On March 24, 2021 Abeona Therapeutics Inc. (Nasdaq: ABEO), a fully-integrated leader in gene and cell therapy, reported financial results for the fourth quarter and full year 2020, and provided an update on recent operational progress (Press release, Abeona Therapeutics, MAR 24, 2021, View Source [SID1234577159]).

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"In 2020, Abeona advanced our three clinical programs toward bringing urgently needed treatments to patients with recessive dystrophic epidermolysis bullosa (RDEB) and Sanfilippo syndrome type A (MPS IIIA) and type B (MPS IIIB) despite the macro disruptions that impacted the world," said Michael Amoroso, Chief Executive Officer of Abeona. "We remain laser focused on executing our strategy and achieving upcoming milestones. Our recent momentum is highlighted by a successful Type B meeting with the FDA where we aligned on co-primary endpoints for the pivotal Phase 3 VIITAL study of EB-101 in RDEB, treatment of the fourth patient in the VIITAL study, and reporting new positive clinical data for both ABO-102 in MPS IIIA and ABO-101 in MPS IIIB at the WORLDSymposium. We look forward to continuing to propel our clinical programs forward and bringing our gene and cell therapies to patients who currently have no approved treatment options. We believe we have sufficient cash resources to build on our momentum and fund our current development and operating plan through the achievement of key anticipated milestones, including the potential for multiple regulatory submissions."

Recent Highlights

Corporate Developments

In March 2021, Michael Amoroso, Executive Vice President, Chief Operating Officer (COO) and principal executive officer at Abeona, was promoted to President, Chief Executive Officer (CEO) and a member of the company’s Board of Directors.
EB-101 (Autologous, Gene-Corrected Cell Therapy)

The fourth patient was treated in Abeona’s EB-101 pivotal Phase 3 VIITAL study for recessive dystrophic epidermolysis bullosa (RDEB). The company currently anticipates completing study enrollment in 2021 of 10 to 15 patients with RDEB, comprising approximately 35 large chronic wound sites treated in total.
Abeona held a successful Type B meeting with the U.S. Food and Drug Administration (FDA) to align with the Agency on the company’s proposal regarding co-primary endpoints of partial wound closure and mean pain reduction for the Phase 3 VIITAL study of EB-101 in RDEB.
ABO-102 and ABO-101 (AAV-based Gene Therapies)

Reported new positive interim data from the ABO-102 Transpher A study for MPS IIIA and the ABO-101 Transpher B study for MPS IIIB. The data was presented in late-breaking platform oral presentations at the 17th Annual WORLDSymposium in February 2021. The presented results from the high dose cohort 3 in the Transpher A study showed evidence of preservation of neurocognitive development within normal range of a non-afflicted child for 2.5 years to 3 years after treatment with ABO-102 in the three young patients treated before 30 months of age. In addition, the data showed a dose-related and sustained reduction in cerebrospinal fluid (CSF) levels of heparan sulfate, denoting transgene expression in the CNS, and a durable reduction of liver volume. The presented results from the Transpher B study continued to show signals of biologic effect with reduction of disease-specific biomarkers in the CSF, plasma and urine and reduction in liver volumes. Abeona expects additional follow-up visits and neurocognitive assessments of patients treated in the high dose cohort 3 in the Transpher A study and additional clinical updates from the Transpher B study in 2021.
The FDA accepted Abeona’s request for a meeting to discuss the data-to-date from the Transpher A study and the potential path to a Biologics License Application (BLA) submission for ABO-102 in MPS IIIA.
As previously reported, target enrollment in the ABO-102 Transpher A study has been achieved. Abeona continues to enroll patients into the study given the lack of treatment options for MPS IIIA and encouraging efficacy and safety data from the high dose cohort 3. To date, 19 patients have been dosed in the Transpher A study, including 13 patients dosed in cohort 3.
The ABO-101 Transpher B study for MPS IIIB is ongoing, and to date, 11 patients have been dosed, including 4 patients dosed in cohort 3.
Preclinical Pipeline

Initiated preclinical research in December 2020 and are assessing AAV capsids in six undisclosed ophthalmic disorders and intend to advance toward IND-enabling studies in two to three indications in 2022. Previously reported preclinical data showed the potential for AIM AAV vectors to efficiently target the retinal epithelium after intravitreal injection, creating the potential for new pipeline candidates that can address multiple ophthalmic disorders.
Fourth Quarter and Full Year 2020 Financial Results

Cash, cash equivalents and short-term investments totaled $95.0 million as of December 31, 2020, compared to $129.3 million as of December 31, 2019. Net cash used in operating activities was $35.0 million for the full year of 2020, compared to $62.8 million for the full year 2019.

License and other revenues for the fourth quarter and full year of 2020 were $3.0 million and $10.0 million, respectively, compared to zero revenues in the same periods in 2019. The increase in revenue was comprised of initial proceeds from agreements with Taysha Gene Therapies in August 2020 relating to ABO-202, a potential gene therapy for for CLN1 disease, and in October 2020 relating to intellectual property directed to a potential gene therapy for Rett syndrome.

Research and development (R&D) expenses were $9.2 million for the fourth quarter of 2020 and $30.1 million for the full year of 2020, compared to $9.6 million and $48.6 million in the same periods in 2019. The decrease in R&D expenses was primarily due to decreased clinical and development work for the company’s gene and cell therapy product candidates as a result of scaled back manufacturing, clinical and non-clinical development activities impacted by the COVID-19 pandemic, and cost savings from the decision to internally manufacture retrovirus for the EB-101 program.

General and administrative (G&A) expenses were $7.4 million for the fourth quarter of 2020 and $23.8 million for the full year of 2020, compared to $4.7 million and $20.7 million in the same periods in 2019. The increase in G&A expenses in the fourth quarter of 2020 was primarily due to increased professional fees and increased share-based compensation expense. The increase in G&A expenses for full year 2020 was primarily due to severance costs associated with management changes and increased professional fees.

Net loss was $15.8 million for the fourth quarter of 2020 and $84.2 million for the full year of 2020, compared to net loss of $16.4 million and $76.3 million for the same periods in 2019. The increase in the full year 2020 net loss is primarily due to the licensed technology non-cash impairment charge of $32.9 million related to the termination of the license agreement with REGENXBIO, partially offset by increased license and other revenues along with decreased clinical and development expenses.

Conference Call Details

Abeona Therapeutics will host a conference call and webcast tomorrow, Thursday, March 25, 2021 at 8:30 a.m. ET, to discuss its fourth quarter 2020 financial results and business update. To access the call, dial 888-506-0062 (U.S. toll-free) or 973-528-0011 (international) and Entry Code: 251720 five minutes prior to the start of the call. A live, listen-only webcast and archived replay of the call can be accessed on the Investors & Media section of Abeona’s website at www.abeonatherapeutics.com. The archived webcast replay will be available for 30 days following the call.

On March 24, 2021 Hutchison China MediTech Limited ("HUTCHMED") (Nasdaq/AIM: HCM) reported that it has reached an agreement with GL Mountrose Investment Two Limited, a company controlled and managed by GL Capital Group ("GL Capital"), to sell its entire indirect interest in Hutchison Whampoa Guangzhou Baiyunshan Chinese Medicine Company Limited ("HBYS"), a non-core and non-consolidated over-the-counter ("OTC") drug joint venture business (Press release, Hutchison China MediTech, MAR 24, 2021, View Source [SID1234577126]).

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"HUTCHMED’s focus is the discovery and development of novel therapies in oncology and immunology. Over the past 20 years, we have invested in establishing one of the leading innovation-driven, global biopharmaceutical companies based in China," said Simon To, Chairman of HUTCHMED. "The sale of our shares in HBYS, and exit from the OTC drug arena, will allow us to focus our organization and resources on our primary aim of accelerating investment in our Oncology/Immunology assets in China and beyond."

Jeffrey Li, Founder and Chief Executive Officer of GL Capital, said, "As a long-term shareholder and supporter of HUTCHMED, GL is pleased to acquire its share in one of the best-known OTC businesses in China. This transaction is in-line with GL’s strategy of building a leadership position in the OTC drug area to serve patients’ needs for self-medication and cost containment of their overall healthcare budget."

The aggregate amount to be received by HUTCHMED of approximately $169 million in cash represents about 22 times HBYS’ adjusted net profit attributable to HUTCHMED equity holders of $7.7 million in 2020[1]. Of the proceeds, approximately $127 million is related to its shareholding in HBYS with the approximately $42 million balance related to distributions of the previously announced land compensation and prior year undistributed profits.

A deposit of $15.9 million is payable by GL Capital immediately following signing of the agreement which will be credited against the proceeds due on closing of the transaction. The transaction is subject to regulatory approval in China and is expected to close in mid-2021.

On March 24, 2021 Exacis Biotherapeutics Inc., a development-stage immuno-oncology company working to democratize access to the most advanced and effective cancer treatments, reported several important steps in the preclinical development of its ExaNK engineered NK cell-therapy candidates (Press release, EirGenix, MAR 24, 2021, View Source [SID1234577107]).

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ExaNK cells are generated from induced pluripotent stem cells (iPSCs) that are made using mRNA-based cell-reprogramming and gene-editing technologies. ExaNK cells are designed to resist rejection when administered to patients, with the goal of reducing or eliminating the need for costly and dangerous lymphodepletion, a procedure normally performed using cytotoxic chemotherapy, which carries risks of neurotoxicity and infection. These engineered iPSCs will form the basis for Exacis’ tumor-targeted ExaCAR-NK cells as well as non-CAR-bearing ExaNK cells designed to improve the effect of monoclonal antibodies against both liquid and solid tumors.

Exacis produces rejection-resistant ExaNK cells by performing functional editing of key stealthing targets in its proprietary mRNA-reprogrammed iPSCs. These engineered iPSCs are then differentiated to the final NK-cell product using Exacis’ proprietary high-yield differentiation process. The resulting ExaNK cells show higher tumor cell-killing activity and cytokine production in vitro than peripheral blood-derived NK cells, with no evidence of self-killing (i.e., "fratricide").

Exacis develops its off-the-shelf products using iPSCs to avoid the need for donors. This approach aims to lower the cost and increase the availability and consistency of engineered immuno-oncology cell therapies in comparison to currently approved products.

The discoveries announced today were made in collaboration with Exacis’ parent company, Factor Bioscience, and have been submitted for presentation at a major conference later this year. Exacis has also disclosed the details of these discoveries in a provisional patent application filed earlier this month.

"These results illustrate the sound scientific basis for Exacis’ approach to the development of next-generation engineered NK-cell therapies," said Matt Angel, PhD, CEO of Factor Bioscience and Chair of Exacis’ Scientific Advisory Board. "Combining cell reprogramming with gene editing allows the production of a near-unlimited supply of genetically uniform engineered cells, while using mRNA for both the cell-reprogramming and gene-editing steps uniquely enables the generation of footprint-free cells with no risk of vector integration."

"We continue to be encouraged by the rapid progress we are making towards developing accessible, next-generation engineered cell therapies that will improve patient experiences and outcomes. This first opportunity to expand our substantial intellectual property portfolio marks a key milestone for the company," stated Gregory Fiore MD, CEO and President of Exacis Biotherapeutics.

Next steps for Exacis include scaling the iPSC expansion and differentiation processes to prepare for IND-enabling studies to be conducted later this year.

On March 24, 2021 United Therapeutics Corporation (Nasdaq: UTHR) reported that Dr. Gil Golden, Executive Vice President and Chief Medical Officer of United Therapeutics, will provide an overview and update on the company’s business during a fireside chat session at the 10th Annual J.P. Morgan Napa Valley Forum (Press release, United Therapeutics, MAR 24, 2021, View Source [SID1234577106]).

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The session will take place virtually on Wednesday, March 31, 2021, from 12:00 p.m. to 12:45 p.m., Eastern Daylight Time, and can be accessed via a live webcast on the United Therapeutics website at View Source An archived, recorded version of the session will be available approximately 24 hours after the session ends and can be accessed at the same location for 30 days.

On March 24, 2021 CASI Pharmaceuticals, Inc. (Nasdaq: CASI), a U.S. biopharmaceutical company focused on developing and commercializing innovative therapeutics and pharmaceutical products, reported the pricing of an underwritten public offering of 15,853,658 shares of its common stock at a price to the public of $2.05 per share (Press release, CASI Pharmaceuticals, MAR 24, 2021, View Source [SID1234577105]). CASI has granted the underwriters an option to purchase up to an additional 2,378,048 shares of common stock, which terminates on the earlier of 30 days and the day before CASI files to the U.S. Securities and Exchange Commission ("SEC") its Annual Report on Form 10-K for the fiscal year ended December 31, 2020. The offering is expected to close on or about March 26, 2021, subject to satisfaction of customary closing conditions. The gross proceeds to CASI from the offering, excluding any exercise by the underwriters of their option to purchase additional shares, are expected to be approximately $32.5 million, before deducting underwriting discounts and commissions and other offering expenses payable by CASI.

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Oppenheimer & Co. Inc., Mizuho Securities USA LLC, and BTIG LLC are acting as joint book-running managers for the offering.

CASI intends to use the net proceeds of the offering for working capital and general corporate purposes, which include, but are not limited to advancing CASI’s product portfolio, acquiring the rights to new product candidates and general and administrative expenses.

The securities described above are being offered by CASI pursuant to a shelf registration statement on Form S-3, including a base prospectus, that was filed on November 20, 2020 and declared effective by the SEC on December 2, 2020. The offering is being made only by means of a written prospectus and prospectus supplement that form a part of the registration statement. 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. Copies of the preliminary prospectus supplement and the accompanying prospectus relating to the offering, when available, may also be obtained from Oppenheimer & Co. Inc., Attention: Syndicate Prospectus Department, 85 Broad Street, 26th Floor, New York, NY, 10004, by telephone at (212) 667-8055, or by email at [email protected]; or Mizuho Securities USA LLC, Attention: Equity Capital Markets, 1271 Avenue of the Americas, 3rd Floor, New York, NY, 10020; by phone at (212) 205-7600; or by email at [email protected].

Before investing in the offering, you should read in their entirety the preliminary prospectus supplement and the accompanying prospectus and the other documents that CASI has filed with the SEC that are incorporated by reference in the preliminary prospectus supplement and the accompanying prospectus, which provide more information about CASI and the offering.

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