for newly diagnosed, unfit patients with AML and RARA gene overexpression. The data slice comes from 19 patients, though the trial is set to ultimately enroll 80 people.

Of nine evaluable patients treated with the tamibarotene and standard-of-care, 100% had a complete response rate (CR) or complete response with incomplete hematologic recovery (CRi)—the trial’s primary endpoint measure. This is compared to 70% of the 10 patients treated with just venetoclax and azacitidine. Syros did not share if the response rates were statistically significant.

Of the nine patients receiving the tamibarotene, venetoclax and azacitidine combination, seven (78%) had a CR while two (22%) had a CRi. That’s compared to three of the 10 control patients (30%) having a CR and four (40%) with a CRi.

The median time to CR/CRi response was 21 days for patients treated with the combination of tamibarotene, venetoclax and azacitidine, as compared to 25 days for the control group. 

Syros said the safety profile is consistent with prior clinical experience and that tamibarotene in combo with venetoclax and azacitidine is generally well tolerated, with no additive toxicities or new safety signals. Most of the non-hematologic adverse events were low-grade and reversible, and rates of serious adverse events were similar between the study arms, according to the biotech.

Syros continues to enroll patients in the trial and expects to share more data in 2024.  

“I am highly encouraged by the initial data from the randomized portion of SELECT-AML-1,” Thomas Cluzeau, M.D., Ph.D., head of hematology at Nice University Hospital in France, said in a Dec. 6 release. “Despite the recent advances in treatment for unfit AML patients, there remains a substantial need for options that offer higher response rates and improved overall survival, particularly for the one-third of patients who do not respond to existing standard-of-care. I believe tamibarotene may offer a significant therapeutic advance for the treatment of AML and I am eager to continue enrolling patients in the ongoing SELECT-AML-1 trial.”

Since sharing the data snapshot, Syros’ stock has shot up from $2.80 per share at market close yesterday to $3.40 as of 10 a.m. ET today.

The data offers a sliver of hope for the company amid an objectively challenging year.

The biotech, co-founded by Flagship Pioneering in 2011, laid off 35% of staff in early October and sharpened its focus on tamibarotene for higher-risk myelodysplastic syndrome (HR-MDS) in a phase 3 pivotal trial and AML. The company paused development on another clinical asset, known as SY-2101, an oral form of arsenic trioxide that Syros had been developing in acute promyelocytic leukemia.   

The cuts came after Incyte ended a five-year collaboration with Syros in August. The companies had identified seven targets, but Incyte ultimately opted not to take any into further development. Pfizer also walked away from a sickle cell program with Syros earlier this year. The New York Big Pharma had inherited the partnership from Global Blood Therapeutics, which it acquired in 2022.

The changes didn’t stop there, though. Just a few days ago, Syros’ former Chief Commercial Officer and Chief Business Officer Conley Chee took over as CEO after former leader Nancy Simonian, M.D., retired.