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."

On January 10, 2020 iCo Therapeutics (TSXV: ICO) (OTCQB: ICOTF) ("iCo" or the "Company"), reported that granted 2,000,000 stock options to directors, officers and an advisor (Press release, iCo Therapeutics, JAN 10, 2020, View Source [SID1234553650]).

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The stock options are exercisable at the price of $0.08 and will expire on January 10, 2025. The stock options shall vest as follows: 1/5 on today’s date (the "Effective Date") and then 1/5 every six months until all options are vested. The closing price of the Company’s shares on the TSX Venture Exchange on January 10, 2020 was $0.08. After giving effect to this option grant, a total of 2,975,000 options will be issued and outstanding with 710,357 remaining for future issuance under the Company’s stock option plan.

On January 10, 2020 Moffitt Cancer Ctr reported that patients with advanced melanoma who develop metastases in the leptomeninges, the fluid filled membranes surrounding the brain and spinal cord, have an extremely dismal prognosis (Press release, Moffitt Cancer Ctr, JAN 10, 2020, View Source [SID1234553269]). Most patients only survive for 8 to 10 weeks after diagnosis. One reason for this poor prognosis is that very little information is known about the molecular development of leptomeningeal melanoma metastases (LMM), making it difficult to develop effective therapies. Researchers in Moffitt Cancer Center’s Donald A. Adam Melanoma and Skin Cancer Center of Excellence and the Department of Neuro-Oncology sought to change this by performing an extensive analysis of the molecular characteristics of the cerebrospinal fluid of patients with LMM. Their findings were published in Clinical Cancer Research, a journal of the American Association for Cancer Research (AACR) (Free AACR Whitepaper).

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Cancer development and progression are highly regulated by intricate interactions between cancer cells and the surrounding environment. Melanoma cells that invade and metastasize into the leptomeninges interact with the surrounding cerebrospinal fluid. Moffitt researchers wanted to improve their understanding of the development of LMM by analyzing the protein and RNA composition of cerebrospinal fluid from patients with LMM. They compared the molecular profiles of 8 control patients without LMM to 8 patients with LMM, including one LMM patient who had an extraordinary response to treatment and was still alive more than 35 months after diagnosis.

They discovered that the cerebrospinal fluid from LMM patients was enriched for proteins involved in innate immunity, proteases and the IGF-signaling pathway. The most commonly altered protein was TGF-β1. Interestingly, the one patient who had an extraordinary response to treatment displayed high levels of these proteins at baseline, but expression levels decreased as the patient responded to treatment. However, the protein expression patterns in the remaining LMM patients who had poor responses to treatment were high at baseline and remained high throughout treatment and disease progression.

The researcher team, led by Keiran Smalley, Ph.D., director of the Donald A. Adam Melanoma and Skin Cancer Center of Excellence and Peter Forsyth, M.D., Chair of the Department of Neuro-Oncology, hypothesized that the cerebrospinal fluid of LMM patients could impact melanoma cells by modulating their molecular profile. They confirmed this hypothesis by incubating cerebrospinal fluid from the LMM patients with melanoma cells and discovered that the fluid was able to induce activation of proteins and signaling pathways involved in malignant progression, including the PI3K/AKT pathway, integrins, B cell signaling, mitotic cell cycle progression, TNFR, TGF-β and oxidative stress.

Their findings demonstrate that the cerebrospinal fluid from LMM patients who did not respond to treatment promoted survival of melanoma cells, while the cerebrospinal fluid from the extraordinary responder did not promote survival. These observations suggest that molecules exist within the cerebrospinal fluid that can stimulate melanoma cell survival and prevent cell death. The researchers reported that one of these survival molecules is TGF-β. The patient who responded well to treatment had very low to undetectable levels of cerebrospinal fluid TGF-β, while those patients who did not respond to treatment had much higher levels of TGF-β.

The researchers hope that their data will provide important knowledge about LMM and offer insights into potential therapeutic targets. "It is likely that the environment of LMM is a key regulator of both disease progression and therapeutic response. Improved knowledge about the microenvironment of LMM may allow novel therapeutic strategies to be developed that can delay disease progression," explained Smalley.

This study was supported by grants from the National Institutes of Health, the Department of Defense and the State of Florida.