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.

On May 12, 2023 Inmune Bio presented its corporate presentation (Presentation, INmune Bio, MAY 12, 2023, View Source [SID1234631599]).

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On May 12, 2023 IN8bio, Inc. (Nasdaq: INAB), a leading clinical-stage biopharmaceutical company focused on innovative gamma-delta T cell therapies, reported its financial results and operational highlights for the first quarter ended March 31, 2023 (Press release, In8bio, MAY 12, 2023, View Source [SID1234631598]). In addition, the Company provided an overview of recent corporate developments through May 2023.

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"We made significant progress in advancing our gamma-delta T-cell programs in the first quarter of 2023," said William Ho, CEO and co-founder of IN8bio. "Recently we announced positive clinical data from our Phase 1 trial of INB-100, which demonstrated long-term complete remissions and corresponding biomarker data demonstrating elevated gamma-delta T cell levels in all patients. For the first time, we have demonstrated persistence and in vivo expansion with an allogeneic donor derived cell therapy product. We will continue the ongoing expansion of this study, with updated data expected at a major medical meeting later this year."

Mr. Ho continued, "In addition, we are honored to have been selected as one of only a handful of oral abstract presentations for a Phase 1 clinical trial at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) 2023 Annual Meeting. Our team is committed to advancing the potent tumor-targeting capabilities of our gamma-delta T cell platform, with the aim of improving outcomes for patients in need of new and innovative treatment options. We continue to explore the potential of gamma-delta T cells to treat multiple cancer types, as evidenced by our recent preclinical progress in solid tumors and in AML. The IN8bio team is excited about our unique next generation technologies and look forward to providing additional updates on our progress in the coming months."

Business Highlights and Recent Developments

New preclinical data in ovarian cancer will be presented in a poster at the American Society of Cell & Gene Therapy (ASGCT) (Free ASGCT Whitepaper) 26th Annual Meeting. The abstract, to be presented on May 17, 2023, details new preclinical data from the INB-400 program demonstrating the potential of gamma-delta T cells to target and kill solid tumor cells outside the brain.

New INB-200 Phase 1 data will be featured in an oral abstract presentation at the ASCO (Free ASCO Whitepaper) 2023 Annual Meeting. Abstract details including new data from the Phase 1 study evaluating INB-200 in patients with newly diagnosed GBM will be available on May 25, 2023.

Received FDA Orphan Drug Designation for INB-400 (autologous) and INB-410 (allogeneic) for the treatment of a broad spectrum of malignant gliomas, including newly diagnosed GBM. This milestone marks the first time a genetically modified gamma-delta T cell therapy has received this designation, qualifying the Company for incentives, including potential seven year market exclusivity. Enrollment in the autologous arm of the Phase 2 trial targeting newly diagnosed GBM is anticipated to begin in the second half of 2023.

Presented encouraging preclinical data for novel non-signaling CAR (nsCAR) platform and announced the launch of the INB-330 program in AML at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting. Preliminary findings demonstrated that a CD33 targeting nsCAR construct (ns33CAR) was able to selectively differentiate between tumor and healthy tissue, potentially overcoming a critical challenge associated with this previously "undruggable" target.

Presented new, positive INB-100 data from the Phase 1 study showing long-term CRs and elevated gamma-delta T cells in 100% (n=7) of evaluable patients at the 49th Annual Meeting of the European Society for Blood and Marrow Transplantation (EBMT). INB-100 treatment achieved durable CRs in 100% of treated patients, including high-risk AML patients and a patient with acute lymphoblastic leukemia (ALL) who had failed 4 prior lines of therapy, including CAR-T. All evaluable patients remain alive at last assessment and one patient surviving beyond 3 years.
Corporate Updates

Appointed Jeremy R. Graff, Ph.D., to the Company’s Board of Directors. Dr. Graff brings significant drug development and leadership experience in the biotechnology and pharmaceutical sectors including 17 years at Eli Lilly and Company, where he built and led the translational oncology group, supporting and advancing 31 clinical assets in Eli Lilly’s oncology portfolio.
First Quarter 2023 Financial Highlights

Research and Development (R&D) expenses: R&D expenses were $4.4 million for the three months ended March 31, 2023, compared to $2.4 million for the comparable prior year period. The increase in R&D expenses was primarily due to (i) contract research organization expenses related to our INB-400 and INB-100 clinical programs, (ii) increased facility-related costs from opening our new laboratory space in Birmingham, Alabama and (iii) increased personnel-related costs, including salaries, benefits, and stock-based compensation due to increased headcount.
General and administrative expenses: General and administrative expenses were $3.5 million for the three months ended March 31, 2023, compared to $3.8 million for the comparable prior year period. The decrease was primarily due to a decrease in professional services and facility related costs.
Net loss: The Company reported a net loss of $7.5 million, or $0.30 per basic and diluted common share, for the three months ended March 31, 2023, compared to a net loss of $6.1 million, or $0.33 per basic and diluted common share, for the comparable prior year period.
Cash position: As of March 31, 2023, the Company had cash of $10.9 million, compared to $18.2 million as of December 31, 2022. Subsequent to March 31, 2023, the Company raised an additional $9.9 million in net proceeds through the Company’s at-the-market (ATM) program.

On May 12, 2023 IGM Biosciences, Inc. (Nasdaq: IGMS), a clinical-stage biotechnology company focused on creating and developing engineered IgM antibodies, reported its financial results for the first quarter ended March 31, 2023 and provided an update on recent developments (Press release, IGM Biosciences, MAY 12, 2023, View Source [SID1234631597]).

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"We are pleased to be underway in our randomized clinical trial of IGM-8444 in combination with standard of care FOLFIRI chemotherapy and bevacizumab in second line metastatic colorectal cancer patients, and we look forward to presenting additional clinical data in the middle of 2023 from our non-randomized Phase 1 clinical study of 3 mg/kg of IGM-8444 plus FOLFIRI, with and without bevacizumab," said Fred Schwarzer, Chief Executive Officer of IGM Biosciences. "We are also looking forward to the start of clinical testing of imvotamab in autoimmune diseases, where we believe the clinical safety and efficacy profile of imvotamab positions us very well for the exciting new area of treating autoimmune disease with T cell engagers."

Pipeline Updates

IGM-8444 (DR5)

Clinical development of IGM-8444 advances. The Company continues to advance the clinical development of IGM-8444, the Company’s IgM agonist antibody targeting death receptor 5 (DR5).
Dosing ongoing in the randomized colorectal cancer clinical trial. The Company is currently treating patients in a randomized clinical trial of IGM-8444 plus FOLFIRI and bevacizumab in second line metastatic colorectal cancer. The Company plans to assess both the 3 mg/kg and 10 mg/kg dose levels of IGM-8444 with a primary endpoint of progression free survival and secondary endpoints of overall response rate and overall survival as compared to the current standard of care treatment arm.
Dosing ongoing in the venetoclax combination. The Company is currently treating patients with acute myeloid leukemia in its IGM-8444 plus venetoclax and azacytidine Phase 1 combination cohort.
Dosing ongoing in the fifth birinapant dose cohort. The Company is also currently treating patients in its fifth birinapant Phase 1 combination dose escalation cohort.
Additional clinical data expected in middle of 2023. The Company plans to present additional clinical data from the 3 mg/kg of IGM-8444 plus FOLFIRI cohort, with and without bevacizumab, of its non-randomized Phase 1 clinical trial in the middle of 2023.
Presented preclinical data. In April 2023, the Company presented preclinical data on IGM-8444 at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting. The data was featured in a poster presentation titled "Characterization of the synergistic tumor cytotoxicity of agonist DR5 IgM antibody IGM-8444 with chemotherapeutic agents".
Imvotamab (CD20 x CD3)

Advancing into multiple autoimmune clinical trials. The Company is planning to begin clinical testing of imvotamab in severe systemic lupus erythematosus and severe rheumatoid arthritis, subject to IND clearance.
Presented preclinical data. In April 2023, the Company presented preclinical data on imvotamab at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting. The data was featured in a poster presentation titled "Depletion of tissue-resident B cells by a CD20xCD3 IgM bispecific T cell engager in cynomolgus monkeys demonstrates effective tissue penetration and potent target cell killing".
IGM-7354 (IL-15 x PD-L1)

Phase 1 trial. The Company continues enrolling patients in a Phase 1 clinical trial exploring the safety, efficacy and biomarker activity of IGM-7354, an IgM targeted immunostimulatory IL-15 cytokine, in the treatment of patients with solid tumors.
Presented preclinical data. In April 2023, the Company presented preclinical data on IGM-7354 at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting. The data was featured in a poster presentation titled "IGM-7354, an immunocytokine with IL-15 fused to an anti-PD-L1 IgM, induces NK and CD8+ T cell-mediated cytotoxicity of PD-L1-positive tumor cells".
IGM-2644 (CD38 x CD3)

Phase 1 trial. The Company is initiating a clinical trial exploring the safety and efficacy of IGM-2644, a CD38 x CD3 IgM T cell engaging antibody, in patients with recurrent or refractory multiple myeloma.
Presented preclinical data. In April 2023, IGM presented preclinical data evaluating IGM-2644 at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting. The data was featured in a poster presentation titled "Novel CD38xCD3 Bispecific IgM T Cell Engager, IGM-2644, Potently Kills Multiple Myeloma Cells Though Complement and T Cell Dependent Mechanisms".

First Quarter 2023 Financial Results

Cash and Investments: Cash and investments as of March 31, 2023 were $373.4 million, compared to $427.2 million as of December 31, 2022.
Collaboration Revenue: For the first quarter of 2023, collaboration revenues were $0.5 million, compared to no revenue for the same period in 2022.
Research and Development (R&D) Expenses: For the first quarter of 2023, R&D expenses were $50.9 million, compared to $38.9 million for the same period in 2022.
General and Administrative (G&A) Expenses: For the first quarter of 2023, G&A expenses were $13.0 million, compared to $13.1 million for the same period in 2022.
Net Loss: For the first quarter of 2023, net loss was $59.3 million, or a loss of $1.33 per share, compared to a net loss of $51.9 million, or a loss of $1.53 per share, for the same period in 2022.

2023 Financial Guidance

The Company expects full year 2023 GAAP operating expenses of $275 million to $285 million, including estimated non-cash stock-based compensation expense of approximately $45 million, and full year collaboration revenue of approximately $3 million related to the Sanofi agreement. The Company expects to end 2023 with a balance of more than $200 million in cash and investments, and for the balance to enable it to fund its operating expenses and capital expenditure requirements into the fourth quarter of 2024.