PureTech Provides End of Year Report on Key Progress
PureTech Provides End of Year Report on Key Progress
Rapid advancement of
Catalyst-rich 2023 anticipated, with results from late-stage trial of LYT-100 in idiopathic pulmonary fibrosis and Phase 1b trial of LYT-200 in leukemia. Initiation of clinical trials planned with LYT-300 and LYT-310 (oral cannabidiol) targeting neurological conditions as well as the combination portion of the Phase 1 trial of LYT-200 in solid tumors
Continued momentum across Founded Entities, including Karuna's positive Phase 3 results for KarXT in schizophrenia, commercial progress for Gelesis' Plenity® and Akili's EndeavorRx® and Vor's promising initial data in acute myeloid leukemia
Board composition and committee changes also noted
"Over the next 12 months, we anticipate multiple important catalysts that will further guide how we prioritize our pipeline, informing our decisions regarding which programs we will drive to commercial launches ourselves and which programs could be most successfully advanced through other avenues such as a partnership, sale, or spinout into another entity."
Key Wholly Owned Program Updates
· LYT-100 (deupirfenidone) is in development for the potential treatment of conditions involving inflammation and fibrosis, including IPF, for which current standards of care are associated with significant tolerability issues, resulting in approximately three out of four patients in the
o Announced results from a randomized, double-blind crossover study in healthy older adults demonstrating that approximately 50% fewer subjects treated with
o Initiated a global, randomized double blind placebo-controlled trial of LYT-100 in patients with IPF. This trial is expected to serve as the first of two registration-enabling trials, and topline results are expected by the end of 2023.
o Initiated a preclinical study of LYT-100 for the prevention and treatment of radiation induced fibrosis, an indication for which
o Completed a Phase 2a trial of LYT-100 in patients with breast cancer-related, upper limb secondary lymphedema, which met the primary endpoint of safety and tolerability, adding to the growing body of data demonstrating a favorable tolerability profile for LYT-100. Secondary endpoints assessed exploratory biomarkers related to lymphedema, and the data did not provide support for a clear development path for this indication. Given the Company's current priorities in IPF, where the regulatory path is well-defined and there is a wealth of strong efficacy data with pirfenidone, the Company has deprioritized lymphedema as an indication.
· LYT-200 (anti-galectin-9 mAb) is in development for the potential treatment of metastatic solid tumors that have poor survival rates as well as hematological malignancies, such as acute myeloid leukemia (AML), where more than 50% of patients either don't respond to initial treatment or experience relapse after responding to initial treatment.
o Completed the bi-monthly and weekly monotherapy dose escalation portion of the Phase 1 program assessing the safety and tolerability of escalating doses of LYT-200 as a potential treatment for metastatic solid tumors. No dose-limiting toxicities were reported, and the full results will be presented in an upcoming scientific forum. Evaluation of LYT-200 in combination with tislelizumab is expected to begin in the first quarter of 2023.
o Shared new preclinical data supporting the clinical potential of LYT-200 as a therapeutic agent for the treatment of leukemia in a scientific poster at the
o Given the expansion of the clinical evaluation of LYT-200, the Company has deprioritized preclinical development of LYT-210.
· LYT-300 (oral allopregnanolone) is in development for the potential treatment of neurological and neuropsychological conditions where there is a need for more effective treatments that work quickly, have more favorable tolerability and can be administered conveniently.
o Completed the multi-part Phase 1 trial of LYT-300, which demonstrated oral bioavailability in healthy adults, achieving blood levels of allopregnanolone at or above those associated with therapeutic effect and ninefold greater than orally administered allopregnanolone, based on third-party published data. The data also demonstrated target engagement with GABAA receptors, which are known to regulate mood and other neurological conditions. LYT-300 was generally well-tolerated with no treatment-related severe or serious adverse events observed. A Phase 1b/2a trial is expected to begin in the first half of 2023.
· LYT-310 (oral cannabidiol [CBD]) is in development to expand the therapeutic application of CBD across a range of neurological disorders.
o Announced nomination of a new therapeutic candidate, LYT-310, which is expected to enter the clinic in Q4 of 2023. As with LYT-300,
· LYT-503/IMB-150 (partnered non-opioid pain program) is being advanced through a collaboration for the potential treatment of interstitial cystitis/bladder pain syndrome, a chronic bladder condition that consists of discomfort or pain in the bladder or surrounding pelvic region that is not adequately controlled for many patients.
o First patient, first visit was achieved in the Phase 1 clinical trial evaluating LYT-503/IMB -150 as a potential non-opioid treatment for female patients with Interstitial Cystitis/Bladder Pain Syndrome.
· LYT-510 (oral inflammation-targeting formulation of tacrolimus) is in development for the potential treatment of chronic pouchitis and inflammatory bowel disease (IBD), a condition for which current treatments require multiple injections over time and are associated with several limitations, including dose-limiting adverse events, loss of efficacy over time, a lack of efficacy entirely and the potential for opportunistic infections or malignancies.
o LYT-510 is the lead candidate generated from the Alivio™ Platform, with two others, LYT-503/IMB-150 and LYT-500, also developed using
Key Founded Entity Updates:
· Karuna (Nasdaq: KRTX)
o Announced positive topline Phase 3 data evaluating the efficacy, safety and tolerability of KarXT in adults with schizophrenia, meeting its primary endpoint and key secondary endpoints. Karuna plans to submit a New Drug Application (NDA) for KarXT in schizophrenia with the
o Announced the initiation of its Phase 3 ADEPT program, which is evaluating KarXT for the treatment of psychosis related to Alzheimer's Disease (AD.)
· Gelesis (NYSE: GLS)
o Announced that based on the extensive safety data and differentiation due to affordability, broad label and safety profile, it is preparing an application to the FDA to broaden the classification of Plenity in the
· Vor (Nasdaq: VOR)
o Announced initial clinical data from VBP101, Vor's Phase 1/2a multicenter, open-label, first-in-human study of tremtelectogene empogeditemcel or "trem-cel" (formerly VOR33) in patients with AML. The data observed from the first treated patient support the potential of a trem-cel transplant to be successfully manufactured, to engraft normally and to maintain blood counts following treatment with the CD33-targeted therapy Mylotarg. The clinical trial continues to enroll patients and additional data are expected in 2023.
· Akili (Nasdaq: AKLI)
o Deployed first wave of its EndeavorRx4 go-to-market sales force in 14 priority territories across the
o Announced that Highmark, the fourth largest
o Announced that its partner, Shionogi, started a pivotal Phase 3 randomized, controlled study of SDT-001 in children with attention-deficit hyperactivity disorder (ADHD).
o Opened a new facility designed to manufacture clinical and commercial supply for its therapeutic portfolio, including for the planned Phase 3 study (expected to begin in the first half of 2023) and potential commercial launch of lead candidate, VE303, in Clostridioides difficile infection. Vedanta believes it was the first company to manufacture CGMP-grade defined bacterial consortia in powdered form, which enables stable, consistent oral formulations.
Board Composition & Committee Changes:
· As noted in
For more information, visit www.puretechhealth.com or connect with us on Twitter @puretechh.
Cautionary Note Regarding Forward-Looking Statements
This press release contains statements that are or may be 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 those related to the Company's LYT-100 development program and the timing for results from ongoing clinical trials of LYT-100, the LYT-200 development program and the timing for results from ongoing clinical trials of LYT-200, the planned initiation of clinical trials for LYT-300 and LYT-310, the potential therapeutic benefits of the product candidates within Company's Wholly Owned Programs, the Company's plan related to the prioritization of programs and activities associated with its pipeline, the Company's approach to potential partnerships or spinouts of its platforms or candidates and our future prospects, developments and strategies. 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
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 References to "Wholly Owned Programs" refer to the Company's six therapeutic candidates (LYT-100, LYT-200, LYT-300, LYT-310, LYT-510 and LYT-503/IMB-150), lymphatic and inflammation technology platforms and potential future therapeutic candidates and platforms that the Company may develop or obtain. References to "Wholly Owned Pipeline" refer to LYT-100, LYT-200, LYT-300, LYT-310, LYT-510 and LYT-503/IMB-150. On
Founded Entities represent companies founded by
 Important Safety Information about Plenity: Patients who are pregnant or are allergic to cellulose, citric acid, sodium stearyl fumarate, gelatin, or titanium dioxide should not take Plenity. To avoid impact on the absorption of medications: For all medications that should be taken with food, take them after starting a meal. For all medications that should be taken without food (on an empty stomach), continue taking on an empty stomach or as recommended by your physician. The overall incidence of side effects with Plenity was no different than placebo. The most common side effects were diarrhea, distended abdomen, infrequent bowel movements, and flatulence. Contact a doctor right away if problems occur. If you have a severe allergic reaction, severe stomach pain, or severe diarrhea, stop using Plenity until you can speak to your doctor. Rx Only. For the safe and proper use of Plenity or more information, talk to a healthcare professional, read the Patient Instructions for Use, or call 1-844-PLENITY.
 EndeavorRx is the first-and-only FDA-authorized treatment delivered through a video game experience. EndeavorRx is indicated to improve attention function as measured by computer-based testing in children ages 8 to 12 years old with primarily inattentive or combined-type ADHD, who have a demonstrated attention issue. Patients who engage with EndeavorRx demonstrate improvements in a digitally assessed measure Test of Variables of Attention (TOVA®) of sustained and selective attention and may not display benefits in typical behavioral symptoms, such as hyperactivity. EndeavorRx should be considered for use as part of a therapeutic program that may include clinician-directed therapy, medication, and/or educational programs, which further address symptoms of the disorder. EndeavorRx is available by prescription only. It is not intended to be used as a stand-alone therapeutic and is not a substitution for a child's medication. The most common side effect observed in children in EndeavorRx's clinical trials was a feeling of frustration, as the game can be quite challenging at times. No serious adverse events were associated with its use. EndeavorRx is recommended to be used for approximately 25 minutes a day, 5 days a week, over initially at least 4 consecutive weeks, or as recommended by your child's health care provider. To learn more about EndeavorRx, please visit EndeavorRx.com.
 Dempsey, T. M., Payne, S., Sangaralingham, L., Yao, X., Shah, N. D., & Limper, A. H. (2021). Adoption of the Antifibrotic Medications Pirfenidone and Nintedanib for Patients with Idiopathic Pulmonary Fibrosis. Annals of the
 Margaritopoulos, G. A., Trachalaki, A., Wells, A. U., Vasarmidi, E., Bibaki, E., Papastratigakis, G., Detorakis, S., Tzanakis, N., &
 The use of the Animal Rule is intended for drugs and biological products developed to reduce or prevent serious or life-threatening conditions caused by exposure to lethal or permanently disabling toxic chemical, biological, radiological or nuclear substances
 Walter, R. B., Othus, M., Burnett, A. K., Löwenberg, B., Kantarjian, H. M., Ossenkoppele, G. J., Hills, R. K., Ravandi, F., Pabst, T., Evans, A., Pierce, S. R., Vekemans, M. C., Appelbaum, F. R., & Estey, E. H. (2015). Resistance prediction in AML: analysis of 4601 patients from MRC/NCRI, HOVON/SAKK, SWOG and
 Brexanolone NDA 211371 Multi-disciplinary Review and Evaluation, FDA CDER, 2018.
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