On March 3, 2022 Priothera, a late-clinical stage biotechnology company pioneering the development of its S1P receptor modulator drug, mocravimod, reported that the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) have both granted orphan drug designation (ODD) to mocravimod for the treatment of Acute Myeloid Leukemia (AML) in patients undergoing allogeneic hematopoietic stem cell transplantation (HSCT) (Press release, Priothera, MAR 3, 2022, View Source [SID1234609425]). EMA’s ODD follows a recommendation from the Committee for Orphan Medicinal Products (COMP).

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Florent Gros, Co-Founder and CEO of Priothera, commented: "The orphan drug designations we received for mocravimod from both the FDA and EMA are important milestones towards addressing the urgent, unmet needs of AML patients. Allogenic stem cell transplantation is the only potentially curative approach for AML patients but has unacceptably high mortality rates with current treatments. We are looking forward to initiating the global Phase 2b clinical trial with mocravimod in multiple centers in the US, Europe and Asia in the coming months."

Mocravimod, a sphingosine 1 phosphate (S1P) receptor modulator which has been previously tested in multiple autoimmune indications, is being developed to enhance the curative potential of HSCT. Moreover, it has shown a clinically relevant benefit in an early clinical study in patients with hematologic malignancies undergoing HSCT.

A multicenter Phase 2b study evaluating the efficacy and safety of mocravimod as an adjunctive and maintenance therapy to HSCT in adult AML patients is planned for the second half of 2022. The study will include approximately 250 patients in several countries in Europe, the US and Asia, upon approvals from respective health authorities.

Orphan drug designation is reserved for medicines treating rare, life-threatening or chronically debilitating diseases.

About mocravimod
Mocravimod (also known as KRP203), is a novel, synthetic, sphingosine 1-phosphate receptor (S1PR) modulator with a long duration in the body. Phase 1 and Phase 2 trials successfully assessed mocravimod for safety and tolerability in several autoimmune indications. Promising data from a Phase 1b/2a clinical study in patients with hematological malignancies led Priothera to further develop mocravimod for the treatment of blood cancers.

Mocravimod will be investigated in a Phase 2b/3 study as a potential treatment for patients with Acute Myeloid Leukemia (AML) receiving allogeneic hematopoietic stem cell transplantation (HSCT). Allogenic HSCT is the only potentially curative approach for AML patients, but current treatments have unacceptably high mortality and morbidity rates.

Priothera leverages S1PR’s unique mode of action to maintain anti-leukemia activity – graft-versus leukemia (GVL) – while reducing tissue damage resulting from graft-versus-host disease (GVHD), a consequence of allogenic HSCT. This novel treatment approach – the only S1PR modulator treating blood cancers – tackles a high unmet medical need and intends to add quality life to patients.

On March 3, 2022 Herantis Pharma Plc ("Herantis"), developing disease modifying therapies for Parkinson’s disease, reported the company’s Year-end report for 2021 (Press release, Herantis Pharma, MAR 3, 2022, View Source,c3517887 [SID1234609424]).

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A live webinar (in English) will be held today, at 10:00 EET / 9:00 CET.

Link to Registration: Herantis’ FY 2021 Report Webinar

Highlights January-December 2021
Significant progress has been made with HER-096:

Selection of HER-096 as the lead candidate for development based on efficacy, safety, pharmacokinetic, pharmacodynamic and stability criteria.
Demonstration of HER-096 penetrating the blood brain barrier and entering the brain in therapeutic concentrations after a single subcutaneous administration in a rat model.
Confirmation of a unique mechanism of action, similar to CDNF, resulting in a significant reduction of alpha-synuclein deposition, neuroinflammation, and remarkable restoration of dopaminergic neurons in preclinical disease models
Identification of novel biomarker candidates in the Phase 1b Cerebral Dopamine Neurotrophic Factor (CDNF) study aimed at supporting future clinical development of HER-096
Other operational highlights:

A collaboration with Nanoform Finland Plc. was formed to investigate the feasibility of applying the nanoforming process to rhCDNF. In 2021, we established that nanoformed rhCDNF nanoparticles retain their stability and activity.
Formation of Herantis Scientific Advisory Board (SAB), consisting of leading experts in the development of therapies for Parkinson’s disease from industry and academia: Dr.Anders Gersel Pedersen M.D. (chair), Dr. Daniele Bravi M.D., Prof. David Dexter, PhD, and Prof. Alberto Espay M.D. MSc
Hilde Furberg, MSc, an experienced biopharma executive, was elected to the Board of Directors
Completion of a private placement exceeding EUR 4.0 million
Herantis published several papers and scientific posters in scientific media and conferences during 2021
Events after year end 2021
On January 20th, 2022, Herantis Pharma’s Board of Directors appointed Frans Wuite, MD, MBA, as the interim CEO, effective immediately. Wuite is also the Vice Chairman of Herantis’ Board of Directors and will continue in this role. Herantis’ CEO until January 20th, 2022, Dr. Craig Cook, left the company following the Board of Directors’ decision. A search for a permanent CEO will be launched. In connection with the CEO transition and following strong preclinical data in 2021, Herantis’ Board of Directors has decided to focus a significant majority of the company’s development efforts to advance HER-096, a small, synthetic peptidomimetic molecule derived from the active site of CDNF. HER-096 combines the unique mode of action of the CDNF protein to target Parkinson’s disease with the ease of subcutaneous administration.
Group’s Key Figures:

Year-end 2021 Report Webinar Details

Interim CEO, Frans Wuite, MD will present Herantis and comment on the Year-end 2021 report followed by a live Q&A session. Please join the webinar a few minutes in advance.

On March 3, 2022 Genocea Biosciences, Inc. (NASDAQ: GNCA), a biopharmaceutical company developing next-generation cancer immunotherapies, reported that it will host its fourth quarter 2021 financial results and corporate update conference call and live audio webcast on Thursday, March 10th at 8:30 a.m. E.T (Press release, Genocea Biosciences, MAR 3, 2022, View Source [SID1234609423]).

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Interested participants may access the conference call by dialing (844) 826-0619 (domestic) or (315) 625-6883 (international) and referring to conference ID number 1066689. To join the live webcast, please visit the presentation page of the investor relations section of the Genocea website at View Source

A webcast replay will be available on the Genocea website beginning approximately two hours after the event and will be archived for 90 days.

On March 3, 2022 Magenta Therapeutics (Nasdaq: MGTA), a clinical-stage biotechnology company developing novel medicines designed to bring the curative power of stem cell transplants to more patients, reported that the company will participate in a Cell Therapy Panel Discussion at the 42nd Annual Cowen Healthcare Conference, to be held virtually, on Wednesday, March 9th, 2022 at 9:10 a.m. ET (Press release, Magenta Therapeutics, MAR 3, 2022, View Source [SID1234609422]).

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A live webcast of the panel can be accessed on the Magenta Therapeutics website at View Source The webcast replay will be available for 30 days following the event.

On March 2, 2022 Akari Therapeutics, Plc (Nasdaq: AKTX), a late-stage biopharmaceutical company focused on innovative therapeutics to treat orphan autoimmune and inflammatory diseases where complement (C5) and/or leukotriene (LBT4) systems are implicated, reported the appointment of Rachelle Jacques as President and Chief Executive Officer of Akari Therapeutics (Press release, Akari Therapeutics, MAR 2, 2022, View Source [SID1234609517]). Ms. Jacques will also join the company’s Board of Directors. Her appointment begins at the end of March 2022.

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Outgoing Chief Executive Officer, Clive Richardson, will continue to serve the Company, supporting Rachelle to accelerate business development and ensuring a smooth transition.

"I am very pleased to be able to welcome Rachelle as CEO of Akari Therapeutics. She has considerable biotechnology experience including a proven track record of bringing advanced therapies for rare diseases to the market," said Ray Prudo, M.D., Akari Therapeutics’ founder and Chairman of the company’s Board of Directors. "With her combined experience in C5 inhibition, immunology and rare diseases, Rachelle has a deep understanding of the potential of Akari’s pipeline. This, together with her strategic experience, affords a springboard to advance our Phase 3 pipeline through potential regulatory approval and towards commercialization, as well as and bring forward our earlier stage pipeline. I very much look forward to working with Rachelle who will be based in the Boston area."

"It’s an exciting time at Akari and I look forward to building on the robust research and development work that has already been completed to advance nomacopan, a unique drug differentiated by its dual mode of action which has the potential to deliver benefits beyond standard complement inhibition," stated Ms. Jacques. "Our pipeline, including two late-stage pivotal programs, has tremendous potential for patients with significant unmet needs and we are working with urgency to realize that promise for them."

Before this appointment, Ms. Jacques served as Chief Executive Officer of Enzyvant Therapeutics Inc., a commercial-stage biotechnology company developing transformative regenerative therapies for rare diseases. Prior to Enzyvant, she served as the Senior Vice President and Global Complement Franchise Head at Alexion Pharmaceuticals, Inc., where she was responsible for global franchise strategy development and execution of the C5 complement inhibitors, eculizumab and ravulizumab, across the therapeutic areas of hematology, nephrology and neurology. She was Vice President of U.S. Hematology Marketing at Baxalta Inc. and then Shire plc, following Shire’s acquisition of Baxalta in 2016. At Baxalta, she served as Vice President of Business Operations after its spinoff from Baxter International Inc. Ms. Jacques held multiple leadership positions at Baxter, including Vice President of Finance, U.S. BioScience Business. Earlier in her career, Ms. Jacques served in various roles at Dow Corning Corporation, including operational management positions in the U.S., Europe, and China.

Ms. Jacques serves on the boards of directors of uniQure N.V. (Nasdaq: QURE) and Corbus Pharmaceuticals (Nasdaq: CRBP). She is co-chair of the Alliance for Regenerative Medicine (ARM) Tissue Engineering & Biomaterials Committee and is a founding member of the ARM Action for Equality Task Force. Ms. Jacques received her B.A. in business administration from Alma College.