On March 8, 2023 TScan Therapeutics, Inc. (Nasdaq: TCRX), a clinical-stage biopharmaceutical company focused on the development of T cell receptor (TCR)-engineered T cell therapies (TCR-T) for the treatment of patients with cancer, reported financial results for the three months and full year ended December 31, 2022, and highlighted upcoming anticipated milestones (Press release, TScan Therapeutics, MAR 8, 2023, View Source [SID1234628321]).

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"This will be a pivotal year for TScan. We expect to have patients enrolled in all three arms of our hematologic malignancies program and plan to provide a clinical update by mid-year," said David P. Southwell, President and Chief Executive Officer. "Our solid tumor program is also advancing to the clinic this year. TScan is the first company to launch a trial that will treat cancer patients with multiplexed TCR-T cell therapies matched to the HLA and target profile of their tumors. This approach should help overcome target and HLA loss, which are known mechanisms of resistance. Following the clearance of the first three INDs for our solid tumor program in January, including our T-Plex IND enabling the combination of different TCRs, we are rapidly populating our ImmunoBank to enable a broad range of multiplexed T cell therapies for patients. We plan to file INDs for four additional solid tumor TCRs throughout the year. With $120 million of cash on hand, we are well capitalized to execute on these upcoming milestones."

"We are now actively recruiting patients into our hematologic malignancies program," commented Debora Barton, M.D., Chief Medical Officer. "Several sites are currently recruiting patients, with additional sites to be activated throughout this year. We expect that we will reach the recommended Phase 2 dose for TSC-100 and TSC-101 and report further clinical safety and biomarker data for the program by the end of 2023. Many of the clinical sites enrolling patients in our hematologic malignancies program are planning to join our solid tumor study. That study is planned to begin enrolling patients into the screening protocol by the middle of this year, with patient dosing expected to commence in the third quarter. We plan to share initial safety and biomarker data for single agent TCRs by the end of 2023, with initial multiplexing data expected in the first half of 2024."

"TScan’s TCR-T cell products have been designed to improve upon first-generation TCR-T in two important ways: by increasing depth of response and by extending duration of response," said Gavin MacBeath, Ph.D., Chief Scientific and Operations Officer. "Our non-viral, in-house manufacturing platform allows us to add enhancements to our TCRs, including CD8α/β to

increase depth of response by enlisting helper T cells, and a dominant negative form of TGFβRII to overcome inhibition of T cells by TGFβ in the hostile tumor microenvironment. To address antigen heterogeneity and HLA loss, our recently approved T-Plex IND allows us to multiplex our enhanced TCR-Ts across targets and HLA types at the same time, bringing us closer to our goal of providing customized TCR-T therapy to treat patients with a wide variety of solid tumor malignancies."

Recent Corporate Highlights

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TScan filed three investigational new drug (IND) applications with the U.S. Food and Drug Administration (FDA) for its solid tumor program in December 2022. The IND applications for T-Plex, TSC-204-A0201, and TSC-204-C0702 have now been cleared by the FDA. T-Plex will serve as the primary IND for TScan’s solid tumor program, enabling customized mixtures of TCR-Ts to be administered to patients based on tumor antigen positivity and HLA expression. Along with T-Plex, the FDA has approved secondary INDs for two TCR-T products, TSC-204-A0201 and TSC-204-C0702, that target melanoma-associated antigen 1 (MAGE-A1) presented on HLA A*02:01 and C*07:02, respectively. MAGE-A1 is a validated cancer-associated antigen overexpressed in 45% of head and neck cancers and 50% of melanoma, cervical, and non-small cell lung cancers. TScan believes that TSC-204-C0702 is the first TCR to enter clinical trials for an epitope of MAGE-A1 presented on an HLA type other than A*02:01.

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In December 2022, the Company presented a poster on the clinical trial design and trial in progress for the Phase 1 umbrella trial of TSC-100 and TSC-101 to treat residual leukemia and prevent relapse after hematopoietic cell transplant (HCT) at the 64th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting 2022.

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In November 2022, the Company presented two posters at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) 37th Annual Meeting:

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Discovery of PRAME-specific TCR-T cell therapy candidates for the treatment of solid tumors, presented by Mollie Jurewicz, Ph.D.
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Multiplexed TCR-T cell therapy targeting MAGE-A1 and PRAME enhances the activity of adoptive T cell therapy in pre-clinical models, presented by Antoine Boudot, Ph.D.

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During the fourth quarter of 2022, TScan was named the best mid-sized biotechnology company in the Top Places to Work for 2022 by The Boston Globe. Top Places to Work recognizes the most admired workplaces in the state voted on by the people who know them best – their employees. The survey measures employee opinions about their Company’s direction, execution, connection, management, work, pay and benefits, and engagement.

Upcoming Anticipated Milestones

Hematologic Malignancies Program:

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Expects to enroll patients in all three arms in the Phase 1 umbrella trial (NCT05473910) for TSC-100 and TSC-101 and start reporting preliminary safety and biomarker data mid-year 2023.
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Expects to reach the recommended Phase 2 dose for TSC-100 and TSC-101 and report interim clinical data for the program by the end of 2023.

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The Company expects to report prevention of relapse data in 2024.

Solid Tumor Program:

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Anticipate further expansion of the ImmunoBank by filing INDs for four TCRs throughout 2023.
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Anticipate sharing preliminary safety and biomarker data for the most advanced TCRs by the end of 2023.
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The Company expects to report initial multiplex therapy data for its first combination of TCRs under T-Plex in the first half of 2024.

Fourth Quarter 2022 Financial Results

Revenue for the three months ended December 31, 2022, was $3.1 million, compared to $2.9 million for the three months ended December 31, 2021 (2021 Quarter). This increase is due to the timing of research activities related to our target discovery collaboration with Novartis (the "Novartis Agreement").

Research and development expenses for the three months ended December 31, 2022, were $15.6 million, compared to $12.6 million for the 2021 Quarter. The increase of $3.0 million was primarily driven by an increase in personnel expense, expansion of leased facilities, and preclinical activities to support solid tumors, and Phase 1 study start-up activities for TSC-100 and TSC-101.

General and administrative expenses for the three months ended December 31, 2022, were $6.1 million, compared to $4.4 million for the 2021 Quarter. The increase of $1.7 million in general and administrative expenses was primarily driven by increased personnel and facilities related costs to support the progress of the Company into the clinic.

For the three months ended December 31, 2022, TScan Therapeutics reported a net loss of $18.7 million, compared to a net loss of $14.2 million for the 2021 Quarter.

Full Year 2022 Financial Results

As of December 31, 2022, TScan Therapeutics had cash and cash equivalents of $120.0 million excluding $5.0 million of restricted cash. Based on current operating plans, the Company believes that existing cash and cash equivalents will be sufficient to fund its operating expenses and capital expenditure requirements into the second quarter of 2024.

Revenue for the year ended December 31, 2022, was $13.5 million, compared to $10.1 million for the year ended December 31, 2021 (2021 Period). This increase is due to the timing of research activities related to the Novartis Agreement.

Research and development expenses for the year ended December 31, 2022, were $59.8 million, compared to $45.0 million for the 2021 Period. The increase of $14.8 million was primarily driven by an increase in personnel expense, expansion of leased facilities, preclinical activities to support solid tumors, and Phase 1 study start-up activities for TSC-100 and TSC-101.

General and administrative expenses for the year ended December 31, 2022, were $20.4 million, compared to $13.8 million for the 2021 Period. The increase of $6.5 million in general and administrative expenses was primarily driven by increased personnel and facilities related costs to support the progress of the company into the clinic

For the year ended December 31, 2022, TScan Therapeutics reported a net loss of $66.2 million, compared to a net loss of $48.6 million for the 2021 Period

As of December 31, 2022, the Company had issued and outstanding a combined total of 24,225,954 shares of voting common stock and non-voting common stock.

On March 8, 2023 Tessa Therapeutics Ltd. (Tessa), a clinical-stage cell therapy company developing next-generation cancer treatments for hematological malignancies and solid tumors, reported entry into a Cooperative Research and Development Agreement (CRADA) with the U.S. National Cancer Institute (NCI), part of the National Institutes of Health (NIH), to investigate TT11X, Tessa’s allogeneic "off-the-shelf" CD30.CAR-modified Epstein-Barr virus-specific T-cell (EBVST) therapy, in multiple subtypes of non-Hodgkin lymphoma (Press release, Tessa Therapeutics, MAR 8, 2023, View Source [SID1234628320]).

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Under terms of the CRADA, Tessa will collaborate with the NCI’s Division of Cancer Treatment and Diagnosis (DCTD) to identify potential opportunities to expand the applicability of TT11X as a treatment of non-Hodgkin lymphoma. In this collaboration, NCI Cancer Therapy Evaluation Program (CTEP) will serve as the regulatory sponsor and conduct mutually approved clinical trials through NCI funded clinical network groups and using drug supply and other necessary support provided by Tessa. Tessa is currently advancing a Phase 1 clinical trial in the United States investigating TT11X in CD30-positive lymphomas. Data from the Phase 1 study presented at the 64th Annual Meeting of the American Society of Hematology (ASH) (Free ASH Whitepaper) demonstrated TT11X to be well-tolerated at all dosing levels, eliciting a 79% overall response rate and 43% complete response rate among 14 heavily pre-treated CD30-positve Hodgkin lymphoma patients.

"We are pleased to enter into a CRADA with NCI.," said Tessa President and CEO Thomas Willemsen. "The collaboration is a strong incentive and encouragement for us to continue developing our scientific platform and will significantly expand our ability to conduct clinical trials with TT11X in a wide number of indications. Based on compelling safety and efficacy data from the Phase 1 trial of TT11X in patients with CD30-positive Hodgkin lymphoma, we believe there is a substantial opportunity to direct our ‘off-the-shelf’ therapy to other CD30-positive subtypes of non-Hodgkin lymphoma, where there is high patient need. We look forward to working with the clinical research team at the DCTD as well as DCTD’s funded extramural clinical network groups and benefitting from their breadth of research experience and scientific resources."

Tessa is advancing a pipeline of products that utilize CD30.CAR-modified EBVSTs, including its lead allogeneic cell therapy, TT11X, which is being co-developed with the Baylor College of Medicine for the treatment of relapsed or refractory CD30-positive lymphomas (NCT04288726). Tessa plans to extend its allogeneic EBVST platform to other cancer indications, including solid tumors.

Tessa’s proprietary "off-the-shelf" CD30.CAR-EBVST allogeneic cell therapy platform is based on decades-long research and development by researchers at Baylor College of Medicine into the unique properties of virus-specific T-cells (VSTs). These highly specialized T cells have the ability to recognize and kill infected cells while activating other parts of the immune system for a coordinated response. CD30.CAR-modified allogeneic EBVSTs without gene editing have demonstrated a strong safety profile and efficacy in early trials with minimal risk of graft-versus-host disease (GVHD).

On March 8, 2023 Synlogic, Inc. (Nasdaq: SYBX), the leading company advancing therapeutics based on synthetic biology, reported that Aoife Brennan, M.B. Ch.B., Synlogic President and Chief Executive Officer, and members of the leadership team, will provide a company presentation at the Oppenheimer 33rd Annual Healthcare Conference being held virtually from March 13-15, 2023 (Press release, Synlogic, MAR 8, 2023, View Source [SID1234628319]). Presentation details are provided below:

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Date: Monday, March 13, 2023
Time: 3:20-3:50 p.m. ET
Webcast: Register for the presentation here.

A replay of the presentation will be available for 90 days. Recorded presentations, if available, can be found on the Events section of the Synlogic website.

On March 8, 2023 Replimune Group, Inc. (NASDAQ: REPL), a clinical stage biotechnology company pioneering the development of a novel portfolio of tumor-directed oncolytic immunotherapies, reported that members from the Replimune management team will present and host investor meetings at the following two conferences (Press release, Replimune, MAR 8, 2023, View Source [SID1234628318]).

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Barclays Global Healthcare Conference
Date: Wednesday, March 15, 2023
Fireside Chat Time: 2:05 pm EDT
Location: The Loews Miami Beach Hotel, Miami, FL

Jefferies Biotech on the Bay Summit
Date: Thursday, March 16, 2023
Location: 1 Hotel South Beach, Miami, FL
In-person 1×1 investor meetings only

On March 8, 2023 Protara Therapeutics, Inc. (Nasdaq: TARA), a clinical-stage company developing transformative therapies for the treatment of cancer and rare diseases, reported financial results for the full year and fourth quarter ended December 31, 2022, and provided a business update (Press release, Protara Therapeutics, MAR 8, 2023, View Source [SID1234628317]).

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"With key data and milestones expected from our clinical programs for TARA-002, including data from the Phase 1a portion of the ADVANCED-1 trial in non-muscle invasive bladder cancer (NMIBC) in the second quarter of 2023, we believe this year will be a particularly exciting time for Protara," said Jesse Shefferman, Chief Executive Officer of Protara Therapeutics. "We look forward to leveraging data from the ADVANCED-1 trial to inform the design of further clinical studies in our NIMBC program. For our program in lymphatic malformations (LMs), a highly underserved pediatric population for which we believe TARA-002 could serve as a meaningful intervention, we have begun Phase 2 clinical trial start up activities and anticipate initiating the trial in the second half of this year. We believe we are well positioned to successfully execute on our pipeline programs and look forward to providing updates in due course."

Recent Highlights

TARA-002 in NMIBC

In December 2022, the Company’s Phase 1 ADVANCED-1 clinical trial of TARA-002, Protara’s investigational cell-based immunopotentiator for the treatment of NMIBC, was featured in a Trials in Progress poster at the Annual Meeting of the Society of Urologic Oncology.
The Company expects to report data from the Phase 1a portion of the trial in the second quarter of 2023 and move rapidly into the Phase 1b expansion portion of the trial, which will evaluate safety and efficacy in patients with carcinoma in situ (CIS).
TARA-002 in LMs

The Company has initiated study start up activities for a Phase 2 clinical trial of TARA-002 in pediatric patients with macrocystic and mixed-cystic LMs.
IV Choline Chloride in Intestinal Failure Associated Liver Disease (IFALD)

Protara’s prospective study to enhance understanding of the incidence of IFALD in patients dependent on parenteral nutrition is ongoing with results expected in the third quarter of 2023.
The Company plans to leverage results from the prospective study, as well as its completed retrospective study, to inform next steps for the IV Choline Chloride development program.
Corporate Updates

In January 2023, Protara announced the appointment of Patrick Fabbio as Chief Financial Officer. Mr. Fabbio brings to Protara more than 30 years of experience at various life science and pharmaceutical companies and most recently served as President and Chief Financial Officer at NYSE-listed Rafael Holdings, Inc.
Fourth Quarter and Full Year 2022 Financial Results

As of December 31, 2022, cash, cash equivalents and marketable debt securities totaled $102.3 million. The Company expects its cash, cash equivalents, and marketable debt securities will be sufficient to fund its planned operations and data milestones into 2025.

Research and development expenses for the fourth quarter of 2022 increased to $5.0 million from $4.1 million for the prior year period, and for the full year decreased to $16.8 million compared to $21.1 million for 2021. The fourth quarter increase was primarily due to an increase in non-clinical studies performed in the quarter versus the comparable period. The full year decrease was primarily due to a reduction in clinical manufacturing expenses.

General and administrative expenses for the fourth quarter of 2022 decreased to $5.0 million from $6.2 million for the prior year period, and for the full year decreased to $20.7 million compared to $26.4 million for 2021. The fourth quarter and full year decreases were primarily due to a reduction in stock based compensation expense and market development activities.

For the fourth quarter of 2022, Protara reported a net loss of $39.0 million, or $3.46 per share, compared with a net loss of $10.2 million, or $0.91 per share, for the same period in 2021. Net loss in the fourth quarter of 2022 included a non-cash goodwill impairment charge of $29.5 million associated with the accounting for the reverse merger transaction in January of 2020. Net loss for the year ended December 31, 2022 was $66.0 million, or $5.86 per share, compared with a net loss of $47.3 million, or $4.21 per share, for the year ended December 31, 2021. Net loss for the fourth quarter included approximately $1.4 million of stock-based compensation expenses. Net loss for the year ended December 31, 2022 included approximately $6.7 million of stock-based compensation expenses.
About TARA-002

TARA-002 is an investigational cell therapy in development for the treatment of NMIBC and LMs for which it has been granted Rare Pediatric Disease Designation by the U.S. Food and Drug Administration. TARA-002 was developed from the same master cell bank of genetically distinct group A Streptococcus pyogenes as OK-432, a broad immunopotentiator marketed as Picibanil in Japan and Taiwan by Chugai Pharmaceutical Co., Ltd. Protara has successfully demonstrated manufacturing comparability between TARA-002 and OK-432.

When TARA-002 is administered, it is hypothesized that innate and adaptive immune cells within the cyst or tumor are activated and produce a strong immune cascade. Neutrophils, monocytes and lymphocytes infiltrate the abnormal cells and various cytokines, including interleukins IL-2, IL-6, IL-8, IL-10, IL-12, interferon (IFN)-gamma, tumor necrosis factor (TNF)-alpha, granulocyte colony-stimulating factor, and granulocyte-macrophage colony-stimulating factor are secreted by immune cells to induce a strong local inflammatory reaction and destroy the abnormal cells.

About Non-Muscle Invasive Bladder Cancer (NMIBC)

Bladder cancer is the 6th most common cancer in the United States, with NMIBC representing approximately 80% of bladder cancer diagnoses. Approximately 65,000 patients are diagnosed with NMIBC in the United States each year. NMIBC is cancer found in the tissue that lines the inner surface of the bladder that has not spread into the bladder muscle.

About Lymphatic Malformations (LMs)

LMs are rare, congenital malformations of lymphatic vessels resulting in the failure of these structures to connect or drain into the venous system. Most LMs are present in the head and neck region and are diagnosed in early childhood during the period of active lymphatic growth, with more than 50% detected at birth and 90% diagnosed before the age of 3 years. The most common morbidities and serious manifestations of the disease include compression of the upper aerodigestive tract, including airway obstruction requiring intubation and possible tracheostomy dependence; intralesional bleeding; impingement on critical structures, including nerves, vessels, lymphatics; recurrent infection, and cosmetic and other functional disabilities.

About IV Choline Chloride and Intestinal Failure-associated Liver Disease (IFALD)

IV Choline Chloride is an investigational, intravenous (IV) phospholipid substrate replacement therapy initially in development for patients receiving parenteral nutrition (PN) who have IFALD. Choline is a known important substrate for phospholipids that are critical for healthy liver function. Because PN patients cannot sufficiently absorb adequate levels of choline and no available PN formulations contain sufficient amounts of choline to correct this deficiency, PN patients often experience a prolonged progression to hepatic failure and death, with the only known intervention being a dual small bowel/liver transplant. If approved, IV Choline Chloride would be the first approved therapy for IFALD. It has been granted Orphan Drug Designations (ODDs) by the FDA for the treatment of IFALD and the prevention of choline deficiency in PN patients.