On August 8, 2023 Acorda Therapeutics, Inc. (Nasdaq: ACOR) reported a business update and reported its financial results for the second quarter ended June 30, 2023 (Press release, Acorda Therapeutics, AUG 8, 2023, View Source [SID1234633949]).

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"INBRIJA’s growth in the first half of 2023 improved significantly versus the first half of 2022, including a 42% increase in new prescription request forms, or PRFs. New PRFs are a leading indicator of future revenue growth, which we expect will accelerate going forward. We are seeing the impact of the new sales and marketing programs we launched this year; our streaming TV commercial has had approximately 8 million views in its first 4 months, and 165 physicians have prescribed INBRIJA for the first time in 2023 since they or their patients viewed the commercial," said Ron Cohen, M.D., Acorda’s President and Chief Executive Officer. "U.S. net revenue in the first half of the year increased less quickly than we projected, and we are therefore revising our guidance for 2023 INBRIJA U.S. net revenue to $34 million-$38 million, from $38 million-$42 million, and as a result we do not expect to be cash flow neutral this year. The new range continues to represent significant growth over 2022 and, in addition, we have continued to control costs, enabling us to revise our guidance for operating expenses to $93 million-$98 million from $93 million-$103 million."

Dr. Cohen added, "We were also pleased that Ampyra continued to perform well, with flattening of its attrition curve. And we have continued to communicate with our bondholders to enable constructive approaches to servicing the Company’s convertible debt."

Second Quarter 2023 Financial Results

For the quarter ended June 30, 2023, the Company reported INBRIJA worldwide net revenue of $9.1 million, a 2% decrease, of which $8.3 million was from sales in the U.S., a 12% increase, compared to the same quarter in 2022. The first quarter of 2022 included initial channel loading shipments for the launch in Germany, whereas initial shipments for the launch in Spain occurred largely in the first quarter of 2023. The Company also reported ex-U.S. INBRIJA net revenue of $0.8 million in the second quarter related to the launch in Spain.

For the quarter ended June 30, 2023, the Company reported AMPYRA net revenue of $16.9 million, a 7% decrease compared to $18.2 million for the same quarter in 2022. Additionally, for the quarter ended June 30, 2023, the Company reported FAMPYRA royalty revenues of $2.9 million, a 6% decrease compared to the same quarter in 2022. As previously disclosed, AMPYRA lost its exclusivity when generics entered the market in 2018, and the Company expects AMPYRA revenue to continue to decline, although at a slower rate.

Research and development (R&D) expenses for the quarter ended June 30, 2023 was $1.6 million, compared to $1.5 million for the quarter ended June 30, 2022. Sales, general and administrative (SG&A) expenses for the quarter ended June 30, 2023 were $21.8 million, compared to $30.1 million for the same quarter in 2022.

Total operating expenses for the quarter ended June 30, 2023 was $33.3 million, compared to $45 million for the same quarter in 2022.

Non-GAAP adjusted operating expenses (adjusted OPEX) for the quarter ended June 30, 2023 was $23.4 million, compared to $31.6 million for the same quarter in 2022. This quarterly non-GAAP measure, more fully described below under "Non-GAAP Financial Measures," excludes costs of goods sold, amortization of intangible assets, change in fair value of derivative liability, and change in fair value of acquired contingent liability. A reconciliation of the GAAP operating expenses to non-GAAP operating expenses is included with the attached financial statements.

Benefit from income taxes for the quarter ended June 30, 2023 was $2 million, compared to a provision for income taxes of $26.6 million for the same quarter in 2022.

The Company reported a net loss of $9.4 million for the quarter ended June 30, 2023, or a net loss of ($7.55) per share on both a basic and diluted basis. Net loss in the same quarter of 2022 was $46.7 million, or a net loss of ($54.01) per share on both a basic and diluted basis.

At June 30, 2023, the Company had cash, cash equivalents, and restricted cash of $26.4 million, compared to $44.7 million at year end 2022.

2023 Financial Guidance

For the full year 2023, the Company revised INBRIJA U.S. net revenue guidance to be $34-$38 million, from $38-$42 million. Adjusted OPEX guidance was revised to be $93-$98 million, from $93-$103 million. Ending cash balance guidance was revised to be $39-44 million, from $43-$47 million. The Company reaffirms guidance for AMPYRA net revenue to be $65-$70 million. The Company does not expect to be cash flow neutral in 2023.

Reverse Split and Nasdaq Minimum Bid Price Compliance

On June 2, 2023, the Company completed a 1-for-20 reverse stock split of its outstanding and authorized shares of common stock, and it began trading on a split-adjusted basis at the market open on June 5, 2023. The Company subsequently received notification from the Nasdaq Stock Market that as of June 20, 2023 it had regained compliance with the minimum bid price requirement set forth in Nasdaq Listing Rule 5450(a)(1), and the common stock continues to be listed and traded on the Nasdaq Global Select Market.

Board of Directors

At Acorda’s Annual Meeting of Stockholders on June 22, 2023, Tom Burns, the Senior Vice President of Finance and Chief Financial Officer of XOMA Corporation, was elected to the Company’s Board of Directors. Jeff Randall, who had served on Acorda’s Board since 2006, rotated off the Board.

Webcast and Conference Call

The Company will host a webcast/conference call in conjunction with its second quarter 2023 update and financial results today at 4:30 p.m. ET.

To participate in the Webcast, please use the following registration link:

View Source
If you register for the Webcast, you will have the opportunity to submit a written question for the Q&A portion of the presentation. After you have registered, you will receive a confirmation email with the Webcast details. On the day of the Webcast, you will receive an email 2 hours prior to the start of the Webcast with the link to join. The presentation will be available on the Investors section of www.acorda.com.

A replay of the call will be available from 8:30 p.m. ET on August 8, 2023, until 11:59 p.m. ET on September 7, 2023. To access the replay, please dial 1 866 813 9403 (domestic) or +1 929 458 6194 (international); access code 649308. The archived webcast will be available in the Investor Relations section of the Acorda website at www.acorda.com.

On August 8, 2023 Abeona Therapeutics Inc. (Nasdaq: ABEO) reported financial results for the second quarter of 2023 and provided an update on progress toward achieving key corporate objectives (Press release, Abeona Therapeutics, AUG 8, 2023, View Source [SID1234633948]).

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"Abeona is at a pivotal moment in our history as we are busy finalizing and planning to soon submit the BLA for EB-101 for the treatment of recessive dystrophic epidermolysis bullosa," said Vish Seshadri, Chief Executive Officer of Abeona. "The recent capital raise allows us to initiate preparations for the commercial launch of EB-101 in the U.S., while also extending our cash runway well beyond the anticipated regulatory approval time for EB-101."

Second Quarter and Recent Progress

Strengthened balance sheet, preparing for commercialization of EB-101 in the U.S.

● In July, Abeona raised $25 million in a registered direct offering priced at-the-market with select existing investors to primarily fund initiation of the Company’s launch preparations in anticipation of the EB-101 BLA submission and potential approval.

EB-101 for recessive dystrophic epidermolysis bullosa (RDEB)

● Completed the Process Performance Qualification manufacturing runs for both retroviral vector (RVV) and EB-101 drug product manufacturing to demonstrate Abeona’s validated process and readiness for commercial production. This data will be included in the chemistry, manufacturing, and control (CMC) module for the EB-101 BLA submission.
● Generated the additional data requested by the U.S. Food and Drug Administration (FDA) that supports comparability between two RVV sources used in EB-101 clinical studies. This data will be included in the CMC module for the EB-101 BLA submission.
● Abeona submitted the briefing package for the pre-BLA meeting with the FDA in August 2023 for its anticipated BLA submission of EB-101 in the treatment of RDEB. Abeona anticipates submitting the BLA in the third quarter of 2023.
● Additional efficacy and safety data from the pivotal Phase 3 VIITAL study of investigational EB-101 in RDEB were presented at the International Societies for Investigative Dermatology (ISID) and Society for Pediatric Dermatology (SPD) meetings. The positive top-line data from the VIITAL study were reported in November 2022.

Preclinical programs

● Completed pre-Investigational New Drug Application (IND) meetings with the FDA regarding the preclinical development plans and regulatory requirements to support first-in-human trials for two preclinical gene therapy product candidates from its adeno-associated virus (AAV) ophthalmology program. The Company intends to initiate IND-enabling studies in the second half of 2023.
● Abeona presented new preclinical data at the 26th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT) (Free ASGCT Whitepaper), including data on three internally developed preclinical gene therapy product candidates from its AAV ophthalmology program.

Second Quarter Financial Results

Cash, cash equivalents, restricted cash and short-term investments totaled $37.1 million as of June 30, 2023, prior to the $25 million registered direct offering in July 2023, as compared to $40.7 million as of March 31, 2023. Abeona estimates that its cash and cash equivalents, restricted cash and short-term investments as of June 30, 2023, in addition to the proceeds from the $25 million registered direct offering in July 2023, are sufficient resources to fund operations into the fourth quarter of 2024.

License and other revenues in the second quarter of 2023 were $3.5 million, compared to $1.0 million in the second quarter of 2022. Research and development expenses for the three months ended June 30, 2023 were $8.5 million, compared to $6.7 million for the same period of 2022. General and administrative expenses were $5.0 million for the three months ended June 30, 2023, compared to $3.5 million for the same period of 2022. Net loss attributable to common shareholders was $16.7 million for the second quarter of 2023, or $0.92 loss per common share as compared to a net loss attributable to common shareholders of $7.9 million, or $1.36 loss per common share, in the second quarter of 2022.

Conference Call Details

Abeona Therapeutics will host a conference call and webcast today, August 8, 2023, at 8:30 a.m. ET, to discuss its 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: 446072 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 August 8, 2023 Race Oncology Limited ("Race") reported to shareholders with a strategic update, including an overview of revisions to corporate strategy, designed to optimise use of existing resources, while driving bisantrene’s commercial partnering and collaboration potential (Press release, Race Oncology, AUG 8, 2023, View Source [SID1234633884]).

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Key highlights:

New corporate strategy launched, detailing Race’s optimised clinical program and key areas of discovery, placing Race at the heart of cancer care
Lead clinical focus on investigating anti-cancer, plus cardio-protective and FTO properties in metastatic breast cancer, using the peripherally administered bisantrene formulation, RC220 with a view to improving therapeutic outcomes and heart health for patients with cancer
Secondary focus on pursuing increased anti-cancer / FTO benefits of bisantrene combined with current standard of care therapies in acute myeloid leukaemia (AML)
Targeted R&D program designed to maximise bisantrene’s partnering potential
Video update, presentation and shareholder information sessions available to provide additional background and offer Q+A opportunities
Investors are also invited to visit the Interactive Announcements page in Race’s Investor Hub to submit questions View Source
Read the strategic update summary and investor presentation here.

Non-Executive Chair, Mary Harney commented: "Through the course of conducting this review of strategy, Race drew upon a rich set of insights. We further reviewed our deep historic clinical dataset, as well as many preclinical studies across a range of indications. We investigated the many market opportunities for bisantrene and engaged with key opinion leaders to inform our view of unmet patient need. Finally, and of critical importance, we reviewed the paths to market which would position our lead asset most strongly with potential partners.

This strategic update delivers committed and planned programs, designed to optimally leverage bisantrene RC220’s potential in the areas of anti-cancer, cardio-protection and FTO inhibition. This has been a significant body of work – I thank the Race team for their dedication in driving toward the best outcome and am also grateful for the ongoing interest and support of our shareholders."

Race CEO and Managing Director, Damian Clarke-Bruce commented: "Race’s revised development program has been designed in such a way that our new bisantrene formulation can fit straight into a global pharmaceutical partner’s pipeline. Through our rigorous clinical development plan, Race will investigate bisantrene RC220 in both metastatic breast cancer and continue our clinical experience in AML, while investigating and expanding our knowledge of its mechanism of action. We have a clear regulatory pathway and a program that is supported by international key opinion leaders, for a drug candidate which has already been shown to improve patients’ lives.

We look forward to presenting the strategy to existing and potential new shareholders over the coming weeks as we seek to advance our position as a commercial partner with a life-changing therapeutic asset in bisantrene RC220."

On August 7, 2023 Aichi Cancer Center and NEC Corporation’s research group with Gifu University, Toyama University and Kitasato University Medical Center, reported to have developed a method for efficiently identifying the lung cancer antigens and the antigen-specific T cells that recognize the antigens through both a single-cell analysis of Tumor Infiltrating Lymphocytes (TILs) and NEC’s AI-based antigen prediction system that predicts immune response (Press release, NEC, AUG 8, 2023, View Source [SID1234633878]). Our paper describing the results of this study was published on August 6, 2023 in the "Journal for ImmunoTherapy of Cancer," which is the official journal of the Society of Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) in the United States.

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■ Research Background
Lung cancer is one of the most common cancers and one of the leading causes of cancer death worldwide. There are many types of cancer treatment, such as surgery, chemotherapy, radiation therapy, molecular targeted therapy, immunotherapy, and combinations of these. Recently developed immune checkpoint inhibitors (ICI) have attracted attention as a new therapy, and lung cancer is one of the most sensitive cancers to ICI, but it is effective in only a subset of individuals. Accordingly, new effective immunotherapies are required for lung cancer.

Cytotoxic T lymphocytes (CTLs) in TIL are crucial immune cells that can specifically recognize and eliminate tumor cells. Antigens targeted by CTL include patient-specific neoantigens and common antigens commonly expressed among patients such as cancer-testis antigens (CTA). In general, it is not easy to identify any antigens. If these antigens can be efficiently identified, a combination therapy with ICIs and antigen-specific immunotherapy may enhance the efficacy of treatment.

■ Contents and results of this research
In this study by Aichi Cancer Center and NEC, we performed a single-cell analysis to determine the TILs characteristics of patients with surgically resected non-small cell lung cancer (NSCLC) (n=3) (Figure. 1). Then, we divided the TILs into 10 clusters based on gene expression profile, and identified the exhausted T cell cluster (Tex cluster) characterized by the expression of the genes called exhaustion markers (Figure. 2). We synthesized the TCRs contained in the identified exhausted T cell cluster and induced each of the TCRs into each corresponding T cell, and examined the immune responses to neoantigens predicted by NEC’s AI-based antigen prediction system and typical CTAs. It was confirmed that NEC’s AI-based antigen prediction system can accurately predict the antigens that cause the immune responses, and we identified four TCRs recognizing KK-LC-1 (one of the CTAs, *2), and five TCRs recognizing the neoantigens.

In addition, by re-clustering of TCR clones (n=140) that express nine TCRs, it was discovered that even antigen-specific TCR clones have different differentiation stage and functional status among individual TCR clones (Figure 4A), and that there is a bias in differentiation and function of TCRs for each antigen.

TIL single-cell analysis and AI to predict cancer antigens will facilitate the identification of lung cancer antigens and may lead to the development of personalized cancer vaccine therapies and engineered T cell therapies in NSCLC in the future.

■Researcher’s comment

Dr. Hirokazu Matsushita, Chief, Division of Translational Oncoimmunology, Aichi Cancer Center
In collaboration with NEC and leading research institutes, the Aichi Cancer Center has developed a procedure for efficiently identifying antigens and antigen-specific T cells using surgical samples from patients with cancer. Looking ahead, we will add a spatial analysis of the cancer microenvironment that contains antigens and antigen-specific T-cells to this system to further clarify the nature of the T-cells infiltrating the cancer. In addition to aiming to develop innovative cancer immunotherapies from these studies, we will apply the knowledge gained to other cancers as well.
Yoshiko Yamashita, Ph.D. Senior Professional, AI Drug Development Division, NEC Corporation
NEC is conducting clinical trials of personalized cancer vaccine therapies targeting neoantigens. We believe that we have taken a step forward in realizing even more sophisticated personalized cancer vaccine immunotherapies and engineered T cell therapies by using a method to identify antigen-specific T cells constructed in this study and an Attentive Variational Information Bottleneck (AVIB) method (*3) to predict interactions between TCRs and antigens using AI newly developed by NEC. We will continue to accelerate research and development to provide effective treatments to patients.

■Research support

Aichi Cancer Center Priority Project Research
NEC Corporation
Subsidy Program for Scientific Research by the Japan Society for the Promotion of Science
Japan Respiratory Foundation
Uehara Memorial Life Science Foundation

On August, 7, 2023 DotBio, the biopharmaceutical company pushing the boundaries of antibody therapeutic modalities to bring more effective therapies to patients, reported the closing of a USD 5.6 million Pre-Series A financing round (Press release, DotBio, AUG 7, 2023, View Source [SID1234646735]). The oversubscribed round was led by specialist life sciences investors Proxima Ventures, Gaorong Capital and AIM-HI Accelerator Fund.

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The funds will be used to commence pre-clinical studies, including animal efficacy and Chemical, Manufacturing and Controls studies, that will validate DotBio’s therapeutic candidates ahead of future clinical development. In addition, DotBio’s proprietary, modular DotBody platform will be scaled and automated to allow for the screening of thousands of multi-functional antibody prototypes against diverse extracellular and intracellular immune-oncology targets that are suited as next-generation clinical candidates.

Proxima Ventures, a specialist seed-to-growth healthcare Venture Capital firm based in Shanghai, led the financing. Haolin Sung, EMBA, a serial biotech entrepreneur and Partner of Proxima Ventures, joins DotBio as a member of the Board. Gaorong Capital is focused on early and growth-stage investments, with a specialty in new consumption, new technology and healthcare. Dr Beilin Le, PhD, an Executive Director of Gaorong Capital’s Healthcare Division, will join DotBio as a Board Observer. Dr Sujuan Ba, PhD, Co-Founder and CEO of AIM-HI Accelerator Fund, a US-based global not-for-profit organization dedicated to promoting breakthrough technologies and therapies in the cancer space, will join DotBio as a Strategic Advisor.

Ignacio Asial, Chief Executive Officer at DotBio, commented: "We are pleased to be supported by leading life science investors to close our pre-Series A financing round, allowing us to accelerate the development of our internal pipeline of multi-functional antibodies with the potential to treat a wide range of cancers and improve patient lives. We are making significant progress in developing intracellular antibody therapies, work that was highlighted in our Nature Communications publication in 2022. This round will allow us to accelerate these efforts, with the aim of revolutionizing how we target intracellular oncogenes. With partnered programs expected to enter the clinic in the near future, our humanized domain antibody continues to be de-risked, allowing us to develop more ambitious therapeutic modalities. I’m very pleased to welcome Mr. Haolin Sung to our Board of Directors, whose global experience in biotech venture creation, management, and funding will be of great value as we enter this next phase of growth."

Haolin Sung, Partner at Proxima Ventures, replied: "I’m pleased to work with the DotBio team at a time when new, multi-targeted therapeutic modalities are gaining global momentum and pushing the boundaries of what is possible in therapeutic discovery. DotBio’s approach to identifying and derisking multi-specific antibodies by comparing thousands of prototype molecules in a data-driven approach is at the cutting edge of science. I’m excited about the Company’s modular technology platform and believe it will open up unprecedented possibilities for patients around the world through the facilitation of rapid prototyping of potential new therapies that target both extracellular and intracellular disease drivers. I look forward to supporting the company on its mission to deliver more effective oncology therapies that can reach patients faster."

Beilin Le, Executive Director at Gaorong Capital, added: "The DotBio team has demonstrated that their humanized domain antibody technology platform is unique, differentiated, and has the potential to revolutionize the multi-functional antibody space. Their partnerships with CStone Pharmaceuticals, Junshi Biosciences, and Uni-Bio Science Group highlight the company’s value and expertise. Remarkably, DotBio’s out-licensing of a DotBody module to Junshi Biosciences in 2022 resulted in the rapid development of JS207, a bi-specific antibody for oncology indications, which has already progressed to Investigational New Drug (IND) filing in 2023. As JS207 moves through clinical trials, it will further validate the effectiveness of DotBody technology. I can’t wait to see the company scale and advance its internal pipeline".