On January 12, 2020 Iovance Biotherapeutics, Inc. (NASDAQ: IOVA), a late-stage biotechnology company developing novel T cell-based cancer immunotherapies, and Cellectis (Euronext Growth: ALCLS – NASDAQ: CLLS), a clinical-stage biopharmaceutical company focused on developing immunotherapies based on gene-edited allogeneic CAR T-Cells (UCART), reported that the companies have entered into a research collaboration and exclusive worldwide license agreement whereby Iovance will license certain TALEN technology from Cellectis in order to develop tumor infiltrating lymphocytes (TIL) that have been genetically edited to create more potent cancer therapeutics (Press release, Cellectis, JAN 12, 2020, View Source [SID1234553027]).

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The worldwide exclusive license enables Iovance Biotherapeutics’ use of TALEN technology addressing multiple gene targets to modify TIL for therapeutic use in several cancer indications. Financial terms of the license include development, regulatory and sales milestone payments from Iovance Biotherapeutics to Cellectis, as well as royalty payments based on net sales of TALEN-modified TIL products.

"We are very excited to be collaborating with Cellectis in applying the TALEN gene-editing technology to Iovance’s TIL product. We believe that we can genetically modify TIL to make a more tumor-reactive anti-cancer product," said Maria Fardis, Ph.D., MBA, President and Chief Executive Officer of Iovance Biotherapeutics. She added: "We plan to move a TALEN-edited TIL therapy into a clinical trial as rapidly as possible."

"We are thrilled to be working with Iovance and believe that applying our TALEN technology to TIL-based products will yield even better treatments for a variety of cancers," added André Choulika, Ph.D., Chairman and CEO, Cellectis. "Patients remain at the heart of our company and it is our sincere hope that this collaboration can help provide more efficacious options to those in need."

On January 12, 2020 BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) reported that the company will provide updates on berotralstat, an oral kallikrein inhibitor for hereditary angioedema (HAE), and BCX9930, an oral Factor D inhibitor for complement-mediated diseases, this week at the 38th Annual J.P. Morgan Healthcare Conference in San Francisco (Press release, BioCryst Pharmaceuticals, JAN 12, 2020, View Source [SID1234553026]).

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"BioCryst is positioned for transformation in 2020 with multiple global approvals and launches of berotralstat, and PNH proof of concept data with BCX9930. The $100 million in additional capital we brought into the company in Q4 2019 provides a foundation for progress and value creation in 2020," said Jon Stonehouse, president and chief executive officer of BioCryst.

Berotralstat Program Updates:

New drug application (NDA) submitted to U.S Food and Drug Administration in December 2019

APeX-J trial in Japan met its primary endpoint (p=0.003) for prevention of HAE attacks, and berotralstat was safe and generally well-tolerated

JNDA submission to Japanese Pharmaceuticals and Medical Devices Agency (PMDA) on-track for Q1 2020

Marketing authorization application to European Medicines Agency (EMA) on-track for Q1 2020
BCX9930 Program Updates:

As previously announced, results from an ongoing three part Phase 1 trial of BCX9930 showed rapid, sustained and >95% suppression of the alternative pathway (AP) of the complement system at 100 mg every 12 hours, as measured by the AP Wieslab assay.

In two initial multiple ascending dose (MAD) assessment cohorts, healthy volunteers received 50 mg or 100 mg of oral BCX9930 or placebo (each MAD cohort randomized 10:2) administered every 12 hours for seven days. Healthy volunteers in the MAD cohorts were prophylactically dosed with the broad-spectrum antibiotic, amoxicillin/clavulanate. BCX9930 was safe and generally well tolerated at all doses studied in single ascending dose and MAD cohorts. There were no serious adverse events. A clinically benign rash was observed in some healthy volunteers in the MAD (two in the 50 mg cohort, seven in the 100 mg cohort), which was self-limited and resolved in 4-8 days after onset.

The company has now completed an additional MAD cohort with 50 mg of oral BCX9930 or placebo administered every 12 hours for 14 days, with vaccination instead of an antibiotic. Key observations from the additional MAD cohort include:

Benign rash (similar to prior MAD cohorts) that was self-limited and resolved in 4 to 8 days post-onset seen in seven healthy volunteers

Successfully dosed-through benign rash, with rash resolving on-drug, in both patients who continued dosing, per protocol

Biopsies of rashes from multiple subjects confirm benign assessment
The company is on-track to report proof of concept data in paroxysmal nocturnal hemoglobinuria (PNH) patients in 1H 2020.

Additional details can be found on slides , which can be accessed at may be accessed in the Investors section of BioCryst’s website at http://www.biocryst.com.

On January 12, 2020 Atara Biotherapeutics, Inc. (Nasdaq: ATRA), a leading off-the-shelf, allogeneic T-cell immunotherapy company developing novel treatments for patients with cancer, autoimmune and viral diseases, reported President and Chief Executive Officer Pascal Touchon will share details of the company’s mission, scientific platform and development pipeline during a presentation at the 38th Annual J.P. Morgan Healthcare Conference on Thursday, January 16 at 8:30 a.m. PST at the Westin St. Francis Hotel in San Francisco (Press release, Atara Biotherapeutics, JAN 12, 2020, View Source [SID1234553025]).

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"As we enter into this new year, we are proud of our progress building a leading off-the-shelf, allogeneic T-cell immunotherapy company with a robust pipeline," said Pascal Touchon, President and Chief Executive Officer of Atara Biotherapeutics. "Backed by our innovative EBV T-cell platform, next-generation CAR T technologies, state-of-the-art manufacturing capabilities and dedication to developing transformative T-cell immunotherapies for patients, we are confident in our ability to execute on our 2020 plans."

Anticipated key milestones across Atara’s four strategic priorities in 2020 include:

Tab-cel (tabelecleucel)

Submitted clinical trial applications (CTAs) to several European countries in November 2019 to enable opening EU clinical sites in 2020
Begin enrollment in a Phase 2 multi-cohort study including patients with other EBV+ cancers in the second half of 2020
Initiate FDA Biologics License Application (BLA) submission for patients with EBV+ PTLD in the second half of 2020
ATA188

Initiate enrollment of randomized, double-blind, placebo-controlled Phase 1b study in patients with progressive MS in the second or third quarter of 2020
Present six- and twelve-month ATA188 Phase 1a clinical results for cohorts 3 and 4 in the first and second halves of 2020, respectively
ATA2271

Submit next-generation mesothelin-targeted autologous CAR T IND for patients with advanced mesothelioma in the second or third quarter of 2020
EBV CAR T Platform

Academic presentation of an off-the-shelf, allogeneic CD19 CAR T clinical proof-of-principle using a partially HLA matched Epstein-Barr virus T cell academic construct for patients with relapsed/refractory B-cell malignancies at the 2020 Transplantation and Cellular Therapy (TCT) Meetings to be held February 19-23 in Orlando, Fla.
A live audio webcast of the J.P. Morgan Healthcare Conference presentation will be available by visiting the Investors and Media section of the Atara website. An archived replay of the webcast will be available on the Company’s website for 14 days following the live presentation.

On January 12, 2020 Agios Pharmaceuticals, Inc. (NASDAQ: AGIO) reported its "Agios 2025" six-year strategic vision focused on creating and commercializing differentiated medicines to treat hematologic malignancies, solid tumors and rare genetic diseases (Press release, Agios Pharmaceuticals, JAN 12, 2020, View Source [SID1234553024]). Under this plan, by the end of 2025, the company expects to have four marketed products across at least eight indications, at least six molecules in clinical development and be cash-flow positive. Agios will present at the 38th Annual J.P. Morgan Healthcare Conference in San Francisco on Monday, January 13 at 7:30 a.m. PT (10:30 a.m. ET), and a live webcast will be available at investor.agios.com.

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"We are entering an exciting new chapter for Agios as we advance our first rare genetic disease program across three opportunities in PK deficiency, thalassemia and sickle cell disease and continue our work to expand the benefit of IDH inhibitors to solid tumors as well as to additional indications in hematologic malignancies," said Jackie Fouse, Ph.D., chief executive officer of Agios. "We will realize our 2025 vision by continuing to leverage our unmatched expertise in cellular metabolism, early translational research and our passionately patient-focused team, the same attributes that enabled us to discover, develop and market two targeted oncology medications in just 10 years. With both near- and long-term value drivers, Agios enters 2020 with strong momentum that will continue as we work toward achieving our 2025 strategic vision."

"AGIOS 2025" STRATEGIC VISION

The "Agios 2025" strategic vision delineates the company’s view for growth over the next six years with established and expanding franchises focused on treating hematologic malignancies, solid tumors and rare genetic diseases. As part of this vision, Agios expects to achieve the following milestones by the end of 2025:

4 marketed medicines discovered and developed at Agios
Approvals in 8+ indications spanning hematologic malignancies, solid tumors and rare genetic diseases
6+ molecules in the clinic generated by the company’s internal research discovery engine
Cash-flow positive within the six-year timeframe
ANTICIPATED 2020 KEY MILESTONES

Agios announced today that it expects to achieve the following key milestones in 2020:

Hematologic Malignancies

Deliver full-year U.S. revenue for TIBSOVO of $105-115 million
Receive European Medicines Agency CHMP opinion for TIBSOVO in relapsed or refractory acute myeloid leukemia (AML) with an IDH1 mutation by year-end
Complete enrollment of Phase 3 AGILE trial of TIBSOVO in combination with azacitidine in adult patients with previously untreated IDH1 mutant AML by year-end
Complete enrollment of the relapsed or refractory myelodysplastic syndrome arm of the TIBSOVO Phase 1 study of IDH1 mutant advanced hematologic malignancies by year-end
Solid Tumors

File supplemental new drug application (sNDA) for TIBSOVO in previously treated IDH1 mutant cholangiocarcinoma by year-end
Rare Genetic Diseases

Announce topline data for ACTIVATE and ACTIVATE-T pivotal trials for mitapivat in adults with pyruvate kinase (PK) deficiency by year-end
Submit updated data from the Phase 2 study of mitapivat in thalassemia for presentation at the European Hematology Association (EHA) (Free EHA Whitepaper) Congress and finalize pivotal development strategy by year-end
Achieve proof of concept for mitapivat in sickle cell disease by mid-2020
Receive investigational new drug (IND) clearance for AG-946, a next generation PKR activator, and initiate first-in-human study in healthy volunteers in the first half of 2020
Research

Achieve at least one new development candidate by year-end
RECENT MILESTONES

The company also provided an update on the following 2019 key milestones:

Completed enrollment of ACTIVATE-T, a single-arm trial evaluating mitapivat in regularly transfused adults with PK deficiency
Expect to complete enrollment in ACTIVATE, a 1:1 randomized, placebo-controlled trial in adult PK deficiency patients who do not receive regular transfusions, in the first quarter of 2020
Initiated the registration-enabling Phase 3 INDIGO study of vorasidenib in patients with Grade 2 non-enhancing glioma with an IDH mutation
2019 Year-End Cash and Guidance
Agios ended 2019 with approximately $718 million of cash, cash equivalents and marketable securities. The company expects that its cash, cash equivalents and marketable securities as of December 31, 2019, together with anticipated product and royalty revenue, interest income and expense reimbursements under our collaboration agreements, but excluding any additional program-specific milestone payments, will enable the company to fund its planned operating expenses and capital expenditure requirements through at least the end of 2021.

Presentation at 38th Annual J.P. Morgan Healthcare Conference
Agios will webcast its corporate presentation and break out session from the 38th Annual J.P. Morgan Healthcare Conference in San Francisco on Monday, January 13, 2020 at 7:30 a.m. PT (10:30 a.m. ET). A live webcast of the presentation can be accessed under "Events & Presentations" in the Investors section of the company’s website at agios.com. A replay of the webcast will be archived on the Agios website for at least two weeks following the presentation.

On January 10, 2020 BridgeBio Pharma, Inc. (NASDAQ: BBIO) reported a collaboration agreement with Catalent to establish dedicated gene therapy development and manufacturing capacity at Catalent’s Paragon Gene Therapy clinical and commercial manufacturing center in Harmans, Maryland (Press release, BridgeBio, JAN 10, 2020, View Source [SID1234576232]). The agreement is intended to support the clinical and commercial manufacturing needs for BridgeBio’s gene therapy product candidates for congenital adrenal hyperplasia, BBP-631, and Canavan disease, BBP-812.

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Catalent’s commercial facility is fully compliant with cGMP requirements and allows for up to 5000 liters of production. The over 400,000 square feet footprint is complete with all necessary support functions for storage and fill finish for final product supply.

"Having flexibility and greater certainty in manufacturing capacity is critical to success in gene therapy," said Eric David, M.D., J.D., CEO of BridgeBio’s gene therapy subsidiaries. "Catalent’s Paragon Gene Therapy arm has been our trusted partner for almost two years, and this expansion of our relationship is intended to allow for smoother clinical and commercial development, as well as an acceleration of our pipeline programs, helping us move faster to address critical unmet health needs for patients and their families."

"Catalent’s expertise in the cGMP manufacturing of viral vectors complements our internal investment in the CMC process and analytical development to support our gene therapy portfolio," said Fred Porter, Ph.D., senior vice president of CMC and technical development of BridgeBio’s gene therapy subsidiaries. "Securing dedicated capacity for the delivery of clinical and commercial supply is critical to our long-term strategy."

Pete Buzy, president of Paragon Gene Therapy, commented, "It is Catalent’s continued goal to grow with our customers and to be able to offer them secure, state-of-the-art gene therapy facilities for their critical clinical and commercial needs. For gene therapies, the manufacturing scale-up process is complex and unique. Therefore, for our partners, having access to advanced adeno-associated virus production expertise and experience is vital to progress these pioneering treatments towards commercialization and the patients who need them."