Syros Receives Fast Track Designation from the FDA for Tamibarotene for the Treatment of Newly Diagnosed Unfit AML with RARA gene overexpression

Rhea-AI Summary

Syros Pharmaceuticals receives Fast Track Designation from FDA for tamibarotene in combination with azacitidine and venetoclax for newly diagnosed AML patients with RARA overexpression. Positive results from SELECT-AML-1 trial show 100% CR/CRi rate with no added toxicity, potentially offering a new frontline treatment option.

Positive

• Fast Track Designation granted by FDA for tamibarotene in combination with azacitidine and venetoclax for newly diagnosed AML patients with RARA overexpression.
• Initial randomized data from SELECT-AML-1 trial show a 100% CR/CRi rate with the triplet regimen compared to 70% with venetoclax and azacitidine alone.
• Tamibarotene, venetoclax, and azacitidine combination demonstrates rapid response with no added toxicity or new safety signals.
• Enrollment for pivotal analysis in SELECT-MDS-1 Phase 3 trial completed, with complete response data expected in the fourth quarter of 2024.
• FDA previously granted Fast Track Designation to tamibarotene for the treatment of HR-MDS patients with RARA overexpression.

Negative

• None.

Insights

Oncology Doctor

The Fast Track Designation granted by the FDA to Syros Pharmaceuticals for tamibarotene is a significant milestone in the treatment of acute myeloid leukemia (AML), especially for a subset of patients who are over 75 years old or have comorbidities that make intensive chemotherapy a non-viable option. The designation is a testament to the unmet medical need in this patient population and the potential of tamibarotene to improve clinical outcomes. The high complete response rates reported in the SELECT-AML-1 clinical trial indicate a promising advancement in AML therapeutics, particularly given the lack of additional toxicity when tamibarotene is added to the existing treatment regimen of venetoclax and azacitidine.

From a medical standpoint, the ability to achieve a 100% CR/CRi rate is remarkable and if these results are consistent in larger trials, it could lead to a shift in standard care practices. The rapid response time and favorable safety profile further support the potential of this therapeutic approach. However, it's important to remain cautious until the final data from the SELECT-AML-1 trial are available, as early trial results sometimes do not translate into the same level of effectiveness in a broader population.

Medical Research Analyst

The Fast Track Designation is a clear indicator of the FDA's recognition of tamibarotene as a potential significant advancement in AML treatment. This status not only expedites the development process but also enhances interactions with the FDA, which could lead to priority review and accelerated approval. For investors and stakeholders, this regulatory milestone could be a harbinger of shortened time-to-market and an increased probability of commercial success, pending the outcome of ongoing trials.

It is also noteworthy that the SELECT-AML-1 trial has shown a stark contrast in CR/CRi rates between the tamibarotene combination therapy and the control group. These results could potentially lead to a strong value proposition for Syros Pharmaceuticals in the AML market. Furthermore, the lack of additive toxicity suggests that if approved, tamibarotene could be well-received in the clinical setting, potentially leading to widespread adoption and significant market penetration.

Looking ahead, the anticipated data from the SELECT-MDS-1 Phase 3 trial for higher-risk myelodysplastic syndrome (MDS) patients will be another critical factor for Syros' trajectory. The dual Fast Track designations for AML and HR-MDS underscore the strategic positioning of tamibarotene across multiple hematologic malignancies, potentially diversifying the company's portfolio and revenue streams.

Market Research Analyst

The biopharmaceutical sector is highly competitive and regulatory milestones such as Fast Track Designation can have a significant impact on a company's market position. For Syros Pharmaceuticals, the Fast Track status for tamibarotene may enhance investor confidence and could lead to an uptick in stock valuation as the market anticipates the potential for a new treatment option in a space with high unmet need. The AML treatment market is forecasted to grow and tamibarotene's promising clinical results position Syros to capture a portion of this market, especially among patients unsuitable for intensive chemotherapy.

However, market success depends on various factors, including the final clinical trial outcomes, the competitive landscape, reimbursement scenarios and the ability of Syros to effectively commercialize tamibarotene. Investors should monitor the upcoming data releases and regulatory interactions closely, as these will provide further insight into the drug's commercial viability and potential impact on Syros' financial performance.

CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Syros Pharmaceuticals (NASDAQ:SYRS), a biopharmaceutical company committed to advancing new standards of care for the frontline treatment of hematologic malignancies, today announced that the United States Food and Drug Administration (FDA) has granted Fast Track Designation to tamibarotene in combination with azacitidine and venetoclax for the treatment of newly diagnosed acute myeloid leukemia (AML) with RARA overexpression as detected by an FDA approved test in adults who are over age 75 years or who have comorbidities that preclude the use of intensive induction chemotherapy. Tamibarotene, an oral first-in-class selective retinoic acid receptor alpha (RARα) agonist, is currently being evaluated in combination with venetoclax and azacitidine for the treatment of newly diagnosed AML patients with RARA gene overexpression.

“We are pleased to receive Fast Track designation for tamibarotene for the treatment of AML. This designation reflects the tremendous need for a safe and effective therapy, which can improve the clinical outcomes and prognosis among people diagnosed with AML, many of whom cannot tolerate intensive treatment,” said David A. Roth, M.D., Chief Medical Officer of Syros Pharmaceuticals. “We are particularly encouraged to secure Fast Track designation following initial randomized data from a prespecified interim analysis of our ongoing SELECT-AML-1 clinical trial, in which treatment with our RARα agonist, tamibarotene, in combination with venetoclax and azacitidine resulted in a 100% CR/CRi rate compared with a 70% CR/CRi rate for the comparator of venetoclax and azacitidine. Additionally, tamibarotene in combination with venetoclax and azacitidine demonstrated no added toxicity relative to venetoclax and azacitidine alone. We look forward to sharing additional data from SELECT-AML-1 later this year, and to potentially accelerate the delivery of tamibarotene as a new frontline option for the approximately 30% of AML patients who are positive for RARA overexpression.”

Fast Track is a process designed by the FDA to facilitate the development and expedite the review of drug candidates intended to treat serious conditions and for which nonclinical or clinical data demonstrate the potential to address unmet medical need. The purpose is to facilitate development and expedite review of drugs to treat serious and life-threatening conditions, to bring the approved products to patients earlier. A therapeutic candidate that receives Fast Track designation may be eligible for more frequent interactions with the FDA to discuss the therapeutic candidate’s development plan. Therapeutic candidates with Fast Track designation may also be eligible for priority review and accelerated approval if supported by clinical data.

Syros is evaluating tamibarotene in combination with venetoclax and azacitidine in newly diagnosed, unfit AML patients with RARA overexpression in the ongoing SELECT-AML-1 Phase 2 trial. In December 2023, Syros announced initial randomized data from SELECT-AML-1, demonstrating a 100% CR/CRi (complete response/complete response with incomplete hematologic recovery) rate in response-evaluable patients (nine of nine) treated with the triplet regimen of tamibarotene, venetoclax and azacitidine, as compared to 70% among patients (seven of ten) treated with venetoclax and azacitidine alone, with corresponding CR rates of 78% vs 30%, respectively. The median time to CR/CRi response was rapid; all patients treated with the triplet regimen achieved a CR/CRi by the end of cycle one. Consistent with prior clinical experience, tamibarotene in combination with approved doses of venetoclax and azacitidine was generally well tolerated, and the overall safety profile demonstrated no additive toxicities or new safety signals, and no evidence of increased myelosuppression compared to treatment with the doublet combination of venetoclax and azacitidine. Syros expects to report additional data from SELECT-AML-1 in 2024.

Syros is also evaluating tamibarotene in combination with azacitidine in newly diagnosed higher-risk myelodysplastic syndrome (MDS) patients with RARA overexpression in the SELECT-MDS-1 Phase 3 trial. As recently announced, enrollment to support the pivotal primary efficacy analysis was completed in the first quarter of 2024, and pivotal complete response data is expected by the middle of the fourth quarter of 2024. In January 2023, the FDA granted Fast Track Designation to tamibarotene for the treatment of HR-MDS patients with RARA overexpression.

About Syros Pharmaceuticals
Syros is committed to developing new standards of care for the frontline treatment of patients with hematologic malignancies. Driven by the motivation to help patients with blood disorders that have largely eluded other targeted approaches, Syros is developing tamibarotene, an oral selective RARα agonist in frontline patients with higher-risk myelodysplastic syndrome and acute myeloid leukemia with RARA gene overexpression. For more information, visit www.syros.com and follow us on Twitter (@SyrosPharma) and LinkedIn.

Cautionary Note Regarding Forward-Looking Statements

This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995, including without limitation statements regarding Syros’ clinical development plans, the timing to report clinical data, the ability to commercialize tamibarotene and deliver benefit to patients, and the potential benefits of receiving Fast Track designation. The words “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “hope,” “intend,” “may,” “plan,” “potential,” “predict,” “project,” “target,” “should,” “would,” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Actual results or events could differ materially from the plans, intentions and expectations disclosed in these forward-looking statements as a result of various important factors, including Syros’ ability to: advance the development of its programs under the timelines it projects in current and future clinical trials; demonstrate in any current and future clinical trials the requisite safety, efficacy and combinability of its drug candidates; sustain the response rates and durability of response seen to date with its drug candidates; successfully develop a companion diagnostic test to identify patients with the RARA biomarker; obtain and maintain patent protection for its drug candidates and the freedom to operate under third party intellectual property; obtain and maintain necessary regulatory approvals; identify, enter into and maintain collaboration agreements with third parties; manage competition; manage expenses; raise the substantial additional capital needed to achieve its business objectives; attract and retain qualified personnel; and successfully execute on its business strategies; risks described under the caption “Risk Factors” in Syros’ Annual Report on Form 10-K for the year ended December 31, 2023, which is on file with the Securities and Exchange Commission; and risks described in other filings that Syros makes with the Securities and Exchange Commission in the future.

View source version on businesswire.com: https://www.businesswire.com/news/home/20240409254657/en/

Syros

Karen Hunady

Director of Corporate Communications & Investor Relations

1-857-327-7321

khunady@syros.com

Investor

Hannah Deresiewicz

Stern Investor Relations, Inc.

212-362-1200

hannah.deresiewicz@sternir.com

Source: Syros Pharmaceuticals

FAQ

What is the significance of the Fast Track Designation granted to Syros Pharmaceuticals by the FDA for tamibarotene?

The Fast Track Designation aims to expedite the review and development of drug candidates for serious conditions with unmet medical needs, potentially bringing approved products to patients earlier.

What are the key findings from the initial randomized data of the SELECT-AML-1 trial regarding tamibarotene, venetoclax, and azacitidine combination?

The trial showed a 100% CR/CRi rate with the triplet regimen compared to 70% with venetoclax and azacitidine alone, demonstrating rapid response with no added toxicity.

What is the status of the enrollment and expected data release for the SELECT-MDS-1 Phase 3 trial evaluating tamibarotene in combination with azacitidine?

Enrollment for pivotal analysis in the SELECT-MDS-1 trial has been completed, with complete response data expected by the middle of the fourth quarter of 2024.

When did the FDA grant Fast Track Designation to tamibarotene for the treatment of HR-MDS patients?

The FDA granted Fast Track Designation to tamibarotene for the treatment of HR-MDS patients in January 2023.