On April 22, 2021 Aptorum Group Limited (Nasdaq: APM, Euronext Paris: APM) ("Aptorum Group" or "Aptorum"), a clinical-stage biopharmaceutical company, through its affiliate, reported that it has entered into a material transfer and option agreement ("Agreement") with Yale University ("Yale") to evaluate a group of preclinical stage novel immunomodulators (Press release, Aptorum, APR 22, 2021, View Source [SID1234578372]). The novel immunomodulators may potentially target autoimmune and oncology diseases, including but not limited to, rheumatoid arthritis, lupus and sclerosis, as well as a variety of cancers, all subject to further preclinical development and testing. Aptorum also obtained an exclusive option to in-license the novel immunomodulators and its affiliated intellectual property rights including its patent rights and know-how, based on licensing terms pursuant to a binding term sheet incorporated into the Agreement.

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Under the arrangement, Aptorum will be responsible for assessing Yale’s originally developed novel immunomodulators against lupus, arthritis, inflammatory bowel diseases, neurodegenerative diseases or other oncology indications. Upon exercising its option to license, Aptorum will undertake to develop and commercialize one or more of these novel immunomodulators, as supported by Yale.

Mr. Ian Huen, Chief Executive Officer of Aptorum Group, commented: "Aptorum is delighted to partner with Yale to validate and develop its novel class of immunomodulators. Aptorum believes that these immunomodulators have the potential to advance first-in-class drug discovery and development of such drugs for potential autoimmune and oncology diseases in particular. For example, it has been widely reported that COVID-19 infections often trigger a cytokine storm in patients leading to undesirable and often fatal autoiummune reactions1 and we will also explore the potential applications of the immunomodulators for the treatment of these cytokine storms. Through this initiative, we are looking forward to strategically expanding our development footprint in the treatment of those diseases, which have other potential applications such as in oncology and infectious diseases which are already core focuses of our company."

On April 22, 2021 ITM AG reported that the successful close of a convertible loan financing totaling EUR 90 million (USD 109 million) led by Petrichor Healthcare Capital Management with participation of additional undisclosed investors (Press release, ITM Isotopen Technologien Munchen, APR 22, 2021, View Source [SID1234578371]). The proceeds will support the expansion of ITM’s precision oncology pipeline as well as fund late-stage development and market access activities for lead candidate, n.c.a. 177Lu-Edotreotide, currently in a phase III trial in gastroenteropancreatic neuroendocrine tumors (GEP-NETs). Under the terms of the agreement, the loan will convert into shares of ITM in the event of certain future financial or other corporate development transactions. Further details of the financing were not disclosed.

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"This funding represents our commitment to being at the forefront of demonstrating the therapeutic potential of Targeted Radionuclide Therapy and reflects our ambition to translate our leadership as a global radioisotope provider into developing innovative radiopharmaceuticals to meet the needs of patients with difficult-to-treat tumors. The investment from Petrichor and the syndicate recognizes our clinical and corporate achievements to date and enables us to further leverage the full potential of nuclear medicine for cancer patients," commented Steffen Schuster, Chief Executive Officer of ITM.

In conjunction with the financing, Tadd Wessel, Founder and Managing Partner at Petrichor, will join ITM’s supervisory board. He brings more than 20 years of experience in the healthcare financing sector and was formerly a Managing Director at OrbiMed Advisors, where he led the build-out of the healthcare special situations and structured finance business.

Mr. Wessel added: "ITM has established itself as a dominant global provider of therapeutic radioisotopes. We expect the company’s isotope business to benefit from tremendous growth of targeted radiotherapy in the years ahead, while serving as a platform for ITM’s deep pipeline of precision oncology Targeted Radionuclide Therapies and companion diagnostics. Targeted radiotherapy has demonstrated immense success to date, and we expect the drug class to continue to gain recognition as one of the most promising areas within precision oncology. We believe that ITM’s unparalleled expertise and technological capabilities are evidenced by their market leadership, and we look forward to supporting the team in their pursuit of better patient outcomes."

ITM, founded in 2004, is a company built on longstanding experience in the production and supply of high-quality medical radioisotopes for cancer treatment and diagnosis with an established global supply network. The company has forward-integrated to develop Targeted Radionuclide Diagnostics and Therapies designed to provide medical benefit for difficult-to-treat cancer indications.

On April 22, 2021 NeuBase Therapeutics, Inc. (Nasdaq: NBSE) ("NeuBase"), a biotechnology company accelerating the genetic revolution with a new class of precision genetic medicines, reported the pricing of an underwritten public offering of 8,000,000 shares of its common stock, at a price to the public of $5.00 per share (Press release, NeuBase Therapeutics, APR 22, 2021, View Source [SID1234578370]). The gross proceeds to NeuBase from the offering, before deducting the underwriting discounts and commissions and offering expenses payable by NeuBase, are expected to be approximately $40 million, excluding any exercise of the underwriters’ option to purchase additional shares of common stock. In addition, NeuBase has granted the underwriters a 30-day option to purchase up to an additional 1,200,000 shares of its common stock at the public offering price, less underwriting discounts and commissions, to cover over-allotments, if any. All of the shares of common stock in the offering are being sold by NeuBase.

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RBC Capital Markets, Oppenheimer & Co. Inc. and Chardan are acting as the joint book-running managers for the offering, and National Securities Corporation is acting as co-manager.

NeuBase intends to use the net proceeds from the offering for general corporate purposes, working capital and development of its product candidates and pipeline expansion. This offering is expected to close on or about April 26, 2021, subject to the satisfaction of customary closing conditions.

The securities described above were offered by NeuBase pursuant to a shelf registration statement on Form S-3 (File No. 333-254980) previously filed with the Securities and Exchange Commission (the "SEC") on April 1, 2021 and declared effective by the SEC on April 14, 2021. The securities may be offered only by means of a prospectus. A preliminary prospectus supplement and the accompanying prospectus relating to and describing the offering has been filed with the SEC. Electronic copies of the preliminary prospectus supplement and, when available, copies of the final prospectus supplement and the accompanying prospectus relating to the offering may be obtained by visiting the SEC’s website at www.sec.gov or by contacting RBC Capital Markets, Attention: Equity Syndicate, 200 Vesey Street, 8th Floor, New York, NY 10281, or by telephone at (877) 822-4089 or by e-mail at [email protected], Oppenheimer & Co. Inc., Attention: Syndicate Prospectus Department, 85 Broad Street, 26th Floor, New York, NY 10004, or by telephone at (212) 667-8055 or by e-mail at [email protected], or Chardan, 17 State Street, 21st floor, New York, New York 10004, by telephone at (646) 465-9032 or by e-mail at [email protected].

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

On April 22, 2021 Blueprint Medicines Corporation (NASDAQ: BPMC), reported that it will host a live conference call and webcast at 8:30 a.m. ET on Thursday, April 29, 2021 to report its first quarter 2021 financial results and provide a corporate update (Press release, Blueprint Medicines, APR 22, 2021, View Source [SID1234578369]).

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To access the live conference call, please dial (855) 728-4793 (domestic) or (503) 343-6666 (international), and refer to conference ID 9292306. A webcast of the call will also be available under "Events and Presentations" in the Investors & Media section of the Blueprint Medicines website at View Source The archived webcast will be available on Blueprint Medicines’ website approximately two hours after the conference call and will be available for 30 days following the call.

On April 22, 2021 Onconova Therapeutics, Inc. (NASDAQ: ONTX), a clinical-stage biopharmaceutical company focused on discovering and developing novel products for patients with cancer, reported that the first patient has been dosed in an investigator-initiated Phase 2 study to assess the efficacy and safety of rigosertib in patients with recessive dystrophic epidermolysis bullosa (RDEB)-associated locally advanced/metastatic squamous cell carcinoma (SCC) (Press release, Onconova, APR 22, 2021, View Source [SID1234578368]). The patient was dosed at the EB House Austria, a center of expertise for epidermolysis bullosa at the University Hospital Salzburg, Austria. Additional sites are anticipated to be opened in the UK and in the US to study this rare and genomically driven devastating disease.

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In this open-label investigator-initiated study, 12 patients will receive either oral or intravenous rigosertib at the clinician’s discretion given the various clinical manifestations of the disease, which may dictate the need for either oral or intravenous administration of rigosertib. These patients have skin desquamation making intravenous access difficult, or may form esophageal strictures, which make oral administration difficult. Patients will receive either oral rigosertib in four-week cycles (three weeks on, one week off) for up to 13 cycles, with 560 mg of oral rigosertib in the morning and again in the afternoon, for a total of 1,120 mg/day. Alternatively, patients will receive intravenous (IV) rigosertib as a 72-hour IV infusion on days 1, 2 and 3 of eight 2-week cycles, and on days 1, 2 and 3 of nine 4-week cycles thereafter, with each 24-hour infusion consisting of 1,800 mg of rigosertib.

The study has two primary endpoints. The first is to determine the anti-tumor activity of rigosertib in RDEB patients with advanced SCC who have failed prior standard of care through the overall response rate (ORR), defined as the proportion of patients who achieve either a complete response (CR) or a partial response (PR). The second primary endpoint is to evaluate the safety and tolerability of rigosertib in this population. Secondary study endpoints include quality of life and a biomarker analysis performed on archival tissue from all patients. Patients will be dosed for up to one year, with trial duration anticipated to be approximately two-and-a-half years.

"We are pleased with the advancement of our investigator-initiated programs with rigosertib, and to provide rigosertib in support of this important Phase 2 investigator-sponsored study," said Steven M. Fruchtman, M.D., President and Chief Executive Officer of Onconova Therapeutics. "Recessive dystrophic epidermolysis bullosa is a genetic skin blistering disease that often results in squamous cell carcinoma in severe subtypes. In this patient cohort, squamous cell carcinoma is the leading cause of death. We have previously identified polo-like kinase 1 as a therapeutic target in skin SCC, including RDEB SCC, so we are encouraged by the start of this trial. We hope rigosertib can prove beneficial to this rare patient population with a tremendous unmet medical need."

In addition to Onconova Therapeutics, the study is being supported by DEBRA International. "The aggressive course and poor prognosis of skin cancer in our patients emphasize the urgent need for potent therapies," stated Professor Johann W. Bauer, M.D., Principal investigator of the trial. "We hope that rigosertib as an innovative approach provides benefit to this devastating illness that currently lacks effective therapies."

"Basic research has provided understanding into the etiology of Recessive Dystrophic Epidermolysis Bullosa-associated cancer," stated Andrew South Ph.D., Associate Professor, Department of Dermatology & Cutaneous Biology, Thomas Jefferson University. "I would like to thank both the Debra Foundations for funding this work as well as Onconova for providing a research drug that may target the life-threatening cancers arising in these patients."