TScan Therapeutics Reports Fourth Quarter and Full Year 2024 Financial Results and Provides Business Update

On March 5, 2025 TScan Therapeutics, Inc. (Nasdaq: TCRX), a clinical-stage biotechnology company focused on the development of T cell receptor (TCR)-engineered T cell (TCR-T) therapies for the treatment of patients with cancer, reported financial results for the three months and full year ended December 31, 2024, and provided a business update (Press release, TScan Therapeutics, MAR 5, 2025, View Source [SID1234650906]).

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"The progress we achieved across our pipeline in 2024 has paved the way for a transformative year ahead. We are encouraged by the ALLOHA heme data presented at ASH (Free ASH Whitepaper) with only 2 of 26 patients having relapsed compared to 4 of 12 control-arm subjects. We look forward to presenting additional data from the Phase 1 trial by the end of the year, including two-year relapse data on the initial patients," said Gavin MacBeath, Ph.D., Chief Executive Officer. "For the PLEXI-T solid tumor program, we continue to enroll patients investigating seven different TCR-Ts, including the recently added MAGE-A4 TCR-T (TSC-202-A0201). We look forward to treating our first patient with multiplex therapy in the first half of 2025 and sharing safety and response data for multiplex therapy in the second half of the year."

Recent Corporate Highlights


The Company recently presented updated results from the ongoing ALLOHA trial of TSC-100 and TSC-101 at the 66th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition.

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Infusions with TSC-100 and TSC-101 were well-tolerated with no dose limiting toxicities and adverse events were consistent with hematopoietic cell transplantation (HCT).
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TSC-100 and TSC-101 TCR-T cells have been detected >1 year post infusion and have a clear dose-persistence relationship.
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2 of 26 (8%) treatment-arm subjects relapsed as compared to 4 of 12 (33%) control-arm subjects.

Median time to relapse was not evaluable in TCR-T-treated subjects vs 160 days in the control arm.

Event-free survival strongly favors the treatment arm (HR=0.30).


In December 2024, the Company refinanced its previous convertible debt facility maturing in 2026 with a non-dilutive term loan for up to $52.5 million from Silicon Valley Bank (SVB), a division of First Citizens Bank, of which $32.5 million was drawn at closing. The SVB term loan allows for monthly interest-only payments through September 30, 2027, and matures on September 1, 2029.


In December 2024, the Company completed a $30 million registered direct offering with Lynx1 Capital Management LP (Lynx1), a large existing shareholder of the Company, and an investment fund advised by Lynx1, for pre-funded warrants to purchase up to 7,500,000 shares of the Company’s voting common stock at a price of $4.00 per pre-funded warrant, representing a premium of 37% to the previous closing price of TScan Therapeutics’ common stock. Net proceeds from the offering extended the Company’s cash runway into the first quarter of 2027.


The Company announced that it has been named one of the Top Places to Work in Massachusetts for the third consecutive year in the 17th annual, employee-based survey from The Boston Globe. The 2024 Top Places to Work issue can be found online at Globe.com/TopPlaces.

Upcoming Anticipated Milestones

Heme Malignancies Program: TScan’s two lead TCR-T therapy candidates, TSC-100 and TSC-101, are designed to treat residual disease and prevent relapse in patients with acute myeloid leukemia (AML), acute lymphoblastic leukemia (ALL), or myelodysplastic syndrome (MDS) undergoing allogeneic HCT (the ALLOHA trial, NCT05473910).


Opened expansion cohorts at dose level 3 to further characterize safety and evaluate translational and efficacy endpoints.

Plans to continue development of TSC-101 only, as TSC-101 enables treatment of ~98% of patients with HLA type A*02:01.

Initiate a registration trial for TSC-101, pending further feedback from regulatory authorities, in the second half of 2025.

Plans to present additional data from the Phase 1 trial by the end of the year, including two-year relapse data on the initial patients.

Plans to file an investigational new drug (IND) application for TSC-102-A0301, a TCR-T targeting CD45 on HLA-A*03:01, in the second half of 2025.

Solid Tumor Program: TScan continues to expand the ImmunoBank, a collection of TCR-T therapy candidates that target different cancer-associated antigens presented on diverse HLA types. TScan’s strategy is to treat patients with multiple TCR-T therapy candidates to overcome tumor heterogeneity and resistance that may arise from either target or HLA loss (the PLEXI-T trial, NCT05973487).


IND filing for TCR targeting MAGE-A4 on HLA-A*02:01 (TSC-202-A0201) recently cleared by U.S. Food and Drug Administration (FDA).

The Company now has seven TCR-Ts cleared for clinical development in its PLEXI-T Phase 1 trial.

Progressing through initial dose levels across the TCR-T therapy candidates.

Plans to dose first patient with multiplex TCR-T therapy in the first half of 2025.

Safety and response data for multiplex TCR-T therapy anticipated in the second half of 2025.

Financial Results

Revenue: Revenue for the fourth quarter of 2024 was $0.7 million, compared to $7.2 million for the fourth quarter of 2023, and $2.8 million for the full-year 2024, compared to $21.0 million for the full-year 2023. The decrease in both periods was primarily due to timing of research activities pursuant to the Company’s collaboration agreement with Amgen which commenced in May 2023 compared to the collaboration and license agreement with Novartis which ended in March 2023.

R&D Expenses: Research and development (R&D) expenses for the fourth quarter of 2024 were $29.4 million, compared to $22.4 million for the fourth quarter of 2023, and $107.4 million for the full-year 2024, compared to $88.2 million for the full-year 2023. The period over period increases were primarily driven by an increase in clinical studies expense associated with the ongoing enrollment of our ALLOHA Phase 1 heme trial and start-up activities and initial enrollment in our PLEXI-T Phase 1 solid tumor clinical trial, as well as an increase in personnel expenses due to additional headcount in support of our expanded research and development activities. R&D expenses included non-cash stock compensation expense of $1.3 million and $0.9 million for the fourth quarter of 2024 and 2023, respectively, and $4.8 million and $2.9 million for the full-year 2024 and 2023, respectively.

G&A Expenses: General and administrative (G&A) expenses for the fourth quarter of 2024 were $8.0 million, compared to $6.2 million for the fourth quarter of 2023, and $30.3 million for the full-year 2024, compared to $26.4 million for the full-year 2023. The period over period increases were primarily driven by an increase in personnel expenses due to increased headcount to support business activities. G&A expenses included non-cash stock compensation expense of $1.4 million and $0.6 million for the fourth quarter of 2024 and 2023, respectively, and $4.7 million and $2.3 million for the full-year 2024 and 2023, respectively.

Net Loss: Net loss was $35.8 million for the fourth quarter of 2024, compared to $19.6 million for the fourth quarter of 2023, and included net interest income of $2.0 million and $1.7 million, respectively. Net loss for the full-year 2024 was $127.5 million, compared to $89.2 million for the full-year 2023, and included net interest income of $8.4 million and $4.2 million, respectively. Net loss for the fourth quarter of 2024 and full-year 2024 included a $1.1 million loss on extinguishment of debt.

Cash Position: Cash, cash equivalents, and marketable securities as of December 31, 2024 were $290.1 million, excluding $5.0 million of restricted cash. The Company believes that its existing cash resources will be sufficient to fund its current operating plan into the first quarter of 2027.

Share Count: As of December 31, 2024, the Company had issued and outstanding shares of 56,590,627, which consists of 52,314,039 shares of voting common stock and 4,276,588 shares of non-voting common stock, and outstanding pre-funded warrants to purchase 73,087,945 shares of voting common stock at an exercise price of $0.0001 per share.

Purple Biotech Granted New U.S. Patent for NT219 in Combination with EGFR Antibody Therapy, Enhancing Global IP Protection in Major Markets

On March 5, 2025 Purple Biotech Ltd. ("Purple Biotech" or "the Company") (NASDAQ/TASE: PPBT), a clinical-stage company developing first-in-class therapies that seek to overcome tumor immune evasion and drug resistance, reported that the U.S. Patent and Trademark Office has issued a patent for NT219 used in combination with epidermal growth factor receptor (EGFR) antibodies for treating cancer patients who have acquired resistance to EGFR therapies (Press release, Purple Biotech, MAR 5, 2025, View Source [SID1234650905]). The patent, titled "Combinations of IRS/STAT3 dual modulators and anti-cancer agents for treating cancer" addresses NT219 in combination with cetuximab (Erbitux) as well as other EGFR antibodies such as panitumumab (Vectibix) and necitumumab (Portrazza) in treating tumors resistant to EGFR therapy and in preventing or delaying cancer tumor recurrence following cessation of treatment with an EGFR therapy.

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Purple Biotech’s NT219 is a first-in-class small molecule drug designed to target key cancer resistance mechanisms by degrading IRS1/2 and blocking IGF1R/IRS1 and STAT3 survival pathways.

NT219 recently advanced into a Phase 2 study in second-line patients with recurrent and/or metastatic head and neck cancer to evaluate the drug in combination with standard-of-care head and neck cancer therapies, including cetuximab and pembrolizumab. The head and neck cancer treatment market is forecast to reach $5 billion by 2030.

"This latest U.S. patent completes geographic patent protection of NT219 used in combination with cetuximab in major markets such as the United States, Europe, China and Japan," stated Gil Efron, Purple Biotech CEO. "We believe this additional patent strongly positions us for the potential future commercialization of our novel NT219 therapy."

Purple Biotech’s growing intellectual property portfolio for NT219 in combination with anti-cancer standard-of-care therapies includes a previously issued U.S. patent that covers the pharmaceutical combinations of NT219 with small molecule EGFR inhibitors. This patent has also been granted or allowed in major markets.

Protara Therapeutics Announces Fourth Quarter and Full Year 2024 Financial Results and Provides a Business Update

On March 5, 2025 Protara Therapeutics, Inc. (Nasdaq: TARA), a clinical-stage company developing transformative therapies for the treatment of cancer and rare diseases, reported a business update and announced financial results for the fourth quarter and full year ended December 31, 2024 (Press release, Protara Therapeutics, MAR 5, 2025, View Source [SID1234650904]).

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"Following a year of significant progress and execution across our pipeline, we remain well positioned to deliver on our mission to bring transformative therapies to patients with cancer and rare diseases," said Jesse Shefferman, Chief Executive Officer of Protara Therapeutics. "Notably, supported by positive six-month data from our Phase 2 ADVANCED-2 trial in non-muscle invasive bladder cancer (NMIBC), we continue to believe TARA-002 could represent a differentiated, meaningful addition to the treatment paradigm, both as a monotherapy and in potential combination with other agents. We continue to expect to report initial data from 12-month evaluable patients in our ADVANCED-2 trial by mid-year."

Mr. Shefferman added, "Beyond our NMIBC program, we remain on track to commence our pivotal THRIVE-3 trial of intravenous (IV) Choline Chloride in the first half of the year. We also look forward to providing an interim update from our ongoing Phase 2 STARBORN-1 trial of TARA-002 in lymphatic malformations (LMs) by the end of the first half of 2025. With our cash runway extending into 2027, we look forward to achieving many critical milestones across all of our development programs as we work to deliver new treatment options to patients in need."

Recent Progress and Highlights

The Company is on track to report 12-month data by mid-year from its ongoing Phase 2 open-label ADVANCED-2 trial in evaluable NMIBC patients with carcinoma in situ or CIS (± Ta/T1) who are Bacillus Calmette-Guérin (BCG)-Unresponsive and BCG-Naïve. In December 2024, the Company reported positive interim results in which the complete response (CR) rate across BCG exposures was 72% (13/18) at six months and 70% (14/20) at any time, with 100% (9/9) of patients maintaining a CR from three months to six months. In addition, two of three patients maintained a CR at nine months. TARA-002 showed a favorable safety profile, with no Grade 2 or greater treatment-related adverse events and no treatment discontinuations due to adverse events.
Interim results from approximately 25 six-month evaluable BCG-Unresponsive patients are expected to be announced by the end of 2025. As previously communicated, the BCG-Unresponsive cohort is designed to be registrational in alignment with the 2024 BCG-Unresponsive Non-muscle Invasive Bladder Cancer: Developing Drugs and Biological Products for Treatment Draft Guidance for Industry issued by the U.S. Food and Drug Administration (FDA).
Following regulatory alignment with the FDA, the Company expects to provide an update on the design of its planned BCG-Naïve registrational trial by the end of the first half of 2025.
Protara continues to investigate systemic subcutaneous dosing through priming and maintenance combined with intravesical dosing, as well as exploring combination treatment with TARA-002 in NMIBC patients with CIS. Given what has been observed to date with TARA-002’s mechanism of action and strong safety profile, the Company believes it holds significant potential for use in both systemic dosing and combination therapy and is finalizing various opportunities.
IV Choline Chloride for Patients on Parenteral Support (PS)

The Company plans to initiate THRIVE-3, a registrational Phase 3 clinical trial, in the first half of 2025. THRIVE-3 is a seamless Phase 2b/3 trial with a dose confirmation portion (n=24) followed by a double-blinded, randomized, placebo-controlled portion to assess the efficacy and safety of IV Choline Chloride over 24 weeks in adolescents and adults on long-term PS when oral or enteral nutrition is not possible, insufficient, or contraindicated (n=100). IV Choline Chloride was previously granted Fast Track designation by the FDA.
TARA-002 in LMs

Protara remains on track to provide an interim update on the Phase 2 STARBORN-1 trial of TARA-002 in pediatric patients with macrocystic and mixed cystic LMs by the end of the first half of 2025. The Company previously announced the completion of the study’s first safety cohort, in which TARA-002 showed promising results and was generally well-tolerated.
Fourth Quarter and Full Year 2024 Financial Results

As of December 31, 2024, cash and cash equivalents, and marketable debt securities totaled $170.3 million. The Company expects its cash and cash equivalents, and marketable debt securities will be sufficient to fund its planned operations and data milestones into 2027.
Research and development expenses for the fourth quarter of 2024 increased to $9.5 million from $6.4 million for the prior year period, and for the full year increased to $31.7 million compared to $25.0 million for 2023. The fourth quarter and full year increases were primarily due to an increase in expenses related to clinical trial and non-clinical activities for TARA-002 and IV Choline Chloride.
General and administrative expenses for the fourth quarter of 2024 increased to $4.8 million from $4.7 million for the prior year period, and for the full year decreased to $17.5 million compared to $18.6 million for 2023. The full year decrease was primarily due to a decrease in personnel-related expenses.
For the fourth quarter of 2024, Protara incurred a net loss of $12.8 million, or $0.48 per share, compared with a net loss of $10.2 million, or $0.90 per share, for the same period in 2023. Net loss for the year ended December 31, 2024 was $44.6 million, or $2.17 per share, compared with a net loss of $40.4 million, or $3.57 per share, for the year ended December 31, 2023. Net loss for the fourth quarter of 2024 included approximately $0.9 million of stock-based compensation expenses. Net loss for the year ended December 31, 2024 included approximately $4.1 million of stock-based compensation expenses.
About TARA-002

TARA-002 is an investigational cell therapy in development for the treatment of NMIBC and of 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 by Chugai Pharmaceutical Co., Ltd. Protara has successfully shown 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 pro-inflammatory response with release of cytokines such as tumor necrosis factor (TNF)-alpha, interferon (IFN)-gamma IL-6, IL-10, IL-12. TARA-002 also directly kills tumor cells and triggers a host immune response by inducing immunogenic cell death, which further enhances the antitumor immune response.

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 three 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

IV Choline Chloride is an investigational, intravenous phospholipid substrate replacement therapy in development for patients receiving parenteral support (PS). Choline is a known important substrate for phospholipids that are critical for healthy liver function that also play an important role in modulating gene expression, cell membrane signaling, brain development and neurotransmission, muscle function, and bone health. PS patients are unable to synthesize choline from enteral nutrition sources, and there are currently no available PS formulations containing choline. Approximately 80% of patients dependent on PS are choline-deficient and of those approximately 63% have some degree of liver dysfunction, which can lead to hepatic failure. Every year in the U.S. there are approximately 90,000 people who require PS at home and of those approximately 30,000 are on long-term PS. IV Choline Chloride has the potential to become the first U.S. Food and Drug Administration (FDA) approved IV choline formulation for PS patients. It has been granted Orphan Drug Designation by the FDA for the prevention and/or treatment of choline deficiency in patients on long-term PN and been granted Fast Track Designation as a source of choline when oral or enteral nutrition is not possible, insufficient, or contraindicated. The U.S. Patent and Trademark Office has issued us a U.S. patent claiming a choline composition and a U.S. patent claiming a method for treating choline deficiency with a choline composition, each with a term expiring in 2041.

Corporate overview

On March 5, 2025 Olema oncology presented its corporate presentation (Presentation, Olema Oncology, MAR 5, 2025, View Source [SID1234650903]).

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Jazz Pharmaceuticals to Acquire Chimerix, Further Diversifying Oncology Portfolio

On March 5, 2025 Jazz Pharmaceuticals plc (Nasdaq: JAZZ) ("Jazz" or the "Company") and Chimerix (Nasdaq: CMRX) ("Chimerix"), reported the companies have entered into a definitive agreement for Jazz to acquire Chimerix for $8.55 per share in cash, representing a total consideration of approximately $935 million (Press release, Jazz Pharmaceuticals, MAR 5, 2025, View Source [SID1234650902]). The transaction has been approved by both companies and is expected to close in the second quarter of 2025.

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Chimerix’s lead clinical asset is dordaviprone, a novel first-in-class small molecule treatment in development for H3 K27M-mutant diffuse glioma, a rare, high-grade brain tumor that most commonly affects children and young adults. There are no U.S. Food and Drug Administration (FDA)-approved therapies specifically for H3 K27M-mutant diffuse glioma patients; radiation is the most common treatment approach. A New Drug Application (NDA) for accelerated approval of dordaviprone in recurrent H3 K27M-mutant diffuse glioma was recently accepted and granted Priority Review by FDA. FDA has set a target Prescription Drug User Fee Act (PDUFA) action date of August 18, 2025. If approved in the U.S., dordaviprone may be eligible for a Rare Pediatric Disease Priority Review Voucher (PRV). Separately, dordaviprone is being studied in the ongoing Phase 3 ACTION trial, evaluating its use in newly diagnosed, non-recurrent H3 K27M-mutant diffuse glioma patients following radiation treatment, potentially extending this treatment option into the front-line setting.

"Adding dordaviprone to our oncology R&D pipeline will further diversify our portfolio with a medicine that addresses a significant unmet need with no other FDA-approved therapies and limited treatment options for this patient population. If approved, dordaviprone has the potential to rapidly become a standard of care for a rare oncology disease and also contribute durable revenue beginning in the near-term," said Bruce Cozadd, chairman and chief executive officer, Jazz Pharmaceuticals. "We are encouraged by the dordaviprone clinical trial results to date and look forward to closing the proposed acquisition and working with our new colleagues from Chimerix to fully leverage our combined R&D and commercial expertise to deliver this novel therapy to patients, beginning as early as the second half of this year."

"We are excited to reach this agreement with Jazz Pharmaceuticals as they bring global scale to broaden our dordaviprone commercial strategy," said Mike Andriole, President and CEO of Chimerix. "The transaction, if approved, provides the opportunity to advance access to dordaviprone to reach more patients globally. This announcement is the culmination of years of scientific work by our incredibly talented team, and will deliver significant and certain value to our shareholders."

Key Highlights


Strategic fit that will strengthen Jazz’s presence in the rare oncology space and reinforces commitment to patients with rare diseases with significant unmet need


Dordaviprone has been shown to benefit patients with recurrent H3 K27M-mutant diffuse glioma across several clinical studies, with a consistently favorable safety profile both as monotherapy and in combination with other treatment approaches including radiation


With no currently approved therapies for H3 K27M-mutant diffuse glioma patients, dordaviprone has the potential to rapidly become a standard of care and a meaningful therapy for patients with limited treatment options


Potential near-term commercial launch in the U.S., if approved


FDA has accepted an NDA for dordaviprone seeking accelerated approval for treatment of H3 K27M-mutant diffuse glioma in adult and pediatric patients with progressive disease following prior therapy


NDA has been granted Priority Review and assigned a PDUFA target action date of August 18, 2025


Following the closing of the proposed acquisition and in collaboration with its new colleagues from Chimerix, Jazz plans to leverage its combined development and commercial capabilities to continue advancing the dordaviprone clinical trial program and execute a strong commercial launch, if approved in the U.S.


Will create an additional durable revenue opportunity for Jazz with patent protection into 2037, with potential to receive patent term extension


Ongoing Phase 3 ACTION trial has potential to confirm clinical benefit of dordaviprone in recurrent H3 K27M-mutant diffuse glioma and extend its use to front-line patients

Transaction Terms

Under the terms of the merger agreement, Jazz will commence an all-cash tender offer to acquire all outstanding shares of Chimerix’s common stock, whereby Chimerix shareholders will be offered $8.55 per share in cash, representing a total consideration of approximately $935 million. This reflects an approximately 72% premium based on the closing trading price on March 4, 2025. Upon the successful completion of the tender offer, Jazz will acquire all shares not acquired in the tender through a second-step merger for the same consideration per share paid in the tender offer.

Jazz expects to fund the transaction through existing cash and investments.

Closing Conditions

The transaction is subject to customary closing conditions, including the tender of a majority of the outstanding shares of Chimerix’s voting common stock and other conditions. Chimerix’s Board of Directors unanimously recommends that Chimerix shareholders tender their shares in the tender offer.

Advisors

Guggenheim Securities is serving as financial advisor to Jazz Pharmaceuticals, and Wachtell, Lipton, Rosen & Katz is serving as legal advisor.

Centerview Partners LLC is serving as financial advisor to Chimerix, and Skadden, Arps, Slate, Meagher & Flom LLP and Cooley LLP are serving as legal advisors.

About Dordaviprone

Dordaviprone (ONC201) is a novel first-in-class small molecule imipridone that selectively targets the mitochondrial protease ClpP and dopamine receptor D2 (DRD2). Dordaviprone’s unique mechanism of action includes alterations of key epigenetic modifications such as reversal of H3 K27me3-loss, which is the hallmark of H3 K27M-mutant gliomas.