On May 12, 2023 Veracyte, Inc. (Nasdaq: VCYT) reported that new data show the use of the Prosigna Breast Cancer Assay altered treatment decisions for patients with early-stage breast cancer, including significantly reducing the use of chemotherapy among those with clinically high-risk disease (Press release, Veracyte, MAY 12, 2023, View Source [SID1234631604]). The findings are from EMIT, a prospective, multi-year, population-based study in Norway that is investigating the impact of molecular testing – specifically, the Prosigna test – on breast cancer care and outcomes. These initial data focused on treatment decisions and were shared in a poster (#103P) today at ESMO (Free ESMO Whitepaper) Breast Cancer Congress 2023, taking place May 11-13 in Berlin.

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"Our findings demonstrate the important role that gene expression testing, and in particular the Prosigna assay, can play in offering physicians better prognostic information to help guide next steps for their patients with breast cancer," said Bjørn Naume, M.D., Professor at the Department of Oncology, Rikshospitalet Oslo University Hospital. "Specifically, Prosigna test results changed physicians’ treatment decisions in all patient clinical-risk groups with early breast cancer, regardless of whether they were low-, intermediate-, or high-risk, and reduced treatment discrepancies across hospitals."

For the study, researchers evaluated data for 2,164 women in Norway with early-stage (node-negative) breast cancer, recording physicians’ treatment decisions for these patients before and after a Prosigna test result. Prior to Prosigna results, physicians directed 27% to no systemic treatment (low-risk patients), 38% to endocrine therapy only (intermediate-risk patients) and 35% to chemotherapy followed by endocrine therapy (high-risk patients). After Prosigna test results, these treatment decisions changed to 25%, 51% and 24%, respectively. The researchers found that use of Prosigna changed adjuvant therapy decisions for almost one-third (29%) of patients. Notably, for patients assigned to chemotherapy prior to Prosigna results, 45% were de-escalated to endocrine therapy following Prosigna results.

The Prosigna assay analyzes the activity of the PAM50 gene signature, along with clinical-pathological features, to provide a hormone-receptor positive early breast cancer patient and her physician with a prognostic score indicating the probability of cancer recurrence during the next 10 years if treated with endocrine therapy alone. The Prosigna test is performed on the nCounter Analysis System and is available to laboratories in Europe and elsewhere to enable local testing for physicians and their patients.

"We are honored that the Prosigna test was selected to be part of EMIT, which we believe is among the most rigorous studies to evaluate the impact of gene expression testing on clinical decision-making and patient outcomes in breast cancer," said Kelly Marcom, M.D., Veracyte’s medical director for Breast Cancer. "The findings presented at the ESMO (Free ESMO Whitepaper) Breast conference add to the growing body of evidence demonstrating the clinical utility of Veracyte’s Prosigna assay. We look forward to additional data that will come out of the EMIT trial as researchers continue to follow these patients over multiple years."

On May 12, 2023 Carisma Therapeutics Inc. (the "Company"), reported to have entered into an Amended and Restated Open Market Sale AgreementSM (the "A&R Sale Agreement") with Jefferies LLC, as sales agent ("Jefferies"), pursuant to which the Company may offer and sell shares of its common stock, $0.001 par value per share (the "Shares"), from time to time through Jefferies (Filing, 8-K, Carisma Therapeutics, MAY 12, 2023, View Source [SID1234631603]). The Company has also filed a prospectus supplement (the "Prospectus Supplement") under the Company’s universal shelf registration statement on Form S-3 (File No. 333-271295) that was originally filed with the Securities and Exchange Commission (the "SEC") on April 17, 2023 and declared effective by the SEC on May 2, 2023 (the "Registration Statement"). Pursuant to the Prospectus Supplement, the Company may offer and sell Shares having an aggregate offering price of up to $100.0 million.

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The Company previously filed prospectus supplements with the SEC under the Company’s universal shelf registration statements on Form S-3/A (File No. 333-223750) on November 29, 2020 and February 7, 2021 and on Form S-3 (File No. 333-255943) on June 1, 2021 and July 14, 2021 relating to the offering and sale of the Company’s common stock pursuant to the Open Market Sale AgreementSM, dated as of November 29, 2019, as amended, with Jefferies (the "Original Sale Agreement"). Prior to entering into the A&R Sale Agreement, the Company had remaining capacity to sell up to approximately $97.8 million of the Company’s common stock pursuant to the Original Sale Agreement under the prior prospectus supplement dated July 14, 2021. The Prospectus Supplement supersedes and replaces all prior prospectus supplements relating to the offering and sale of shares of the Company’s common stock pursuant to the Original Sale Agreement and the A&R Sale Agreement supersedes and replaces the Original Sale Agreement in all respects.

Upon delivery of an issuance notice and subject to the terms and conditions of the A&R Sale Agreement, Jefferies may sell the Shares at market prices by any method deemed to be an "at the market offering" as defined in Rule 415(a)(4) promulgated under the Securities Act of 1933, as amended (the "Securities Act").

The Company or Jefferies may suspend or terminate the offering of Shares upon notice to the other party, subject to certain conditions. Jefferies will act as sales agent on a commercially reasonable efforts basis consistent with its normal trading and sales practices.

The Company has agreed to pay Jefferies commissions for its services of acting as sales agent of up to 3.0% of the gross proceeds from the sale of the Shares pursuant to the A&R Sale Agreement. The Company has also agreed to provide Jefferies with customary indemnification and contribution rights.

A copy of the A&R Sale Agreement is attached as Exhibit 1.1 hereto and is incorporated herein by reference. The foregoing description of the material terms of the A&R Sale Agreement is qualified in its entirety by reference to such exhibit.

Wilmer Cutler Pickering Hale and Dorr LLP, counsel to the Company, has issued a legal opinion relating to the Shares. A copy of such legal opinion, including the consent included therein, is attached as Exhibit 5.1 hereto.

The Shares will be sold pursuant to the Registration Statement, and offerings of the Shares will be made only by means of the Prospectus Supplement. This Current Report on Form 8-K shall not constitute an offer to sell or solicitation of an offer to buy the Shares, nor shall there be any sale of the Shares in any state in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities law of such state or jurisdiction.

On May 12, 2023 Precision BioSciences, Inc. (Nasdaq: DTIL), a clinical stage gene editing company developing ARCUS-based ex vivo allogeneic CAR T and in vivo gene editing therapies, reported that its late-breaking abstract presenting preclinical in vivo gene editing data for its PBGENE-DMD program, being developed for the potential treatment of Duchenne muscular dystrophy (DMD), is available through the American Society of Gene & Cell Therapy (ASGCT) (Free ASGCT Whitepaper) 26th Annual Meeting website at View Source (Press release, Precision Biosciences, MAY 12, 2023, View Source [SID1234631602]).

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An oral presentation, ARCUS-Mediated Excision of the "Hot Spot" Region of the Human Dystrophin Gene for the Treatment of Duchenne Muscular Dystrophy (DMD), will be presented as part of the Late-breaking Abstracts 2 Session on May 19, 2023.

On May 12, 2023 PharmaMar (PHM:MSE) reported that the European Commission has designated lurbinectedin as an Orphan Medicinal Product for the treatment of Soft Tissue Sarcoma (STS), following a positive opinion from the Committee for Orphan Medicinal Products (COMP) of the European Medicines Agency (EMA) (Press release, PharmaMar, MAY 12, 2023, View Source [SID1234631601]).

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STS is a rare type of cancer. Currently, one of the most common subtypes of sarcoma is Leiomyosarcoma (LMS), which accounts for 16% of the pathology, and most frequently affects middle-aged adults and older, predominantly women.

Orphan drug designation is a status granted by the EMA to drugs intended to treat rare or uncommon diseases that affect fewer than 5 people per 10,000 inhabitants in the European Union. To obtain this designation, the drug must demonstrate its ability to treat a disease, that has no satisfactory therapeutic options and is expected to benefit patients suffering from the disease. Orphan drug designation offers several benefits to drug manufacturers, including exemption from some EMA fees, scientific and regulatory advice during drug development, and the possibility of receiving tax benefits and market exclusivity for a period of 10 years in the European Union.

On May 12, 2023 Mustang Bio, Inc. ("Mustang") (Nasdaq: MBIO), a clinical-stage biopharmaceutical company focused on translating today’s medical breakthroughs in cell and gene therapies into potential cures for hematologic cancers, solid tumors and rare genetic diseases, reported its financial results and recent corporate highlights for the first quarter ended March 31, 2023 (Press release, Mustang Bio, MAY 12, 2023, View Source [SID1234631600]).

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Manuel Litchman, M.D., President and Chief Executive Officer of Mustang, said, "The first quarter of 2023 was marked by the continued advancement of our lead clinical candidate MB-106, a CD20-targeted, autologous CAR T cell therapy to treat relapsed or refractory B-cell non-Hodgkin lymphomas ("B-NHL") and chronic lymphocytic leukemia ("CLL"), through two parallel tracks. The first is the ongoing Phase 1/2 single-institution clinical trial at Fred Hutchinson Cancer Center ("Fred Hutch"), in which MB-106 continues to demonstrate compelling safety and efficacy. The second track is Mustang’s multicenter, open-label, non-randomized Phase 1/2 clinical trial, which continues to accrue. The final dose level escalation is anticipated in the Phase 1 indolent lymphoma arm in the third quarter of this year. MB-106 remains an attractive potential therapeutic option when compared to approved autologous CAR Ts, which are generating an annualized run rate of $3 billion in net sales, based on reported sales in the third quarter of 2022. We expect data from the Fred Hutch clinical trial to be presented at medical meetings in the second quarter of 2023, and initial data from the multicenter clinical trial to be disclosed shortly as well."

Financial Results:

● As of March 31, 2023, Mustang’s cash and cash equivalents and restricted cash totaled $58.8 million, compared to $76.7 million as of December 31, 2022, a decrease of $17.9 million year-to-date.
● Research and development expenses were $14.0 million for the first quarter of 2023, compared to $16.3 million for the first quarter of 2022. Non-cash, stock-based expenses included in research and development were $0.1 million for the first quarter of 2023, compared to $0.5 million for the first quarter of 2022.
● General and administrative expenses were $2.3 million for the first quarter of 2023, compared to $3.3 million for the first quarter of 2022. Non-cash, stock-based expenses included in general and administrative expenses were $0.1 million for the first quarter of 2023, compared to $1.0 million for the first quarter of 2022.
● Net loss attributable to common stockholders was $16.7 million, or $2.06 per share, for the first quarter of 2023, compared to a net loss attributable to common stockholders of $19.8 million, or $2.71 per share, for the first quarter of 2022.
Recent Corporate Highlights:

● Mustang’s lead clinical candidate is MB-106, a CD20-targeted, autologous CAR T cell therapy to treat relapsed or refractory B-NHL and CLL. MB-106 data to date include an overall response rate of 96% and complete response rate of 75% in a wide range of hematologic malignancies, including Waldenstrom macroglobulinemia ("WM"), in a clinical trial conducted by Mustang’s collaborators at Fred Hutch. In parallel, Mustang’s multicenter, open-label, non-randomized Phase 1/2 clinical trial evaluating the safety and efficacy of MB-106 continues to accrue, and Mustang anticipates escalation to the final dose level in the Phase 1 indolent lymphoma arm in the third quarter of this year. The FDA granted Orphan Drug Designation to MB-106 for the treatment of WM, and Mustang has
treated the first WM patient in the indolent lymphoma arm of the trial. Results from this arm are expected to support an accelerated Phase 2 registration strategy for WM, with the first pivotal Phase 2 WM patient potentially to be treated in the first quarter of 2024. In the second quarter of this year, Mustang plans to report safety and efficacy data from the indolent lymphoma arm.
● Phase 1/2 data from the Fred Hutch clinical trial on MB-106, a CD20-targeted, autologous CAR T cell therapy for patients with relapsed or refractory B-NHL and CLL, will be presented at the European Hematology Association (EHA) (Free EHA Whitepaper) Hybrid Congress ("EHA2023") taking place June 8-11, 2023, in Frankfurt, Germany and at the International Conference on Malignant Lymphoma ("ICML") taking place June 13-17, 2023, in Lugano, Switzerland. Data from the Waldenstrom macroglobulinemia cohort were selected for poster presentation at EHA (Free EHA Whitepaper)2023 and outpatient treatment of follicular lymphoma was selected for oral presentation at ICML.
● Mustang continues to collaborate with the Mayo Clinic to progress its exclusively licensed novel in vivo CAR T technology platform that may be able to transform the administration of CAR T therapies and has the potential to be used as an off-the-shelf therapy. Mustang anticipates the publication of proof-of-concept research in a murine tumor model in 2023.
● In April 2023, Mustang effected a 15-for-1 reverse stock split of its issued and outstanding common stock. Mustang’s common stock began trading on a split-adjusted basis on the Nasdaq Capital Market as of the commencement of trading on April 4, 2023.
● In April 2023, Mustang terminated the Loan and Security Agreement, as Amended, with Runway Growth Finance Corp. ("Runway") upon receipt by Runway of a payoff amount of $30.7 million from the Company representing the applicable final payment.