On June 11, 2024 Monopar Therapeutics Inc. (Nasdaq: MNPR), a clinical-stage radiopharma company, and NorthStar Medical Radioisotopes, LLC, a global innovator in development and commercial production of medical radioisotopes, reported an amendment and expansion to their existing collaboration (Press release, Monopar Therapeutics, JUN 11, 2024, View Source [SID1234644265]). Under terms of the revised collaboration, the Companies entered into a long-term, non-exclusive master supply agreement for NorthStar to provide Monopar with the powerful therapeutic radioisotope actinium-225 (Ac-225). The amendment builds on NorthStar’s significant investment in Ac-225 manufacturing and Monopar’s promising preclinical therapeutic results with Ac-225 in its lead MNPR-101 radiopharma program.

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The amendment also clarifies certain economic terms and those related to jointly developed intellectual property rights for Monopar’s MNPR-101 for radiopharmaceutical use. Monopar has acquired those rights from NorthStar, together with certain broad, jointly developed intellectual property pertaining to MNPR-101, giving Monopar full ownership and title to its lead MNPR-101 radiopharmaceutical platform. Both companies will share ownership of the filed patent application on the use of PCTA as a linker with Ac-225, which has shown superior binding and yield with Ac-225 over the current industry-leading linker, DOTA.

"We are excited about the evolution of our collaboration, the promising potential of the MNPR-101 radiopharma platform, and the long-term access to a high-quality source of Ac-225 that can support our current development programs and potential future commercial products," said Chandler Robinson, MD, Monopar’s Chief Executive Officer.

"Using our proprietary electron accelerator technology, NorthStar is poised to be the first commercial-scale producer of non-carrier added (n.c.a.) Ac-225. We’re delighted to continue and extend our collaboration with Monopar and support development of its exciting MNPR-101 radiopharma platform focused on therapeutic agents to treat aggressive cancers," said Frank Scholz, PhD, NorthStar’s Chief Executive Officer.

On June 11, 2024 Nykode Therapeutics ASA (OSE: NYKD), a clinical-stage biopharmaceutical company dedicated to the discovery and development of novel immunotherapies, reported updates on its mRNA platform at the Cancer Immunotherapy meeting in Boston (Press release, Nykode Therapeutics, JUN 11, 2024, View Source [SID1234644264]). The company highlighted the significant advantages of its innovative APC-targeted neoantigen vaccine, delivered in a mRNA-lipid nanoparticle (LNP) format.

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This latest study demonstrated that the APC-targeted vaccine consistently achieved a broader and more robust immune response across doses compared to an untargeted neoantigen vaccine. It also provided superior tumor control and resulted in dramatically improved survival rates in a mouse model of colorectal cancer

Chief Scientific Officer, Agnete Fredriksen, commented on the findings, "Our latest data not only emphasize the effectiveness of the APC-targeted approach but also confirm our platform’s potential across modalities to significantly advance the treatment landscape for cancer. The strong data demonstrating Nykode’s potential to improve mRNA vaccines underscore our commitment to leading the way in next-generation immunotherapies."

Nykode presented a poster detailing these findings at the meeting, with further information available on the Nykode website: View Source

On June 11th, 2024 – Orphelia Pharma, a pharmaceutical company dedicated to the development and marketing of pediatric and orphan medicines, reported that KIZFIZO, temozolomide oral suspension, will be included in the MetroWILMS-1906 study (EudraCT: 2021-002540-67, NCT05384821) (Press release, ORPHELIA Pharma, JUN 11, 2024, View Source;utm_medium=rss&utm_campaign=orphelia-pharma-to-collaborate-with-oscar-lambret-clinical-center-to-study-kizfizo-the-first-pediatric-drinkable-formulation-of-temozolomide-for-the-treatment-of-refractory-or-relapsed-nephrob [SID1234644263]). MetroWILMS is a French, multicentric, non-randomized, Phase 1-2 clinical trial to study the efficacy and safety of metronomic chemotherapy, including temozolomide, for the treatment of refractory or relapsed nephroblastoma (Wilms tumor). MetroWILMS is sponsored by the Oscar Lambret clinical center on behalf of the Société Française des Cancers de l’Enfant (SFCE), Nephroblastoma committee.

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Pediatric renal (kidney) tumors account for 4%-11% of all childhood cancers, the most common of which being nephroblastoma or Wilms tumor. It affects young children, most of the cases being diagnosed before 5 years of age. Despite a good prognosis, 10 to 15 % of patients present a refractory Wilms tumor or experience relapse and bear a dismal prognosis.

As part of the MetroWILMS protocol, KIZFIZO (under the name KIMOZO currently in use in France) will be administered for the treatment of refractory/relapsed Wilms tumors (after 1 or 2 lines of treatment or after 1 line for high risk relapse for which there would not be any curative therapy), using a metronomic schedule and in combination with other drugs (vincristine, irinotecan, etoposide and cis retinoic acid). MetroWILMS will include 28 pediatric patients, with a recruitment planned until October 2028.

"There is a major clinical need for an oral liquid form of temozolomide in various pediatric oncology indications, including in refractory or relapsed Wilms tumor", said Hélène Sudour-Bonnange, onco-pediatrician at Oscar-Lambret clinical center and principal investigator of MetroWILMS trial. "We believe that KIZFIZO oral suspension is especially well suited to treat our young patients."

"Refractory or relapsed nephroblastoma is a very rare indication, but this is well in line with Orphelia’s missions to help pediatric patients with rare cancers", said Jérémy Bastid, chief medical officer at Orphelia Pharma. "Should MetroWilms demonstrate a clinical benefit, we could consider expanding the label of KIFZIZO in this indication".

About KIZFIZO 40 mg/ml

KIZFIZO (temozolomide oral suspension, 40 mg/ml) is a ready-to-use oral liquid pediatric formulation of temozolomide developed for use in the treatment of relapsed or refractory high-risk neuroblastoma, which carry a very poor prognosis. This age-adapted and taste-masked formulation delivers an accurate dose in a small volume, while avoiding drug handling and caregiver exposure to temozolomide. It is the result of a collaboration between the pharmacists and clinicians at Gustave Roussy hospital and the development team at Orphelia Pharma.

In March 2022, KIZFIZO (under the trade name KIMOZO) was granted Early Access Authorization (Autorisation d’Accès Précoce) by the French authorities, for the treatment of refractory and relapsed high-risk neuroblastoma as monotherapy or in combination with irinotecan or topotecan.

KIZFIZO has received an Orphan Drug Designation from the EMA and the FDA and the formulation is covered by granted patents in Europe and the US. The company filed an MAA with the EMA in the summer of 2023.

On June 11, 2024 TRACON Pharmaceuticals (NASDAQ: TCON), a clinical stage biopharmaceutical company utilizing a cost-efficient, CRO-independent Product Development Platform (PDP) to advance its pipeline of novel targeted cancer therapeutics and to partner with other life science companies, reported the publication of Phase 2 clinical data of its DNA damage repair inhibitor drug candidate, TRC102, in patients with glioblastoma in Clinical Cancer Research (Press release, Tracon Pharmaceuticals, JUN 11, 2024, View Source [SID1234644262]). The article, entitled, "Evaluating the Base Excision Repair Inhibitor TRC102 and Temozolomide for patients with Recurrent Glioblastoma in the Phase 2 Adult Brain Tumor Consortium Trial BERT," highlights the activity of TRC102 given in combination with Temodar chemotherapy in patients with recurrent glioblastoma: View Source

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TRC102 was evaluated in a Phase 2 trial in combination with Temodar in 19 patients with progressive or recurrent glioblastoma following surgical resection, Temodar and external beam radiotherapy. Extended survival was observed in two patients (progression-free survival ≥ 17 months and overall survival > 32 months), both of whom demonstrated significantly enriched signatures of DNA damage response (DDR), chromosomal instability, and cellular proliferation by RNA sequencing prior to initiating treatment with Temodar and TRC102. The study was completed by the Adult Brain Tumor Consortium and led by Manmeet Alhuwalia, MD while he was Chair of Neuro-Oncology at Cleveland Clinic prior to his appointment as Chief of Medical Oncology, Chief Scientific Officer, and Deputy Director of the Miami Cancer Institute. The authors concluded the study findings confirm the safety and feasibility of TRC102 given with Temodar for recurrent glioblastoma patients and warrant further evaluation of combination therapy in biomarker-enriched trials enrolling glioblastoma patients with baseline hyperactivated DDR pathways.

"We believe that the data generated to date provides a strong rationale for studying TRC102 in combination with Temodar and radiotherapy in newly diagnosed patients with malignant glioma," said James Freddo, M.D., Chief Medical Officer of TRACON. "The Clinical Cancer Research publication supports prior data published in Cancer Cell (View Source) that patients whose cancers demonstrate activation of DDR pathways may be particularly sensitive to the pharmacologic effects of TRC102."

Based on a 100% response rate (including a 20% complete response rate) in a Phase 1 clinical trial combining TRC102 with pemetrexed, cisplatin and radiation therapy in 15 patients with stage III non-squamous non-small cell lung cancer (View Source), TRC102 is currently being studied in a randomized Phase 2 clinical trial in combination with chemotherapy (pemetrexed, cisplatin or carboplatin) and radiation therapy for stage III non-squamous non-small cell lung cancer (NCT05198830: View Source;rank=6). This trial is sponsored by the National Cancer Institute (NCI) through a Cooperative Research and Development Agreement (CRADA). Determination of the primary endpoint of progression free survival is expected in 2025. TRACON and the NCI have a longstanding history of partnership to develop TRC102, whereby the NCI has funded six Phase 1 or Phase 2 trials through the CRADA.

About TRC102

TRC102 (methoxyamine) is a novel, clinical-stage small molecule inhibitor of the DNA base excision repair pathway, which is a pathway that causes resistance to alkylating and antimetabolite chemotherapeutics. TRC102 is currently being studied in multiple randomized Phase 2 clinical trial entitled Testing the Addition of an Anti-Cancer Drug, TRC102, to the Usual Chemotherapy Treatment (Pemetrexed, Cisplatin or Carboplatin) During Radiation Therapy for Stage III Non-Squamous Non-Small Cell Lung Cancer (NCT05198830) that is sponsored by the NCI through a CRADA. TRC102 was granted orphan drug designation by the US FDA for the treatment of malignant glioma in 2020. For more information about the clinical trials, please visit TRACON’s website at www.traconpharma.com/clinical-trials.

About Malignant Glioma and Glioblastoma
Glioblastoma (GBM) is a rapidly growing malignant glioma that develops from glial cells (astrocytes and oligodendrocytes) that support the health of the nerve cells within the brain. GBM is the most invasive type of glial tumors, rapidly growing and commonly invading into nearby brain tissue. The National Cancer Institute estimates that approximately 22,850 adults are diagnosed with brain and other nervous system cancers annually in the U.S. and approximately 15,320 of these diagnoses will result in death. GBM has an incidence of two to three per 100,000 adults per year in the U.S., and accounts for 52 percent of all primary brain tumors.

On June 11, 2024 Tonix Pharmaceuticals Holding Corp. (Nasdaq: TNXP) ("Tonix" or the "Company"), a fully-integrated biopharmaceutical company, reported that it intends to offer and sell shares of its common stock (or pre-funded warrants in lieu thereof) (Press release, TONIX Pharmaceuticals, JUN 11, 2024, View Source [SID1234644261]). All of the securities to be sold in the offering are to be offered by Tonix. The offering is subject to market conditions, and there can be no assurance as to whether or when the offering may be completed, or as to the actual size or terms of the offering.

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The Company intends to use the net proceeds from the offering for working capital and general corporate purposes, including the preparation of the new drug application relating to its Tonmya product candidate in patients with fibromyalgia, and the satisfaction of any portion of its existing indebtedness.

Dawson James Securities, Inc. is the sole placement agent for the offering.

This offering is being made pursuant to an effective shelf registration statement on Form S-3 (File No. 333-266982) previously filed with the U.S. Securities and Exchange Commission (the "SEC"). The offering will be made only by means of a prospectus supplement and accompanying prospectus. The preliminary prospectus supplement and accompanying prospectus describing the terms of the proposed offering will be filed with the SEC and will be available on the SEC’s website located at View Source Electronic copies of the preliminary prospectus supplement may be obtained, when available, from Dawson James Securities, Inc., 101 North Federal Highway, Suite 600, Boca Raton, FL 33432 or by telephone at (561) 391-5555, or by email at [email protected]. Before investing in this offering, interested parties should read in their entirety the prospectus supplement and the accompanying prospectus and the other documents that Tonix has filed with the SEC that are incorporated by reference in such prospectus supplement and the accompanying prospectus, which provide more information about Tonix and such offering.

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