On October 20, 2021 Ionis Pharmaceuticals, Inc. (NASDAQ: IONS) reported that it will host a live webcast on Wednesday, November 3rd at 11:30 a.m. Eastern Time to discuss its third quarter 2021 financial results and report on pipeline and business progress (Press release, Ionis Pharmaceuticals, OCT 20, 2021, View Source [SID1234591616]).

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Interested parties may access the webcast at View Source A webcast replay will be available for a limited time at the same address.

On October 20, 2021 Celdara Medical, LLC reported the awarding of six DRIVEN Accelerator Hub grants to outstanding researchers and innovators in the Northeast region (Press release, Celdara Medical, OCT 20, 2021, View Source [SID1234591669]). In this most recent round of funding, two Acceleration Awards (up to $50,000 per early-stage company) were awarded to Olfax Medical (Vermont) and ResusciTech (Rhode Island) and 4 Ignition Awards (up to $25,000 per faculty innovator) were awarded to Andre Khalil, PhD (University of Maine), Gian Ignacio, MD Candidate (Brown), Michael Cole, PhD (Dartmouth), and Yina Huang, PhD (Dartmouth). Awardees were selected from a competitive pool of applicants from the five states DRIVEN serves: DE, RI, VT, ME, NH.

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"We are very pleased to provide recognition and funding to these innovators," said Julie Coleman, PhD, Program Lead at Celdara Medical. "This is a great opportunity for Celdara Medical to deliver on our commitment to give back to the community by supporting early-stage biomedical companies and researchers in their mission to improve patient outcomes and save lives. I am assured that these awards will have a tangible impact on advancing critical technologies, ultimately bringing treatments closer to patients. This round of awardees addresses an array of indications including migraine, myocardial infarction, and multiple cancer types. I am very excited to see the how the DRIVEN awards accelerate these innovative projects."

"On behalf of Celdara Medical and the DRIVEN Accelerator Hub, I’m delighted to recognize this outstanding group of DRIVEN awardees," said Jake Reder, Celdara Medical CEO and Principal Investigator of the DRIVEN Accelerator Hub. "The entrepreneurial spirit – the momentum – in our region is palpable. Engaging with this cohort of world-changing scientists and entrepreneurs has been exceptionally rewarding."

Since the DRIVEN Accelerator Hub’s founding in 2018, 12 awards have been made across Northeast IDeA states. A complete list of awardees with brief technology description are listed below.

Acceleration Fund Awardees ($50,000 to early-stage companies):
Clairways (New Hampshire): Clairways is developing a small, unobtrusive, chest-worn lung health monitor that provides continuous, burden-free tracking of common respiratory signs, including coughing, wheezing, and respiratory patterns, which cannot be practically monitored today.
Extrave Bioscience (Delaware): Extrave uses extracellular vesicles (EV) as a delivery platform to treat damaged/degenerative muscle tissue in patients with muscle diseases, with a primary initial focus on Duchenne muscular dystrophy.
Lodestone Biomedical (New Hampshire): Lodestone enables immediate, in vivo, real-time monitoring of treatment response in immuno-oncology by measuring immune modulators in the tumor microenvironment with a needle implantable biosensor and external magnetic reader.
Olfax Medical (Vermont): Olfax is developing a novel, fast-acting migraine treatment for individuals of all ages who have been diagnosed with acute migraine and cluster headaches.
ResuciTech (Rhode Island): ResusciTech is addressing shortcomings in current CPR training and out-of-hospital cardiac arrest bystander response. The team is marketing a product that utilizes smartphone sensors to measure the depth and rate of chest compressions during CPR and to simultaneously provide audio-visual feedback. This feedback provides the user information needed to adjust compressions for the best possible outcome: saving a life.
Ignition Fund Awardees ($25,000 to innovative faculty)
Andre Khalil (University of Maine): Diagnostic technology that may detect breast cancer 1-4 years earlier than current practices, leading to pre-detection of the disease.
Brent Osborne (University of Vermont): First-in-class PKG-targeted therapies for the treatment of hypertension.
Gian Ignacio (Brown University): Embolic protection device for cardiothoracic procedures to prevent major and minor cerebral complications (e.g., strokes).
Louis Lapierre (Brown University): Targeting XPO1 and the autophagy pathway as a treatment for Alzheimer’s Disease.
Michael Cole (Dartmouth College): Novel platform technology to identify small molecules as inhibitors of the MYC:TRAPP interaction for cancer treatment.
Paul Baures (Keene State University): Fatty acid synthase (FASN) inhibitor to augment cancer chemotherapy and improve clinical outcomes.
Yina Huang (Dartmouth College): Novel CAR T cell platform that enables treatment of solid tumors including metastatic melanoma.

On October 20, 2021 TCR2 Therapeutics Inc. (Nasdaq: TCRR), a clinical-stage cell therapy company with a pipeline of novel T cell therapies for patients suffering from solid tumors, reported new programs and provided highlights from its emerging TRuC pipeline programs during its first virtual R&D Day (Press release, TCR2 Therapeutics, OCT 20, 2021, View Source [SID1234591601]).

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"At TCR2, our mission is to build the next great cell therapy company in solid tumors based on the early success of our mesothelin franchise and an emerging pipeline which will extend our reach into new cancer patient populations and beyond," said Garry Menzel, Ph.D., President and Chief Executive Officer of TCR2 Therapeutics. "Today we will review our narrowed focus on solid tumors and unveil new strategies to potentially further enhance the persistence and efficacy of our TRuC-T cells. In addition, we will introduce compelling preclinical data for our TRuC Tregs, which could expand our footprint into the autoimmune disease setting. We believe that TCR2 is already helping to change the treatment paradigm for patients with treatment-refractory solid tumors and, through continued innovation, will progress our re-prioritized pipeline to patients with a variety of unmet medical needs."

Pipeline Updates

Gavo-cel:

TCR2 announced today the completion of the 3-patient cohort at the new dose level 3.5A (3×108/m2 following lymphodepletion) using a split dosing approach. Two patients were evaluable for safety. In both cases, gavo-cel was well-tolerated with no patients experiencing on-target, off tumors toxicities or Grade ≥3 cytokine release syndrome (CRS) non-hematologic toxicities.
TCR2 anticipates the identification of the RP2D in 4Q21.
TC-110:

TCR2 announced today that, in alignment with its pipeline prioritization on solid tumors, the Company has deprioritized the development of TC-110 for the treatment of patients with CD19+ non-Hodgkin lymphoma or adult acute lymphoblastic leukemia and plans instead to evaluate business development options.
TC-510:

TCR2 announced today the Company anticipates the IND filing for its first TRuC-T cell enhanced with a PD1xCD28 switch receptor to be in 1Q22.
TC-520:

TCR2 announced today the selection of its lead candidate targeting CD70 co-expressing an IL-15 enhancement as TC-520. In new preclinical data highlighted at the R&D Day, TC-520 enhanced with membrane-bound IL-15 resulted in a significant increase in TC-520 cells with a CD8+ naïve/T memory stem cells phenotyope, improved autonomous persistence as well as increased expansion following repeated stimulation with CD70-expressing cancer cell lines.
The Company anticipates initiating IND-enabling studies for TC-520 with an indication focus on renal cell carcinoma in 2022.
Allogeneic:

TCR2 announced today new preclinical data demonstrating allogeneic (off-the-shelf) TRuC-T cells targeting mesothelin that utilized a CRISPR/Cas9 endonucleases approach and the use of fully human TCRγ/δ domains reduced the risk of immunogenicity and host rejection, lacked alloreactivity while maintaining clearance of tumor cells comparable to autologous TRuC-T cells targeting mesothelin.
TCR2 is currently evaluating the combination of enhancements with allogeneic TRuC-T cells to potentially improve persistence.
The Company anticipates the identification of a lead candidate for its allogeneic program in 2022.
TRuC Tregs:

TCR2 announced today new preclinical data demonstrating proof-of-concept for TRuC Treg cells targeting HLA-A*02 for the prevention of Graft versus Host Disease (GvHD). In in vitro and in vivo experiments, TRuC Tregs utilizing the full TCR signaling complex promoted and stabilized Tregs by suppressing the proliferation of mismatched effector cells and inhibiting the production of cytokines in a dose dependent manner.
TCR2 plans to evaluate business development options to enable the treatment of patients with GvHD and other autoimmune diseases.

On October 20, 2021 PTC (Nasdaq: PTC) reported that it will release its fiscal 2021 fourth quarter and full year results on Wednesday, November 3rd after the stock market closes. Senior management will host a live webcast and conference call to review the results on Wednesday, November 3rd at 5pm Eastern Time (Press release, PTC Therapeutics, OCT 20, 2021, View Source [SID1234591617]). The earnings press release, accompanying earnings presentation, and financial data tables will be accessible prior to the conference call and webcast on the Investor Relations section of the Company’s web site at PTC Investor Relations.

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What: PTC Fiscal Q4’21 Conference Call and Webcast

When: Wednesday, November 3rd, 2021 at 5:00pm (ET)

Webcast: View Source

Replay: To access the replay via webcast, please visit View Source.

Please note that statements made on the conference call and webcast are as of the date of the conference call and webcast and PTC does not assume any obligation to update any statements made live or the archived call. Matters discussed may include forward-looking statements about PTC’s anticipated financial results and growth, as well as about the development of products and markets, which are based on current plans and assumptions. Actual results in future periods may differ materially from current expectations due to a number of risks and uncertainties, including those described from time to time in reports filed by PTC with the U.S. Securities and Exchange Commission, including PTC’s most recent reports on Form 10-K and 10-Q.

On October 20, 2021 SpringWorks Therapeutics, Inc. (Nasdaq: SWTX), a clinical-stage biopharmaceutical company focused on developing life-changing medicines for patients with severe rare diseases and cancer, reported that it has entered into an exclusive worldwide license agreement with Dana-Farber Cancer Institute (Dana-Farber) and a sponsored research agreement (SRA) with Stanford Medicine for a portfolio of novel small molecule inhibitors of Epidermal Growth Factor Receptor (EGFR) designed for the treatment of EGFR-mutant lung cancers (Press release, SpringWorks Therapeutics, OCT 20, 2021, View Source [SID1234591670]). The portfolio, which was originally developed in the laboratory of Nathanael Gray, Ph.D., Professor of Chemical and Systems Biology, Co-Director of Cancer Drug Discovery, and Co-Leader of the Cancer Therapeutics Research Program at Stanford Medicine, includes several differentiated, first-in-class chemical series representing novel approaches to targeting mutant EGFR and is designed to address both de novo oncogenic drivers and emerging resistance mutations to existing EGFR inhibitors. The most advanced asset in the portfolio is currently in lead optimization and targets the C797S resistance mutation.

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"We are very pleased to expand our portfolio with the in-license of these selective and differentiated EGFR inhibitors, which are complementary to our strategy of developing novel targeted therapies for patients with biomarker-defined solid tumors," said Saqib Islam, Chief Executive Officer of SpringWorks. "In addition to the monotherapy opportunities in lung cancer, where a significant unmet medical need remains, these EGFR inhibitors provide several avenues for combination therapy development as well."

"Since our lab discovered the initial compounds that could address EGFR T790M mutations, we have been exploring additional strategies to create next-generation agents for patients with EGFR-mutant lung cancers in order to address the resistance mutations that inevitably arise upon treatment with osimertinib and other therapies," said Dr. Gray. "I am grateful for the SRA support from SpringWorks and continued collaboration with my former colleagues at Dana-Farber to further this exciting science with the goal of translating our initial discoveries into meaningful new therapies for cancer patients."

Under the terms of the license agreement, SpringWorks will provide Dana-Farber with an upfront payment and Dana-Farber will be eligible to receive development and commercial milestones and royalties based on any future net sales. Concurrent with this license, SpringWorks has entered a multi-year SRA to fund continued research and development in Dr. Gray’s laboratory at Stanford Medicine as well as collaborating laboratories at Dana-Farber, including those of Pasi Jänne, M.D., Ph.D., Director of the Lowe Center for Thoracic Oncology at Dana-Farber and Professor of Medicine at Harvard Medical School, Michael Eck, M.D., Ph.D., Professor of Biological Chemistry and Molecular Pharmacology at Dana-Farber and Harvard Medical School, and Jarrod Marto, Ph.D., Principal Investigator at Dana-Farber and Associate Professor of Pathology at Brigham and Women’s Hospital and Harvard Medical School. This SRA is intended to support lead optimization and translational biology efforts as the portfolio advances towards development candidate nomination.

"Drs. Gray, Jänne, Eck and Marto have been pioneers in the discovery and development of precision medicines for cancer patients. Through this license agreement and multi-year research collaboration, we have the opportunity to advance programs at the leading edge of targeted oncology development, potentially delivering first- and best-in-class medicines to address emerging drivers of therapeutic resistance," said Mike Burgess M.B.Ch.B., Ph.D., Head of Research & Development at SpringWorks.