On April 28, 2020 Adaptimmune Therapeutics plc (Nasdaq:ADAP), a leader in cell therapy to treat cancer, reported that the European Medicine Agency’s (EMA) Committee for Orphan Medicinal Products (COMP) has adopted a positive opinion for Orphan Drug Designation for ADP-A2M4 for the treatment of soft tissue sarcomas (Press release, Adaptimmune, APR 28, 2020, View Source [SID1234556698]).

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!

Adaptimmune’s SPEARHEAD-1 trial with ADP-A2M4 for people with synovial sarcoma and myxoid/round cell liposarcoma (MRCLS) is actively enrolling at approximately 25 clinical sites in Canada, France, Spain, the United Kingdom, and the US. The SPEARHEAD-1 trial is intended to support the registration of ADP-A2M4 for the treatment of advanced synovial sarcoma and MRCLS.

"Outcomes with currently available treatments remain unsatisfactory for patients with inoperable or metastatic soft tissue sarcoma, and there is a high unmet medical need for new treatment options for patients with this disease," said Dennis Williams, PharmD, Adaptimmune’s SVP, Late Stage Development. "ADP-A2M4 has the potential to offer substantial improvement in the treatment of advanced soft tissue sarcoma and the COMP’s adoption of a positive opinion for Orphan Drug Designation for ADP-A2M4 is another important milestone for this program."

The COMP adopts an opinion on the granting of orphan drug designation, after which the opinion is submitted to the European Commission for endorsement. This designation by the European Commission provides certain regulatory and financial incentives for companies to develop and market therapies that treat a life-threatening or chronically debilitating condition affecting no more than five in 10,000 persons in the European Union, and where the treatment provides a significant benefit to those affected by the condition or no satisfactory treatment is available.

Earlier this year, the United States (US) Food and Drug Administration (FDA) granted Orphan Drug Designation (ODD) to SPEAR T-cells targeting MAGE-A4 for the treatment of soft tissue sarcomas and Regenerative Medicine Advanced Therapy (RMAT) designation for the treatment of synovial sarcoma.

On April 28, 2020 AMAG Pharmaceuticals, Inc. (NASDAQ: AMAG) reported that its Board of Directors has appointed Scott Myers as AMAG’s President and Chief Executive Officer, and member of the Board, effective immediately (Press release, AMAG Pharmaceuticals, APR 28, 2020, View Source [SID1234556697]). Mr. Myers succeeds William Heiden, who is stepping down from such roles in light of Mr. Myers’ appointment.

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!

"Scott is a seasoned leader with exceptional experience, skill set and passion, and it became evident to the Board through its rigorous search that Scott is the ideal candidate to lead AMAG forward," said Gino Santini, Chairman of AMAG’s Board. "Scott’s track record of success, coupled with his experience leading companies with development stage and commercialized assets, uniquely positions him to lead the Company. The Board is confident that Scott will maximize AMAG’s strengths and proven capabilities and enable the Company to better serve patients."

Mr. Santini added, "The Board of Directors would like to thank Bill for his dedication and leadership over the past eight years. We appreciate Bill’s contributions to AMAG and we wish him the best in his future endeavors."

Mr. Myers is a proven executive who brings nearly three decades of global pharmaceutical and medical technology experience to AMAG. Mr. Myers most recently served as Chairman and Chief Executive Officer of Rainier Therapeutics, a clinical-stage biotechnology company focused on metastatic bladder cancer. Prior to joining Rainier, Mr. Myers served as Chief Executive Officer, President and as a director of Cascadian Therapeutics Inc. Mr. Myers also served as Chief Executive Officer of Aerocrine AB, a medical device company from 2011 to 2015. While at the helm of these companies, Mr. Myers drove transformation that maximized growth and delivered significant long-term value.

"AMAG is at a pivotal point in its evolution and I look forward to working with AMAG’s Board, its leadership team and its dedicated employees to advance the strategic plan, further define the Company’s future, optimize its current products and bring innovative therapies to patients in need," said Mr. Myers. "We have a significant opportunity ahead of us to unlock the potential of AMAG’s pipeline and leverage its existing commercial strengths to drive long-term growth and shareholder value."

Mr. Myers is currently an independent director of Selecta Biosciences where he serves as the Chair of the Compensation and Benefits Committee, as well as a member of the Nominating and Governance Committee. Mr. Myers also serves as an independent director for Harpoon Therapeutics, a clinical stage biotechnology company where he serves on the Audit Committee. Mr. Myers began his career in management consulting and then moved into the pharmaceutical industry through senior leadership roles at Johnson & Johnson, DOV Pharmaceuticals and UCB.

Mr. Myers holds a Bachelor of Arts in Biology from Northwestern University in Evanston, Illinois, and a Master of Business Administration from The University of Chicago Graduate School of Business (Booth) in Chicago, Illinois.

Inducement Equity Awards
The Board of Directors of AMAG approved an inducement award to Mr. Myers of an option to purchase 1,000,000 shares of common stock. The option will have an exercise price equal to the closing price of AMAG’s common stock on the grant date and will be exercisable in four equal annual installments beginning on the first anniversary of April 28,2020 (the grant date). The option will have a ten-year term and be subject to the terms and conditions of the stock option agreement pursuant to which the option will be granted. This equity award will be granted without stockholder approval as inducements material to the employee entering into employment with AMAG in accordance with NASDAQ Listing Rule 5635(c)(4).

On April 28, 2020 MacroGenics, Inc. (Nasdaq: MGNX), a clinical-stage biopharmaceutical company focused on discovering and developing innovative monoclonal antibody-based therapeutics for the treatment of cancer, reported that the Company will release its financial results for the first quarter 2020 after the market closes on Tuesday, May 5, 2020 (Press release, MacroGenics, APR 28, 2020, View Source [SID1234556696]). MacroGenics will host a conference call to discuss the financial results and recent corporate progress on Tuesday, May 5, 2020 at 4:30 p.m. ET. The conference call can be accessed by dialing (877) 303-6253 (domestic) or (973) 409-9610 (international) five minutes prior to the start of the call and providing the Conference ID 2993147.

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 listen-only webcast of the conference call can be accessed under "Events & Presentations" in the Investor Relations section of the Company’s website at View Source A recorded replay of the webcast will be available shortly after the conclusion of the call and archived on the Company’s website for 30 days following the call.

On April 28, 2020 Eagle Pharmaceuticals, Inc. (Nasdaq: EGRX) ("Eagle" or the "Company") and Teva Pharmaceutical Industries Ltd. (Nasdaq: TEVA) ("Teva") reported that on April 27, 2020, the U.S. District Court for the District of Delaware (the "Court") has issued a patent decision in favor of Eagle and Teva for BENDEKA (bendamustine hydrochloride injection, or bendamustine HCl), a liquid, low-volume (50 mL) and short-time 10-minute infusion formulation of bendamustine hydrochloride (Press release, Eagle Pharmaceuticals, APR 28, 2020, View Source [SID1234556695]).

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 Court upheld the asserted patent claims as valid and found that the defendants’ proposed ANDA products would infringe those claims. Under this decision, the patent defendants – Slayback Pharma LLC, Apotex Inc. and Apotex Corp., Fresenius Kabi USA, LLC, and Mylan Laboratories Limited – will not be able to launch their ANDA products before 2031.

"We are delighted with the Court’s decision upholding our patents for BENDEKA, and further protecting the longevity of this important product," said Scott Tarriff, Chief Executive Officer of Eagle Pharmaceuticals. "With this decision, BENDEKA’s value is likely to be intact for many years, thus ensuring our continued ability to invest in our growing research program and product pipeline," concluded Tariff.

On April 28, 2020 Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel products for rare and ultra-rare diseases, reported that clinical, preclinical and manufacturing data from its investigational gene therapy programs will be presented at the American Society of Gene & Cell Therapy (ASGCT) (Free ASGCT Whitepaper) 23RD Annual Meeting, which will be held virtually May 12-15, 2020 (Press release, Ultragenyx Pharmaceutical, APR 28, 2020, View Source [SID1234556694]). Information will be available at www.asgct.org.

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!

Nine Ultragenyx abstracts have been accepted for virtual presentation, including:

An oral presentation of available data from the confirmatory cohort of the Phase 1/2 study of DTX401, an AAV-based gene therapy for the treatment of glycogen storage disease Type Ia (GSDIa) (Abstract #1306)
An oral presentation of new data from the first three cohorts of the Phase 1/2 study of DTX301, an investigational adeno-associated virus (AAV) gene therapy for the treatment of ornithine transcarbamylase (OTC) deficiency (Abstract #505)
Multiple presentations, including two orals, highlighting the HeLa producer cell line platform and improvements in the HEK293 triple transfection system (Abstracts #539 and #543)
"We have made meaningful progress across all of our gene therapy programs, including continued improvements to both the HeLa PCL and HEK293 transient transfection manufacturing technology platforms which we believe will enable highly reproducible, more consistent and scalable gene therapy manufacturing," said Emil D. Kakkis, M.D., Ph.D., Chief Executive Officer and President of Ultragenyx. "In addition to the presentations that support our gene therapy manufacturing approach, we look forward to sharing first-time results from the confirmatory cohort of our GSDIa study and updated clinical data from the OTC study."

Details of the nine presentations are as follows:

Tuesday, May 12
Oral Presentation
Abstract #98: AAV9/hCDKL5 delivery to cerebrospinal fluid of juvenile CDKL5-deficient mice improves learning and memory and motor function in adult mice

Time: 5:15-5:30 PM ET
Poster Presentation
Abstract #172: Characterization and correction of an in vitro model of Wilson Disease by recombinant adeno-associated virus (rAAV) delivered ATP7B transgene

Time: 5:30-6:30 PM ET
Poster Presentation
Abstract #451: Rapid CMC development and pre-commercial considerations for rAAV gene therapy products for rare diseases

Time: 5:30-6:30 PM ET
Wednesday, May 13
Oral Presentation
Abstract #539: HeLa 3.0: CRISPR knockout of genes modulating titer in established rAAV-producing cell lines

Time: 4:00-4:15 PM ET
Oral Presentation
Abstract #505: AAV8 gene therapy as a potential treatment in adults with late-onset OTC deficiency: results from a Phase 1/2 clinical trial

Time: 4:15-4:30 PM ET
Oral Presentation
Abstract #543: Characterization of phenotypic and genotypic stability of rAAV producing HeLa cell lines

Time: 4:45-5:00 PM ET
Poster Presentation
Abstract #586: Gene therapy for Wilson Disease using rAAV to restore ATP7B gene function

Time: 5:30-6:30 PM ET
Thursday, May 14
Poster Presentation
Abstract #1008: Elongation of the Rep-Cap cassette with a cellular intron reduces reverse-packaged Rep-Cap trans plasmid sequences and increases therapeutic vector genome packaging in a HEK293 triple transfection rAAV vector production system

Time: 5:30-6:30 PM ET
Friday, May 15
Oral Presentation
Abstract #1306: AAV8-mediated liver-directed gene therapy as a potential therapeutic option in adults with glycogen storage disease type Ia (GSDIa): results from a Phase 1/2 clinical trial

Time: 10:30-10:45 AM ET