Mustang Bio Reports First Quarter 2021 Financial Results and Recent Corporate Highlights

On May 14, 2021 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 financial results and recent corporate highlights for the first quarter ended March 31, 2021 (Press release, Mustang Bio, MAY 14, 2021, View Source [SID1234580016]).

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Manuel Litchman, M.D., President and Chief Executive Officer of Mustang, said, "In the first quarter of 2021, we successfully executed our clinical and regulatory strategies and achieved milestones that we believe will drive Mustang’s continued progress. We announced encouraging MB-107 and MB-207 clinical updates from our investigator-IND trials for X-Linked Severe Combined Immunodeficiency ("XSCID"), as well as additional consistent safety and efficacy data pertaining to our lentiviral vector gene therapies that we have seen over the more than eight years since the first patient was treated in 2012. In January, the U.S. Food and Drug Administration ("FDA") lifted a Chemistry, Manufacturing and Controls ("CMC") hold on the MB-107 Investigational New Drug ("IND") application for newly diagnosed infants under the age of two and we expect to dose the first infant in the multicenter trial soon. We also plan to file an IND for our MB-207 clinical trial for patients with XSCID who have been previously treated with hematopoietic stem cell transplantation ("HSCT") and for whom re-treatment is indicated."

Dr. Litchman continued, "Notably, we are thrilled with the progress of our MB-106 CD20-targeted CAR T cell therapy program for relapsed or refractory CD20+ B-cell non-Hodgkin lymphomas ("B-NHL") and chronic lymphocytic leukemia ("CLL"). Earlier this month, we announced that the FDA approved Mustang’s IND to initiate a multicenter Phase 1/2 clinical trial investigating the safety and efficacy of MB-106 for relapsed or refractory CD20+ B-NHL and CLL. Also, we look forward to the Fred Hutchinson Cancer Research Center presentation at the European Hematology Association (EHA) (Free EHA Whitepaper) 2021 ("EHA2021") Virtual Congress next month, where Dr. Mazyar Shadman will present updated interim data from their ongoing CD20-targeted CAR T clinical trial. We are very pleased to have three active Mustang-sponsored INDs, and we anticipate having four active Mustang-sponsored INDs by the end of the year."

Financial Results:

As of March 31, 2021, Mustang’s cash and cash equivalents and restricted cash totaled $130.4 million, compared to $98.8 million as of December 31, 2020, an increase of $31.6 million year-to-date.
Research and development expenses including license acquisitions were $11.6 million for the first quarter of 2021, compared to $9.6 million for the first quarter of 2020. Non-cash, stock-based expenses included in research and development were $0.7 million for the first quarter of 2021, compared to $0.4 million for the first quarter of 2020.
General and administrative expenses were $3.5 million for the first quarter of 2021, compared to $2.0 million for the first quarter of 2020. Non-cash, stock-based expenses included in general and administrative expenses were $1.5 million for the first quarter of 2021, compared to $0.5 million for the first quarter of 2020.
Net loss attributable to common stockholders was $15.0 million, or $0.19 per share, for the first quarter of 2021, compared to a net loss attributable to common stockholders of $11.9 million, or $0.28 per share, for the first quarter of 2020.
Recent Corporate Highlights:

In February 2021, Mustang announced encouraging MB-107 and MB-207 clinical updates from its investigator-IND XSCID trials, as well as additional consistent safety and efficacy data. On January 28, 2021, the FDA removed a CMC hold on the MB-107 Phase 2 clinical trial IND after reviewing a comprehensive CMC package that was submitted by Mustang in late December 2020. The company expects to enroll the first patient in this pivotal multicenter trial in the second quarter of 2021 and is targeting topline data from the trial in the second half of 2022. The company also expects to file an IND in the second quarter of 2021 for its pivotal multicenter Phase 2 clinical trial of MB-207.
In May 2021, Mustang announced that the FDA approved its IND application to initiate a multicenter Phase 1/2 clinical trial investigating the safety and efficacy of MB-106, a CD20-targeted CAR T therapy for high-risk B-NHL and CLL.
Also in May 2021, Mustang announced that CD20-targeted CAR T therapy data were selected for presentation at the EHA (Free EHA Whitepaper)2021 Virtual Congress scheduled to take place in June. Dr. Mazyar Shadman of Fred Hutch will present updated interim data from the ongoing Phase 1/2 clinical trial for B-NHL and CLL. A copy of the abstract can be viewed online through the EHA (Free EHA Whitepaper)2021 website here.

Odonate Therapeutics Announces Financial Results for the Three Months Ended March 31, 2021

On May 14, 2021 Odonate Therapeutics, Inc. (NASDAQ: ODT) reported financial results for the three months ended March 31, 2021 (Press release, Odonate Therapeutics, MAY 14, 2021, View Source [SID1234580048]).

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Odonate recently announced the discontinuation of development of tesetaxel and its intent to wind down tesetaxel-related operations. Odonate is working with clinical sites to transition patients in ongoing tesetaxel studies to appropriate alternative therapies or facilitate continuation of treatment with tesetaxel under compassionate use programs where appropriate.

As of March 31, 2021, Odonate had $133.2 million in cash compared to $157.3 million as of December 31, 2020. This decrease in cash resulted primarily from cash used in operating activities for the three months ended March 31, 2021 of $24.9 million. Odonate’s net loss for the three months ended March 31, 2021 and 2020 was $33.8 million and $30.2 million, or $0.90 and $0.99 per share, respectively.

Japan’s Ministry of Health, Labour and Welfare Grants Priority Review for Enfortumab Vedotin New Drug Application

On March 14, 2021 Astellas Pharma Inc. (TSE: 4503, President and CEO: Kenji Yasukawa, Ph.D., "Astellas") reported Japan’s Ministry of Health, Labour and Welfare (MHLW) has granted priority review for the company’s New Drug Application (NDA), which was submitted in March (Press release, Astellas, MAY 14, 2021, View Source [SID1234579945]). If approved, enfortumab vedotin would be the first antibody-drug conjugate (ADC) available in Japan for the treatment of patients with locally advanced or metastatic urothelial cancer that has progressed after anti-cancer medication.

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Priority reviews are granted by MHLW for applications based on their clinical usefulness and the seriousness of the diseases for which they are indicated.1 The NDA includes data from the phase 3 EV-301 trial and the phase 2 EV-201 trial, both global clinical trials with investigational sites in Japan.

"The decision by the Ministry of Health, Labour and Welfare to evaluate enfortumab vedotin under priority review reflects the urgent need for new medicines to treat advanced urothelial cancer in Japan, where an estimated 9,500 people die from urothelial cancer each year," said Andrew Krivoshik, M.D., Ph.D., Senior Vice President and Oncology Therapeutic Area Head, Astellas.2

Urothelial cancer makes up approximately 90 percent of cases of bladder cancer.3 Locally advanced or metastatic urothelial cancer is an aggressive disease that is associated with poor survival and high healthcare costs.4

About the EV-301 Trial
The EV-301 trial (NCT03474107) is a global, multicenter, open-label, randomized phase 3 trial designed to evaluate enfortumab vedotin versus physician’s choice of chemotherapy (docetaxel, paclitaxel or vinflunine) in approximately 600 patients with locally advanced or metastatic urothelial cancer who were previously treated with a PD-1/L1 inhibitor and platinum-based therapy.5 The primary endpoint is overall survival and secondary endpoints include progression-free survival, overall response rate, duration of response and disease control rate, as well as assessment of safety/tolerability and quality-of-life parameters.

About the EV-201 Trial
The EV-201 trial (NCT03219333) is a single-arm, dual-cohort, pivotal phase 2 clinical trial of enfortumab vedotin for patients with locally advanced or metastatic urothelial cancer who have been previously treated with a PD-1 or PD-L1 inhibitor, including those who have also been treated with a platinum-containing chemotherapy (cohort 1) and those who have not received a platinum-containing chemotherapy in this setting and who are ineligible for cisplatin (cohort 2). The trial enrolled 128 patients in cohort 1 and 91 patients in cohort 2 at multiple centers internationally.6 The primary endpoint is confirmed objective response rate per blinded independent central review. Secondary endpoints include assessments of duration of response, disease control rate, progression-free survival, overall survival, safety and tolerability.

About Enfortumab Vedotin
Enfortumab vedotin is an antibody-drug conjugate (ADC) that is directed against Nectin-4, a protein located on the surface of cells and highly expressed in bladder cancer.7,8 Nonclinical data suggest the anticancer activity of enfortumab vedotin is due to its binding to Nectin-4 expressing cells followed by the internalization and release of the anti-tumor agent monomethyl auristatin E (MMAE) into the cell, which result in the cell not reproducing (cell cycle arrest) and in programmed cell death (apoptosis).7

About the Astellas and Seagen Collaboration
Astellas and Seagen Inc. are co-developing enfortumab vedotin under a 50:50 worldwide development and commercialization collaboration. In the United States, Astellas and Seagen co-promote enfortumab vedotin under the brand name PADCEV (enfortumab vedotin-ejfv). In the Americas outside the US, Seagen holds responsibility for commercialization activities and regulatory filings. Outside of the Americas, Astellas holds responsibility for commercialization activities and regulatory filings.

Aurinia Pharmaceuticals to Participate in Two Upcoming Investor Conferences

On May 14, 2021 Aurinia Pharmaceuticals Inc. (NASDAQ: AUPH / TSX: AUP) (the "Company") reported that members of the executive management team will participate in two upcoming investor conferences (Press release, Aurinia Pharmaceuticals, MAY 14, 2021, View Source [SID1234580001]):

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RBC Capital Markets Global Healthcare Conference on Wednesday, May 19, 2021 at 10:55 a.m. ET; and
Oppenheimer Rare & Orphan Disease Summit on Friday, May 21, 2021 at 10:45 a.m. ET.
In order to participate in the audio webcast, interested parties can access the live webcast under "News/Events" through the "Investors" section of the Aurinia corporate website at www.auriniapharma.com. A replay of the webcast will be available on Aurinia’s website.

NeuBase Therapeutics to Participate in Upcoming Investor Conferences in May 2021

On May 14, 2021 NeuBase Therapeutics, Inc. (Nasdaq: NBSE) ("NeuBase"), a biotechnology company accelerating the genetic revolution with a new class of precision genetic medicines, reported that Dietrich A. Stephan, Ph.D., Chief Executive Officer of NeuBase, will participate in a virtual fireside chat at the RBC Capital Markets Virtual Global Healthcare Conference, as well as present a corporate overview and business update at the Oppenheimer Rare & Orphan Disease Summit, which are both being held in May 2021 (Press release, NeuBase Therapeutics, MAY 14, 2021, View Source [SID1234580017]).

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Conferences Details:

Event: RBC Capital Markets Virtual Global Healthcare Conference
Format: Fireside Chat
Date: Wednesday, May 19th
Time: 11:30 a.m. ET
Location: Webcast Link – or at the company’s website (click here)

Event: Oppenheimer Rare & Orphan Disease Summit
Format: Presentation
Date: Friday, May 21st
Location: Webcast Link – or at the company’s website (click here)