On May 11, 2023 Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a leader in the field of cellular metabolism pioneering therapies for rare diseases, reported that the company and its collaborators will present a broad range of clinical and translational data at the European Hematology Association (EHA) (Free EHA Whitepaper) Hybrid Congress, hosted virtually and in person in Frankfurt, Germany, June 8-11, 2023, and continuing virtually on the Congress platform June 14-15, 2023 (Press release, Agios Pharmaceuticals, MAY 11, 2023, View Source [SID1234631478]).

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The accepted abstracts are listed below and are available online on the EHA (Free EHA Whitepaper) meeting library website. All posters will be presented during the poster session on Friday, June 9 at 16:30-17:45 CEST. All presentations can be accessed on demand by registered meeting attendees on the EHA (Free EHA Whitepaper) Virtual Congress platform until Aug. 15, 2023.

Agios-led Abstracts:

Poster Presentations

Title: Healthcare Resource Use, Economic Burden and In-Patient Mortality in Patients with Alpha- and Beta-Thalassemia Compared to Matched Controls in the Real-World Setting
Abstract: P1463
Presenting Author: Louise Lombard, M Nutr, Agios Pharmaceuticals, Cambridge, MA, United States

Title: Clinically Relevant Hemoglobin Response in Adults with Pyruvate Kinase Deficiency Treated with Mitapivat – A Sub-Analysis of the ACTIVATE Trial
Abstract: P1473
Presenting Author: Hanny Al-Samkari, M.D., Division of Hematology, Massachusetts General Hospital, Harvard Medical School, Boston, MA, United States

Title: The Clinical Characteristics and Overall Survival of Pyruvate Kinase Deficiency Patients in the UK: A Real-World Study
Abstract: P1476
Presenting Author: Sarah Higa, PharmD, M.S., Agios Pharmaceuticals, Cambridge, MA, United States

Title: Mitapivat Efficacy in Adults with Pyruvate Kinase Deficiency and Baseline Hemoglobin Levels >10 g/dL
Abstract: P1477
Presenting Author: Rachael F. Grace, M.D., Dana-Farber/Boston Children’s Cancer and Blood Disorders Center, Harvard Medical School, Boston, MA, United States

Title: Comorbidities and Complications in Adults with Pyruvate Kinase Deficiency According to Hemoglobin Strata – A Descriptive Analysis from the Peak Registry
Abstract: P1479
Presenting Author: Dagmar Pospíšilová, M.D., Ph.D., Department of Pediatrics, Palacky University and University Hospital, Olomouc, Czech Republic

Title: PKM And PKR Expression During Hematopoiesis and Erythropoiesis
Abstract: P1485
Presenting Author: Erin Tsai, M.S., Agios Pharmaceuticals, Cambridge, MA, United States

Title: Mitapivat Improves Iron Overload in Patients with Pyruvate Kinase Deficiency Who Are Regularly Transfused
Abstract: P1497
Presenting Author: Eduard J. van Beers, M.D., Center for Benign Haematology, Thrombosis and Haemostasis, Van Creveldkliniek, University Medical Center Utrecht, Utrecht University, Utrecht, The Netherlands

Publication Only

Title: Estimating Utility Values for Health States in Pyruvate Kinase Deficiency
Abstract: PB2547
Lead Author: Sarah Higa, PharmD, M.S., Agios Pharmaceuticals, Cambridge, MA, United States

Collaborator-led Abstracts:

Poster Presentations

Title: AG946, A Pyruvate Kinase (PK) Activator Improves PK Properties and Red Blood Cell (RBC) Metabolism Upon Ex Vivo Treatment of RBCs from Patients with Myelodysplastic Syndromes
Abstract: P717
Presenting Author: Jonathan de Wilde, MSc, University Medical Center Utrecht, Utrecht University, Utrecht, Netherlands

Title: One-Year Follow-Up of a Phase 2 Study of Mitapivat, an Oral Pyruvate Kinase Activator, for the Treatment of Sickle Cell Disease
Abstract: P1424
Presenting Author: Myrthe J. van Dijk, Ph.D., Division Laboratories, Pharmacy and Biomedical Genetics, CDL en Van Creveldkliniek , University Medical Center Utrecht, Utrecht University, Utrecht, Netherlands

Title: Rare Anaemia Disorders European Epidemiological Platform (RADeep): Distribution of Patients Affected by RADs in Europe
Abstract: P1427
Presenting Author: Maria Del Mar Mañú Pereira, Ph.D., Vall d’Hebron University Hospital, Group of Translational Research in Cancer and Blood disorders in Children, Barcelona, Spain

Title: Mitapivat Ameliorates In Vitro Human β Thalassemic Erythroid Maturation Index and Modulates the Expression of Peroxiredoxin-2
Abstract: P1458
Presenting Author: Lucia De Franceschi, M.D., Department of Medicine, University of Verona, and Azienda Ospedaliera Universitaria Verona, Policlinico GB Rossi, Verona, Italy

Publication Only

Title: Glycolytic Activity and Effect of Ex-Vivo Treatment with the Pyruvate Kinase (PK) Activator AG-946 in Red Blood Cells (RBC) From Low-Risk Myelodysplastic Syndromes (LR-MDS) Patients: A Proof-Of-Concept Study
Abstract: PB1990
Lead Author: Bruno Fattizzo, Fondazione IRCCS Ca’ Granda Ospedale Maggiore Policlinico, University of Milan, Milan, Italy

Title: A Pilot Study of the International Hemoglobinopathy Research Network (INHERENT)
Abstract: PB2515
Lead Author: Petros Kountouris, Ph.D., The Cyprus Institute of Neurology and Genetics, Nicosia, Cyprus

On May 11, 2023 Aeglea BioTherapeutics, Inc. (NASDAQ:AGLE) reported financial results for the first quarter 2023 (Press release, Aeglea BioTherapeutics, MAY 11, 2023, View Source [SID1234631477]).

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First Quarter 2023 Financial Results

As of March 31, 2023, Aeglea had available cash and cash equivalents, marketable securities and restricted cash of $39.8 million.

Aeglea recognized development fee and royalty revenues of $0.2 million in the first quarter of 2023, as a result of its license and supply agreement with Immedica Pharma AB for the commercial rights to pegzilarginase in Europe and several countries in the Middle East (License and Supply Agreement). The revenues recorded in the first quarter of 2023 are related to the PEACE Phase 3 trial and royalties from an early access program in France. Aeglea recognized $1.4 million for the first quarter of 2022 in development fee revenues.

Research and development expenses totaled $13.8 million for the first quarter of 2023 and $17.0 million for the first quarter of 2022. The decrease was primarily related to a decrease in activities related to the PEACE Phase 3 trial and Biologics License Application for pegzilarginase.

General and administrative expenses totaled $5.2 million for the first quarter of 2023 and $8.8 million for the first quarter of 2022. This decrease was primarily due to a reduction in headcount and related expenses and decrease in commercialization activities for pegzilarginase.

Net loss totaled $18.4 million and $24.4 million for the first quarter of 2023 and 2022, respectively, which includes non-cash stock compensation expense of $1.7 million and $2.1 million for the first quarter of 2023 and 2022, respectively.

On May 11, 2023 Actinium Pharmaceuticals, Inc. (NYSE AMERICAN: ATNM) (Actinium or the Company), a leader in the development of targeted radiotherapies, reported that an abstract has been accepted for oral presentation at the upcoming European Hematology Association (EHA) (Free EHA Whitepaper) 2023 Congress (EHA) (Free EHA Whitepaper), which will be held in Frankfurt, Germany June 8-11, 2023 (Press release, Actinium Pharmaceuticals, MAY 11, 2023, View Source [SID1234631476]). The abstract includes data from Actinium’s SIERRA controlled phase 3 study comparing the efficacy of Iomab-B based conditioning, a first-in-class targeted radiotherapy, versus physician’s choice of conventional care in older, relapsed/refractory acute myeloid leukemia with active disease. This latest acceptance of the SIERRA trial results at EHA (Free EHA Whitepaper), a major medical conference, is helping spread awareness about the potential of Iomab-B in facilitating transplants for patients who are currently not transplantable.

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Details of the EHA (Free EHA Whitepaper) presentation are as follows:

Presentation Title: SIERRA trial results with a targeted radiotherapy, Iomab-B, a myeloablative conditioning with reduced intensity tolerability yields high CR, long term survival in HSCT ineligible active r/r AML
Session Type/Title: Oral / SCT Clinical
Date and Time: June 10, 11:30am – 12:45pm CET

On May 11, 2023 Acorda Therapeutics, Inc. (Nasdaq: ACOR) reported a business update and provided its financial results for the first quarter ended March 31, 2023 (Press release, Acorda Therapeutics, MAY 11, 2023, View Source [SID1234631475]).

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"We were very pleased to see INBRIJA’s strong performance in the quarter, with U.S. net revenue up by 52% over the same quarter of 2022. In addition, new prescription request forms increased 45% over the first quarter of 2022," said Ron Cohen, M.D., Acorda’s President and Chief Executive Officer. "We believe that our new marketing programs are having an impact. In April, we launched an INBRIJA television commercial on approximately 50 streaming services, accompanied by a digital ‘surround sound’ campaign to encourage viewers to take an action after viewing the commercial, such as connecting with a nurse educator or speaking to their physician. The initial response has been highly encouraging; in the first six weeks, the commercial has been viewed over 2.5 million times and is already driving substantial traffic to the INBRIJA website."

"Our agreement with Chance Pharmaceuticals is an important step in bringing INBRIJA to those living with Parkinson’s in China; by 2030, it is estimated there will be approximately 5 million people with Parkinson’s disease in that country," Dr. Cohen continued. "We are also very pleased that Tom Burns, who has decades of finance and accounting experience in biotech and high tech, will stand for election to Acorda’s board in June; we are deeply grateful to Jeff Randall for his superb contributions to the Company since joining the Board in 2006, and we wish him the very best."

First Quarter 2023 Financial Results

For the quarter ended March 31, 2023, the Company reported INBRIJA worldwide net revenue of $6.1 million, of which $5.6 million was derived from sales in the U.S., a 52.2% increase compared to the same quarter in 2022. The Company also reported ex-U.S. INBRIJA net revenue of $0.5 million in the first quarter related to the recent launch in Spain in February.

For the quarter ended March 31, 2023, the Company reported AMPYRA net revenue of $12.6 million, a 15.4% decrease compared to $14.9 million for the same quarter in 2022. Additionally, for the quarter ended March 31, 2023, the Company reported FAMPYRA royalty revenues of $2.9 million, a 9.3% 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.

Research and development (R&D) expenses for the quarter ended March 31, 2023 were $1.4 million, compared to $1.7 million for the same quarter in 2022. Sales, general and administrative (SG&A) expenses for the quarter ended March 31, 2023 were $22.5 million, compared to $26.9 million for the same quarter in 2022.

Non-GAAP adjusted operating expenses (adjusted OPEX) for the quarter ended March 31, 2023 was $23.9 million, compared to $28.6 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 March 31, 2023 was $2 million, compared to a provision for income taxes of $0.3 million for the same quarter in 2022.

The Company reported a net loss of ($16.8) million for the quarter ended March 31, 2023, or a net loss of ($0.69) per share on both a basic and diluted basis. Net loss in the same quarter of 2022 was ($24.5) million, or a net loss of ($1.85) per share on both a basic and diluted basis.

At March 31, 2023, the Company had cash, cash equivalents, and restricted cash of $37.8 million, compared to $44.7 million at year end 2022. Restricted cash includes $6.2 million in escrow related to the semi-annual interest payment to the holders of its 6.00% convertible senior secured notes (Convertible Notes).

2023 Financial Guidance

For the full year 2023, Acorda continues to target INBRIJA U.S. net revenue to be $38 – $42 million, AMPYRA net revenue to be $65 – $70 million, adjusted OPEX to be $93 – $103 million, and ending cash balance to be $43 – $47 million.

INBRIJA Commercialization Agreement in China

Under the terms of the agreement, Acorda will receive an up-front payment of $2.5 million, a near term milestone payment of up to $6 million, $3 million upon regulatory approval, up to $132.5 million in sales milestones, and a fixed fee for each carton of INBRIJA supplied to Chance. By 2030, it is estimated that China will have approximately 5 million people with Parkinson’s disease due to its aging population1. Chance plans to seek marketing authorization as quickly as possible.

Board of Directors

Jeff Randall, who has served on Acorda’s Board since 2006, and currently serves as Chair of the Audit Committee, will be rotating off the Board as of the Company’s June 2023 annual meeting of stockholders. Tom Burns, the Senior Vice President of Finance and Chief Financial Officer of XOMA Corporation, will stand for election to the Board at that meeting. Tom is responsible for all financial matters affecting or involving the XOMA companies, including directing XOMA’s financial strategy, accounting, budgeting, financial planning and analysis, and investor relations functions. Mr. Burns has 25 years of experience in accounting and finance in both biotechnology and high technology companies.

Annual Meeting of Stockholders

Acorda’s Annual Meeting of Stockholders will take place on Thursday, June 22, 2023 at 9:00am ET. Stockholders are encouraged to vote by internet, telephone, mail, or in person as described in the materials sent to them so that all shares will be represented at the Annual Meeting.

Webcast and Conference Call

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 May 11, 2023 until 11:59 p.m. ET on June 10, 2023. To access the replay, please dial 1 866 813 9403 (domestic) or +44 204 525 0658 (international); access code 270385. The archived webcast will be available in the Investor Relations section of the Acorda website at www.acorda.com.

Non-GAAP Financial Measures

This press release includes financial results prepared in accordance with accounting principles generally accepted in the United States (GAAP) and also certain historical and forward-looking non-GAAP financial measures. Non-GAAP financial measures are not an alternative for financial measures prepared in accordance with GAAP, and the calculation of the non-GAAP financial measures included herein may differ from similarly titled measures used by other companies. The Company believes that the presentation of these non-GAAP financial measures, when viewed in conjunction with actual GAAP results, provides investors with a more meaningful understanding of our ongoing and projected operating performance because it excludes (i) expenses that pertain to corporate restructurings not routine to the operation of our business, (ii) non-cash charges that are substantially dependent on changes in the market price of our common stock, and (iii) other items as set forth above that are not ascertainable at the present time. We believe these non-GAAP financial measures help indicate underlying trends in the Company’s business and are important in comparing current results with prior period results and understanding expected operating performance. Also, management uses these non-GAAP financial measures to establish budgets and operational goals, and to manage the Company’s business and evaluate its performance. In addition, management believes that adjusted OPEX is important in evaluating the administrative costs of operating the Company’s business.

Adjusted OPEX includes (i) research and development expenses and (ii) selling, general, and administrative expenses, and excludes (i) costs of goods sold, (ii) amortization of intangible assets, (iii) change in fair value of derivative liability, and (iv) change in fair value of acquired contingent liability. We are unable to reconcile our guidance for this non-GAAP measure to GAAP due to the forward-looking nature of the adjustments that are needed to determine this information, which includes information regarding future compensation charges, future changes in the market price of our common stock, and changes in the fair value of derivative and contingent liabilities, none of which are available at this time.

On May 11, 2023 Abeona Therapeutics Inc. (Nasdaq: ABEO) reported its financial results for the first quarter of 2023 and provided an update on progress toward achieving key corporate objectives (Press release, Abeona Therapeutics, MAY 11, 2023, View Source [SID1234631474]).

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"We remain focused on completing our EB-101 Biologics License Application for recessive dystrophic epidermolysis bullosa (RDEB) and are making good progress towards this goal," said Vish Seshadri, Chief Executive Officer of Abeona. "We are also excited to be presenting encouraging results from animal proof-of-concept studies from our AAV ophthalmology program at the ASGCT (Free ASGCT Whitepaper) annual meeting next week."

First Quarter and Recent Portfolio Update

EB-101 for RDEB

● Abeona continues to make progress toward submitting a BLA for EB-101 to the U.S. Food and Drug Administration (FDA) in late second quarter to early third quarter of 2023. The Company has submitted a pre-BLA meeting request to the FDA in advance of the anticipated BLA submission. If the BLA is approved, Abeona anticipates being granted a Priority Review Voucher (PRV), which can be used to receive expedited review by the FDA of a subsequent marketing application for a different product or sold to another company.
● Abeona announced today in a separate press release that additional data from the pivotal Phase 3 VIITAL study of investigational EB-101 in RDEB was presented during an oral session at the International Societies for Investigative Dermatology (ISID) Meeting. The positive top-line efficacy and safety data from the VIITAL study was reported in November 2022.
● As part of its commercial planning, the Company continues to engage with stakeholders across the healthcare system, including public and private payors, and healthcare providers to better understand market access and pricing for EB-101.

Preclinical programs

● Abeona’s preclinical programs are investigating the use of novel adeno-associated virus (AAV) capsids in AAV-based therapies for serious genetic eye diseases. The Company has been granted pre-Investigational New Drug Application meetings for two of its programs to take place in the second quarter of 2023.
● Abeona will present new preclinical data at the 26th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT) (Free ASGCT Whitepaper) taking place from May 16-20, 2023 in Los Angeles, CA. The Company’s presentations will include data on three internally developed investigational preclinical gene therapy product candidates from its AAV ophthalmology program, including ABO-504 for Stargardt disease, ABO-503 for X-linked retinoschisis (XLRS) and ABO-505 for autosomal dominant optic atrophy (ADOA).

First Quarter Financial Results

Cash, cash equivalents, restricted cash and short-term investments totaled $40.7 million as of March 31, 2023, compared to $52.5 million as of December 31, 2022. Net cash used in operating activities was $11.7 million for the three months ended March 31, 2023. Abeona estimates that its current cash and cash equivalents, restricted cash and short-term investments are sufficient resources to fund operations into the third quarter of 2024.

"Our operating cash burn for the first quarter of 2023 and projected operating cash burn for the second quarter of 2023 include BLA submission and personnel costs, which we expect will be substantially lower in the second half of 2023," said Joe Vazzano, Chief Financial Officer of Abeona.

License and other revenues in the first quarter of 2023 were nil, compared to $0.3 million in the first quarter of 2022. Research and development expenses for the three months ended March 31, 2023 were $8.0 million, compared to $10.5 million for the same period of 2022. General and administrative expenses were $4.0 million for the three months ended March 31, 2023, compared to $4.2 million for the same period of 2022. Net loss was $9.1 million for the first quarter of 2023, or $0.54 loss per common share as compared to a net loss of $22.0 million, or $3.80 loss per common share, in the first quarter of 2022.

Portfolio Update Conference Call

The Company will host a conference call and webcast on Tuesday, May 23, 2023, at 8:30 a.m. ET, to discuss the first quarter and recent portfolio update, and its data presentations at the ISID and ASGCT (Free ASGCT Whitepaper) meetings. To access the call, dial 888-506-0062 (U.S. toll-free) or 973-528-0011 (international) and Entry Code: 885338 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.