FDA clears Imugene IND to commence onCARlytics first-in-class clinical study

On May 19, 2023 Imugene Limited (ASX:IMU), a clinical stage immuno oncology company, reported that it has received US Food and Drug Administration (FDA) Investigational New Drug (IND) clearance to initiate a Phase 1 clinical study of its oncolytic virotherapy candidate, onCARlytics (on-CAR-19, CF33-CD19, HOV4) (Press release, Imugene, MAY 19, 2023, View Source [SID1234631874]).

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The FDA clearance of the IND allows Imugene to start patient recruitment and dosing in a first-in-class Phase 1 clinical study for the onCARlytics platform in patients with solid tumours: "A Phase I, Dose Escalation and Dose Expansion, Safety and Tolerability Study of onCARlytics (CF33-CD19), Administered Intravenously or Intratumorally in Combination with Blinatumomab in Adults with Advanced or Metastatic Solid Tumors (OASIS)."

Imugene’s CF33-CD19 oncolytic virus, when combined with the CD19 targeting bispecific monoclonal antibody blinatumomab (Blincyto), has the potential to target and eradicate solid tumours that otherwise cannot be treated with Blincyto therapy alone.

Pacylex Pharmaceuticals Announces First Patient Dosed in a Phase 2a Study of PCLX-001 in Patients with Relapsed/Refractory Non-Hodgkin Lymphoma

On May 18, 2023 Pacylex Pharmaceuticals, a clinical-stage company developing PCLX-001, a first-in-class oral investigational small molecule for leukemia, lymphoma, and solid tumor cancers, reported the first patient has been dosed in a Phase 2a expansion study in patients with relapsed/refractory B-cell Non-Hodgkin Lymphoma (R/R NHL) (Press release, Pacylex Pharmaceuticals, MAY 18, 2023, View Source [SID1234645057]).

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The Company’s Phase 1 multiple ascending dose (MAD) clinical trial evaluating PCLX-001 monotherapy in patients with R/R NHL or advanced solid tumors successfully completed six dose escalations without any dose-limiting toxicities (DLTs) and reached a drug exposure level expected to show clinical activity. Three patients with advanced solid tumors in the highest dose level are continuing to receive PCLX-001.

The Company received a No Objection Letter from Health Canada earlier this year to initiate the expansion cohort, designed to evaluate a potential Phase 2 dose in patients with R/R NHL. Four Canadian clinical sites have been activated and the first patient has been dosed. The expansion study will enroll up to 20 patients to assess the preliminary clinical activity of PCLX-001.

"The first stage of this first-in-human study assessed the safety of PCLX-001 with repeated dosing. We now transition our clinical investigations from primarily establishing safety to both safety and efficacy at a potential Phase 2 dose," said Dr. John Mackey, CMO of Pacylex.

"This milestone represents a major step in the development of PCLX-001 as a first-in-class oral cancer therapy" said Pacylex CEO Dr. Michael Weickert. "Our pre-clinical work showed that PCLX-001 at an equivalent dose in animals regressed R/R NHL tumors so it is exciting to see these patients finally receiving this as an investigational therapy" added Dr. Luc Berthiaume, Pacylex CSO and Professor at the University of Alberta.

Twenty-one patients have received PCLX-001 through 6 dose level escalations with no dose limiting toxicities, therefore dose escalation will continue in patients with advanced solid tumors in parallel with the Phase 2a study in patients with R/R NHL. Pacylex anticipates that it will present an update from the Phase 1 dose escalation portion of the study at a scientific conference this year.

About PCLX-001
PCLX-001 is a first-in-class, oral, small molecule N-myristoyltransferase (NMT) inhibitor being developed to treat patients with leukemia and lymphoma. PCLX-001 selectively kills cancer cells in vitro and have been shown to regress hematologic malignancies and inhibit the growth of lung and breast cancer tumors in animal models. PCLX-001 appears to be particularly effective against Acute Myeloid Leukemia (AML) stem cells in animal disease models.
The Phase 1/2a study (NCT04836195) is an open-label study designed to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, and clinical activity of PCLX-001 in patients with R/R NHL and advanced solid malignancies who have progressed on all available standard therapies.

Mustang Bio Announces Strategic Manufacturing Partnership and Portfolio Updates

On May 18, 2023 Mustang Bio, Inc. ("Mustang" or the "Company") (Nasdaq: MBIO), a clinical-stage biopharmaceutical company focused on translating today’s medical breakthroughs in cell and gene therapies into potential cures for difficult-to-treat cancers and rare genetic diseases, reported a strategic update, including anticipated milestones for 2023 (Press release, Mustang Bio, MAY 18, 2023, View Source [SID1234633166]). Mustang intends to optimize the allocation of its resources and focus on MB-106, MB-109, and in vivo CAR T platform technology. Additionally, Mustang announced a partnership with uBriGene (Boston) Biosciences Inc. ("uBriGene"), the U.S. subsidiary of uBriGene Group, a leading cell and gene therapy contract development and manufacturing organization ("CDMO"), which includes the sale of the Company’s development, manufacturing and analytical testing facility in Worcester, Massachusetts to uBriGene.

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Under the terms of an asset purchase agreement between Mustang and uBriGene, uBriGene will acquire Mustang’s state-of-the-art clinical- and commercial-scale cell and gene therapy manufacturing facility in Worcester, Massachusetts, for a total consideration of $11 million. This consideration includes $6 million payable upfront plus an additional $5 million payable upon Mustang raising $10 million in gross proceeds from equity raises following the closing of the transaction. The closing of the transaction is subject to the satisfaction of certain conditions, including approval of transfer of the Company’s lease to uBriGene by the owner of the building (an affiliate of the University of Massachusetts Chan Medical School) and the acceptance of offers of employment with uBriGene or its affiliates by certain key current Mustang employees. Subject to satisfaction of conditions, the Company expects the transactions to close in June 2023.

Subject to closing, the parties will enter into a manufacturing supply agreement, under which uBriGene will manufacture Mustang’s lead product candidates, including continuing to support MB-106 manufacturing for the ongoing multi-center Phase 1/2 trial.

Mustang’s Worcester facility is a 27,000 square foot, cutting edge cGMP facility supporting process development, manufacturing and analytical testing, designed with the flexibility to expand and support various cell and gene therapy production requirements and capacities. uBriGene intends to expand the Worcester site’s capabilities while leveraging Mustang’s experienced staff and robust quality and operating systems to manufacture a broader portfolio of advanced modalities. uBriGene will also offer their expertise in preclinical research services and late-stage and commercial manufacturing of advanced therapy products with respect to product and process characterization, and regulatory inspections.

Manuel Litchman, M.D., President and Chief Executive Officer of Mustang, commented, "We are very pleased to have found a great partner for the manufacturing of our CAR T cell and gene therapies, and we believe that this strategic partnership with uBriGene will meet our portfolio manufacturing needs to reach critical upcoming data inflection points, while extending our cash runway. I want to thank our manufacturing team for their dedication in building and growing our Worcester facility since it opened in 2018. While we are optimizing our resources at Mustang, we look forward to continuing to work with many of our colleagues in this new capacity, as our CDMO."

"This acquisition is important to uBriGene’s commitment to support the development, clinical, and commercial supply of cell and gene therapies to sustain industry demand and provide new CDMO options," said Alex Chen, President of uBriGene. "We look forward to working together with the University of Massachusetts Chan Medical School and local biotechnology companies to continue to advance the manufacturing ecosystem in the Greater Boston region. This partnership enables us to expand rapidly to create a North American presence and offer the same high-quality cell and gene therapy development and manufacturing capabilities for the U.S. that we currently provide in Asia, including to support Mustang Bio’s lead clinical-stage CAR-T program."

Mustang Bio Strategic Portfolio Updates

CAR T Cell Therapies
After a review of its portfolio of product candidates to determine the future strategy of its programs and the proper allocation of its resources, Mustang will discontinue development of its MB-102, its CD123-targeted CAR T cell therapy, as well as its HER2-, CS1- and PSCA-targeted CAR T cell therapy programs, comprising a portion of the Company’s portfolio of CAR T cell therapies being developed by the Company in partnership with City of Hope.

Mustang will continue to work with Fred Hutchinson Cancer Center ("Fred Hutch") to develop MB-106 (CD20-targeted CAR T cell therapy) and with Mayo Clinic to develop its in vivo CAR T platform technology. Mustang will also continue to work with City of Hope and with Nationwide Children’s Hospital on the development of MB-109 (MB-101 CAR T cell therapy targeting IL13Rα2 on malignant glioma cells + MB-108 oncolytic virus to potentially make these tumors more susceptible to killing by the CAR-T cells).

Gene Therapies
Additionally, based on a review of the data from the investigator-sponsored clinical trials of the gene therapy for X-linked severe combined immunodeficiency ("XSCID") that has been licensed to Mustang Bio, enrollment to these trials has been paused. We await data from new investigator-sponsored trials being planned by our partners that will test a modified version of the current lentiviral vector prior to initiating multicenter Mustang-sponsored trials in both the newborn and previously transplanted patient populations. No safety concerns in the trials utilizing the current vector have been noted to date and no insertional mutagenesis or malignancy has been detected in either of the two investigator-sponsored trials. However, Mustang has made the decision to delay initiating its own sponsored trials out of an abundance of caution, and once we have had the opportunity to review the emerging data from the planned trials utilizing the modified vector, Mustang expects to provide more information on timelines. The delayed start of Mustang’s multicenter trials for XSCID will allow the Company to utilize the safest known vector available in its clinical trials and reduce Mustang’s near-term operating expense.

In addition, in 2023 we look forward to treating a second RAG1-SCID patient with our MB-110 LV-RAG1 ex vivo lentiviral gene therapy in the ongoing investigator-sponsored Phase 1/2 multicenter clinical trial taking place in Europe. Furthermore, we hope to provide an update regarding the research collaboration with Frank J. Staal, Ph.D., from Leiden University to develop additional lentiviral gene therapies.

"Upon completion of a thorough, strategic review of our portfolio of CAR T and gene therapies, it was determined that Mustang’s resources should be focused and allocated to benefit our lead clinical-stage CAR T programs, which could provide potential curative treatment options for certain hematologic cancers and solid tumors, supported by data-to-date. As previously reported, MB-106 continues to demonstrate high efficacy and a favorable safety profile in a Phase 1 investigator-sponsored trial at Fred Hutch, with an overall response rate of 96% and complete response rate of 75% in a wide range of hematologic malignancies, including Waldenstrom macroglobulinemia ("WM"). Given this, Mustang plans to treat patients with WM in the Phase 1 portion of its multicenter MB-106 clinical trial to support a fast-to-market Phase 2 strategy for this indication, with the first pivotal Phase 2 WM patient potentially to be treated in the first quarter of 2024. Data from the Fred Hutch clinical trial also support the potential of MB-106 to be administered as outpatient therapy and provide a best-in-class immunotherapy option for patients treated previously with CD19-directed CAR T cell therapy. We look forward to data from the Fred Hutch clinical trial to be presented at medical meetings in the second quarter of 2023, and initial data from Mustang’s multicenter clinical trial to be disclosed shortly as well. Phase 1 clinical trials of MB-101 at City of Hope and of MB-108 at the University of Alabama at Birmingham continue to enroll patients with recurrent GBM. Additionally, Mustang is excited about advancing the preclinical investigation of these two programs as the combination therapy MB-109 and plans to file an IND for this therapy this year. Concentrating our priorities and postponing the initiation of the MB-107 and MB-207 pivotal trials, along with maintaining a reduced headcount, reduces Mustang’s burn and extends our cash runway. This allows Mustang to allocate more resources to advance our lead clinical-stage programs and potentially expedite the achievement of several near-term milestones across our portfolio of product candidates for difficult-to-treat cancers," said Dr. Litchman.

General Corporate
Mustang expects to incur severance charges related to the facility transaction of approximately $2.1 million, which Mustang expects will be offset by future annualized operating savings of at least $24 million including savings related to personnel, facility and clinical operations and optimization of the development portfolio. The Company also reduced annual interest expense by approximately $4.3 million in April 2023 after repaying and terminating its Loan and Security Agreement with Runway Growth Finance Corp.

BeyondSpring and Leading Cancer Center to Present Poster at 2023 ASCO Annual Meeting

On May 18, 2023 BeyondSpring Inc. (NASDAQ: BYSI) ("BeyondSpring" or the "Company"), a global clinical-stage biopharmaceutical company focused on using a groundbreaking technology platform for drug discovery and developing innovative therapies to improve clinical outcomes for patients with high unmet medical needs, reported that the Company and Memorial Sloan Kettering Cancer Center (MSK) will present a poster at the 2023 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting, taking place on June 2 through 6 in Chicago, IL (Press release, BeyondSpring Pharmaceuticals, MAY 18, 2023, View Source;utm_medium=rss&utm_campaign=beyondspring-and-leading-cancer-center-to-present-poster-at-2023-asco-annual-meeting [SID1234631908]).

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The poster, titled, "​​Plinabulin to shorten neutropenia and improve quality of life peri-autologous hematopoietic cell transplant," will be presented by Dr. Gunjan Shah, MD, MS, Assistant Attending Physician at MSKCC and Principal Investigator of the BeyondSpring-MSKCC pilot trial evaluating Plinabulin in combination with pegfilgrastim in multiple myeloma patients who undergo an autologous hematopoietic cell transplant (AHCT).

Additional presentation details are as follows:

Poster Session Date / Time: Monday, June 5, 2023, at 9 a.m. to 12 p.m. ET
Location: McCormick Place Convention Center, Hall A
Track: Hematologic Malignancies
Abstract / Poster Board No.: 8023 / 15

Legend Biotech Reports First Quarter 2023 Results and Recent Highlights

On May 18, 2023 Legend Biotech Corporation (NASDAQ: LEGN) (Legend Biotech), a global biotechnology company developing, manufacturing and commercializing novel therapies to treat life-threatening diseases, reported its first quarter 2023 unaudited financial results (Press release, Legend Biotech, MAY 18, 2023, View Source [SID1234631875]).

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"We are extremely pleased to announce that we have recently raised $762 million in funding. With this substantial capital infusion, we are poised to embark on a critical chapter in our company’s growth to advance CARVYKTI toward its full potential, and we look forward to presenting the latest data from our CARTITUDE clinical development programs at ASCO (Free ASCO Whitepaper) and EHA (Free EHA Whitepaper) this June," said Ying Huang, Chief Executive Officer of Legend Biotech. Dr. Huang continued, "We would like to extend our deepest gratitude to our investors for their overwhelming support and confidence in our company’s mission and remain committed to creating long-term value for all of our stakeholders."

Financial Results for Quarter Ended March 31, 2023

Cash and Cash Equivalents, Time Deposits, and Short-Term Investments
As of March 31, 2023, prior to giving effect to the registered direct offering, private placements or warrant exercise noted above, Legend Biotech had approximately $854 million of cash and cash equivalents, time deposits, and short-term investments.

Revenue
Total revenue for the three months ended March 31, 2023 was $36.3 million compared to $50.0 million for the three months ended March 31, 2022. Collaboration revenue recognized in the first quarter of 2023 was from CARVYKTI sales primarily in the U.S. License revenue recognized in first quarter of 2022 was due to the achievement of commercial milestone for FDA approval in the U.S. in connection with the license and collaboration agreement (the "Janssen Agreement") with Janssen Biotech, Inc. ("Janssen").

Collaboration Cost of Revenue
Collaboration cost of revenue for the three months ended March 31, 2023 was $35.6 million. Legend Biotech did not have any collaboration cost of revenue in the three months ended March 31, 2022. The $35.6 million is a combination of Legend’s portion of collaboration cost of sales in connection with collaboration revenue under the Janssen Agreement along with expenditures to support the manufacturing capacity expansion which cannot be capitalized.

Research and Development Expenses
Research and development expenses for the three months ended March 31, 2023 were $84.9 million compared to $81.5 million for the three months ended March 31, 2022. This increase of $3.4 million was primarily due to higher patient enrollment for Phase 3 clinical trials for cilta-cel in the first quarter of 2023.

Administrative Expenses
Administrative expenses for the three months ended March 31, 2023 were $22.2 million compared to $12.7 million for the three months ended March 31, 2022. The increase of $9.5 million was primarily due to continued investment in building global information technology infrastructure along with non-recurring financial and legal fees related to Legend Biotech’s restatement of its historical financial statements as reported by Legend Biotech in February 2023.

Selling and Distribution Expenses
Selling and distribution expenses for the three months ended March 31, 2023 were $18.0 million compared to $21.3 million for the year three months ended March 31, 2022. This decrease of $3.3 million was primarily due to non-recurring launch expenses incurred in the first quarter of 2022 to support the commercialization in the U.S market.

Other Income and Gains
Other income and gains for the three months ended March 31, 2023 were $8.2 million compared to $1.0 million for the three months ended March 31, 2022. The increase of $7.2 million was primarily due to increase in interest income and gain on investments.

Other Expenses
Other expenses for the three months ended March 31, 2023 were $10.7 million compared to $1.5 million for the three months ended March 31, 2022. The increase was primarily due to unrealized foreign currency exchange loss in the quarter.

Finance Costs
Finance costs for the three months ended March 31, 2023 were $5.1 million compared to $1.0 million for the three months ended March 31, 2022. The increase was primarily due to interest on advance funding, which is interest-bearing borrowings funded by Janssen under the Janssen Agreement and constituted by principal and applicable interests upon such principal.

Fair Value Gain of Warrant Liability
Fair value gain of warrant liability for the three months ended March 31, 2023 was $20.0 million caused by changes in the fair value of a warrant that Legend Biotech issued to an institutional investor through a private placement transaction in May 2021 with an initial fair value of $81.7 million at the issuance date. The warrant was assessed as a financial liability with a fair value of $47.0 million as of March 31, 2023. On May 12, 2023, Legend Biotech announced that the warrant had been exercised in full.

Loss for the Period
For the three months ended March 31, 2023, net loss was $112.1 million, or $0.34 per share, compared to a net loss of $32.3 million, or $0.10 per share, for the three months ended March 31, 2022.