On December 9, 2024 Protara Therapeutics, Inc. (Nasdaq: TARA) ("Protara"), a clinical-stage company developing transformative therapies for the treatment of cancer and rare diseases, reported the pricing of its underwritten public offering of 13,690,000 shares of its common stock at a price to the public of $6.25 per share and pre-funded warrants to purchase 2,325,372 shares of common stock at a price of $6.249 per pre-funded warrant, which represents the per share price for the common stock less the $0.001 per share exercise price for each such pre-funded warrant (Press release, Protara Therapeutics, DEC 10, 2024, View Source [SID1234648992]). In addition, Protara has granted the underwriters a 30-day option to purchase up to an additional 2,402,305 shares of common stock at the public offering price, less underwriting discounts and commissions. All shares and pre-funded warrants in the offering are being sold by Protara. The gross proceeds from the offering are expected to be approximately $100 million before deducting underwriting discounts and commissions and offering expenses payable by Protara and excluding any exercise of the underwriters’ option to purchase additional shares and the exercise of any pre-funded warrants. The offering is expected to close on December 11, 2024, subject to satisfaction of customary closing conditions. Protara intends to use the net proceeds received from the offering to fund the clinical development of TARA-002, as well as the development of other clinical programs. Protara may also use the net proceeds from the offering for working capital and other general corporate purposes.

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TD Cowen, Cantor, LifeSci Capital, Oppenheimer & Co. and Scotiabank are acting as joint book-running managers of the offering.

The shares of common stock and the pre-funded warrants will be issued pursuant to an effective shelf registration statement on Form S-3 (File No. 333-275290) that was declared effective on November 14, 2023 by the U.S. Securities and Exchange Commission (the "SEC"). The offering is being made only by means of a prospectus supplement and the accompanying prospectus. A final prospectus supplement relating to the offering will be filed with the SEC and will be available on the SEC’s website, located at www.sec.gov. Copies of the final prospectus supplement and the accompanying prospectus relating to the offering, when available, may be obtained from the offices of TD Securities (USA) LLC, 1 Vanderbilt Avenue, New York, New York 10017, by email at [email protected] or by telephone at (855) 495-9846; Cantor Fitzgerald & Co., 110 East 59th Street, 6th Floor, New York, New York 10022, Attention: Capital Markets, or by email at [email protected]; or LifeSci Capital LLC, 1700 Broadway, 40th Floor, New York, New York 10019, or by email at [email protected].

This press release shall not constitute an offer to sell or a solicitation of an offer to buy any of these securities, nor shall there be any sale of these securities in any state or jurisdiction in which such an offer, solicitation or sale would be unlawful prior to registration or qualification under the applicable securities laws of such state or jurisdiction.

On December 10, 2024 NextCure, Inc. (Nasdaq: NXTC), a clinical-stage biopharmaceutical company committed to discovering and developing novel, first-in-class, and best-in-class therapies to treat cancer, reported that the U.S. Food and Drug Administration (FDA) accepted an Investigational New Drug (IND) application for initiation of a Phase 1 clinical trial to evaluate LNCB74, a B7-H4-targeting antibody-drug conjugate (ADC) as a therapeutic for treating multiple cancers (Press release, NextCure, DEC 10, 2024, View Source [SID1234648990]).

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"Acceptance of the IND application for LNCB74 represents an important milestone for NextCure as we focus our resources on advancing our ADC program," said Michael Richman, NextCure’s president and CEO. "The IND application leverages LNCB74 preclinical data that highlights the differentiation of our B7-H4 ADC from other ADCs targeting B7-H4. We believe LNCB74 has the potential to transform treatment for patients and we look forward to advancing LNCB74 into clinical development."

An IND is a request submitted to the regulatory authorities seeking permission to test a new drug or therapeutic substance in humans. The IND includes detailed information about the drug, its composition, pharmacology, toxicology, data from preclinical studies, proposed clinical trial protocols, and information on manufacturing and quality control. With the IND application acceptance now complete, NextCure can proceed with the Phase I trial.

LNCB74 is being developed in partnership with LigaChem Biosciences, Inc. as part of a collaboration and co-development agreement.

On December 10, 2024 Neogap Therapeutics AB, a Swedish clinical-stage biotechnology company, reported that the European Medicines Agency (EMA) has granted Advanced Therapy Medicinal Product (ATMP) classification to its cell therapy, pTTL (personalised Tumour Trained Lymphocytes) (Press release, Neogap Therapeutics, DEC 10, 2024, View Source,c4079677 [SID1234648989]). This classification confirms that pTTL meets the regulatory criteria for a somatic cell therapy product, providing a clear regulatory pathway for its further development.

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The ATMP classification is a key milestone for companies developing advanced therapies. It signifies that the EMA formally recognises the product as an advanced therapy under EU regulations. This designation offers access to the EMA’s tailored scientific advice, ensuring that the therapy’s safety, quality, and efficacy meet stringent regulatory standards. For Neogap, the classification sets a clear direction for preparing upcoming clinical trials and advancing towards future market authorisation.

Neogap’s personalised cell therapy is currently being evaluated in a Phase I/II clinical trial focused on assessing its safety and tolerability in patients with advanced colorectal cancer. This study is a critical step in Neogap’s mission to provide curative treatments for cancer patients with limited remaining treatment options.

"The ATMP classification is a strong validation of our innovative approach and pTTL’s potential as a breakthrough cancer therapy," says Samuel Svensson, CEO of Neogap Therapeutics. "It provides regulatory clarity and invaluable guidance from the EMA, enabling us to advance development efficiently and effectively. We are now well-positioned to continue our work towards delivering transformative treatments to cancer patients."

On December 10, 2024 Leapfrog Bio, a clinical-stage precision oncology company identifying novel therapies for undruggable cancer-driving mutations, reported that Tomas Babak, PhD, the Company’s Chief Scientific Officer, will present evidence that Leapfrog Bio’s Precision PGx Platform discovered a clinically significant link between the EP300 gene and bromodomain and extra-terminal domain (BET) protein inhibitors (Press release, Leapfrog Bio, DEC 10, 2024, View Source [SID1234648988]). The presentation titled, "EP300 loss of function is a pan-cancer sensitizer to BET inhibition," will take place at the AACR (Free AACR Whitepaper) Special Conference in Cancer Research in Toronto, Canada and will focus on the discovery, characterization and clinical development plan for Leapfrog Bio’s BET inhibitor LFB-190 to treat solid tumors with EP300 loss of function.

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Loss of transcriptional control is a hallmark of cancer and can be an essential driver of cancer initiation and progression. BET proteins regulate the expression of multiple cancer-related genes and pathways; several molecules targeting this family of proteins have been tested in early phase clinical trials, although these molecules had shown limited clinical success as monotherapies. After conducting hundreds of pharmacogenetic screens using its Precision PGx Platform, Leapfrog Bio discovered an interaction between BET protein inhibition and the EP300 gene loss of function (LOF) similar to PARP-BRCA1/HRD in significance. The Company believes it has also discovered the mechanism of action for this synthetically lethal relationship between BET protein inhibition and EP300 LOF.

"It was gratifying to identify this essential interaction, as there has been so much research and activity related to BETs inhibitors," said Tomas Babak, PhD, CSO of Leapfrog Bio. "Discovering a strong interaction between BET proteins and the mutated EP300 gene resulting in LOF—as well as the mechanism that drives it—using the Precision PGx Platform is a meaningful validation of the platform’s ability to identify therapies for previously undruggable cancers caused by LOF mutations. Two-thirds of all cancers are caused by mutations in tumor suppressor genes, such as what we see here with EP300, yet less than one percent of LOF tumor suppressor genes have been drugged to date. With Leapfrog Bio’s platform, we can repeatedly uncover candidates that address the unmet needs of patients with cancers caused by LOF mutations."

Leapfrog’s Lead Candidate, LFB-190, exploits the relationship between BET inhibition and EP300 LOF mutations. The Company’s Phase 2-ready compound was clinically studied prior to Leapfrog’s research and a recommended Phase 2 dose has been established.

Greg Vontz, CEO of Leapfrog Bio added, "This discovery demonstrates the ability of the Precision PGx Platform to uncover potential therapeutic solutions for the 10M patients with cancers driven by LOF mutations. We have filed IP around this discovery and are currently preparing for LFB-190’s entry into Phase 2 in lung, colon and bladder cancers in 2025."

On December 10, 2024 IN8bio, Inc. (Nasdaq: INAB), a clinical-stage biopharmaceutical company developing innovative gamma-delta T cell therapies, reported updated data from the ongoing Phase 1 trial of INB-100, an allogeneic, haploidentical gamma-delta T cell therapy in older patients with hematologic malignancies undergoing haploidentical stem cell transplant (HSCT) with reduced intensity conditioning (RIC) at the 2024 American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting, being hosted in San Diego, CA (Press release, In8bio, DEC 10, 2024, View Source [SID1234648986]).

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"This data demonstrates the potential of allogeneic INB-100 gamma-delta T cells to provide durable relapse-free remissions in high-risk or relapsed AML patients undergoing HSCT," said Dr. Joseph P. McGuirk, Schutte-Speas Professor of Hematology-Oncology, Division Director, Hematologic Malignancies and Cellular Therapeutics Medical Director, Blood and Marrow Transplant, The University of Kansas Cancer Center. "Older, frailer patients who receive non-myeloablative, reduced intensity conditioning regimens typically have a significant risk of relapse. Historically, approximately 25% of AML patients undergoing HSCT would be expected to have a leukemic relapse within the first 100 days post-transplant, with up to nearly 50% of such patients experiencing relapse by one-year, which remains the primary cause of death. The longer AML patients remain in remission post-HSCT, the greater their probability of survival. These observed long-term durable remissions using allogeneic gamma-delta T cells are very encouraging and we look forward to announcing additional data next year."

In a poster presentation, IN8bio reported that there have been no newly reported deaths or relapses as of September 30, 2024. As of that cutoff date, median CR was at 16.4 months following a median of 19.2 months of follow-up. As previously reported, all patients (n=10) remained alive, progression-free, and in durable CR through one-year. 100% of AML patients remain in CR after a median 19.7 months of follow-up with three patients with high-risk cytogenetic AML and receiving no maintenance therapy remaining in mCR for greater than three years.

INB-100 continues to demonstrate in vivo expansion and persistence of an haplo-matched allogeneic, or donor-derived cellular, therapy at 365 days with blood levels of gamma-delta T cells surpassing levels previously observed to be associated with greater survival. The persistence of these cells is suggestive of continued gamma-delta T cell surveillance against leukemic relapse.

In addition to the reported complete responses, INB-100 continued to demonstrate a well-tolerated safety profile with no cytokine release syndrome (CRS) or neurotoxicity (ICANS) observed and limited mild infections. Based upon these encouraging results, the INB-100 trial has been expanded to enroll additional patients at Dose Level (DL) 2, the recommended Phase 2 dose (RP2D). Enrollment of additional patients into the expansion cohort is on-going and updated data, are expected to be reported in the first half of 2025.

Summary of Data Presented at ASH (Free ASH Whitepaper)

The Phase 1 investigator-sponsored trial enrolled and treated ten patients at one of two dose levels (D1 or D2). The median age was 68 years with the majority of patients diagnosed with AML in CR1. Two patients (009 and 011) had TP53 mutations, a tumor suppressor that results in poor prognosis, rapid progression and reduced lifespan due to an inability to respond to mutated or damaged DNA.

The latest INB-100 trial data on immune reconstitution continues to show significant allogeneic gamma-delta T cell expansion and persistence in patients through the first 365 days post-treatment. As of September 30, 2024, 100% of patients (n=10) surpassed one-year survival following their haplo-matched transplant and treatment with INB-100. Historically, approximately 25% of patients relapse by 100 days and 40-50% of patients relapse by one year.

Updated safety data includes:

No dose limiting toxicities (DLTs) and no treatment related deaths were observed.
Low grade (1-2) acute graft versus host disease (GvHD) observed in 60% of patients treated. Cases were all steroid responsive.
Treatment-related serious adverse events included Grade 2 rash (60%) and Grade 3 nausea (20%).
One patient death previously reported due to idiopathic pulmonary syndrome likely related to the underlying HSCT at 15.5 months, without disease progression.
No ICAN, CRS, or major infections were observed.
Seven patients across DL 1 and DL 2 remained on study and in CR, with three having surpassed three years, including one now remaining progression free for over four years.