PureTech Receives Orphan Drug Designation for Wholly Owned Candidate LYT-200 for the Treatment of Pancreatic Cancer

Rhea-AI Summary

PureTech Health plc announced that the FDA granted orphan drug designation for its cancer treatment candidate, LYT-200, targeting pancreatic cancer. LYT-200, a human IgG4 monoclonal antibody, addresses poor survival rates in challenging solid tumors. Currently in a Phase 1/2 clinical trial, it has not yet reached the maximum tolerated dose. The designation allows for potential tax credits and seven years of market exclusivity upon approval. Topline results from the trial are expected in H1 2022.

Positive

• FDA granted orphan drug designation for LYT-200, enhancing its potential market prospects.
• LYT-200 targets pancreatic cancer, colorectal cancer, and cholangiocarcinoma, addressing high unmet medical needs.
• Phase 1/2 trial progressing with no maximum tolerated dose reached yet.

Negative

• Topline results from the Phase 1 trial are still pending, indicating uncertainty in timelines for clinical progress.

LYT-200 is being advanced in a range of difficult-to-treat solid tumors including pancreatic cancer, colorectal cancer and cholangiocarcinoma

Phase 1 portion of its adaptive Phase 1/2 trial with LYT-200 in solid tumors continues to progress, with a maximum tolerated dose not yet reached

BOSTON--(BUSINESS WIRE)-- PureTech Health plc (Nasdaq: PRTC, LSE: PRTC) ("PureTech" or the "Company"), a clinical-stage biotherapeutics company dedicated to discovering, developing and commercializing highly differentiated medicines for devastating diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation for its wholly owned clinical therapeutic candidate, LYT-200, for the treatment of pancreatic cancer.

LYT-200 is a fully human IgG4 monoclonal antibody (mAb) targeting a foundational immunosuppressive protein, galectin-9, for the potential treatment of solid tumors, including pancreatic ductal adenocarcinoma (PDAC), colorectal cancer (CRC) and cholangiocarcinoma (CCA), that are difficult to treat and have poor survival rates. LYT-200 is currently being evaluated in the first stage of an adaptive Phase 1/2 trial, with topline results from the Phase 1 portion expected in the first half of 2022 to allow for continued dose escalation as a maximum tolerated dose has not yet been reached.

“The FDA’s decision to grant orphan drug designation for LYT-200 reflects its potential as a novel anti-cancer therapy designed to block multiple immunosuppressive pathways in the tumor microenvironment,” said Julie Krop, M.D., Chief Medical Officer at PureTech. “Too many pancreatic cancer patients do not respond to existing immunotherapy agents and other standard of care regimens. We are looking forward to advancing LYT-200 through the clinic in hopes of meeting this substantial need.”

The FDA grants orphan drug designation to novel drug and biologic products for the treatment, diagnosis or prevention of conditions affecting fewer than 200,000 persons in the U.S. Orphan drug designation qualifies PureTech for incentives under the Orphan Drug Act, including tax credits for some clinical trials and eligibility for seven years of market exclusivity in the U.S., if the drug is approved.

About LYT-200

LYT-200 is a fully human IgG4 monoclonal antibody targeting a foundational immunosuppressive protein, galectin-9, for the potential treatment of solid tumors, including pancreatic ductal adenocarcinoma, colorectal cancer and cholangiocarcinoma, that are difficult to treat and have poor survival rates. PureTech has presented preclinical data demonstrating high expression of galectin-9 across breast cancer, pancreatic and cholangiocarcinoma samples and found that the highest levels of galectin-9 correlated with shorter time to disease relapse and poor survival. These data suggest that galectin-9 could be significant both as a therapeutic target for a range of cancers and as a cancer biomarker. Preclinical and patient-derived organoid tumor models also showed the potential efficacy of LYT-200 and the importance of galectin-9 as a target. LYT-200 is currently being evaluated in a Phase 1/2 adaptive design trial. Results from the Phase 1 dose escalation portion of the trial are expected in the first half of 2022.

About PureTech Health

PureTech is a clinical-stage biotherapeutics company dedicated to discovering, developing and commercializing highly differentiated medicines for devastating diseases, including inflammatory, fibrotic and immunological conditions, intractable cancers, lymphatic and gastrointestinal diseases and neurological and neuropsychological disorders, among others. The Company has created a broad and deep pipeline through the expertise of its experienced research and development team and its extensive network of scientists, clinicians and industry leaders. This pipeline, which is being advanced both internally and through PureTech's Founded Entities, is comprised of 25 therapeutics and therapeutic candidates, including two that have received both U.S. FDA clearance and European marketing authorization, as of the date of PureTech's most recently filed Half Year Report and corresponding Form 6-K. All of the underlying programs and platforms that resulted in this pipeline of therapeutic candidates were initially identified or discovered and then advanced by the PureTech team through key validation points based on the Company's unique insights into the biology of the brain, immune and gut, or BIG, systems and the interface between those systems, referred to as the BIG Axis.

For more information, visit www.puretechhealth.com or connect with us on Twitter @puretechh.

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. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including without limitation statements that relate to our expectations regarding the potential therapeutic benefits of LYT-200 in patients with solid tumors, the design of the Company’s adaptive design Phase 1/2 trial for LYT-200, the progression and expected timing of results from our Phase 1/2 trial of LYT-200, and the potential incentives for the Company as a result of LYT-200 receiving an orphan drug designation. The forward-looking statements are based on current expectations and are subject to known and unknown risks, uncertainties and other important factors that could cause actual results, performance and achievements to differ materially from current expectations, including, but not limited to, those risks, uncertainties and other important factors described under the caption "Risk Factors" in our Annual Report on Form 20-F for the year ended December 31, 2020 filed with the SEC and in our other regulatory filings. These forward-looking statements are based on assumptions regarding the present and future business strategies of the Company and the environment in which it will operate in the future. Each forward-looking statement speaks only as at the date of this press release. Except as required by law and regulatory requirements, we disclaim any obligation to update or revise these forward-looking statements, whether as a result of new information, future events or otherwise.

View source version on businesswire.com: https://www.businesswire.com/news/home/20211111005395/en/

Investors

Allison Mead Talbot

+1 617 651 3156

amt@puretechhealth.com

U.S. Media

Nichole Sarkis

+1 774 278 8273

nichole@tenbridgecommunications.com

Source: PureTech Health plc

FAQ

What is LYT-200 and its significance for PureTech Health (PRTC)?

LYT-200 is a monoclonal antibody targeting galectin-9, significant for treating difficult solid tumors like pancreatic cancer.

What is the current status of the clinical trials for LYT-200?

LYT-200 is in the Phase 1 portion of an adaptive Phase 1/2 trial, with the maximum tolerated dose not yet reached.

What does orphan drug designation mean for PRTC and LYT-200?

The orphan drug designation qualifies PureTech for incentives like tax credits and potential seven years of market exclusivity upon FDA approval.

When can we expect results from the LYT-200 clinical trial?

Topline results from the Phase 1 trial are expected in the first half of 2022.