On March 31, 2022 Abeona Therapeutics Inc. (Nasdaq: ABEO), a fully-integrated leader in cell and gene therapy, reported a business and strategy update and reported 2021 financial results (Press release, Abeona Therapeutics, MAR 31, 2022, View Source [SID1234611234]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"We are committed to developing novel cell and gene therapies for patients with rare diseases with no approved treatment options," said Vish Seshadri, Ph.D., Chief Executive Officer of Abeona. "We are focused on EB-101 and, having recently achieved target enrollment in our pivotal Phase 3 VIITAL study, have increased confidence that we will share topline results in the third quarter of 2022. We also expect animal proof-of-concept data from our preclinical eye programs beginning in the second half of 2022 that could support pre-IND meetings with the FDA. We believe the strategic steps announced today reflect the operating discipline needed to extend our cash runway beyond these near-term catalysts."

Strategy and Business Update

●Following a comprehensive portfolio review, Abeona announced today that it is focusing research and development (R&D) resources on the topline data readout for its EB-101 pivotal Phase 3 VIITAL study while the Company actively pursues a potential commercialization partner for EB-101.
●Target enrollment was achieved in Abeona’s EB-101 pivotal Phase 3 VIITAL study for recessive dystrophic epidermolysis bullosa (RDEB). The Company anticipates topline results for the co-primary endpoints related to wound healing and pain reduction measured at 24 weeks post treatment in the third quarter of 2022. Abeona received positive feedback from the U.S. Food and Drug Administration (FDA) related to a Type B meeting on the proposed Chemistry, Manufacturing and Controls (CMC) requirements of the EB-101 development program, gaining alignment on the characterization and validation plans that could support a potential Biologics License Application (BLA) for EB-101 in RDEB.

●In connection with its shift in priorities, the Company has intensified its pursuit of a strategic partnership to take over development activities for ABO-102, and has ceased build-out of additional AAV manufacturing space. As part of the FDA’s feedback on the Transpher A Statistical Analysis Plan (SAP) in January 2022, the agency recommended that all participants be followed to an age of at least 60 months, which would shift timing of the neurocognitive outcomes data readout to late-2024/early-2025, as compared to the Company’s prior projection of the second quarter of 2023.
●As part of the Company’s portfolio prioritization, Abeona will discontinue development of ABO-101 for MPS IIIB.
●Abeona plans to continue development of AAV-based gene therapies designed to treat ophthalmic and other diseases and next-generation AAV-based gene therapies using the novel AIM capsid platform and internal AAV vector research programs. Abeona will present results from testing of novel AAV capsids in non-human primates at the Association for Research in Vision and Ophthalmology (ARVO) 2022 Annual Meeting being held on May 1-4, 2022 in Denver, CO. The preclinical data could support pre-IND meetings with the FDA for Abeona’s undisclosed eye gene therapy indications. The Company previously reported preclinical data showing the potential for AIM AAV vectors to efficiently target the photoreceptor and retinal epithelium cell layers after intravitreal injection, creating the potential for new pipeline candidates that can address multiple ophthalmic disorders.
●The strategic changes will reduce the Company’s operating expenses and extend the estimated runway of current cash resources to mid-2023.
●In December 2021, Abeona raised approximately $17.5 million in aggregate gross proceeds, before underwriting discounts and commissions and other offering expenses, from an underwritten public offering of common stock.
●Joseph Vazzano was appointed as Chief Financial Officer (CFO) at Abeona, and will serve as the Company’s principal financial officer and principal accounting officer effective March 31, 2022. Mr. Vazzano previously served as CFO of Avenue Therapeutics, Inc. (Nasdaq: ATXI), where he secured multiple equity financings for Avenue and served in a leadership role for signing a complex, two-stage acquisition of the company with future contingent value rights.

Full Year 2021 Financial Results

Cash, cash equivalents, restricted cash and short-term investments totaled $50.9 million as of December 31, 2021, compared to $96.0 million as of December 31, 2020. Net cash used in operating activities was $65.7 million for the full year of 2021, including a $20 million payment in November 2021 in accordance with a settlement agreement with REGENXBIO. Net cash used in operating activities was $35.0 million for the full year of 2020.

License and other revenues for the full year of 2021 were $3.0 million, compared to $10.0 million in 2020. The revenue in 2021 resulted from a clinical milestone achieved in December 2021 under a sublicense agreement with Taysha Gene Therapies for ABO-202 for CLN1 disease.

R&D expenses were $34.3 million for the full year of 2021, compared to $30.1 million in 2020. General and administrative (G&A) expenses were $22.8 million for the full year of 2021, compared to $23.8 million in 2020.

Net loss was $84.9 million for the full year of 2021, or a $0.86 basic and diluted loss per common share as compared to a net loss of $84.2 million, or a $0.91 basic and diluted loss per common share, in 2020. The net loss in 2021 included a non-cash goodwill impairment charge of $32.5 million. The impairment charge has no impact on the Company’s cash position, cash flow from operating activities, and does not have any impact on future operations.

Conference Call Details

Abeona Therapeutics will host a conference call and webcast today, Thursday, March 31, 2022 at 8:30 a.m. ET, to discuss its full year 2021 financial results and business update. To access the call, dial 877-545-0320 (U.S. toll-free) or 973-528-0002 (international) and Entry Code: 851784 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 31, 2022 Fresenius Kabi reported that it has agreed to acquire a stake of 55% of mAbxience Holding S.L. ("mAbxience") (Press release, Fresenius, MAR 31, 2022, View Source [SID1234611221]). The purchase price will be a combination of €495 million upfront payment and milestone payments, strictly tied to the achievement of commercial and development targets. The contractual provisions also include a put / call option scheme regarding the current owners’ remaining shares in mAbxience (45%).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

mAbxience is a leading international biopharmaceutical company, focused on the rapidly developing biosimilars market. The company was founded in 2010 by Dr. Hugo Sigman and Dr. Silvia Gold as the biotechnology division of Insud Pharma S.L. mAbxience has established itself as a leader in the development and manufacturing of biological drugs, with two commercialized biosimilar products (Rituximab and Bevacizumab) and a mid-single-digit number of molecules across immunology and oncology expected to be launched globally in the years 2024 to 2029. This is supported by internal R&D laboratories and state-of-the-art manufacturing facilities in Spain and Argentina. In addition to highly competitive production costs for the internal programs, the manufacturing platform allows mAbxience to offer third party biological CDMO services, including a recent contract with AstraZeneca to produce the drug substance for its COVID-19 vaccine in Latin America. The company currently employs approximately 600 staff and generated sales of approx. €255 million in 2021.

The acquisition of a majority stake in mAbxience follows Fresenius Kabi’s recently unveiled Vision 2026 strategy, delivering on one of the core growth vectors – to "Broaden Biopharma" – by expanding along the value chain and further enhancing the existing Fresenius Kabi biosimilars pipeline.

Fresenius Kabi expects, through its in-house biosimilars programs and through its investment in mAbxience, to capture an overproportionate share of the underlying rapid growth in the biopharmaceutical market. Fresenius Kabi’s footprint in biopharmaceuticals will be significantly strengthened by broadening its biosimilars portfolio and by gaining access to the distinctive manufacturing capabilities of mAbxience. It will also allow Fresenius Kabi to provide end-to-end integrated biopharmaceutical solutions for customers from its state-of-the-art facilities.

mAbxience operates three state-of-the-art facilities for the production of biologic drug substance. This addresses a critical gap in Fresenius Kabi’s value chain, adding flexible, single-use biologic drug substance capacity that can be leveraged to provide competitive cost of production for the enlarged biosimilars portfolio. This manufacturing capability also offers end-to-end integrated biopharmaceutical solutions for customers and thus establishes a strategic foothold for Fresenius Kabi in the fast-growing biologic CDMO sector, complementing the existing small molecule API and fill & finish operations.

Once completed, the transaction is expected to deliver material operating and cost synergies for Fresenius Kabi, primarily driven by leveraging mAbxience’s manufacturing capabilities for Fresenius Kabi’s existing biosimilars business.

The transaction remains subject to regulatory approvals and other customary closing conditions and is expected to close by mid-2022.

Ivenix strengthens Fresenius Kabi’s MedTech business and accelerates growth

Delivers on core growth vector "Expand MedTech" of Vision 2026
Provides next-generation infusion therapy platform for U.S. market
Complements Fresenius Kabi’s global infusion therapy offering
Provides Fresenius Kabi with key capabilities in hospital connectivity and creates new options for growth of MedTech business
Significant scale and growth synergies expected
Fresenius Kabi announced today that it has agreed to acquire Ivenix, Inc. („Ivenix"), a specialized infusion therapy company. The purchase price will be a combination of US$240 million upfront payment and milestone payments, strictly linked to the achievement of commercial and operating targets.

Ivenix is a privately held company based in North Andover, Massachusetts, USA. The company has developed the technologically most advanced infusion system including a large volume pump ("LVP") with administration sets, infusion management software tools, applications and analytics to inform care and advance efficiency. The Ivenix Infusion System’s innovative design and architecture sets a new standard in infusion safety, simplicity and interoperability. The system is centred around the patient and clinician and is designed to reduce infusion-related errors and drive down the total cost of ownership. After having received the U.S. Food and Drug Administration’s (FDA) approval, the Ivenix Infusion System was successfully launched in late 2021.

Ivenix’ Infusion System provides access to attractive growth potential for Fresenius Kabi in the large and growing infusion therapy market. The combination of Ivenix’ leading hardware and software products with Fresenius Kabi’s offerings in intravenous fluids and infusion devices will create a comprehensive and leading portfolio of premium products, forming a strong basis to enable sustainable growth in the high-value MedTech space.

The transaction is subject to regulatory approvals and other customary closing conditions and is expected to close by mid-2022.

Financing and implications on Group financials

mAbxience is expected to be accretive to Group cash earnings per share (earnings before amortization and integration costs) right after closing. Ivenix is expected to be neutral to Group cash earnings per share in 2025 and accretive from 2026 onwards.

Combined, these acquisitions are expected to be broadly neutral to Group cash earnings per share in 2022 and accretive as of 2023.

The transactions are currently expected to be financed by cash flow and available liquidity.

Conference Call

A telephone conference on the acquisition of a majority stake in mAbxience Holding S.L. and the acquisition of Ivenix, Inc. will be held on March 31, 2022 at 1:30 p.m. CEST (7:30 a.m. EDT). All investors are cordially invited to follow the conference call in a live broadcast over the Internet at www.fresenius.com/investors. Following the call, a replay will be available on our website.

On March 31, 2022 TILT Biotherapeutics, a clinical-stage biotechnology company developing oncolytic immunotherapy for enabling therapies based on T cells, such as immune checkpoint inhibitors and adoptive cell therapies, reported that positive interim progress and safety data from its phase 1 clinical trials in metastatic melanoma (T215 – the ‘TUNINTIL’ trial) and solid tumors (T115 – the ‘TUNIMO’ trial) (Press release, TILT Biotherapeutics, MAR 31, 2022, View Source [SID1234611203]). These two trials, with patients in Denmark, France, and Finland, are of the company’s oncolytic immunotherapy asset TILT-123, designed to stimulate T-cells to better fight cancer.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

TILT Biotherapeutics’ CEO, Akseli Hemminki, a biotech entrepreneur and cancer clinician who has personally treated almost 300 patients with ten different oncolytic viruses, said, "We are delighted that our phase 1 trial in solid tumors (T115), which consists of a total of 11 patients being treated to date, is progressing at pace. Our first-in-human melanoma (T215) trial is showing equally positive results, with 10 patients being treated and some now being treated under an extension protocol. I am thankful for the excellent work by the clinical teams. We are showing that the treatment is well tolerated, has prominent biological effects on tumors, and has the potential to increase the efficacy of immunotherapies, and deliver the anti-tumor benefits of armed oncolytic viruses."

The ‘TUNINTIL’ clinical trial (1) is a phase 1, open-label, dose-escalation study of the company’s oncolytic adenovirus coding for Tumor Necrosis Factor Alpha (TNF alpha) and Interleukin 2 (IL-2), known as TILT-123. A total of about 15 metastatic melanoma patients receive TILT-123 as an initial monotherapy over one month, followed by up to two administrations of tumor infiltrating lymphocytes (TILs) in the second month as well as ongoing consecutive doses of TILT-123, for up to 24 months.

The ‘TUNIMO’ clinical trial (2) is also a phase 1 trial of TILT-123. A total of 15-20 patients with solid tumors receive TILT-123 as a monotherapy over a three-month period, and, as with the TUNINTIL protocol, followed up by a 24- month extension period.

The primary objective of both trials is to evaluate the safety of TILT-123 and to deliver insights about the behavior of TILT-123 in humans, such as systemic tumor transduction following intravenous delivery and virus replication in the tumor, as well as immunological responses.

To date, most frequent adverse events across both trials have been fever, chills, and fatigue, consistent with the administration of an oncolytic adenoviral immunotherapy, with no treatment related serious adverse events.

The heart of TILT’s innovative approach revolves around the use of armed oncolytic adenoviruses, using cytokines and other molecules to boost the patient’s immune response to better enable it to find and destroy cancer cells. The company is advancing its pipeline of other assets and programs with TILT-123 towards further clinical trials, including in combination with the immune checkpoint inhibitors (3) (4).

On March 30, 2022 Integra Therapeutics reported the company has closed an additional €1.5-million investment from venture capital firm Columbus Venture Partners (Press release, Integra Therapeutics, MAR 30, 2022, View Source [SID1234654530]). This investment comes on top of the €4.5 million Integra Therapeutics raised last December from AdBio Partners, Invivo Capital and Takeda Ventures and completes its seed round in line with the company’s set goals.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

The new funds will go to support development of the FiCAT platform prototype and preclinical validation with in vivo and ex vivo models. FiCAT is one of the most promising gene writing technologies in the world, as it addresses the current technical limitations of gene therapies (gene size, precision and stability) to make them safer and more efficient at preventing and treating genetic diseases and cancers with unmet medical needs.

The investment from Columbus VP comes through its third fund, Columbus Life Science Fund III FCR, which was presented in 2021 with €120 million. Damià Tormo, managing partner of Columbus VP, will join the Integra Therapeutics board of directors.

"We thank Columbus VP for their trust and enthusiasm and look forward to working with them to achieve safer, more efficient gene therapies for patients as quickly as possible. In rare diseases alone, we know that 72% are caused by genetic defects and that there isn’t currently any effective therapeutic treatment for most of them," explains Dr Avencia Sánchez-Mejías, co-founder and CEO of Integra Therapeutics.

"We believe Integra Therapeutics, with ground breaking science, is destined to be one of the benchmark biotechnology firms in gene writing over the coming years. We can’t wait to start working with the management team at Integra and the current partners," noted Damià Tormo.

Integra Therapeutics also recently brought Professor George Church, a pioneer in human gene editing, on board to join the company’s scientific advisory board.

On March 30, 2022 Oxilio, the pioneering oncology company, repurposing existing drugs to address unmet needs in cancer therapy, reported that it has acquired LSHS Consulting Limited in a share transaction (Press release, Oxilio, MAR 30, 2022, View Source [SID1234621607]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

LSHS is a specialist life science consultancy firm established by Dr Dan Gooding, comprising intellectual property assets relevant to Oxilio’s OXL001 programme.

Dr Gooding was CEO of Nuformix plc until June 2020 and has significant experience in the pre-clinical and clinical development of formulations relevant to OXL001. Through him, Oxilio commenced discussions for the licensing of Nuformix’s NXP-001 cocrystal aprepitant formulation for the treatment of cancer.

Dr Gooding has been instrumental in helping Oxilio make rapid scientific and commercial progress with both our co-crystal and lipid-based formulation routes, in addition to the exploration of further formulation technologies which remain under evaluation. Dr Gooding also introduced Oxilio to TRx Biosciences and facilitated the negotiation of our exclusive licence to use TRX’s lipid platform technology with our aprepitant formulation, again for the treatment of cancer. Since that time, Dr Gooding has become a Director of TRx Biosciences.

As a result of the transaction, Oxilio secures continuing access to Dr Gooding’s expertise and know-how in both our lipid and co-crystal product development routes.

Dr Gooding has undertaken much of our formulation development work with the TRx scientists and provided the additional capability and expertise Oxilio requires to transfer this technology to Quotient Sciences for clinical trials.

We are looking forward to continuing close co-operation with Dr Gooding across a range of scientific developments at Oxilio.