On August 8, 2023 Astex Pharmaceuticals (UK) ("Astex"), a pharmaceutical company dedicated to the discovery and development of novel small molecule therapeutics for oncology and diseases of the central nervous system, reported an exclusive worldwide research collaboration and license agreement with MSD (Merck & Co., Inc., Rahway, N.J., USA) (Press release, Astex Pharmaceuticals, AUG 8, 2023, View Source [SID1234633952]). The goal of the collaboration is to identify small molecule candidates with activity towards a tumour suppressor protein for the treatment of cancer.

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"Astex is applying its fragment-based drug discovery capability to design and generate small molecule modulators tailored to a wide range of potentially important therapeutic targets," said Harren Jhoti, president and CEO of Astex. "This new alliance builds on our existing productive collaboration and allows us to combine our expertise and assets with MSD’s wide-ranging oncology capabilities."

Under the terms of the agreement, Astex will apply its fragment-based drug discovery platform to develop compounds targeting multiple forms of the p53 tumour suppressor protein and provide MSD with lead compounds for further optimization and preclinical development. MSD is granted an exclusive global license to research, develop, and commercialise candidates arising under the collaboration. Astex will receive an upfront payment of $35 million and is eligible for milestone payments associated with the achievement of preclinical, clinical, regulatory and sales milestones, totalling approximately $500 million per program, as well as tiered royalties on sales of any products arising from the collaboration. MSD will assume responsibility for funding all future research and development of lead candidates as well as commercialisation of products globally.

"At MSD we are committed to driving innovation with the goal of improving outcomes for patients with cancer," said George Addona, senior vice president discovery and translational medicine, MSD. "We look forward to building on our collaboration with the Astex team to advance this potentially impactful area of oncology research."

On August 8, 2023 Arvinas, Inc. (Nasdaq: ARVN), a clinical-stage biotechnology company creating a new class of drugs based on targeted protein degradation, reported financial results for the second quarter ended June 30, 2023 and provided a corporate update (Press release, Arvinas, AUG 8, 2023, View Source [SID1234633951]).

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"We continue to make meaningful advances across our entire portfolio and have a data and milestone rich balance to the year," said John Houston, Ph.D., president and chief executive officer at Arvinas. "In addition to sharing promising interim data from our ongoing Phase 1/2 trial with ARV-766, and together with Pfizer, we initiated enrollment for the study lead-in for the first-line Phase 3 trial with vepdegestrant in combination with palbociclib in metastatic breast cancer. We now have two ongoing Phase 3 trials with vepdegestrant with the potential for our first Phase 3 data read-out in the second half of 2024. We are also on track to initiate a Phase 3 trial with bavdegalutamide in the second half of the year. These accomplishments are particularly meaningful as we celebrate our 10-year anniversary this year and reflect our commitment to bringing an entirely new class of transformative medicines to patients."

Recent Developments and Second Quarter Business Highlights

Initiated the study lead-in of the VERITAC-3 Phase 3 trial of vepdegestrant plus palbociclib as a first-line treatment in patients with ER+/HER2- locally advanced or metastatic breast cancer.
Shared data from the Phase 1/2 dose escalation and expansion trial showing that ARV-766 was well-tolerated and demonstrated promising activity in a heavily pre-treated, post-NHA, all-comers patient population:
42% of patients with AR ligand binding domain (LBD) mutations achieved PSA50.
In all patients with L702H mutations, 3 of 5 achieved PSA50; in patients with co-occurring T878/H875/L702 mutations, 3 of 3 achieved PSA50.
RECIST (Response Evaluation Criteria in Solid Tumors) partial responses were observed.
Of four RECIST-evaluable patients with AR LBD mutations, one achieved a confirmed partial response, and one achieved an unconfirmed partial response.
ARV-766 was well tolerated and the majority of treatment-related adverse events (TRAEs) have been Grade 1 or 2, with no Grade ≥4 TRAEs and no dose limiting toxicities.
Low rates of discontinuation (1 of 47) and dose reductions (2 of 47) were observed.
Evaluated and announced preliminary data from Part C of the ongoing Phase 1b/2 ARV-471-mBC-101 study (ClinicalTrials.gov Identifier: NCT04072952).
Continued enrollment in the VERITAC-2 Phase 3 2L+ clinical trial of vepdegestrant as a monotherapy for the treatment of patients with ER+/HER2- metastatic breast cancer (ClinicalTrials.gov Identifier: NCT05654623).
Continued enrollment in the TACTIVE-U study (vepdegestrant in combination with abemaciclib or ribociclib, [ClinicalTrials.gov Identifiers: NCT05548127 and NCT05573555]) and, with Pfizer, entered a collaboration and supply agreement with Carrick Therapeutics to evaluate samuraciclib in combination with vepdegestrant in the TACTIVE-U study.
Continued enrollment in the TACTIVE-E study (vepdegestrant in combination with everolimus; ClinicalTrials.gov Identifier: NCT05501769), and the TACTIVE-N study (vepdegestrant as a monotherapy in the neoadjuvant setting; ClinicalTrials.gov Identifier: NCT05549505).
Announced the inclusion of vepdegestrant in the I-SPY-2 (Investigation of Serial studies to Predict Your Therapeutic Response with Imaging And moLecular analysis 2) trial sponsored by Quantum Leap. The I-SPY-2 Endocrine Optimization Platform (EOP) study (ClinicalTrials.gov Identifier: NCT01042379) includes a vepdegestrant monotherapy arm and a vepdegestrant plus letrozole arm.
Awarded Innovation Passport Designation for vepdegestrant by the U.K. Innovative Licensing and Access Pathway Steering Group.
Announced that John Houston, Ph.D., was named Chairperson replacing Timothy Shannon, M.D., who stepped down from the role. Additionally, Sunil Agarwal, M.D., was appointed to join the company’s Board of Directors and Briggs Morrison, M.D., was appointed Lead Independent Director of the Board.
Anticipated Upcoming Milestones and Expectations

Vepdegestrant (ARV-471)
As part of Arvinas’ global collaboration with Pfizer, the companies plan to:

Present additional data from the Phase 1b combination trial with palbociclib (ClinicalTrials.gov Identifier: NCT04072952) at a medical congress (2H 2023).
Continue enrollment in the study lead-in (SLI) of the VERITAC-3 Phase 3 trial in first-line ER+/HER2- locally advanced or metastatic breast cancer. The SLI will identify the palbociclib dose (75 or 100 mg) to combine with vepdegestrant in the randomized portion of the study.
Initiate Phase 1b/2 trial with vepdegestrant plus Pfizer’s CDK4 (cyclin dependent kinase) inhibitor (2H 2023).
Initiate an additional arm of the Phase 1b/2 combination umbrella trial (TACTIVE-U: ClinicalTrials.gov Identifiers: NCT05548127 and NCT05573555) with Carrick Therapeutics’ CDK7 inhibitor (2H 2023).
Complete enrollment in VERITAC-2 Phase 3 monotherapy trial (ClinicalTrials.gov Identifier: NCT05654623) in patients with metastatic breast cancer (2H 2024).
Androgen Receptor (AR) Franchise (Bavdegalutamide/ARV-110, ARV-766)

Present updated data, including radiographic progression free survival, from the ongoing Phase 1/2 trial with bavdegalutamide at the European Society for Medical Oncology congress in Madrid (October 2023).
Initiate a global Phase 3 trial with bavdegalutamide in mCRPC (2H 2023).
Complete enrollment in the Phase 1b combination study with bavdegalutamide plus abiraterone (2H 2023).
Initiate a Phase 1b/2 dose escalation trial with ARV-766 in combination with abiraterone in patients who have not previously received novel hormonal agents (2H 2023).
Complete enrollment in the Phase 1b combination study with bavdegalutamide plus abiraterone (2H 2023).
Pipeline:

Submit two investigational new drug (IND)/clinical trial authorization (CTA) applications for the Company’s BCL6 (oncology) and LRRK2 (neuroscience) PROTAC protein degraders by year-end 2023.
Progress at least two additional PROTAC protein degrader programs into IND- or CTA-enabling studies by year-end 2023.
Financial Guidance
Based on its current operating plan, Arvinas believes its cash, cash equivalents, restricted cash and marketable securities as of June 30, 2023, is sufficient to fund planned operating expenses and capital expenditure requirements into 2026.

Second Quarter Financial Results

Cash, Cash Equivalents and Marketable Securities Position: As of June 30, 2023, cash, cash equivalents, restricted cash and marketable securities were $1,044.3 million as compared with $1,210.8 million as of December 31, 2022. The decrease in cash, cash equivalents, restricted cash and marketable securities of $166.5 million for the six months ended June 30, 2023 was primarily related to cash used in operations of $172.9 million (net of $2.5 million received from two collaborators), leasehold improvements of $1.7 million and loss on the sale of marketable securities of $0.9 million, partially offset by unrealized gains on marketable securities of $7.0 million and proceeds from the exercise of stock options of $2.0 million.

Research and Development Expenses: Research and development expenses were $103.4 million for the quarter ended June 30, 2023, as compared with $75.3 million for the quarter ended June 30, 2022. The increase in research and development expenses of $28.1 million for the quarter was primarily due to increasing investment in our platform and exploratory programs of $5.4 million, as well as an increases our AR program of $7.4 million, which includes bavdegalutamide and ARV-766, and our ER program of $15.3 million, which is net of the cost sharing of vepdegestrant (ARV-471) under the global Pfizer collaboration agreement to develop and commercialize vepdegestrant that was initiated in July 2021 (Vepdegestrant (ARV-471) Collaboration Agreement).

General and Administrative Expenses: General and administrative expenses were $25.7 million for the quarter ended June 30, 2023, as compared with $24.3 million for the quarter ended June 30, 2022. The increase of $1.4 million was primarily due to an increase in professional fees of $2.0 million and increased investments in our commercial operations of $0.9 million, offset in part by reduced personnel and infrastructure related costs of $1.1 million and reduced insurance costs of $0.3 million.

Revenues: Revenues were $54.5 million for the quarter ended June 30, 2023 as compared with $33.8 million for the quarter ended June 30, 2022. Revenue is related to the Vepdegestrant (ARV-471) Collaboration Agreement, the license and rights to technology fees and research and development activities related to the collaboration and license agreement with Bayer that was initiated in July 2019, the collaboration and license agreement with Pfizer that was initiated in January 2018, the amended and restated option, license and collaboration agreement with Genentech that was initiated in November 2017 and revenue related to our Oerth Bio joint venture which was initiated in July 2019. The increase in revenues of $20.7 million was primarily due to an increase in revenue from the Vepdegestrant (ARV-471) Collaboration Agreement totaling $24.2 million, partially offset by a net decrease in revenue of $1.8 million as the performance period under the collaboration agreement with Genentech has concluded and a decrease of $1.2 million of previously constrained deferred revenue related to our Oerth Bio joint venture.

Income Tax Expense: Income tax benefit was $0.3 million for the quarter ended June 30, 2023, as compared with an income tax expense of $3.4 million for the quarter ended June 30, 2022. Current year tax benefit was driven by expected benefits from state net operating loss carryback claims. Prior year tax expense was driven by revenue recognized in 2022 for tax purposes from the Vepdegestrant (ARV-471) Collaboration Agreement.

Loss from Equity Method Investment: Loss from equity method investment was $1.3 million for the quarter ended June 30, 2023, as compared with $2.5 million for the quarter ended June 30, 2022 due to decreased operating losses incurred by Oerth Bio.

Net Loss: Net loss was $66.6 million for the quarter ended June 30, 2023, as compared with $70.0 million for the quarter ended June 30, 2022. The decrease in net loss for the quarter was primarily due to increased revenue and interest income from our marketable securities, as well as decreased income tax expense, partly offset by increased research and development expenses and general and administrative expenses.

About bavdegalutamide (ARV-110) and ARV-766
Bavdegalutamide (ARV-110) and ARV-766 are investigational orally bioavailable PROTAC protein degraders designed to selectively target and degrade the androgen receptor (AR). Bavdegalutamide and ARV-766 are being developed as potential treatments for men with prostate cancer. Preclinically, both investigational agents have demonstrated activity in models of wild type tumors in addition to tumors with AR mutation or amplification, both common mechanisms of resistance to currently available AR-targeted therapies.

About vepdegestrant (ARV-471)
Vepdegestrant is an investigational, orally bioavailable PROTAC protein degrader designed to specifically target and degrade the estrogen receptor (ER) for the treatment of patients with early and locally advanced or metastatic ER positive/human epidermal growth factor receptor 2 (HER2) negative (ER+/HER2-) breast cancer. Use of vepdegestrant in the ongoing and planned clinical trials will continue to monitor and evaluate patient safety and anti-tumor activity.

In preclinical studies, vepdegestrant demonstrated up to 97% ER degradation in tumor cells, induced tumor shrinkage when dosed as a single agent in multiple ER-driven xenograft models, and showed increased anti-tumor activity when compared to a standard of care agent, fulvestrant, both as a single agent and in combination with a CDK4/6 inhibitor. In July 2021, Arvinas announced a global collaboration with Pfizer for the co-development and co-commercialization of vepdegestrant; Arvinas and Pfizer will equally share worldwide development costs, commercialization expenses, and profits.

On August 8, 2023 Agenus Inc. ("Agenus") (Nasdaq: AGEN), a leader in developing novel immunological agents to treat various cancers, reported results for the second quarter 2023 (Press release, Agenus, AUG 8, 2023, View Source [SID1234633950]).

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Key accomplishments and highlights include:

Botensilimab Update: Botensilimab/balstilimab combination data presented at ESMO (Free ESMO Whitepaper)-GI demonstrated median overall survival (mOS) of 20.9 months and a 23% overall response rate (ORR), both surpassing data reported for standard of care in 3L+ Non-MSI-H colorectal cancer (CRC) in patients without active liver metastases.
Regulatory: The U.S. FDA granted Fast Track designation for the botensilimab/balstilimab combination in 3L+ Non-MSI-H CRC in patients without active liver metastases. Agenus is exploring global accelerated approval strategies for CRC.
Clinical Trial Progress: Ongoing Phase 2 ACTIVATE trials in CRC and melanoma expected to fully enroll by end of year.
AGEN2373 Update: AGEN2373 Phase 1 dose escalation data presented at ASCO (Free ASCO Whitepaper) demonstrated promising monotherapy clinical responses in patients with advanced solid tumors.
"Botensilimab, alone or in combination with balstilimab, continues to display remarkable clinical activity in over 600 patients treated across nine late-stage, treatment resistant solid tumor cancers, demonstrating great potential to revolutionize the role of immunotherapy in cancer treatment," said Chief Executive Officer, Garo Armen, Ph.D. "Agenus is committed to advancing our diverse clinical pipeline with a focus on expediting our first regulatory submission for the botensilimab/balstilimab combination in colorectal cancer. Our data has demonstrated an unprecedented survival benefit over what has been reported for standard of care, underscoring this combination as an important potential treatment option for patients with non-MSI-high colorectal cancer, which represents 85% of the population of patients with colorectal cancer1."

Botensilimab/Balstilimab ("Bot/Bal") Combination in 3L+ Non-MSI-H Metastatic CRC Data Presented at the ESMO (Free ESMO Whitepaper)-GI World Congress on Gastrointestinal Cancer in June 2023:

In 69 evaluable patients with non-MSI-High CRC without active liver metastases, results demonstrated a meaningful improvement over standard of care in both overall response rate (ORR%) and median overall survival (mOS):
mOS of 20.9 months as compared to the recently reported 12.9-month benchmark with standard care.2
ORR of 23% as compared to the recently reported 2.8%1 benchmark with standard care.
In April 2023, the U.S. FDA granted Fast Track designation to the bot/bal combination for patients with non-MSI-H/dMMR metastatic CRC without active liver metastases.
The ACTIVATE Phase 2 trial in this patient population is on target to conclude enrollment by year-end. Planning is underway for a Phase 3 CRC study; with Fast Track designation from the FDA, study design and timing will be determined as we discuss our filing approach with the agency.
A Biologics License Application to the FDA is planned in 2024.
Botensilimab in Other Advanced Solid Tumor Cancers:

Lung: Enrollment continues for a Phase 1b trial targeting PD-(L)1 +/- chemotherapy relapsed/refractory non-small-cell lung cancer (NSCLC); data update planned by year-end.
Melanoma: Ongoing global enrollment in Phase 2 ACTIVATE trial with data update planned in 2024.
Ovarian: Phase 1b trial in patients with platinum-resistant ovarian cancer (PROC) demonstrated a 33% ORR and 67% disease control rate among primarily high-grade, heavily pretreated patients (presented at SGO in March 2023).
Sarcomas: Updated data for a Phase 1b cohort with advanced sarcomas to be presented at ESMO (Free ESMO Whitepaper) on October 21st 2023.
AGEN2373 Data Presented at the ASCO (Free ASCO Whitepaper) 2023 Annual Meeting:

Complete results from the first-in-human monotherapy dose escalation study of AGEN2373, a CD137 agonist, in patients with advanced solid tumors:
Reported responses in metastatic prostate cancer, ampullary carcinoma, and vulvar squamous cell carcinoma highlight the potential impact of AGEN2373.
AGEN2373 is the first CD137 agonist antibody to report single-agent responses with no major toxicities.
Clinical data are consistent with mechanistic design intent of AGEN2373 to avoid hepatic toxicity, a typical constraint of CD137-targeted agonists.
Leadership Appointments:

In alignment with Agenus’ next phase of growth and anticipated commercialization of the bot/bal combination, the company announced the following key executive appointments:

Robin Taylor, Ph.D., Chief Commercial Officer.
Homa Yeganegi, Chief Product Strategy and Global Medical Affairs Officer.
Stephanie Fagan, Chief Communications Officer.
Second Quarter 2023 Financial Results:

We ended our second quarter 2023 with a cash, cash equivalent and short-term investment balance of $157.6 million, compared to $193.4 million at December 31, 2022. In addition, we have raised $20.3 million through sales of our common stock under our at market issuance sales agreement since the end of Q2 2023.

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.