On May 11, 2020 Rubius Therapeutics, Inc. (Nasdaq:RUBY), a clinical-stage biopharmaceutical company that is genetically engineering red blood cells to create an entirely new class of cellular medicines, reported first quarter 2020 financial results and provided an overview of operational progress (Press release, Rubius Therapeutics, MAY 11, 2020, View Source [SID1234557554]).

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"During the first quarter, we made significant progress in advancing our business by reprioritizing our therapeutic areas of focus and progressing our oncology pipeline of Red Cell Therapeutics, including the dosing of our first patient in the RTX-240 clinical trial. RTX-240 is an allogeneic cellular therapy product candidate that is designed to mimic and amplify the functions of the innate and adaptive immune systems by activating and expanding NK cells and T cells to generate a potent anti-tumor response, potentially providing patients with a dual-mechanistic approach to fight their cancer," said Pablo J. Cagnoni, chief executive officer of Rubius Therapeutics. "With the advances in our oncology pipeline, a fully owned and operational manufacturing facility and cash runway that takes us into 2022, we believe we are well positioned to execute our objectives and bring potentially life-saving therapies to patients."

First Quarter and Recent Highlights

·Completed strategic reprioritization of the pipeline to focus on the development of oncology and autoimmune Red Cell Therapeutic programs.
oDevelopment in these therapeutic areas is enabled by the investment in internal manufacturing at the Rubius Smithfield, RI facility.
·Dosed the first patient in the Phase 1/2 clinical trial of RTX-240 for the treatment of patients with relapsed/refractory or locally advanced solid tumors.
oThe cGMP cells used to dose the first patient were produced at the fully owned Rubius manufacturing facility. All subsequent clinical supply for the Company’s oncology programs is expected to be produced from this facility.

·On track to file an Investigational New Drug application for lead artificial antigen-presenting cell program, RTX-321, for the treatment of HPV 16-positive cancers by year-end 2020.
·Unveiled abstracts for the American Society of Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper) 23rd Annual Meeting, highlighting preclinical data from its oncology pipeline of Red Cell Therapeutics, including data supporting RTX-321.
oThe posters will be made available at the beginning of the virtual meeting tomorrow, Tuesday, May 12, 2020, at 6:00 a.m. ET.
·Implemented multiple measures in response to the COVID-19 pandemic to safeguard the health and well-being of employees, their families, business partners and healthcare providers, while continuing to operate its Smithfield, RI manufacturing facility and conduct research and development activities. The extent to which the COVID-19 pandemic may impact Rubius will depend on future developments.

First Quarter 2020 Financial Results

Net loss for the first quarter of 2020 was $48.5 million or $0.60 per common share, compared to $32.6 million or $0.42 per common share in the first quarter of 2019.

In the first quarter of 2020, Rubius invested $36.2 million in research and development (R&D) related to its novel RED PLATFORM and towards expanding and advancing its product pipeline, compared to $20.9 million in the first quarter of 2019. The year-over-year increase was driven by $9.9 million of incremental costs incurred in advancing our cancer programs, including preparation for the Phase 1/2 clinical trial for RTX-240 and preclinical and IND-enabling activities for RTX-321. There was also a $1.6 million increase in costs incurred for our rare disease pipeline prior to the deprioritization of these programs in March 2020. In addition, costs not allocated to programs increased by $3.8 million, resulting from increases in personnel-related costs, stock-based compensation, contract research and development and facility related and other costs. These higher costs were driven by R&D headcount growth and expanded platform development and drug discovery activities.

G&A expenses were $12.7 million during the first quarter of 2020, compared to $13.5 million for the first quarter of 2019. The lower costs were primarily driven by a reduction in stock-based compensation expense.

Cash Position

As of March 31, 2020, cash, cash equivalents and investments were $241.4 million, compared to $283.3 million as of December 31, 2019, providing Rubius with a cash runway into 2022.

During the quarter, the Company used $40.0 million of cash to fund operations and $2.9 million to fund capital expenditures, consisting mostly of payments for assets purchased in 2019.

On May 11, 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 financial results and recent corporate highlights for the first quarter ended March 31, 2020 (Press release, Mustang Bio, MAY 11, 2020, View Source [SID1234557553]).

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Manuel Litchman, M.D., President and Chief Executive Officer of Mustang, said, "We were pleased to announce several important milestones in the first few months of 2020. Most notably, we were excited to submit our Investigational New Drug ("IND") application with the U.S. Food and Drug Administration ("FDA") for MB-107, our lentiviral gene therapy for the treatment of X-linked severe combined immunodeficiency ("XSCID"), also known as bubble boy disease, and we look forward to commencing a multi-center Phase 2 clinical trial in newly diagnosed infants with XSCID under the age of two. We also anticipate filing a second IND in the third quarter of this year for a multi-center trial for the treatment of previously transplanted XSCID patients. Additionally, the European Medicines Agency ("EMA") granted Advanced Therapy Medicinal Product ("ATMP") classification to MB-107, which is an important step in establishing our path to market approval and commercialization in Europe."

"Among our other first quarter accomplishments was the complete response achieved at the lowest dose level in the first subject treated following process optimization in our Phase 1/2 clinical trial of MB-106, our CD20-targeted, autologous CAR T cell therapy, for patients with relapsed or refractory B-cell non-Hodgkin lymphomas. This trial is ongoing, and we hope to announce additional interim data later this year. We are also very pleased to report that MB-105, a PSCA-directed CAR T currently under investigation in a Phase 1 trial at City of Hope, appeared to be active in the first patient to receive the therapy following a standard CAR T conditioning regimen. As we progress through 2020, we look forward to advancing our gene and CAR T cell therapies toward additional potentially value-creating regulatory and clinical milestones in the months ahead," Dr. Litchman concluded.

Recent Corporate Highlights:

·In May 2020, Mustang submitted an IND application with the FDA to initiate a multi-center Phase 2 clinical trial of MB-107 in newly diagnosed infants with XSCID who are under the age of two. The trial is expected to enroll 10 patients who, together with 15 patients enrolled in the current multicenter trial led by St. Jude Children’s Research Hospital, will be compared with 25 matched historical control patients who have undergone hematopoietic stem cell transplant ("HSCT"). The primary efficacy endpoint will be event-free survival. The initiation of this trial is currently on hold pending CMC clearance by the FDA. Mustang is targeting topline data from the trial in the second half of 2022.
·Mustang further expects to file an IND in the third quarter of 2020 for a registrational multi-center Phase 2 clinical trial of its lentiviral gene therapy in previously transplanted XSCID patients. This product will be designated MB-207. Mustang anticipates enrolling 20 patients and comparing them to matched historical control patients who have undergone a second HSCT. Mustang is targeting topline data for this trial in the second half of 2022.
·In the ongoing Phase 1 trial at City of Hope with MB-105, a PSCA-directed CAR T administered systemically to patients with PSCA-positive castration resistant prostate cancer, the first patient to receive the therapy following a standard CAR T conditioning regimen experienced a significant reduction in his prostate-specific antigen ("PSA") at day 28. This PSA response was associated with radiographic improvement of the patient’s metastatic disease.

In April 2020, Mustang announced that the EMA granted ATMP classification to MB-107 for the treatment of XSCID.
·In February 2020, Mustang announced that the first subject treated with the optimized MB-106 (CD20-targeted, autologous CAR T cell therapy) manufacturing process, developed in collaboration between Mustang and Fred Hutchinson Cancer Research Center, achieved a complete response at the lowest starting dose in an ongoing Phase 1/2 clinical trial. The trial is evaluating the safety and efficacy of MB-106 in subjects with relapsed or refractory B-cell non-Hodgkin lymphomas.

Financial Results:

·As of March 31, 2020, Mustang’s cash, cash equivalents and restricted cash totaled $56.8 million, compared to $62.4 million as of December 31, 2019, a decrease of $5.6 million for the first quarter.
·Research and development expenses were $9.3 million for the first quarter of 2020. This compares to $7.0 million for the first quarter of 2019. Non-cash, stock-based compensation expenses included in research and development were $0.4 million for the first quarter of 2020, compared to $0.1 million for the first quarter of 2019.
·Research and development expenses from license acquisitions totaled $0.3 million for the first quarter of 2020, compared to $0.5 million for the first quarter of 2019.
·General and administrative expenses were $2.0 million for the first quarter of 2020. This compares to $2.3 million for the first quarter of 2019. Non-cash, stock-based compensation expenses included in general and administrative expenses were $0.4 million for the first quarter of 2020, compared to $0.7 million for the first quarter of 2019.
·Net loss attributable to common stockholders was $11.9 million, or $0.28 per share, for the first quarter of 2020, compared to a net loss attributable to common stockholders of $9.6 million, or $0.34 per share, for the first quarter of 2019.

On May 11, 2020 Bio-Techne Corporation (NASDAQ:TECH) reported the release of the GMP Cloudz Human T Cell Activation Kit (Press release, Bio-Techne, MAY 11, 2020, View Source [SID1234557552]). This is the first GMP-grade kit within the Cloudz product line addressing the need for robust, yet safe products within the clinical- and commercial-scale of the Cell and Gene Therapy manufacturing process . The kit contains Cloudz particles, which are dissolvable microspheres conjugated to GMP Grade, in-house manufactured CD3 and CD28 antibodies for the expansion of T cells. The GMP Cloudz Human T Cell Activation Kit seamlessly fits within the T cell culture platform, and the particles are easily washed away from cells of interest. Cloudz particles can be added to CD3+ T cells or PBMCs for efficient expansion and purity of cells. The unique properties of the Cloudz microspheres allow them to be readily dissolved and washed away with a GMP-Grade Cloudz Release Buffer.

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Cell and Gene Therapies have gained significant traction over the last decade following the commercial success of CAR-T Cell therapeutics and include a substantial pipeline of therapies within clinical trials. The Cloudz product line is part of the larger cell and gene therapy portfolio that Bio-Techne has created over the last year that now includes several ExCellerate media formulations, GMP proteins, and gene-editing platforms with additional products in the pipeline.

"The GMP Cloudz T Cell activation kit is an important tool within CAR-T and other immune cell therapies providing a much-needed bead-free cell expansion platform," commented Dave Eansor, President of Bio-Techne’s Protein Sciences Segment. "It is an exciting time within Cell and Gene Therapy, and we are proud to be a part of this important area of science."

On May 11, 2020 PharmaCyte Biotech, Inc. (OTCQB: PMCB), a biotechnology company focused on developing cellular therapies for cancer and diabetes using its signature live-cell encapsulation technology, Cell-in-a-Box, reported that cGMP Validation, the company’s GMP consultant, is conducting its final audit of the manufacturing facility in Thailand where PharmaCyte’s clinical trial product was produced by PharmaCyte’s partner, Austrianova Singapore (Austrianova) (Press release, PharmaCyte Biotech, MAY 11, 2020, View Source [SID1234557546]). When the audit is completed, cGMP Validation will give PharmaCyte approval to import the clinical trial product to the company’s supply chain vendor in the United States (U.S.) who will store the product at -80C until it is needed.

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In addition, Austrianova and cGMP Validation have now completed their work together to achieve what has been deemed cGMP compliant batch records for the two manufacturing runs successfully produced by Austrianova. Both worked closely together to revise the batch records that were generated during the two manufacturing runs that produced PharmaCyte’s clinical trial product for its planned Phase 2b clinical trial in locally advanced, inoperable pancreatic cancer (LAPC). A batch record is a detailed written document of a manufactured batch of product, prepared during a pharmaceutical manufacturing process. A batch record contains actual data and the step by step process for manufacturing each batch. The completed manufacturing batch records are proof that the two batches were properly made and checked by quality control personnel at Austrianova according to cGMP standards. This was necessary so that the batches comply with the standards required by the U.S. Food and Drug Administration (FDA) for a batch record for each manufacturing run.

PharmaCyte’s Chief Executive Officer, Kenneth L. Waggoner, said, "We continue to work through a checklist of items that are necessary to submit an acceptable Investigational New Drug application (IND) to the FDA. The work that cGMP Validation and Austrianova are currently performing to audit the manufacturing facility in Thailand and the work they have completed working together to make certain the batch records meet the cGMP requirements for manufacturing a clinical trial product was and continues to be incredibly detailed and must continue to follow strict FDA guidelines.

"We are extremely pleased that the final audit of the manufacturing facility is underway and that our batch records from the two successful manufacturing runs meet all FDA cGMP requirements as the product is not considered a cGMP product unless it meets these stringent standards."

To learn more about PharmaCyte’s pancreatic cancer treatment and how it works inside the body to treat locally advanced inoperable pancreatic cancer, we encourage you to watch the company’s documentary video complete with medical animations at: View Source

On May 11, 2020 Zimmer Biomet Holdings, Inc. (NYSE and SIX: ZBH) reported financial results for the quarter ended March 31, 2020 (Press release, Zimmer Holdings, MAY 11, 2020, View Source [SID1234557544]). The Company reported first quarter net sales of $1.784 billion, a decrease of 9.7% from the prior year period, and a decrease of 8.9% on a constant currency basis – both consistent with the preliminary results disclosed in the Company’s press release on April 6, 2020. Net loss for the first quarter was $509 million, including goodwill impairment. Net earnings on an adjusted basis were $354 million.

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Diluted loss per share was $2.46 for the first quarter. Adjusted diluted earnings per share were $1.70 for the first quarter, a decrease of 9.1% from the prior year period.

"While our full first quarter results have been impacted by the global spread of COVID-19 and the deferral of elective procedures, our operational performance prior to the COVID-19 disruption was trending ahead of our expectations," said Bryan Hanson, President and CEO of Zimmer Biomet. "Over the past two years, we have made significant strides in reshaping and evolving Zimmer Biomet. This progress has better positioned us to address the COVID-19 challenge. We have an innovative and mobilized team, operational scale and increased financial flexibility – all of which we are using to support healthcare professionals and patients. I continue to be so proud of our global team and our collective, unyielding commitment to the ZB mission to better the lives of people around the world."

First quarter performance was negatively impacted by COVID-19, which reached a pandemic level in March and resulted in a significant and sudden global decline in elective procedure volumes.

Please see the attached schedules accompanying this press release for additional details on performance in the quarter, including sales by Zimmer Biomet’s three geographies and five product categories.

Geographic and Product Category Sales

The following sales table provides results by geography and product category for the three-month period ended March 31, 2020, as well as the percentage change compared to the prior year period, on both a reported basis and a constant currency basis.

Cash Flow and Balance Sheet

Operating cash flow for the first quarter was $451 million and free cash flow was $325 million. In the first quarter, the Company paid $50 million in dividends and declared a dividend of $0.24 per share. The Company also recently refinanced $1.5 billion in debt that came due April 1, 2020, renegotiated the terms of its $1.5 billion revolver and secured an additional $1.0 billion credit facility.

Financial Guidance

Zimmer Biomet expects the decline in elective procedure volumes observed in the final weeks of the first quarter to continue to have a significant negative impact in the second quarter of 2020. Given the uncertainty around the scope and duration of COVID-19 and its ongoing impact on the deferral of elective procedures, the Company is currently unable to quantify the expected impact on its results of operations, financial condition and cash flows, which could be material, for 2020 and is therefore not providing full-year financial guidance.

Conference Call

The Company will conduct its first quarter 2020 investor conference call today, May 11, 2020, at 8:30 a.m. Eastern Time. The audio webcast can be accessed via Zimmer Biomet’s Investor Relations website at https://investor.zimmerbiomet.com. It will be archived for replay following the conference call.