On August 21, 2023 CanariaBio Inc., a clinical-stage biopharmaceutical company focused on the development and commercialization of innovative immunotherapies for cancer, reported that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation (ODD) to its investigational drug product, MAb-AR20.5, an IgG1k type murine monoclonal antibody that binds specifically to the circulating and tumor-associated antigen (MUC1) expressed ubiquitously on pancreatic cancer cells (Press release, CanariaBio, AUG 21, 2023, View Source [SID1234634605]). This milestone marks the first monoclonal antibody targeting Mucin1 (MUC1) to receive this designation for pancreatic cancer.

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Pancreatic cancer remains one of the most challenging malignancies to treat, with limited therapeutic options available. The MAb-AR20.5 has shown potential in early studies, by inducing MUC-1-specific immune responses in cancer patients with advanced disease. This specific targeting aims to provide a more focused and potentially more effective treatment strategy for patients diagnosed with this aggressive disease.

"Receiving Orphan Drug Designation for MAb-AR20.5 is a significant milestone for CanariaBio Inc. and speaks to the potential promise of this novel therapeutic approach," said Mike Na, the CEO of CanariaBio Inc. "We are deeply committed to advancing innovative treatments for patients with pancreatic cancer and remain hopeful that MAb-AR20.5 will offer a new lifeline for those affected."

Orphan Drug Designation is granted by the FDA to drugs and biologics intended for the treatment, diagnosis, or prevention of rare diseases or disorders that affect fewer than 200,000 people in the U.S. This designation provides certain benefits, including tax credits, user fee waivers, and eligibility for seven years of marketing exclusivity.

CanariaBio Inc. is planning to initiate clinical trials to assess the safety and efficacy of MAb-AR20.5 in patients with pancreatic cancer. More details about the upcoming studies and trial sites will be made available in the coming months.

On August 21, 2023 Mirati Therapeutics, Inc. (NASDAQ: MRTX), a commercial stage biotechnology company, reported a presentation of two-year follow-up from a pooled analysis of the Phase 1/1b Cohort and Phase 2 Cohort A for the KRYSTAL-1 study evaluating adagrasib in in patients with non-small cell lung cancer (NSCLC) harboring a KRASG12C mutation (Press release, Mirati, AUG 21, 2023, View Source [SID1234634604]).

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Findings will be presented at the 2023 World Conference on Lung Cancer during the press conference on September 10th 10:35 a.m. – 10:40 a.m. SGT / September 9th 7:35 p.m. – 7:40 p.m. PT and as a mini-oral presentation in the "Targeted Therapy: KRAS and Beyond" session occurring on September 10th at 4:22 p.m. SGT / 1:22 a.m. PT.

On August 21, 2023 IDEAYA Biosciences, Inc. (NASDAQ: IDYA), a precision medicine oncology company committed to the discovery and development of targeted therapeutics, reported the clearance of an Investigational New Drug (IND) application with the U.S. Food and Drug Administration (FDA) for initiation of a GSK-sponsored Phase 1/2 clinical trial to evaluate GSK101 (IDE705), a small molecule inhibitor of Pol Theta Helicase, in combination with niraparib, the GSK small molecule inhibitor of poly-(ADP-ribose) polymerase (PARP), for the treatment of patients having tumors with BRCA or other homologous recombination (HR) mutations or homologous recombination deficiency (HRD) (Press release, Ideaya Biosciences, AUG 21, 2023, View Source [SID1234634603]).

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"There remains an unmet medical need for patients having tumors with homologous recombination mutations, such as BRCA1/2 mutations. Based on Pol Theta’s critical role in microhomology-mediated end joining and BRCA reversions, a central mechanism of resistance to PARP inhibitors, we view the niraparib and GSK101 combination as having potential to improve depth and duration of tumor responses that could potentially impact longer-term outcomes for these cancer patients," said Dr. Ramon Kemp, Vice President, Global Head of Oncology Early Development, GSK.

"We are pleased to have our fourth potential first-in-class program advance into the clinic with GSK101, and we believe GSK is well-positioned to maximize the value of GSK101 by creating a potential best-in-class combination with niraparib to treat solid tumors. We are also targeting the Werner Helicase development candidate nomination later this year with GSK, which represents our fifth potential first-in-class program," said Yujiro S. Hata, Chief Executive Officer, IDEAYA Biosciences.

GSK101 is a potential first-in-class small molecule inhibitor of the helicase domain of DNA Polymerase Theta (Pol Theta). The Pol Theta enzyme facilitates DNA repair through microhomology-mediated end joining (MMEJ), an enzymatic function that enables reversion of BRCA mutations. Approximately 30% of patients who progress following treatment with PARP inhibitors have tumors with MMEJ signatures at reversion sites, reflecting a significant medical need and potential commercial opportunity for GSK101 in combination with niraparib.

GSK101 was discovered and preclinically evaluated by IDEAYA in collaboration with GSK. In preclinical studies, the GSK101 and niraparib combination resulted in deeper and more durable regressions or efficacious responses relative to either single agent in BRCA mutant models.

GSK is targeting first-in-human studies for GSK101 in the fourth quarter of 2023. GSK is the sponsor of the IND application and plans to develop GSK101 in combination with niraparib in a Phase 1/2 clinical trial for patients with advanced solid tumors who have exhausted standard of care options and who may benefit from a PARP or POLQ inhibitor, pursuant to the clinical protocol. Enrollment may include patients harboring tumors with BRCA or other homologous recombination (HR) mutations or homologous recombination deficiency (HRD).

GSK will lead clinical development for the Pol Theta program pursuant to its global, exclusive license to develop and commercialize the Pol Theta Helicase Inhibitor DC (GSK Pol Theta License). GSK is responsible for all research and development costs for the program. IDEAYA is eligible to receive a $7 million milestone payment upon acceptance of the IND by the U.S. Food and Drug Administration (FDA), and a potential additional $10 million milestone payment upon initiation of Phase 1 clinical dose expansion. IDEAYA may potentially also receive further aggregate later-stage development and regulatory milestones of up to $465 million.

Upon potential commercialization, IDEAYA will be eligible to receive up to $475 million of commercial milestones and tiered royalties on global net sales by GSK, its affiliates and their sublicensees ranging from high single digit to sub-teen double-digit percentages, subject to certain customary reductions.

On August 21, 2023 ProMIS Neurosciences Inc. (Nasdaq: PMN) ("ProMIS" or the "Company"), a biotechnology company focused on the generation and development of antibody therapeutics targeting toxic misfolded proteins in neurodegenerative diseases such as Alzheimer’s disease (AD), amyotrophic lateral sclerosis (ALS) and multiple system atrophy (MSA), reported that it has entered into a securities purchase agreement with certain new and existing accredited investors to issue and sell an aggregate of approximately $20.4 million of (a) common share units, each consisting of one of the Company’s common shares (the "Common Shares") and one warrant to purchase one Common Share (the "Warrants") (the "Common Share Units") and (b) pre-funded units, each consisting of one pre-funded warrant to purchase one Common Share and one Warrant (the "Pre-Funded Units") (Press release, ProMIS Neurosciences, AUG 21, 2023, View Source [SID1234634602]). The Common Share Units were sold at a price of $1.88 per unit and the Pre-Funded Warrants were sold at a price of $1.87 per unit through a private investment in public equity ("PIPE") financing.

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The PIPE included participation from Affinity Asset Advisors, LLC, Ally Bridge Group, Sphera Healthcare and other institutional and individual accredited investors.

ProMIS anticipates the gross proceeds from the PIPE to be approximately $20.4 million, before deducting fees to the placement agents and other offering expenses payable by the Company. The financing is expected to close on August 23, 2023, subject to customary closing conditions.

Proceeds from the PIPE financing are expected to be used to advance the clinical development of PMN310, ProMIS’ lead therapeutic candidate, as well as for working capital and other general corporate expenses.

Certain directors and officers of the Company are subscribing for up to approximately $145,000 of Common Share Units. The issuance of Common Share Units to insiders will be considered a "related party transaction" within the meaning of Multilateral Instrument 61-101 – Protection of Minority Security Holders in Special Transactions ("MI 61-101"). The Company is relying on exemptions from the formal valuation requirements of MI 61-101 pursuant to section 5.5(a) and the minority shareholder approval requirements of MI 61-101 pursuant to section 5.7(1)(a) in respect of such insider participation as the fair market value of the transaction, insofar as it involves interested parties, does not exceed 25% of the Company’s market capitalization.

The offer and sale of the foregoing securities are being made in a transaction not involving a public offering and have not been registered under the Securities Act of 1933, as amended ("Securities Act"), or any state or other applicable jurisdiction’s securities laws, and may not be offered or sold in the United States absent registration or an applicable exemption from the registration requirements of the Securities Act and applicable state or other jurisdictions’ securities laws. ProMIS Neurosciences has agreed to file a registration statement with the SEC registering the resale of the Common Shares and the Common Shares issuable upon the exercise of the Pre-Funded Warrants and Warrants issued in the PIPE financing.

This press release shall not constitute an offer to sell or the solicitation of an offer to buy these securities, 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 the registration or qualification under the securities law of any such state or other jurisdiction.

On August 21, 2023 Gilead Sciences, Inc. (Nasdaq: GILD) reported that the U.S. Food and Drug Administration (FDA) has placed a partial clinical hold on the initiation of new patients in U.S. studies evaluating magrolimab to treat acute myeloid leukemia (AML) (Press release, Gilead Sciences, AUG 21, 2023, View Source [SID1234634601]).

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The FDA action follows the previously announced discontinuation of the Phase 3 ENHANCE study of magrolimab in higher-risk myelodysplastic syndromes (HR-MDS).

Effective immediately, screening and enrollment of new study participants under the U.S. investigational new drug application (IND 147229) and U.S. Expanded Access Program will be paused. Patients already enrolled in AML clinical studies may continue to receive treatment and be monitored, according to the current study protocol. Global regulatory authorities and clinical trial investigators involved in the studies have been informed of the FDA’s decision. Studies of magrolimab in solid tumors continue without any impact from the FDA action.

Gilead is working with regulatory authorities to determine next steps to release the partial clinical hold for new patient enrollment in the magrolimab AML studies.

Magrolimab is an investigational agent and has not been approved anywhere globally. Its safety and efficacy have not been established.

About Magrolimab

Magrolimab is a potential first-in-class investigational anti-CD47 immunotherapy. The primary mechanism of action of magrolimab is to block the inhibitory CD47-signal regulatory protein (SIRPα) interaction, enhancing the ability of macrophages and other phagocytes to identify and destroy foreign and malignant cells, with the goal of blocking the "don’t eat me" signal used by cancer cells. Magrolimab is being developed as a potential treatment for AML, myeloid malignancies, lymphoma, myeloma, head and neck cancer, colorectal cancer, lung cancer, and breast cancer. More information about clinical trials with magrolimab is available at www.clinicaltrials.gov.