On July 25, 2024 Checkpoint Therapeutics, Inc. ("Checkpoint") (Nasdaq: CKPT), a clinical-stage immunotherapy and targeted oncology company, reported that the U.S. Food and Drug Administration ("FDA") has accepted for review Checkpoint’s resubmission of its Biologics License Application ("BLA") for cosibelimab, its anti-programmed death ligand-1 ("PD-L1") antibody, as a potential new treatment for adults with metastatic or locally advanced cutaneous squamous cell carcinoma ("cSCC") who are not candidates for curative surgery or curative radiation (Press release, Checkpoint Therapeutics, JUL 25, 2024, View Source [SID1234645086]). The resubmission has been accepted as a complete response to the FDA’s December 2023 complete response letter ("CRL") and the FDA has set a Prescription Drug User Fee Act ("PDUFA") goal date of December 28, 2024.

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James F. Oliviero, President and Chief Executive Officer of Checkpoint, said, "We are pleased that the FDA has accepted our BLA resubmission as a complete response after we aligned on our BLA resubmission strategy. We look forward to working closely with the FDA to finalize the review and to the potential opportunity to deliver cosibelimab’s unique dual mechanism of action to patients suffering from cSCC."

In December 2023, the FDA issued a CRL for the cosibelimab BLA, which only cited findings that arose during a multi-sponsor inspection of Checkpoint’s third-party contract manufacturing organization ("CMO") as approvability issues to address in a BLA resubmission. The CRL did not state any concerns about the clinical data package, safety, or labeling for the approvability of cosibelimab.

About Cosibelimab
Cosibelimab is a potential differentiated, high affinity, fully-human monoclonal antibody of IgG1 subtype that directly binds to PD-L1 and blocks the PD-L1 interaction with the programmed death receptor-1 ("PD-1") and B7.1 receptors. Cosibelimab’s primary mechanism of action is based on the inhibition of the interaction between PD-L1 and its receptors PD-1 and B7.1, which removes the suppressive effects of PD-L1 on anti-tumor CD8+ T-cells to restore the cytotoxic T cell response. Cosibelimab is potentially differentiated from the currently marketed PD-1 and PD-L1 antibodies through sustained high tumor target occupancy of PD-L1 to reactivate an antitumor immune response and the additional potential benefit of a functional Fc domain capable of inducing antibody-dependent cellular cytotoxicity ("ADCC") for potential enhanced efficacy.

On July 25, 2024 Amplia Therapeutics Limited (ASX: ATX), ("Amplia" or the "Company"), reported that three (3) patients enrolled in the Company’s Phase 2a clinical trial investigating narmafotinib in the treatment of advanced pancreatic cancer (the ACCENT trial) have recorded a confirmed partial response (Press release, Amplia Therapeutics, JUL 25, 2024, View Source [SID1234645068]). The formal term ‘confirmed partial response’ means there is at least a 30% decrease in the overall size of tumour lesions, and no new tumour lesions, in these patients sustained over a two month period.

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Of the six (6) patients currently assessed at the four month time point, in addition to the three (3) confirmed partial responses, two (2) additional patients have recorded sustained stable disease.

Amplia CEO and MD Dr Chris Burns commented: "To be reporting that three confirmed partial responses have been observed so early in this stage of the trial is extremely encouraging. We are well on track to reach the efficacy threshold of six confirmed partial or complete responses by the end of this quarter, which will then allow us to restart the trial to recruit the full cohort of fifty patients."

The Company recently announced completion of enrolment of the first 26 patients in the trial as part of the industry-standard Simon’s Two-Stage Trial design. Once six (6) confirmed partial or complete responses are obtained then an additional 24 patients will be enrolled, giving a total of 50 patients for the trial.

The Company will provide further updates on the trial as recruitment proceeds.

This ASX announcement was approved and authorised for release by the Board of Amplia Therapeutics.

About Narmafotinib

Narmafotinib (AMP945) is the company’s best-in-class inhibitor of the protein FAK, a protein overexpressed in pancreatic and other cancers, and a drug target gaining increasing attention for its role in solid tumours. The drug, which is a highly potent and selective inhibitor of FAK, has shown promising data in a range of preclinical cancer studies. The drug has successfully completed a healthy volunteer study, and is currently in an open-label Phase 2a trial in pancreatic cancer where a combination of narmafotinib and the chemotherapies gemcitabine and Abraxane is being assessed for safety, tolerability and efficacy.

About the ACCENT Trial

The ACCENT trial is entitled ‘A Phase 1b/2a, Multicentre, Open Label Study of the Pharmacokinetics, Safety and Efficacy of AMP945 in Combination with Nab-paclitaxel and Gemcitabine in Pancreatic Cancer Patients’.

The ACCENT trial explores the use of narmafotinib in combination with standard-of-care chemotherapy of gemcitabine and Abraxane in first-line patients with advanced pancreatic cancer. The trial is a single-arm open label study conducted in two stages. The firststage (Phase 1b), completed in November 2023, identified a 400 mg oral daily dose of narmafotinib, given in the days preceding regular chemotherapy infusion, as safe and well tolerated.

This second stage (Phase 2a) of the trial is designed to assess drug efficacy in combination with gemcitabine and Abraxane. The primary endpoints are Objective Response Rate (ORR) and Duration on Trial (DOT) with secondary endpoints being Progression Free Survival (PFS) and Overall Survival (OS). Safety and tolerability will continue to be assessed.

More information about the ACCENT trial, including a list of participating sites, can be found via the Amplia Therapeutics website and at ClinicalTrials.gov under the identifier NCT05355298.

On July 25, 2024 Chemomab Therapeutics Ltd. (Nasdaq: CMMB) ("Chemomab" or the "Company"), a clinical stage biotechnology company developing innovative therapeutics for fibro-inflammatory diseases with high unmet need, reported that it has entered into a securities purchase agreement for a private investment in public equity ("PIPE") that is expected to result in gross proceeds of approximately $10 million to the Company, before deducting capital market advisor fees and offering expenses (Press release, Chemomab, JUL 25, 2024, View Source [SID1234645087]).

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The PIPE included participation from both new investors, including HBM Healthcare Investments and Sphera Biotech Master Fund LP, and existing investors. Chemomab expects that the net proceeds from the PIPE will extend its cash runway to fund its operations through the beginning of 2026, an extension of approximately one year from current projections, which should fund the Company for approximately one year after the completion of two major milestones expected in early 2025.

Pursuant to the terms of the securities purchase agreement, the Company is selling to certain investors (i) 4,188,867 American Depositary Shares ("ADSs"), each representing twenty (20) ordinary shares of the Company, no par value per share, at a purchase price of $1.235 per share which reflects the average share price on the Nasdaq for the last 4 trading days and (ii), in lieu of ADSs, pre-funded warrants (the "Pre-Funded Warrants") to purchase up to 3,908,300 ADSs at a price per Pre-Funded Warrant of $1.235. The Pre-Funded Warrants have an exercise price of $0.0001 per ADS, are immediately exercisable and remain exercisable until exercised in full. The PIPE is expected to close on or about July 26, 2024, subject to satisfaction of customary closing conditions.

The Company intends to use the net proceeds from the PIPE, together with the Company’s existing cash and cash equivalents, to fund its development programs for CM-101, and for general corporate purposes and working capital.

Oppenheimer & Co. Inc. is acting as Capital Markets Advisor to the Company for the PIPE. Other Advisors included Maxim Group and LifeSci Capital.

The offer and sale of the foregoing securities are being made in a transaction not involving a public offering and the securities have not been registered under the Securities Act of 1933, as amended, and may not be reoffered or resold in the United States except pursuant to an effective registration statement or an applicable exemption from the registration requirements. Concurrently with the execution of the securities purchase agreement, the Company and the investors entered into a registration rights agreement pursuant to which the Company has agreed to file a registration statement with the Securities and Exchange Commission (the "SEC") registering the resale of the ADSs, including ADSs issuable upon exercise of the Pre-Funded Warrants, purchased in the PIPE.

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

On July 25, 2024 AstraZeneca’s Chief Executive Officer, Pascal Soriot reporting on the quarterly results said (Press release, AstraZeneca, JUL 25, 2024, View Source [SID1234645069]):

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"Building on our strong growth in the first half of the year and continued underlying demand for our medicines we are upgrading our FY 2024 guidance for both Total Revenue and Core EPS.

At our Investor Day in May we set out a new revenue ambition to deliver $80 billion of Total Revenue by 2030. This is a clear reflection of the substantial growth potential we see from both our approved medicines and those in our late-stage pipeline. Already this year we have announced five positive, potentially practice-changing Phase III studies that are anticipated to meaningfully contribute to our growth.

In the year to date we have continued to make encouraging progress with several disruptive technologies, including antibody drug conjugates, bispecifics, cell and gene therapies, radioconjugates, and weight management medicines, all of which have the potential to drive our growth beyond 2030."

On July 25, 2024 Ipsen (Euronext: IPN; ADR: IPSEY) and Day One Biopharmaceuticals (Nasdaq: DAWN) (Day One), reported a new global partnership outside the U.S. for tovorafenib, an oral, once-weekly, type II RAF inhibitor for pediatric low grade glioma (pLGG), the most common form of childhood brain cancer, i and any future indications developed by Day One (Press release, Day One, JUL 25, 2024, View Source [SID1234645088]).

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Tovorafenib was granted Orphan Drug Designation and received U.S. FDA approval in April 2024ii as a monotherapy treatment for patients six months and older with relapsed or refractory pLGG harboring a BRAF fusion or rearrangement, or BRAF V600 mutation.iii These BRAF alterations account for more than half of pLGG cases worldwide and there are no approved targeted treatments for people with pLGG harboring BRAF fusions outside the U.S.i,iii,iv Day One will maintain exclusive global development and U.S. commercial rights for tovorafenib.

David Loew, Chief Executive Officer, Ipsen, commented "Today’s announcement marks an exciting addition to our portfolio. Tovorafenib has the potential to make a significant impact on children living with cancer and is an excellent example of our biomarker-driven strategy as we expand our portfolio. Pediatric low-grade glioma is the most common form of childhood brain cancer, and, outside the U.S., there are still no approved targeted treatments for people with pLGG caused by BRAF alterations, including BRAF fusions or V600 in the refractory/relapsed setting. We are delighted to partner with the team at Day One as we work to bring tovorafenib to every eligible patient around the world, who may benefit from this important new treatment option."

Jeremy Bender, Ph.D., Chief Executive Officer, Day One commented, "Our collaboration with Ipsen to bring tovorafenib to patients worldwide highlights our shared commitment to bring novel therapeutics to patients who have limited treatment options. We believe Ipsen’s footprint in Europe and major regions outside of the U.S., in addition to their track record of bringing innovative medicines to market in oncology and rare pediatric diseases, will be an enormous benefit to tovorafenib and to the pediatric oncology community worldwide."

Ipsen’s deep heritage and expertise in oncology means we can accelerate the delivery of this innovation as teams focus on regulatory activities outside the U.S. pLGG is the most common brain tumor diagnosed in children, with patients suffering profound tumor- and treatment-associated morbidities that can impact their life trajectory.i Depending on the tumor’s size, location and growth rate, pLGG can present with a variety of symptoms including vision, hearing and speech problems, neurological symptoms, premature puberty, physical changes and generalized symptoms such as balance problems, fatigue and nausea.v Mortality is relatively rare, however due to the chronic nature of pLGG and potential morbidity associated with treatment, the disease can significantly affect the development, cognition, education and overall quality of life of affected children, whilst negatively impacting the mental health of parents and caregivers.

Under the terms of the agreement, Ipsen will be responsible for the regulatory and commercial activities for tovorafenib in all territories outside of the U.S. Day One will receive an upfront payment of approximately $111 million, which includes approximately $71 million in cash as well as a $40 million equity investment at a premium and up to approximately $350 million in additional launch and sales milestone payments. Day One will receive tiered double-digit royalties starting at mid-teens percentage on sales.