On May 12, 2020 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 two poster presentations at the virtual 23rd Annual Meeting of the American Society of Gene & Cell Therapy ("ASGCT"), being held May 12-15, 2020 (Press release, Mustang Bio, MAY 12, 2020, View Source [SID1234557612]).

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Manuel Litchman, M.D., President and Chief Executive Officer of Mustang, said, "We are extremely pleased with the strides forward that our researchers have made in gaining greater insights into our innovative CS1 chimeric antigen receptor (CAR) T cell therapy (MB-104), which we previously licensed from City of Hope. We commend them on their poster presentations at ASGCT (Free ASGCT Whitepaper) and look forward to learning more as they continue their research to optimize our clinical trials."

Details on the poster presentations are as follows:

Title: CS1 Targeted CAR-T Cells (MB-104) for the Treatment of Multiple Myeloma Shows Antitumor Activity Sparing Normal T-Cells Despite the Common Expression of CS1
Session: Cell Therapies
Abstract number: 421
Date and Time: Tuesday, May 12, 2020, 5:30 PM-6:30 PM ET
Room: Exhibit Hall C & D
Authors: Nathan Gumlaw, Aviva Joseph, James Edinger, Ekta Patel, Research and Translational Sciences, Mustang Bio, Worcester, MA

This poster describes researchers’ investigation into the impact of MB-104 on CS1 positive and negative cells in vitro, as well as T cells due to shared CS1 antigen expansion. The researchers demonstrated MB-104 does not confer biologically significant fratricide and can be successfully manufactured as evident by viability, growth kinetics and fold expansion, despite the shared antigen expression between tumor cells and T cells. CS1 positive T cells are present in culture during the expansion of MB-104, suggesting absence of fratricide. Finally, MB-104 can induce potent anti-tumor cell lysis and proliferates in response to tumor cells but not primary T cells expressing CS1. Taken together, their results demonstrate MB-104 is a novel CS1-targeting CAR T that shows potent anti-tumor cell lysis but spares normal T cells, despite the shared CS1 antigen expression.

Title: Development of an Immunohistochemistry Assay for the Detection of CS-1 Expression in Multiple Myeloma Patients
Session: Pharmacology/Toxicology Studies or Assay Development
Abstract number: 897
Date and Time: Wednesday, May 13, 2020, 5:30 PM-6:30 PM ET
Room: Exhibit Hall C & D
Authors: Bethany Biron Girard, James Edinger, Ekta Patel, Translational Sciences, Mustang Bio, Worcester, MA

This poster details a study in which researchers evaluated commercially available CS1 antibodies for IHC and identified the best clone with high specificity for CS1 to improve screening subjects for CS1 positive tumor expression prior to treatment and correlate efficacy with antigen expression. The researchers, for the first time, developed and optimized a robust immunohistochemistry assay for the assessment of CS1 expression in bone marrow core biopsy samples and plasmacytoma solid tumor samples from multiple myeloma ("MM") patients, which can be used for enrollment into Mustang’s CS1 CAR T clinical trials.

For more information, including abstracts, please visit the ASGCT (Free ASGCT Whitepaper) meeting website at View Source

On May 12, 2020 China Biologic Products Holdings, Inc. (NASDAQ: CBPO) ("China Biologic" or the "Company"), a leading fully integrated plasma-based biopharmaceutical company in China, reported that the Company plans to release its first quarter 2020 financial results on Wednesday, May 20, 2020 after the market closes (Press release, China Biologic Products, MAY 12, 2020, View Source [SID1234557628]).

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The Company’s management will hold a conference call at 7:30 a.m. ET on Thursday, May 21, 2020, which is 7:30 p.m. Beijing Time on May 21, 2020, to discuss first quarter 2020 results. Listeners may access the call by dialing:

A live and archived webcast of the conference call will be available through the Company’s investor relations website at View Source

On May 11, 2020 Infinity Pharmaceuticals, Inc. (NASDAQ: INFI) reported its first quarter 2020 financial results and provided an update on the Company, including its first quarter progress with IPI-549, the Company’s first-in-class, oral immuno-oncology product candidate targeting immune-suppressive tumor-associated myeloid cells through selective phosphoinositide-3-kinase-gamma (PI3K-gamma) inhibition (Press release, Infinity Pharmaceuticals, MAY 11, 2020, View Source [SID1234557456]).

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"While COVID-19 has created challenges for Infinity and the broader life sciences community, it has also highlighted the dedication, commitment and innovation that is allowing the continued advancement of desperately needed treatments for patients," said Adelene Perkins, Chief Executive Officer and Chair of Infinity Pharmaceuticals. "Infinity remains committed to our clinical strategy which is designed to demonstrate the potential benefit of IPI-549 treatment for the patients in our trials who have some of the most challenging cancers and poor prognoses. It is imperative that these patients have continued access to treatment and that we advance the development of potentially promising new therapies. I am particularly proud of our team at Infinity, along with our collaborators and investigators, for prioritizing the safety of our patients while still enabling our progress in the clinic to continue. To this end, following an initial meeting with the MARIO-275 Independent Data Monitoring Committee, we are voluntarily pausing enrollment of patients in MARIO-275 to ensure patient safety while evaluating the potential benefit of IPI-549 plus Opdivo in patients with advanced urothelial cancer. The magnitude of the unmet need and the potential of IPI-549 plus Opdivo to improve outcomes for these patients is reflected in the Fast Track Designation we recently received for this combination in this patient population. Across all our studies, we are working closely with our clinical research organizations to leverage learnings from early patient enrollment and the impact of COVID-19 to address delays in new site initiation and enrollment. While the potential duration of delays in the enrollment of our trials are still being quantified, we anticipate the need for additional financial resources to fund our trials through key data readouts. We expect to provide additional updates on trial status including timelines for enrollment and data readouts as well as financial guidance by our 2Q webcast."

Key 2020 Updates:

Clinical and Regulatory:

Fast Track Designation: Infinity received Fast Track Designation from the U.S. FDA for IPI-549 in combination with the checkpoint inhibitor Opdivo for the treatment of advanced urothelial cancer.
MARIO-275: MARIO-275 is the Company’s ongoing controlled, randomized Phase 2 study evaluating IPI-549 in combination with Opdivo in platinum-refractory, I/O naïve patients with (aUC), in collaboration with Bristol Myers Squibb, or BMS. The objective of this controlled study is to evaluate the potential benefit of adding IPI-549 to Opdivo monotherapy, building on the exploratory biomarker data from the BMS CheckMate-275 study demonstrating an association between high baseline levels of myeloid-derived suppressor cells, or MDSCs, and lower median overall survival as well as Infinity’s MARIO-1 study showing that treatment with IPI-549 reduced MDSC levels in the majority of patients treated.

Independent Data Monitoring Committee (IDMC): Infinity and the MARIO-275 IDMC held the first scheduled IDMC meeting to review safety data on the initial 42 patients treated in the study. Liver enzyme elevations of Grade 3 or higher were seen in seven of 42 patients: five with Grade 3, two with Grade 4 (one is newly observed and has not yet been reviewed by the IDMC). Liver enzyme elevations from the six patients reviewed by the IDMC were reversible and have resolved without sequela. Of 17 patients evaluable for efficacy, objective responses have been observed in patients with Grade 3 and above liver enzyme elevations as well as in patients without these elevations. Infinity is continuing to review this data with the IDMC and plans to continue the treatment of patients now on study with modifications including additional patient monitoring and a dose reduction while voluntarily pausing enrollment and amending the protocol in order to ensure patient safety while evaluating the potential benefit of IPI-549 plus Opdivo.
MARIO-3: MARIO-3 is the Company’s ongoing Phase 2 study in collaboration with Roche/Genentech to evaluate IPI-549 in novel triple combination front-line therapies with Tecentriqand Abraxane in triple negative breast cancer (TNBC) and with Tecentriq and Avastin in renal cell cancer (RCC). The TNBC cohort of MARIO-3 is experiencing initial enrollment and site initiation delays, independent of a COVID-19 impact, which the Company is working closely with its CRO and investigators to evaluate.
MARIO-1: Additional data are expected in 2020 to the extent that they inform potential future IPI-549 development paths.
COVID-19: The Company is continuing to evaluate early enrollment trends in its studies as well as the impact of COVID-19 on its clinical programs. Patients enrolled on MARIO-275, MARIO-3 and MARIO-1 have continued treatment and study visits with limited disruption to date, and Infinity is working closely with trial sites to support the continued treatment of patients in compliance with study protocols. The pandemic is impacting new patient enrollment and site initiation across the Company’s clinical programs. New patient screening and enrollment and new site initiation are being assessed on a case-by-case basis and are ongoing in MARIO-3. There are no anticipated disruptions to drug supply.
Arcus Collaboration: A Phase 1b collaboration study with Arcus Biosciences, which is being conducted by Arcus, is evaluating a checkpoint-inhibitor free, novel triple-combination regimen of IPI-549 + AB928 (dual adenosine receptor antagonist) + Doxil in up to approximately 40 advanced TNBC patients.
Financial:

In January 2020, completed a $20 million asset-backed financing with BVF Partners L.P. with sole recourse in potential royalty payments due on future sales of patidegib, a hedgehog pathway inhibitor discovered by Infinity and licensed to PellePharm in 2013. In addition, Infinity is eligible to receive an additional $5 million payment from BVF based on PellePharm’s ongoing Phase 3 clinical trial of patidegib topical gel in Gorlin Syndrome. PellePharm completed enrollment of its Phase 3 trial in 2019.
Corporate:

Appointed Richard Gaynor, M.D. to Board of Directors. Richard Gaynor, M.D. was most recently the President of Research and Development for Neon Therapeutics (NasdaqGS: NTGN), a biotechnology company developing novel neoantigen-targeted T cell therapies. Prior to his tenure at Neon Therapeutics, Dr. Gaynor spent 15 years in a series of senior roles at Lilly Oncology, most recently as Senior Vice President Clinical Development and Medical Affairs.
Established Clinical Advisory Board with the following initial members:
Chair: Sam Agresta, MD, Infinity Board Member and Chief Medical Officer of Foghorn Therapeutics.
Padmanee (Pam) Sharma, MD, PhD, co-leader of the MD Anderson Cancer Center’s immunotherapy platform and T.C. and Jeanette Hsu Endowed Chair in Cell Biology, Department of Genitourinary Medical Oncology. Dr. Sharma was the principal investigator on the BMS Checkmate-275 study that first demonstrated the association of high myeloid-derived suppressor cells, or MDSCs, to significantly lower overall survival in patients with advanced urothelial cancer (aUC) that provided the inspiration for Infinity’s MARIO-275 study to which Dr Sharma brings unique insight.
Toni Choueiri, MD, Director of the Lank Center for Genitourinary Oncology at Dana-Farber Cancer Institute/Brigham and Women’s Hospital and the Jerome and Nancy Kohlberg Chair and Professor of Medicine at Harvard Medical School.
Michael Postow, MD, co-leader of the Melanoma Disease Management Team at Memorial Sloan Kettering Cancer Center and assistant professor of medicine at Weill Cornell Medical College.
Updated Program and Financial Guidance:

Given that the Company is experiencing delays in MARIO-3 enrollment and site initiation and is voluntarily pausing enrollment in MARIO-275 while amending the MARIO-275 protocol to ensure patient safety, Infinity expects to provide additional updates on trial status including timelines for enrollment and data readouts as well as financial guidance by our 2Q webcast.
As a result of the delays in enrollment in its MARIO-3 and MARIO-275 clinical trials, Infinity anticipates that its existing financial resources will not be sufficient to fully fund the Company through key data readouts on all of its IPI-549 trials, and Infinity will need to find cost savings and/or seek additional capital resources in order to fund studies through key data readouts.
First Quarter 2020 Financial Results:

At March 31, 2020, Infinity had total cash, cash equivalents and available-for-sale securities of $50.3 million, compared to $42.4 million at December 31, 2019.
During the first quarter of 2020, Infinity recognized the $20 million gross proceeds from BVF, net of transaction costs, as a liability that will be amortized using the effective interest method over the life of the arrangement, in accordance with relevant accounting guidance. While recognized as a liability for accounting purposes, the Company is not obligated to repay the $20 million from BVF.
Research and development expense for the first quarter of 2020 was $7.3 million, compared to $5.8 million for the same period in 2019. The increase in R&D expense in 2020 compared to 2019 was primarily due to an increase in clinical and development activities for IPI-549.
General and administrative expense was $3.3 million for the first quarter of 2020, compared to $3.4 million for the same period in 2019.
Net loss for the first quarter of 2020 was $10.9 million, or a basic and diluted loss per common share of $0.19, compared to a net loss of $13.7 million, or a basic and diluted loss per common share of $0.24 for the same period in 2019.
2020 Financial Outlook:

Net Loss: Infinity expects net loss for 2020 to range from $40 million to $50 million.

Cash and Investments: Infinity expects to end 2020 with a year-end cash, cash equivalents and available-for-sale securities balance ranging from $15 million to $25 million.

Cash Runway: Based on its current operational plans, Infinity expects that its existing cash, cash equivalents and available-for-sale securities, will be adequate to satisfy the Company’s capital needs into 2H2021. Infinity’s financial guidance does not include potential additional funding or business development activities, a potential $5 million milestone payment from BVF based on PellePharm’s ongoing Phase 3 clinical trial of patidegib topical gel in Gorlin Syndrome, or any milestones from, or the sale of the Company’s equity interest in, PellePharm.

Conference Call Information

Infinity will host a conference call today, May 11, 2020, at 4:30 p.m. ET to discuss these financial results and company updates. A live webcast of the conference call can be accessed in the "Investors/Media" section of Infinity’s website at www.infi.com. To participate in the conference call, please dial (877) 316-5293 (domestic) and (631) 291-4526 (international) five minutes prior to start time. The conference ID number is 1978798. An archived version of the webcast will be available on Infinity’s website for 30 days.

On May 11, 2020 Acceleron Pharma Inc. (Nasdaq:XLRN), a biopharmaceutical company dedicated to the discovery, development, and commercialization of TGF-beta superfamily therapeutics to treat serious and rare diseases, reported financial results for the first quarter ended March 31, 2020 (Press release, Acceleron Pharma, MAY 11, 2020, View Source [SID1234557505]).

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"Acceleron is off to a strong start in 2020, propelled by one of the most eventful time periods in the Company’s history," said Habib Dable, President and Chief Executive Officer of Acceleron. "The breadth and depth of our hematology program has been on full display, owing to the publication of the MEDALIST and BELIEVE Phase 3 luspatercept trial results in the prestigious New England Journal of Medicine and the recent FDA approval of REBLOZYL for the treatment of anemia in a population of patients with lower-risk MDS. We’re thrilled that this first-in-class erythroid maturation agent, having received approvals in two distinct indications over a span of just five months, is now available in the US to patients who have long needed a new option for treating their chronic anemias."

Added Mr. Dable: "Our scientific expertise in leveraging the therapeutic potential of the TGF-beta superfamily of proteins is delivering equally exciting results in pulmonary disease. With great enthusiasm, we announced that the PULSAR Phase 2 trial of sotatercept in patients with PAH met its primary and key secondary endpoints-underscoring our belief that sotatercept could eventually alter the current treatment paradigm. Following the results, sotatercept was granted Breakthrough Therapy designation by the FDA and PRIME designation by the EMA. We look forward to presenting topline results from PULSAR in a virtual meeting of the ATS as well as interactions with health authorities as we prepare for future Phase 3 development of sotatercept on our path to potential global registration."

Program Highlights

Hematology

REBLOZYL (luspatercept-aamt): Myelodysplastic Syndromes (MDS), Beta-Thalassemia, and Myelofibrosis (MF)
REBLOZYL is the first and only U.S. FDA approved erythroid maturation agent designed to promote red blood cell production through a novel mechanism. Luspatercept-aamt is being developed to treat anemia in patients with beta-thalassemia, MDS, and MF. REBLOZYL is part of the global collaboration between Acceleron and Bristol Myers Squibb.

In April, Acceleron and partner Bristol Myers Squibb announced the FDA approved REBLOZYL for the treatment of anemia failing an erythropoiesis stimulating agent and requiring 2 or more red blood cell (RBC) units over 8 weeks in adult patients with very low- to intermediate-risk MDS with ring sideroblasts (MDS-RS) or with myelodysplastic/myeloproliferative neoplasm with ring sideroblasts and thrombocytosis (MDS/MPN-RS-T).

The New England Journal of Medicine published results from the pivotal Phase 3 MEDALIST and BELIEVE trials.

In April, REBLOZYL received a positive opinion from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) for the treatment of adult patients with:

transfusion-dependent anemia due to very low-, low- and intermediate-risk MDS with ring sideroblasts, who had an unsatisfactory response or are ineligible for erythropoietin-based therapy; and

transfusion-dependent anemia associated with beta thalassemia

Bristol Myers Squibb expects to initiate the Phase 3 INDEPENDENCE trial in patients with MF on concomitant JAK 2 inhibitor therapy who require RBC transfusions by year-end 2020.

The BEYOND Phase 2 trial in adult patients with non-transfusion-dependent beta-thalassemia is ongoing, with topline results expected by year-end 2020 or early 2021.

Enrollment is ongoing in the COMMANDS Phase 3 trial in patients with treatment-naïve, lower-risk MDS, with topline results expected in 2022.

Pulmonary

Sotatercept: Pulmonary Arterial Hypertension (PAH)
Sotatercept is an investigational agent designed to be a selective ligand trap for members of the TGF-beta superfamily to rebalance BMPR2 signaling, which is a key molecular driver of PAH. In preclinical studies of PAH, sotatercept (RAP-011) reversed pulmonary vessel muscularization and improved indicators of right heart failure.

In January, Acceleron reported that the PULSAR Phase 2 trial in patients with PAH met its primary and key secondary endpoints. Sotatercept was generally well tolerated in the trial and adverse events observed were generally consistent with previously published data on sotatercept in other diseases.

PULSAR Phase 2 topline results are expected to be presented in an American Thoracic Society (ATS) 2020 web-based session by the end of June 2020.

Acceleron plans to host a webcast and conference call for investors and analysts following the ATS 2020 virtual presentation to discuss highlights from the PULSAR trial results.

In April, sotatercept was granted Breakthrough Therapy designation by the FDA for the treatment of patients with PAH (World Health Organization Group 1).

The EMA recently granted Priority Medicines (PRIME) designation to sotatercept for the treatment of patients with PAH.

Enrollment is ongoing in the exploratory SPECTRA trial in patients with PAH, with preliminary results expected in 2020.

Financial Results

Cash Position – Cash, cash equivalents and investments as of March 31, 2020 were $415.6 million, as compared to $453.8 million as of December 31, 2019. Based on the Company’s current operating plan and projections, it believes that current cash, cash equivalents and investments will be sufficient to fund projected operating requirements until such time as it expects to receive significant royalty revenue from REBLOZYL sales.

Revenue – Revenue for the first quarter of 2020 was $4.3 million, which includes $2.8 million of cost share revenue, and $1.5 million of royalty revenue from net U.S. sales of REBLOZYL. All revenue was derived from the Company’s partnership with Bristol Myers Squibb.

Costs and Expenses – Total costs and expenses for the first quarter of 2020 were $55.9 million. This includes R&D expenses of $37.7 million and SG&A expenses of $18.3 million.

Net Loss – The Company’s net loss for the first quarter of 2020 was $50.9 million.

Conference Call and Webcast
The Company will host a webcast and conference call to discuss its first quarter 2020 financial results and provide an update on recent corporate activities on May 11, 2020, at 5:00 p.m. EDT.

The webcast will be accessible under "Events & Presentations" in the Investors & Media page of the Company’s website at acceleronpharma.com. Individuals can participate in the conference call by dialing 877-312-5848 (domestic) or 253-237-1155 (international) and referring to the "Acceleron First Quarter 2020 Earnings Call."

The archived webcast will be available for replay on the Acceleron website approximately two hours after the event.

On May 11, 2020 Protagonist Therapeutics, Inc. (Nasdaq: PTGX), a clinical stage biopharmaceutical company, reported that it has commenced an underwritten public offering of 5,000,000 shares of its common stock (Press release, Protagonist, MAY 11, 2020, View Source [SID1234557542]). All of the shares of common stock are being offered by Protagonist. In connection with this offering, Protagonist expects to grant the underwriters a 30-day option to purchase up to 750,000 additional shares of common stock.

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Jefferies LLC and BMO Capital Markets Corp. are acting as joint book-running managers for the offering. H.C. Wainwright & Co., LLC and Nomura Securities International, Inc. are acting as co-lead managers for the offering. The offering is subject to market conditions and there can be no assurance as to whether or when the offering may be completed or the actual size or terms of the offering.

A shelf registration statement relating to the offered shares of common stock was filed with the Securities and Exchange Commission (SEC) on October 31, 2019, and was declared effective on November 22, 2019. A preliminary prospectus supplement and accompanying prospectus relating to the offering will be filed with the SEC and will be available on the SEC’s website, located at www.sec.gov. Prospective investors should read the preliminary prospectus supplement, when available, and the accompanying prospectus and other documents we have filed with the SEC for more complete information about us and the offering. Copies of the prospectus supplement and the accompanying prospectus related to the offering may be obtained, when available, from Jefferies LLC Attention: Equity Syndicate Prospectus Department, 520 Madison Avenue, 2nd Floor, New York, NY 10022, by telephone at 877-547-6340 or by email at [email protected] or from BMO Capital Markets Corp., Attention: Equity Syndicate Department, 3 Times Square, 25th Floor, New York, NY 10036, by telephone at 800-414-3627 or by email at [email protected].

This press release shall not constitute an offer to sell or a solicitation of an offer to buy these securities nor shall there be any sale of these securities in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.