On January 4, 2023 Checkpoint Therapeutics, Inc. ("Checkpoint") (Nasdaq: CKPT), a clinical-stage immunotherapy and targeted oncology company, reported the submission of a Biologics License Application ("BLA") to the U.S. Food and Drug Administration ("FDA") for the approval of cosibelimab, its investigational anti-PD-L1 antibody, as a treatment for patients with metastatic cutaneous squamous cell carcinoma ("cSCC") or locally advanced cSCC who are not candidates for curative surgery or radiation (Press release, Checkpoint Therapeutics, JAN 4, 2023, View Source [SID1234625796]).

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"This is a major milestone for Checkpoint Therapeutics, representing our first submission of a marketing application for one of our investigational medications and furthering our evolution from a development-stage company to a fully integrated commercial organization to support the potential launch of cosibelimab," said James Oliviero, President and Chief Executive Officer of Checkpoint. "cSCC is the second most common type of skin cancer in the United States. While most cases involve localized tumors amenable to curative resection, approximately 40,000 cases will become advanced, and an estimated 15,000 people will die from their disease each year. Based on its compelling and differentiated product profile and the positive data generated to date, we believe cosibelimab has the potential to be an important treatment option for patients. Importantly, we continue to plan for cosibelimab to be positioned, upon regulatory approval, as potentially the first and only price disruptive PD-1/PD-L1 inhibitor in the U.S. market." Mr. Oliviero continued, "We want to thank the patients and their families, as well as the physicians and their research teams, who participated in our trial and contributed immensely to the advancement of cosibelimab."

The BLA submission is based on positive efficacy and safety results from Checkpoint’s ongoing registration-enabling, multi-regional, multicohort clinical trial evaluating cosibelimab administered as fixed doses of either 800 mg every two weeks or 1200 mg every three weeks in patients with selected recurrent or metastatic cancers, including pivotal cohorts in metastatic and locally advanced cSCC.

In January 2022, Checkpoint announced that the metastatic cSCC cohort met its primary endpoint, with cosibelimab demonstrating a confirmed objective response rate ("ORR") of 47.4% (95% CI: 36.0, 59.1) based on independent central review of 78 patients enrolled in the cohort using Response Evaluation Criteria in Solid Tumors version 1.1 ("RECIST 1.1") criteria. In June 2022, Checkpoint announced positive interim results from its locally advanced cSCC cohort, with cosibelimab demonstrating a confirmed ORR of 54.8% (95% CI: 36.0, 72.7) based on independent central review of 31 patients enrolled in the cohort, exceeding a clinically meaningful lower bound of the 95% two-sided confidence interval of 25%. Based upon interactions with the FDA, the BLA submission includes both the metastatic and locally advanced cSCC indications.

About Cutaneous Squamous Cell Carcinoma

Cutaneous squamous cell carcinoma is the second most common type of skin cancer in the United States, with an estimated annual incidence of approximately 1 million cases according to the Skin Cancer Foundation. While most cases are localized tumors amenable to curative resection, approximately 40,000 cases will become advanced, and an estimated 15,000 people will die from their disease each year. In addition to being a life-threatening disease, cSCC causes significant functional morbidities and cosmetic deformities based on tumors commonly arising in the head and neck region and invading blood vessels, nerves and vital organs such as the eye or ear.

About Cosibelimab

Cosibelimab is a potential best-in-class, high affinity, fully-human monoclonal antibody of IgG1 subtype that directly binds to programmed death ligand-1 ("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 >99% target tumor occupancy to reactivate an antitumor immune response and the additional benefit of a functional Fc domain capable of inducing antibody-dependent cell-mediated cytotoxicity ("ADCC") for potential enhanced efficacy in certain tumor types.

On January 4, 2023 BridgeBio Pharma, Inc. (Nasdaq: BBIO) ("BridgeBio" or the "Company"), a commercial-stage biopharmaceutical company focused on genetic diseases and cancers, reported that members of its management team will present at the 41st Annual J.P. Morgan Healthcare Conference in San Francisco, CA on Monday, January 9 at 3:00 pm PT.

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To access the live webcast of BridgeBio’s presentation, please visit the "Events & Presentations" page within the Investors section of the BridgeBio website at View Source A replay of the webcast will be available on the BridgeBio website for 90 days following the event.

On January 4, 2023 Bloom Science, a clinical-stage biotechnology company developing novel medicines for neurological diseases and conditions, and Yeda Research and Development Co Ltd, the Weizmann Institute of Science’s technology transfer arm to advance science for the benefit of humanity, reported to have entered into an agreement in which Bloom obtained from Yeda an exclusive, worldwide license to use specific microbial strains in developing and commercializing live biotherapeutic products (LBPs) to ameliorate amyotrophic lateral sclerosis (ALS) symptoms (Press release, Bloom Science, JAN 4, 2023, View Source [SID1234625792]).

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There is a large unmet need for patients who have not responded to existing therapies for ALS, a disease that causes nerve cells to die, which breaks the neural pathways, and causes extreme mobility loss over time. Bloom’s systematic approach to developing a new generation of therapeutics starts with the gut microbiome.

"The gut microbiome’s influence on the brain offers incredible potential for healing," said Bloom Science founder and CEO Christohper Reyes, PhD. "Bloom’s collaboration with Yeda enables us to advance our program to modulate the gut microbiome interaction with the brain and provide ALS patients with new therapeutic options that help restore brain function."

Bloom’s final lead orally-administered LBP candidate selection for ALS, BL-002, started in 2022 with animal models and will advance towards human clinical trials in 2023. The company is testing multiple drug candidates for ALS.

Bloom’s BL-002 is built on evidence that oxidative stress drives ALS by causing the loss of motor neurons and the dysfunction of mitochondria. Nicotinamide, an important NAD+ pathway metabolite and a derivative of vitamin B3, can counter the effects of ALS.

BL-002 produces this vitamin and Bloom has shown it attenuates motor-neuron loss, and increases lifespan and motor coordination, in ALS model mice.

On January 4, 2023 BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) reported that Jean-Jacques Bienaimé, Chairman and Chief Executive Officer, will present at the 41st Annual J.P. Morgan Healthcare Conference on Monday, January 9, 2023, at 10:30 am PT / 1:30 pm ET, in San Francisco, California (Press release, BioMarin, JAN 4, 2023, View Source,-January-9,-at-10-30-am-PT-1-30-pm-ET,-in-San-Francisco,-CA [SID1234625791]). To access the live webcast, please visit the investor section of the BioMarin website, www.biomarin.com. A replay will also be archived on the site for at least one week following the event.

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On January 4, 2023 Cellectis S.A. ("Cellectis" or the "Company") (Euronext Growth: ALCLS – NASDAQ: CLLS), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies, reported that it has filed a prospectus supplement with the Securities and Exchange Commission ("SEC"), pursuant to which it may offer and sell to eligible investors a maximum gross amount of up to $60.0 million of American Depositary Shares ("ADS"), each representing one ordinary share of Cellectis, nominal value €0.05 per share, from time to time in sales deemed to be an "at the market offering" pursuant to the terms of a sales agreement with Jefferies LLC ("Jefferies"), acting as sales agent (Press release, Cellectis, JAN 4, 2023, View Source [SID1234625795]). The timing of any sales will depend on a variety of factors. The at-the-market ("ATM") program is presently intended to be effective through the expiration of the existing registration statement, i.e. July 6, 2025, unless terminated prior to such date in accordance with the sales agreement or the maximum amount of the program has been reached.

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The ADSs are listed on the Nasdaq Global Market under the symbol "CLLS", and the Company’s ordinary shares are listed on Euronext Growth in Paris under the symbol "ALCLS".

The Company plans to use the net proceeds, if any, of sales of ADSs issued under the ATM program to fund the continued clinical development of UCART 123, UCART22, UCART20x22, and UCARTCS1, and any remainder for working capital and other general corporate purposes.

Jefferies, as sales agent, will use commercially reasonable efforts to arrange on the Company’s behalf for the sale of all ADSs requested to be sold by the Company, consistent with Jefferies’ normal sales and trading practices. Sales prices may vary based on market prices and other factors. Only eligible investors (as described in greater detail below) may purchase ADSs under the ATM program. In any case, the corresponding sales price of the new ordinary shares underlying the ADSs will not be less than the volume weighted-average of the trading prices of the Company’s ordinary shares on Euronext Growth in Paris over the three trading days prior to the relevant pricing date, subject to a maximum discount to such volume weighted-average price of 15%.

The ADSs and the underlying ordinary shares will be issued through a capital increase without shareholders’ preferential subscription rights under the provisions of Article L. 225-138 of the French Commercial Code (Code de commerce) as decided by the board of directors (the "Board") of Cellectis on December 15, 2022 pursuant to the 11th and/or 13th resolutions adopted by the Combined General Meeting of Shareholders held on June 28, 2022 (or any substitute resolutions, adopted from time to time), within the limit of a maximum number of 13,645,293 ordinary shares (being the maximum authorized by the shareholders for each such resolution), representing a maximum potential dilution of approximately 23.04% based on the share capital of the Company as of September 30, 2022.

The ATM program may only be issued to the categories of investors defined in the 11th and/or 13th resolutions (or any similar resolutions that may be substituted to them in the future), comprising (i) any person or legal entity, whether French or foreign (i.e., non-French), that invests on a regular basis or has invested at least €5 million over the preceding 36 months in the health or biotechnology sector and/or (ii) any industrial company, institution or entity, whether French or foreign (i.e., non-French), active in the health or biotechnology sectors or any affiliate thereof. The new ordinary shares will be admitted to trading on the market of Euronext Growth in Paris and the issued ADSs will trade on Nasdaq.

During the term of the ATM program, the Company will include, in the publication of its quarterly results, information about its use of the program during the preceding quarter and will also provide an update after each capital increase on a dedicated location on its corporate website in order to inform investors about the main features of each issue that may be completed under the ATM program from time to time and, as the case may be, will publish a press release if required by applicable law or regulation.

A shelf registration statement on Form F-3 (including a prospectus) relating to Cellectis’ ADSs was filed with the SEC and was declared effective on July 7, 2022. Before purchasing ADSs in the ATM program, prospective investors should read the prospectus supplement and the accompanying prospectus, together with the documents incorporated by reference therein. Prospective investors may obtain these documents for free by visiting EDGAR on the SEC’s website at www.sec.gov. Alternatively, a copy of the prospectus supplement (and accompanying prospectus) relating to the ATM program may be obtained from Jefferies LLC, 520 Madison Avenue, New York, NY 10022 or by telephone at +1 (877) 821-7388 or by email at [email protected]. There will be no prospectus subject to the approval of the Autorité des Marchés Financiers. The Company disclosed in a press release on December 28, 2022 that it entered into a finance contract with the European Investment Bank ("EIB") on December 28, 2022, which is further described, in particular with respect to the warrants to be issued to the EIB, if any, in a report on Form 6-K dated January 4, 2023 and incorporated by reference in the prospectus supplement, and which is available on the Company’s website at View Source Further, the prospectus supplement also incorporates by reference a report on Form 6-K dated January 4, 2022 that on December 31, 2022, Alain Godard informed the Board of his resignation as a member of the Board, effective immediately. Mr. Godard’s resignation from the Board did not result from any disagreement with Cellectis.