Mustang Bio Announces Initial Phase 1 Data on MB-105 for Patients with PSCA-positive Castration Resistant Prostate Cancer

Data presented by City of Hope at 27th Annual Prostate Cancer Foundation Scientific Retreat

WORCESTER, Mass., Oct. 26, 2020 (GLOBE NEWSWIRE) — 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, today announced that one patient‘s experience on the Phase 1 trial of MB-105, a prostate stem cell antigen (PSCA) chimeric antigen receptor (CAR) T administered systemically to patients with PSCA-positive metastatic castration-resistant prostate cancer (mCRPC), was presented at the virtual 27th Annual Prostate Cancer Foundation Scientific Retreat.

Tanya Dorff, M.D., City of Hope Associate Clinical Professor, Department of Medical Oncology & Experimental Therapeutics and Head of its Genitourinary Cancer Program and the trial’s principal investigator, presented a description of the correlative science from the ongoing Phase 1 open-label clinical trial of MB-105, one of the first CAR T trials for prostate cancer in the nation. In a 73-year-old male patient with PSCA-positive mCRPC who was treated with MB-105 and lymphodepletion (a standard CAR T pre-conditioning regimen) after failing eight prior therapies, MB-105 demonstrated on day 28 a 94 percent reduction in prostate-specific antigen (PSA), near complete reduction of measurable soft tissue metastasis by computerized tomography, and improvement in bone metastases by magnetic resonance imaging. The therapy was associated with cytokine release syndrome, which was clinically managed with tocilizumab (anti-IL-6 receptor antibody), and hemorrhagic cystitis requiring transfusion which clinically resolved in 30 days.

Manuel Litchman, M.D., President and Chief Executive Officer of Mustang, said, “We are encouraged by the initial data presented by City of Hope from the ongoing Phase 1 trial of Mustang’s CAR T cell therapy MB-105. We see potential for this PSCA-targeted CAR T in the treatment of prostate cancer, as well as other difficult-to-treat solid tumor cancers. We look forward to the continued progression of this trial and anticipate providing further data in the second half of 2021.”

According to the American Cancer Society (ACS), prostate cancer is the most common cancer in American men, excluding skin cancer. ACS estimates 191,930 new cases of prostate cancer in the U.S. will be diagnosed this year, and roughly one out of every nine men will be diagnosed with prostate cancer during his lifetime. The median survival for men with CRPC is less than two years, according to the American Urological Association.

About MB-105 (PSCA CAR T technology)
MB-105 was developed in the laboratory of Saul Priceman, Ph.D., assistant professor in City of Hope’s Department of Hematology & Hematopoietic Cell Transplantation and associate director of translational sciences in the T Cell Therapeutics Research Laboratory led by Stephen Forman, M.D., leader of City of Hope’s Hematologic Malignancies and Stem Cell Transplantation Institute and the laboratory’s director.

The Phase 1 clinical trial of MB-105 will continue to enroll up to 33 patients. Its primary endpoints are to define safety and optimal dosing of PSCA CAR T cells in treating patients with PSCA-positive mCRPC. Secondary

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Bayer to acquire Asklepios Bio in foray into gene therapy worth up to $4 billion

FRANKFURT (Reuters) – Bayer BAYGn.DE agreed to acquire unlisted U.S. biotech firm Asklepios BioPharmaceutical Inc for as much as $4 billion in a bet on gene therapy with the help of modified viruses.

FILE PHOTO: A bridge is decorated with the logo of a Bayer AG, a German pharmaceutical and chemical maker in Wuppertal, Germany August 9, 2019. REUTERS/Wolfgang Rattay/File Photo

Germany’s Bayer will pay $2 billion upfront and up to an additional $2 billion in milestone payments contingent on development achievements, it said on Monday.

The North Carolina-based takeover target, also known as AskBio, is trying to use the harmless adeno-associated virus as a delivery device to bring genetic repair kits against a range of diseases into the body.Drugs and farming pesticides maker Bayer needs to upgrade its drug development pipeline amid a weaker outlook for agricultural sales and as it seeks to finalise an $11 billion settlement over claims its Roundup weedkiller causes cancer.

Among AskBio’s most advanced projects are early tests on volunteers of prospective treatments against Pompe disease – a rare genetic disease causing buildup of a sugar molecule inside cells – as well as against Parkinson’s disease and congestive heart failure.

Bayer said the deal complements the 2019 acquisition of BlueRock Therapeutics, a developer of stem cell therapies, and underscores Bayer’s intention to create a cell and gene therapy business.

AskBio, which was founded in 2001, and BlueRock will exchange information and collaborate but will each operate as independent entities, prompting a pledge from AskBio’s five main owners, who are co-founders or key scientists, to remain with the firm.

“We are staying on board because of the unique structure that Bayer has provided … We’ll have the ability to make our science decisions,” said Chief Executive Officer and co-founder Sheila Mikhail.

Investors TPG Capital and Vida Ventures are selling a minority stake in the company.

AskBio is also helping other companies with their gene therapy research and production and has licensed experimental drugs to external partners, which has financed much of its own drug development activities. Bayer or AskBio would not provide figures for such fee revenues.

A potential treatment of Duchenne Muscular Dystrophy, invented by AskBio is currently being developed in clinical trials by Pfizer PFE.N and this month won fast track here status from U.S. regulators.

Bayer in 2018 moved to lean more strongly on external firms to improve drug development, which analysts say needs a boost to make up for an expected decline in revenues from its two pharma bestsellers from about 2024.

Credit Suisse was financial advisor while Baker McKenzie was legal counsel to Bayer. JP Morgan was financial advisor to AskBio, while Ropes & Gray was legal counsel.

Reporting by Ludwig Burger; Editing by Frances Kerry

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Goldfinch Bio Presents Clinical Data from Phase 1 Trial Supporting Advancement of GFB-887 as a Precision Medicine for Patients with Kidney Diseases

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Goldfinch Bio, a clinical stage biotechnology company focused on discovering and developing precision medicines for the treatment of kidney diseases, today announced for the first time results from its Phase 1 clinical trial evaluating GFB-887, a first-in-class highly potent and selective inhibitor of Transient Receptor Potential Canonical Channel 5 (TRPC5), in healthy volunteers. The data are being presented today at the virtual American Society of Nephrology (ASN) Kidney Week 2020 Annual Meeting.

Goldfinch Bio is developing GFB-887 as a precision medicine for patients with kidney diseases characterized by overactivation of the TRPC5-Rac1 pathway, including focal segmental glomerulosclerosis (FSGS) and diabetic nephropathy (DN). Overactivation of the TRPC5-Rac1 pathway leads to injury of podocytes, which are cells lining the kidney that, in their healthy state, prevent essential protein loss (proteinuria). Injury to podocytes causes podocyte loss, proteinuria and, eventually, kidney failure. TRPC5-Rac1 pathway overactivation is the key cause of disease in a substantial portion of FSGS and DN patients, and there are currently no approved drugs that specifically target the TRPC5-Rac1 pathway in these diseases.

“We are excited to share these first-in-human data, which demonstrate that GFB-887 is well-tolerated and suggest a dose-dependent reduction in urinary Rac1, confirming GFB-887 target engagement in the podocyte,” said Anthony Johnson, M.D., President and Chief Executive Officer of Goldfinch Bio. “Suppressing the TRPC5-Rac1 pathway has the potential to deliver clinically meaningful benefit to patients by reducing proteinuria and, as a result, preserving native kidney function. Supported by the Phase 1 data, we are now underway with our Phase 2 TRACTION-2 study of GFB-887 in FSGS and DN, as we continue to advance our mission of protecting patients from the inevitability of dialysis and kidney transplant by delivering precision medicines for subsets of kidney disease.”

Data from the Phase 1 Clinical Trial

The primary objective of the randomized, double-blinded, placebo-controlled trial was to assess the safety, tolerability, pharmacokinetic (PK) profile and pharmacodynamics (PD) of GFB-887 in healthy volunteers. A key exploratory objective was to characterize changes in urinary Rac1. Urinary Rac1 concentration may predict therapeutic response to TRPC5 inhibition.

The study enrolled 70 subjects, who were randomized four to one to receive GFB-887 at seven dose levels (ranging from 5 mg to 900 mg) or placebo.

Primary Objective: Safety, Tolerability and PK Data

GFB-887 was observed to be well-tolerated at all doses. There were no dose-limiting toxicities, severe adverse events (AEs) or abnormalities in laboratory or clinical assessments. In total, 38 percent of subjects who received GFB-887 reported AEs, compared to 21 percent of subjects treated with placebo. GFB-887-treated subjects reported headache more frequently than placebo-treated subjects (28.6% versus 7.2% respectively). The only other AE that was reported in two or more GFB-887-treated subjects was nausea (3.6%). Slight, asymptomatic reductions in blood pressure were observed in subjects treated at the highest doses.

The PK profile of GFB-887 is consistent with once-daily dosing, with a half-life ranging from 55 to 68 hours. A single 40 mg dose was also shown to exceed the preclinical

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