TScan Therapeutics Receives FDA’s Regenerative Medicine Advanced Therapy (RMAT) Designation for its Two Lead TCR-T Therapy Candidates for the Treatment of Heme Malignancies
TScan Therapeutics announced that the FDA has granted Regenerative Medicine Advanced Therapy (RMAT) designation to its lead TCR-T therapy candidates, TSC-100 and TSC-101, for treating heme malignancies, including AML, ALL, and MDS. The RMAT designation is based on encouraging data from the ALLOHA trial and aims to expedite the development of transformative therapies. Both candidates target minor histocompatibility antigens HA-1 and HA-2 to eliminate malignant cells in patients undergoing allogeneic HCT with reduced intensity conditioning. Initial trial data show promising results with no relapse in treated patients and no dose-limiting toxicities observed.
- RMAT designation speeds up drug development and review processes.
- Both TSC-100 and TSC-101 show no relapse in patients with over 10 months of follow-up.
- No dose-limiting toxicities observed in treated patients.
- FDA approval could provide early interactions to discuss accelerated approval pathways.
- Potential priority review of an Investigational New Drug (IND) application.
- 40% of patients relapse within two years post-transplant.
- Relapse and death observed in control-arm patients highlight ongoing risks.
- No control-arm patients have shown relapse-free survival similar to the treatment group.
Insights
RMAT designation is a significant step for any development-stage therapy. This FDA recognition acknowledges early evidence of the therapy's potential to address unmet medical needs in serious conditions. In this case, TScan's TSC-100 and TSC-101 being granted this status indicates the agency sees promise in these candidates for treating heme malignancies like AML, ALL and MDS.
The RMAT designation not only expedites the regulatory process but also provides potential avenues for accelerated approval. For retail investors, this means the therapies could reach the market faster if subsequent clinical trials demonstrate efficacy and safety, potentially leading to earlier revenue streams for TScan. The reported initial success in the ALLOHA trial, where treated patients remain relapse-free with no dose-limiting toxicities, further adds optimism about the therapies' future potential.
It's important to note that while RMAT designation is an encouraging milestone, it is not a guarantee of ultimate approval. Future clinical results will need to confirm the early promise shown to date. However, the FDA's recognition helps de-risk the development pathway somewhat.
From a financial perspective, the RMAT designation could lead to increased investor confidence and potentially a positive impact on TScan's stock price. The expedited review timelines and enhanced communication with the FDA can shorten the time to market, reducing uncertainty. This means TScan could potentially generate revenue sooner than previously anticipated, positively affecting its financial outlook.
The announcement can also make the company more attractive to potential investors and partners. For TScan, this early recognition by the FDA might lead to new funding opportunities or strategic partnerships, vital for sustaining operations and further clinical development.
However, it's prudent for investors to keep an eye on subsequent clinical trial results. Early positive data is encouraging, but until these results are consistently replicated in larger cohorts, the long-term financial impact remains speculative. The market's reaction will likely depend on continuous positive updates from ongoing and future trials.
The RMAT designation for TSC-100 and TSC-101 is highly relevant in the context of heme malignancies treatment. These are serious conditions with high relapse rates post-transplant. The novel approach of targeting minor histocompatibility antigens HA-1 and HA-2 is particularly noteworthy. By eliminating all recipient hematopoietic cells (including malignant ones) while sparing donor-derived cells, these therapies offer a potentially transformative treatment pathway.
The early clinical data showing that all eight treated patients remain relapse-free is promising, especially given the typically poor outcomes for relapsed patients. This could herald a shift in post-transplant care, offering new hope to patients who otherwise have limited options. If these initial results hold in larger trials, it could pave the way for a new standard in treating AML, ALL and MDS.
Investors should understand that while early signs are promising, oncology treatments often face numerous hurdles. It will be critical to see how these therapies perform across larger, more diverse patient populations.
RMAT designation granted for both TSC-100 and TSC-101 for the treatment of patients with AML, ALL, and MDS undergoing allogeneic HCT with reduced intensity conditioning
WALTHAM, Mass, May 29, 2024 (GLOBE NEWSWIRE) -- TScan Therapeutics, Inc. (Nasdaq: TCRX), a clinical-stage biopharmaceutical company focused on the development of T cell receptor (TCR)-engineered T cell (TCR-T) therapies for the treatment of patients with cancer, today announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to TSC-100 and TSC-101, the Company’s two lead TCR-T therapy candidates for the treatment of heme malignancies (NCT05473910).
“We are delighted to receive FDA RMAT designation for both candidates in our heme program designed to treat patients with acute myeloid leukemia (AML), acute lymphoblastic leukemia (ALL), and myelodysplastic syndrome (MDS) undergoing allogeneic hematopoietic cell transplantation (HCT) with reduced intensity conditioning, based on encouraging initial data from the ALLOHA trial,” said Chrystal U. Louis, M.D., Chief Medical Officer. “This is an important milestone that recognizes the transformative potential of our engineered TCR-T therapy candidates, TSC-100 and TSC-101, in multiple difficult-to-treat cancers. We look forward to working closely with the FDA in our ongoing commitment to deliver life-changing therapies to patients.”
Established under the 21st Century Cures Act, RMAT designation is a dedicated program designed to expedite the drug development and review processes for promising pipeline products, including gene and cell therapies. A regenerative medicine therapy is eligible for RMAT designation if it is intended to treat, modify, reverse, or cure a serious or life-threatening disease or condition, and preliminary clinical evidence indicates that the drug or therapy has the potential to address unmet medical needs for such a disease or condition. Like Breakthrough Therapy designation, RMAT designation provides the benefits of intensive FDA guidance on efficient drug development, including the ability for early interactions with FDA to discuss surrogate or intermediate endpoints, potential ways to support accelerated approval and satisfy post-approval requirements, potential priority review of an Investigational New Drug (IND) application, and other opportunities to expedite development and review.
TScan has developed two lead TCR-T therapy candidates, TSC-100 and TSC-101, that target minor histocompatibility antigens HA-1 and HA-2, respectively. TScan is prospectively selecting HLA A*02:01-positive transplant patients who are either HA-1- or HA-2-positive, with donors who are negative for these antigens. In this context, TSC-100 and TSC-101 are designed to eliminate all recipient hematopoietic cells, including malignant, pre-malignant or normal cells, that persist post-transplant, while leaving donor-derived cells unaffected. Approximately
On May 13, 2024, the Company provided an update on its Phase 1 heme malignancies program. The update included additional follow-up on all eight treatment-arm patients as well as data on two additional control-arm patients. With a median follow-up of >10 months, all eight patients treated with TSC-100 or TSC-101 remain relapse-free with no detectable disease. No dose-limiting toxicities were observed. In contrast, two of eight control-arm patients relapsed approximately six months post-transplant and one of these patients died approximately three months later. A third control-arm patient required clinical intervention because of concerns of impending relapse, and a fourth control-arm patient died post-transplant.
About TScan Therapeutics, Inc.
TScan is a clinical-stage biopharmaceutical company focused on the development of T cell receptor (TCR)-engineered T cell (TCR-T) therapies for the treatment of patients with cancer. The Company’s lead TCR-T candidates, TSC-100 and TSC-101, are in development for the treatment of patients with hematologic malignancies to prevent relapse following allogeneic hematopoietic cell transplantation. The Company is also developing TCR-T candidates for the treatment of various solid tumors. The Company has developed and continues to expand its ImmunoBank, the Company’s repository of therapeutic TCRs that recognize diverse targets and are associated with multiple HLA types, to provide customized multiplex therapeutic TCR-Ts for patients with a variety of cancers.
Forward-Looking Statements
This release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, including, but not limited to, express or implied statements regarding the Company’s plans, progress, and timing relating to the Company’s hematologic malignancies program, including any discussions with regulatory authorities related to TSC-100 and TSC-101 and any expectations regarding the benefits of RMAT designation; the progress of the hematologic malignancies programs being indicative or predictive of the success of such program; the potential benefits of any of the Company’s proprietary platforms, multiplexing, or current or future product candidates in treating patients; and the Company’s goals and strategy. TScan intends such forward-looking statements to be covered by the safe harbor provisions for forward-looking statements contained in Section 21E of the Securities Exchange Act of 1934 and the Private Securities Litigation Reform Act of 1995. In some cases, you can identify forward-looking statements by terms such as, but not limited to, “may,” “might,” “will,” “objective,” “intend,” “should,” “could,” “can,” “would,” “expect,” “believe,” “anticipate,” “project,” “target,” “design,” “estimate,” “predict,” “potential,” “plan,” “on track,” or similar expressions or the negative of those terms. Such forward-looking statements are based upon current expectations that involve risks, changes in circumstances, assumptions, and uncertainties. The express or implied forward-looking statements included in this release are only predictions and are subject to a number of risks, uncertainties and assumptions, including, without limitation: the beneficial characteristics, safety, efficacy, therapeutic effects and potential advantages of TScan’s TCR-T therapy candidates; TScan’s expectations regarding its preclinical studies being predictive of clinical trial results; TScan’s recently approved INDs being indicative or predictive of bringing TScan closer to its goal of providing customized TCR-T therapies to treat patients with cancer; the timing of the launch, initiation, progress, expected results and announcements of TScan’s preclinical studies, clinical trials and its research and development programs; TScan’s ability to enroll patients for its clinical trials within its expected timeline; TScan’s plans relating to developing and commercializing its TCR-T therapy candidates, if approved, including sales strategy; estimates of the size of the addressable market for TScan’s TCR-T therapy candidates; TScan’s manufacturing capabilities and the scalable nature of its manufacturing process; TScan’s estimates regarding expenses, future milestone payments and revenue, capital requirements and needs for additional financing; TScan’s expectations regarding competition; TScan’s anticipated growth strategies; TScan’s ability to attract or retain key personnel; TScan’s ability to establish and maintain development partnerships and collaborations; TScan’s expectations regarding federal, state and foreign regulatory requirements; TScan’s ability to obtain and maintain intellectual property protection for its proprietary platform technology and our product candidates; the sufficiency of TScan’s existing capital resources to fund its future operating expenses and capital expenditure requirements; and other factors that are described in the “Risk Factors” and “Management’s Discussion and Analysis of Financial Condition and Results of Operations” sections of TScan’s most recent Annual Report on Form 10-K and any other filings that TScan has made or may make with the SEC in the future. Any forward-looking statements contained in this release represent TScan’s views only as of the date hereof and should not be relied upon as representing its views as of any subsequent date. Except as required by law, TScan explicitly disclaims any obligation to update any forward-looking statements.
Contacts
Heather Savelle
TScan Therapeutics, Inc.
VP, Investor Relations
857-399-9840
hsavelle@tscan.com
Maghan Meyers
Argot Partners
212-600-1902
TScan@argotpartners.com
FAQ
What is the significance of TScan's RMAT designation for TSC-100 and TSC-101?
How did TSC-100 and TSC-101 perform in the ALLOHA trial?
What types of cancer are targeted by TScan's TCR-T therapies?
What are the relapse rates post-transplant for patients with AML, ALL, and MDS?
What are the main targets of TSC-100 and TSC-101 therapies?
