SELLAS Announces U.S. FDA Rare Pediatric Disease Designation (RPDD) Granted to SLS009 for the Treatment of Pediatric Acute Myeloid Leukemia

Rhea-AI Summary

SELLAS Life Sciences Group (NASDAQ: SLS) has received Rare Pediatric Disease Designation (RPDD) from the FDA for SLS009, its CDK9 inhibitor, to treat pediatric acute myeloid leukemia (AML). This marks SELLAS' second RPDD, following a designation for ALL last month. The company has opened enrollment for pediatric AML patients in an ongoing Phase 2 clinical trial.

AML is the second most common hematological malignancy in children, often with poorer prognosis compared to other pediatric cancers. The RPDD makes SLS009 eligible for a Priority Review Voucher (PRV) upon marketing approval, which can be transferred or sold. SLS009 already has Orphan Drug and Fast Track Designations for AML treatment.

Positive

• Received Rare Pediatric Disease Designation (RPDD) for SLS009 in pediatric AML
• Opened enrollment for pediatric AML patients in ongoing Phase 2 clinical trial
• Eligibility for Priority Review Voucher (PRV) upon marketing approval
• Previously received Orphan Drug and Fast Track Designations for SLS009 in AML

Negative

• No concrete financial or revenue data provided
• SLS009 still in clinical trial phase, no guaranteed market approval

Oncology Doctor

The Rare Pediatric Disease Designation (RPDD) granted to SLS009 for pediatric acute myeloid leukemia (AML) is significant for several reasons. Firstly, AML is notably aggressive in children, with survival rates particularly low in relapsed or refractory cases. The designation recognizes the serious and life-threatening nature of pediatric AML and the lack of effective treatments currently available. RPDD can be a pivotal step in expediting the development and review of innovative treatments like SLS009, which targets CDK9, a protein important for cell cycle regulation and cancer cell proliferation.

Given the poor prognosis associated with pediatric AML, especially in relapsed cases where survival rates can be as low as 0% after a single chemotherapy course, any new therapeutic development is welcome. Additionally, the potential eligibility for a Priority Review Voucher (PRV) upon marketing approval further incentivizes rapid progression through clinical trials. The PRVs, highly valuable in the biotech industry, can accelerate the review of subsequent drugs, potentially bringing much-needed therapies to market faster or providing substantial financial gains if sold.

The ongoing Phase 2 clinical trial enrollment is important for gathering efficacy and safety data. Successful outcomes in these trials could transform the treatment landscape for pediatric AML, offering new hope for a patient group with limited options.

Financial Analyst

From a financial perspective, the FDA’s Rare Pediatric Disease Designation for SLS009 could have significant implications for SELLAS Life Sciences Group. The potential acquisition of a Priority Review Voucher (PRV) upon FDA approval stands out. PRVs are highly valuable assets in the pharmaceutical industry, recently fetching prices around $100 million in the secondary market. This can provide substantial non-dilutive funding for SELLAS, enhancing its financial position without issuing new shares or incurring debt.

Moreover, the rarity of the pediatric AML market means there is minimal competition, allowing SELLAS to potentially dominate this niche market if the drug proves effective. This exclusivity could translate into higher pricing power and significant revenue streams once the drug receives market approval.

Investors should note that while the designation itself does not guarantee financial success, it signals strong support from the FDA and can lead to faster approval processes. This aligns with SELLAS’ strategic goals and could bolster investor confidence, potentially driving up stock prices as the company progresses through clinical trials.

- Acute Myeloid Leukemia (AML) is the Second Most Common Hematological Malignancy in Children Often Associated with Poorer Prognosis Compared to Other Pediatric Cancers –

- This Recognition Marks SELLAS’ Second RPDD Following the Designation for ALL Received Last Month -

- Opened Enrollment for Pediatric AML Patients in Ongoing Phase 2 Clinical Trial -

- RPDD Provides Eligibility for SLS009 to Receive a Priority Review Voucher (PRV) Upon Marketing Approval that can be Transferred/Sold to Other Parties –

NEW YORK, July 16, 2024 (GLOBE NEWSWIRE) -- SELLAS Life Sciences Group, Inc. (NASDAQ: SLS) (“SELLAS” or the “Company”), a late-stage clinical biopharmaceutical company focused on the development of novel therapies for a broad range of cancer indications, today announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation (RPDD) to SLS009, a highly selective CDK9 inhibitor, for the treatment of pediatric acute myeloid leukemia (AML). The FDA previously granted Orphan Drug and Fast Track Designations to SLS009 for the treatment of AML.

“Receiving our second Rare Pediatric Disease Designation, following pediatric acute lymphoblastic leukemia last month, is another acknowledgment of SLS009’s novel transformational treatment potential to improve the lives of patients, including children with AML,” said Angelos Stergiou, MD, ScD h.c., President and Chief Executive Officer of SELLAS. ”This designation reinforces our dedication to addressing the urgent needs of children with AML, including those with treatment-resistant mutations, highlighting the promise of SLS009 to offer the specialized care and support they require, especially considering the limited treatment options for rare pediatric diseases. We look forward to continued SLS009 development and enrolling pediatric AML patients in our Phase 2 clinical trial.”

AML prognosis with currently available treatments in the refractory and/or relapsed pediatric patient population remains poor. In a representative study, the 5-year overall survival (OS) rate in relapsed pediatric AML was 33% for all patients, and in patients whose remission lasted less than 12 months only 15.7%. In patients who did not achieve complete remission after one course of chemotherapy 5-year overall survival was 0%. About 50% of children with pediatric AML relapse. Generally, the only therapy considered curative in relapsed and refractory patients is a bone marrow transplant and the primary goal of chemotherapy is to achieve remission so that pediatric patients can be transplanted.

Rare Pediatric Disease (RPD) Designation is granted by the FDA for serious or life-threatening diseases that affect fewer than 200,000 people in the United States and in which the serious or life-threatening manifestations primarily affect individuals less than 18 years of age. If, in the future, a New Drug Application (NDA) for SLS009 for the treatment of pediatric AML is approved by the FDA, SELLAS might be eligible to receive a Priority Review Voucher (PRV) that could be redeemed to receive a priority review for any subsequent marketing application. PRVs may be used by the sponsor or sold to another sponsor for their use and have recently sold for approximately $100 million.

About SELLAS Life Sciences Group, Inc.

SELLAS is a late-stage clinical biopharmaceutical company focused on the development of novel therapeutics for a broad range of cancer indications. SELLAS’ other lead product candidate, GPS, is licensed from Memorial Sloan Kettering Cancer Center and targets the WT1 protein, which is present in an array of tumor types. GPS has the potential as a monotherapy and combination with other therapies to address a broad spectrum of hematologic malignancies and solid tumor indications. The Company is also developing SLS009 (formerly GFH009) - potentially the first and best-in-class differentiated small molecule CDK9 inhibitor with reduced toxicity and increased potency compared to other CDK9 inhibitors. Data suggests that SLS009 demonstrated a high response rate in AML patients with unfavorable prognostic factors including ASXL1 mutation, commonly associated with poor prognosis in various myeloid diseases. For more information on SELLAS, please visit www.sellaslifesciences.com.

Forward-Looking Statements

This press release contains forward-looking statements. All statements other than statements of historical facts are “forward-looking statements,” including those relating to future events. In some cases, forward-looking statements can be identified by terminology such as “plan,” “expect,” “anticipate,” “may,” “might,” “will,” “should,” “project,” “believe,” “estimate,” “predict,” “potential,” “intend,” or “continue” and other words or terms of similar meaning. These statements include, without limitation, statements related to the GPS clinical development program, including the REGAL study and the timing of future milestones related thereto. These forward-looking statements are based on current plans, objectives, estimates, expectations, and intentions, and inherently involve significant risks and uncertainties. Actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of these risks and uncertainties, which include, without limitation, risks and uncertainties with oncology product development and clinical success thereof, the uncertainty of regulatory approval, and other risks and uncertainties affecting SELLAS and its development programs as set forth under the caption “Risk Factors” in SELLAS’ Annual Report on Form 10-K filed on March 28, 2024 and in its other SEC filings. Other risks and uncertainties of which SELLAS is not currently aware may also affect SELLAS’ forward-looking statements and may cause actual results and the timing of events to differ materially from those anticipated. The forward-looking statements herein are made only as of the date hereof. SELLAS undertakes no obligation to update or supplement any forward-looking statements to reflect actual results, new information, future events, changes in its expectations, or other circumstances that exist after the date as of which the forward-looking statements were made.

Investor Contact

Bruce Mackle

Managing Director

LifeSci Advisors, LLC

SELLAS@lifesciadvisors.com

FAQ

What is the significance of SELLAS (SLS) receiving Rare Pediatric Disease Designation for SLS009?

The RPDD for SLS009 in pediatric AML acknowledges its potential as a novel treatment for a rare pediatric disease. It makes SELLAS eligible for a Priority Review Voucher upon FDA approval, which could be sold or used to expedite future drug reviews.

What clinical stage is SLS009 currently in for pediatric AML treatment?

SELLAS has opened enrollment for pediatric AML patients in an ongoing Phase 2 clinical trial for SLS009.

How common is Acute Myeloid Leukemia (AML) in children?

AML is the second most common hematological malignancy in children, often associated with a poorer prognosis compared to other pediatric cancers.

What other designations has SELLAS (SLS) received for SLS009 in AML treatment?

In addition to the RPDD, the FDA has previously granted Orphan Drug and Fast Track Designations to SLS009 for the treatment of AML.