On April 26, 2022 Treadwell Therapeutics, a clinical-stage biotechnology company developing novel medicines for unmet needs in cancer, reported that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation to CFI-400945, a first in class inhibitor of Polo-like kinase 4 (PLK4), for the treatment of adult patients with relapsed or refractory Acute Myeloid Leukemia (AML) (Press release, Treadwell Therapeutics, APR 26, 2022, View Source [SID1234612962]).

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"Although several exciting new classes of medicines have emerged in the past decade for patients with AML, there still remains an unmet need for certain patient segments, where survival rates remain low," said Dr. Michael Tusche, Treadwell co-CEO. "CFI-400945, has shown encouraging signs of monotherapy activity in AML patients with adverse cytogenetics. We are grateful for the Fast Track Designation for this exciting program, and look forward to frequent interactions with the FDA to chart our regulatory path forward, as we continue the development of ‘945 in leukemia."

Fast Track designation seeks to streamline the development and accelerate the review of new agents with potential to treat serious or life-threatening diseases and that potentially address an unmet medical need. Drugs that are granted this designation can have more frequent interactions with the FDA, as well as potential pathways for expedited approval.

About AML
AML is a disease characterized by uncontrolled proliferation of malignant clonal hematopoietic stem cells which can lead to anemia, neutropenia, and thrombocytopenia. If left untreated, AML can lead to death within weeks. In the US, an estimated 19,940 new cases of AML were expected to be diagnosed and approximately 11,180 deaths attributed to AML, nearly all in adults. AML is generally a disease of the elderly with an average age of 68 years at the time of diagnosis and is more common in men than women. For adults <65 years of age, the 5-year survival is approximately 33%, but drops dramatically to 4% in adults >65 years of age.

On April 26, 2022 Cellectar Biosciences, Inc. (NASDAQ: CLRB), a late-stage clinical biopharmaceutical company focused on the discovery, development and commercialization of targeted drugs for the treatment of cancer, reported an independent Data Monitoring Committee (DMC) has completed its planned futility/efficacy assessment of the company’s pivotal Phase 2b study of iopofosine in Waldenstrom’s macroglobulinemia (WM) and unanimously recommended continuation of the trial as planned (Press release, Cellectar Biosciences, APR 26, 2022, View Source [SID1234612961]).

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"We remain highly encouraged with the consistent performance of iopofosine I-131 in this difficult-to-treat refractory patient population," said James Caruso, president and chief executive officer of Cellectar. "We have alignment with the FDA on a 20% major response rate hurdle as the primary endpoint of our pivotal study. In a poster presented at ASCO (Free ASCO Whitepaper) 2021, we showed an 83.3% major response rate with one complete response in six WM patients, which served as the basis for our pursuing this indication."

The pivotal trial is a global, non-comparator, single-arm, open-label expansion cohort of the currently ongoing Phase 2 CLOVER-1 study of CLR 131. The study will enroll 50 WM patients. Patients in the trial will receive up to four doses of iopofosine over two cycles (cycle one on days 1, 15, and cycle two on days 57 and 71). The primary endpoint of the trial is major response rate as defined as a partial response (a minimum of a 50% reduction in the biological marker IgM) or better in patients that receive a minimum total body dose of 60 mCi with secondary endpoints of treatment free survival, duration of response and progression free survival.

The DMC is an independent committee of clinical research experts who review data in ongoing clinical trials. The DMC assessment was based on a pre-specified futility analysis within the first 10 patients as defined in the study protocol.

About Waldenstrom’s macroglobulinemia
Waldenstrom’s macroglobulinemia (WM) is a rare and incurable disease defined by specific genotypic subtypes that defines patient responses and long-term outcomes. The U.S. annual incidence is 3,000 (anticipated annual growth rate of approximately 30% through 2025) with a current U.S. prevalence of approximately 45,000 patients. The U.S. represents approximately 43% of the global market. WM is a lymphoma, or cancer of the lymphatic system. The disease occurs in a type of white blood cell called a B-lymphocyte or B-cell, which normally matures into a plasma cell whose job is to manufacture immunoglobulins (antibodies) to help the body fight infection. In WM, there is a malignant change to the B-cell in the late stages of maturing, and it continues to proliferate into a clone of identical cells, primarily in the bone marrow but also in the lymph nodes and other tissues and organs of the lymphatic system. These clonal cells over-produce an antibody of a specific class called IgM.

WM cells have characteristics of both cancerous B-lymphocytes (NHL) and plasma cells (multiple myeloma), and they are called lymphoplasmacytic cells. For that reason, WM is classified as a type of non-Hodgkin’s lymphoma called lymphoplasmacytic lymphoma (LPL). About 95% of LPL cases are WM; the remaining 5% do not secrete IgM and consequently are not classified as WM.

There is no standard treatment for WM. Several drugs have demonstrated activity either alone or in combinations, but only a single drug has received regulatory approval. Treatment is mainly focused on the control of symptoms and the prevention of organ damage. Front-line treatments for WM include rituximab alone or in combination with other agents. In the salvage therapy (second line or later) setting, ibrutinib, combinations of proteosome inhibitors and immunomodulatory drugs and stem cell transplantation are considered. Ibrutinib is the only drug to receive regulatory approval (2015) as a salvage therapy; in late 2019, it was approved for front-line treatment in combination with rituximab. Factors such as long-term cytopenias, age, hyper viscosity, the need for quick disease control, lymphadenopathy, co-morbidities, and IgM-related end-organ damage are key considerations in the choice of treatment.

On April 26, 2022 Deciphera Pharmaceuticals, Inc. (NASDAQ: DCPH), a biopharmaceutical company focused on discovering, developing, and commercializing important new medicines to improve the lives of people with cancer, reported that it intends to offer and sell $150,000,000 of shares of its common stock and pre-funded warrants to purchase shares of its common stock in an underwritten public offering (Press release, Deciphera Pharmaceuticals, APR 26, 2022, View Source [SID1234612960]). In addition, Deciphera intends to grant the underwriters a 30-day option to purchase up to an additional $22,500,000 of shares of common stock offered in the public offering. All of the shares and pre-funded warrants are being offered by Deciphera. The offering is subject to market and other 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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Deciphera intends to use the net proceeds from the offering to continue to fund the development of vimseltinib including in its pivotal Phase 3 MOTION study of vimseltinib in TGCT patients currently underway, additional clinical trials as well as clinical research outsourcing and manufacturing of clinical trial material and pre-commercial and medical affairs capabilities related to vimseltinib; to fund the development of DCC-3116 including multiple Phase 1b combination studies and potential Phase 2 expansion combination cohorts in multiple tumor types as well as clinical research outsourcing and manufacturing of clinical trial material; to fund the research and development of the Company’s pan-RAF program and other new research activities for potential drug candidates from its proprietary kinase switch control inhibitor platform; and the remainder for working capital purposes, including general operating expenses.

J.P. Morgan and Jefferies are acting as joint book-running managers for the offering.

The securities described above are being offered by Deciphera pursuant to a shelf registration statement on Form S-3 (No. 333-236389) that was declared effective by the Securities and Exchange Commission (SEC) on March 10, 2022. A preliminary prospectus supplement and accompanying prospectus 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 preliminary prospectus supplement and the accompanying prospectus relating to this offering may be obtained, when available, by contacting: J.P. Morgan Securities LLC, c/o Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, NY 11717, by telephone at 866-803-9204, or by email at [email protected]; or Jefferies LLC, Attention: Equity Syndicate Prospectus Department, 520 Madison Avenue, New York, NY 10022, by telephone at 877-821-7388 or by email at [email protected].

This press release does 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 registration or qualification under the securities laws of that state or jurisdiction.

On April 26, 2022 Molecular Partners AG (SIX: MOLN; NASDAQ: MOLN), a clinical-stage biotech company developing a new class of custom-built protein drugs known as DARPin therapeutics, reported that Amgen, its collaboration partner for MP0310 (AMG 506), has informed the Company of their decision to return global rights of MP0310 to Molecular Partners following a strategic pipeline review (Press release, Molecular Partners, APR 26, 2022, View Source [SID1234612959]). Molecular Partners is presently conducting a phase 1 study of MP0310 and will look to present full phase 1 data at a scientific conference when available.

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"We would like to thank Amgen for a very fruitful collaboration which has led us to the initial clinical findings that an immunostimulatory receptor, such as 4-1BB, can be activated in a localized fashion," said Patrick Amstutz, Molecular Partners’ CEO. "This collaboration has enabled us to broaden our immuno-oncology expertise, DARPin platform knowledge, and clinical development capabilities which are now being applied across our portfolio. We continue to anticipate the full phase 1 dataset later this year, following which we can begin to explore potential collaborations for the program with new partners."

MP0310 is a dual-targeted compound, targeting both FAP and 4-1BB, that has the potential to activate T-cells and other immune cells, specifically in the tumor microenvironment, aiming to avoid systemic side effects associated with 4-1BB activation. Given this mode of action, future development will call for combination therapy trials. No additional clinical studies of MP0310 have been planned at this time. Following completion of the ongoing Phase 1 study, the Company will look to initiate discussions with potential collaborators.

The collaboration with Amgen was initiated in December 2018, providing an upfront payment of $50 million to Molecular Partners. Per the terms of the agreement, Molecular Partners is conducting the phase 1 clinical trial of MP310. Under the agreement with Amgen, following Phase 1 data, Amgen would have had the right to progress the program into later stage development, including into combination trials. The phase 1 clinical study was initiated in October 2019 in patients with solid tumors. Initial preliminary data from patient biopsies has demonstrated the potential of MP0310 to colocalize with FAP (fibroblast activation protein) and activate 4-1BB in the tumor microenvironment, after a first dose. A second part of the phase 1 clinical trial was initiated to optimize dosing and to demonstrate sustained activity at later time points. Data from the second part of the trial is pending. The remaining patient cohorts will be treated and followed, per the trial protocol, which is expected to be completed in the second half of 2022.

On April 26, 2022 Exelixis, Inc. (Nasdaq: EXEL) reported that its first quarter 2022 financial results will be released on Tuesday, May 10, 2022 after the markets close (Press release, Exelixis, APR 26, 2022, View Source [SID1234612958]). At 5:00 p.m. ET / 2:00 p.m. PT, Exelixis management will host a conference call and webcast to discuss the results and provide a general business update . Access to the event is available via the Internet from the company’s website.

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To access the webcast link, log onto www.exelixis.com and proceed to the News & Events / Event Calendar page under the Investors & Media heading. Please connect to the company’s website at least 15 minutes prior to the conference call to ensure adequate time for any software download that may be required to listen to the webcast. Alternatively, please call 855-793-2457 (domestic) or 631-485-4921 (international) and provide the conference call passcode 6282616 to join by phone.

A telephone replay will be available until 8:00 p.m. ET on May 12, 2022. Access numbers for the telephone replay are: 855-859-2056 (domestic) and 404-537-3406 (international); the passcode is 6282616. A webcast replay will also be archived on www.exelixis.com for one year.