On April 5, 2021 China’s Legend Biotech and Janssen reported that they have finished off the rolling submission to the FDA for their partnered cell therapy hopeful cilta-cel (Press release, Legend Biotech, APR 5, 2021, View Source [SID1234577717]).

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The closely watched asset, the star of the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper)’s meeting a few years’ back, saw Johnson & Johnson’s U.S. submission for adults with relapsed and/or refractory multiple myeloma completed in the first quarter, according to a new form filed with the Securities and Exchange Commission from Legend.

Analysts at Jefferies said this is a "critical first step" toward nabbing an approval for the therapy by year-end. There’s another two months for the biologics license application to be accepted and then, if its nabs a priority review, another six months for approval, meaning it could be given the green light toward the end of 2021, for which it has been previously guided.

This comes just a few weeks after Bristol Myers Squibb and partner bluebird bio nabbed an approval for Abecma—formerly known as idecabtagene vicleucel, or ide-cel—to treat adults whose multiple myeloma has progressed after at least four previous rounds of treatment.

The March approval was the first CAR-T med approved in multiple myeloma and the first CAR-T in the B-cell maturation antigen-targeted class. J&J and Legend are now hoping to be close behind.

In recent data out late last year at the annual meeting of the American Society of Hematology (ASH) (Free ASH Whitepaper), the pair unveiled the first phase 2 readout for its prospect, which banished multiple myeloma in two-thirds of patients and shrank tumors in 97% of patients.

RELATED: ASH (Free ASH Whitepaper): J&J, Legend’s anti-BCMA CAR-T keeps it consistent in phase 2 as FDA filing looms

The data, presented virtually at ASH (Free ASH Whitepaper)’s annual meeting in December, came from 97 patients whose cancer had returned after a median of six prior treatments or had not responded to treatment in the first place.

The investigators followed the patients for a median of one year after treatment. J&J’s Janssen unit previously reported data from 29 patients from the phase 1b part of the trial, showing the treatment—ciltacabtagene autoleucel, or cilta-cel—eliminated tumors in 86% of patients and shrank tumors in all 29 of them for a 100% overall response rate.

Jefferies said in a note to clients that, while the duo won’t be first, "We think round 8-9 months difference in the timing of market arrival does not offer much first mover advantage for [BMS/bluebird bio’s] ide-cel given its inferior clinical profile to cilta-cel."

On April 5, 2021 Rain Therapeutics reported that it filed for a $100 million IPO to advance its lead MDM2 inhibitor RAIN-32 into Phase III testing (Press release, Rain Therapeutics, APR 5, 2021, View Source [SID1234577712]). The news comes months after the company closed a $63 million Series B and inlicensed RAIN-32 from Daiichi Sankyo.

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Rain licenses and develops precision cancer therapies. The new lead program, RAIN-32, previously completed Phase I testing in patients with solid tumors or lymphomas. The influx of cash will support a pivotal Phase III trial in liposarcoma, which Rain intends to launch this year, plus two more Phase II trials in solid tumors and intimal sarcoma, respectively.

Preclinical data published by independent French researchers last year suggested MDM2 inhibitors had therapeutic potential for treating liposarcomas, where MDM2 amplification is common.

MDM2 is an inhibitor of tumor-suppressive p53, a notoriously challenging drug target. Several companies are developing MDM2 inhibitors to act upstream of p53, with limited success, which Rain attributes to dose-limiting hematological toxicities.

Last year, Roche ended development of its MDM2 inhibitor, idasanutlin, then in Phase II testing for patients with acute myeloid leukemia (AML). The most advanced anti-MDM2 therapy is APG-115, which Ascentage Pharma has in multiple Phase Ib/II trials for solid tumors and hematologic malignancies, plus a Phase II combination trial with checkpoint inhibitor Keytruda (pembrolizumab) for anti-PD-1 refractory or relapsed cancers. At least two other companies–Novartis and Aileron–also have MDM2 inhibitors in early clinical testing for several cancers.

According to Rain, the safety and tolerability data from Daiichi Sankyo’s trial suggest RAIN-32 can be dosed longer than other MDM2-targeting programs in development.

Rain also has a preclinical RAD52 inhibitor program, licensed from Drexel University in August 2020. RAD52 plays a role in DNA damage response and the company is exploring therapies for breast and ovarian cancers characterized by BRCA1/2 mutations. The company is aiming to select a lead clinical RAD52 inhibitor candidate in 2022. There are no known RAD52 inhibitors in clinical trials.

As recently as last year, its previous lead candidate tarloxotinib, licensed from the University of Auckland, was in Phase II testing for patients with EGFR and ErbB Exon 20 insertion mutations in solid tumors.

The company has not announced an end to the trial but tarloxotinib is not named in Rain’s Securities and Exchange Commission (SEC) forms or listed on the company’s website. Rain did not respond to a request for clarification in time for publication.

On April 5, 2021 Celsion Corporation (NASDAQ: CLSN) ("Celsion" or the "Company"), reported the closing of its previously announced registered direct offering of 11,538,462 shares of common stock at a purchase price of $1.30 per share, resulting in net proceeds of $13.9 million, after deducting placement agents’ fees but before expenses payable by the Company (Press release, Celsion, APR 5, 2021, View Source [SID1234577628]).

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A.G.P./Alliance Global Partners acted as the lead placement agent for the offering.

JonesTrading Institutional Services LLC and Brookline Capital Markets, a division of Arcadia Securities, LLC, acted as co-placement agents for the offering.

Celsion intends to use the net proceeds for general corporate purposes, including research and development activities, capital expenditures and working capital.

This offering was made pursuant to an effective shelf registration statement on Form S-3 (File No. 333-254515), previously filed with the Securities and Exchange Commission (the "SEC") on March 19, 2021 and declared effective on March 30, 2021. The offering of the shares of common stock were made by means of a prospectus, including a prospectus supplement, forming a part of the effective registration statement. A prospectus supplement and the accompanying prospectus relating to and describing the terms of the offering are filed with the SEC and are available on the SEC’s website at View Source or by contacting A.G.P./Alliance Global Partners, 590 Madison Avenue, 28th Floor, New York, NY 10022, or by telephone at (212) 624-2060, or by email at [email protected].

This press release shall not constitute an offer to sell or a solicitation of an offer to buy 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 April 5, 2021 Prothena Corporation plc (NASDAQ:PRTA), a late-stage clinical company with expertise in protein dysregulation and a pipeline of investigational therapeutics for rare peripheral amyloid and neurodegenerative diseases, reported the appointment of Hideki Garren, MD, PhD, as Chief Medical Officer (Press release, Prothena, APR 5, 2021, View Source [SID1234577627]). Dr. Garren will lead the clinical and medical organizations to advance Prothena’s clinical pipeline.

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"Hideki brings valuable experience and expertise to our team as we look ahead towards transitioning to a fully integrated research, development and commercial biotechnology company," said Gene Kinney, PhD, President and Chief Executive Officer. "His experience leading several late-stage development programs in neurology and rare disease indications through registration and launch is particularly relevant as we advance our pipeline of programs in these areas. We are delighted to welcome him to the team as we prepare for our next stage of growth."

"Prothena’s impressive scientific platform has advanced a rich pipeline of potentially transformational therapeutics, including several programs that have demonstrated benefit on meaningful clinical endpoints in diseases with highly significant unmet medical needs," commented Dr. Garren. "I am excited to join the company during this dynamic period of growth and look forward to working as part of this exceptional team with a rich scientific heritage."

Dr. Garren joins Prothena from F. Hoffmann-La Roche Ltd. (Roche) & Genentech Inc. where he was Vice President, Global Head of Neuroimmunology, leading Roche’s Neuroimmunology franchise team. A neurologist by training, Dr. Garren was most recently responsible for the Phase 3 clinical programs for Ocrevus for Multiple Sclerosis and Enspryng for Neuromyelitis Optica Spectrum Disorder. Dr. Garren also held the role of Executive Director, Translational Medicine Expert in Neuroscience with Novartis Pharma. Prior to Novartis, Dr. Garren served as Co-Founder, Executive Vice President, Chief Scientific Officer, and Chief Operating Officer of Bayhill Therapeutics, Inc., a company he started in 2002 based on a technology platform he co-invented while at Stanford University.

Dr. Garren earned his Bachelor of Science from the California Institute of Technology and his MD and PhD from the University of California, Los Angeles (UCLA). He completed his internship in internal medicine at UCLA, and his residency in neurology and fellowship in neuroimmunology at Stanford University.

On April 5, 2021 XOMA Corporation (Nasdaq: XOMA) ("XOMA" or the "Company") reported it has commenced an underwritten registered public offering of depositary shares, each representing 1/1000th fractional interest in a share of the Company’s Series B Cumulative Perpetual Preferred Stock, with liquidation preference of $25.00 per depositary share, subject to market and certain other conditions (Press release, Xoma, APR 5, 2021, View Source [SID1234577626]). The Company expects to grant the underwriters a 30-day option to purchase additional depositary shares in connection with the offering.

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The Company expects to use the net proceeds of this offering to fund the segregated dividend account and the remaining net proceeds for general corporate purposes, including funding future acquisitions of milestone and royalty rights associated with drug development programs with third-party funding.

B. Riley Securities, Inc., National Securities Corporation, Ladenburg Thalmann & Co. Inc., and William Blair & Company are acting as joint book-runners for this offering. Aegis Capital Corp., Boenning & Scattergood, Inc., Incapital LLC, and Northland Capital Markets are acting as co-managers.

The offering of these securities is being made pursuant to an effective shelf registration statement on Form S-3, which was initially filed with the Securities and Exchange Commission (the "SEC") on March 10, 2021, and declared effective by the SEC on March 19, 2021. The offering of these depositary shares will be made only by means of a prospectus and prospectus supplement. A copy of the prospectus and prospectus supplement relating to these securities may be obtained, when available, from the website of the SEC at View Source or by contacting: B. Riley Securities, Inc., 1300 17th Street North, Suite 1300, Arlington, Virginia 22209, Attn: Prospectus Department, Email: [email protected], Telephone: (703) 312-9580.

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.