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.

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!

"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 Fusion Pharmaceuticals Inc., a privately-held, clinical-stage precision oncology company developing next-generation radiopharmaceuticals, reported that Chief Executive Officer John Valliant, Ph.D., will present an overview of the Company at the 38th Annual J.P. Morgan Healthcare Conference in San Francisco, California (Press release, Fusion Pharmaceuticals, JAN 10, 2020, View Source [SID1234552975]). The presentation will take place on Thursday, January 16, 2020 at 11:00am PST.

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!

On January 10, 2020 Adimab, LLC, the global leader in the discovery and optimization of fully human monoclonal and bispecific antibodies, reported that in 2019 it entered into agreements with nine new companies (Press release, Adimab, JAN 10, 2020, View Source [SID1234552992]). In addition, Adimab announced the expansion of 11 of its current partnerships and the achievement of 62 technical and development milestones across numerous collaborations.

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!

Over the past 11 years, Adimab has partnered with over 70 companies for the discovery of therapeutic IgGs and bispecific antibodies, resulting in more than 315 therapeutic programs originating from the Adimab Platform. In 2019, Adimab and its partners added 55 new therapeutic programs. New alliances for 2019 include collaborations with Amagma Therapeutics, Black Belt Therapeutics, BYOMass Inc., Galderma, iOmx Therapeutics, Nextpoint Therapeutics, TRex Bio, and VelosBio. In addition, Adimab expanded its collaborations with Acceleron, Alector, Biotheus, Boehringer Ingelheim, Celgene (now a Bristol-Myers Squibb company), Innovent Biologics, Inc., Novartis, Regeneron, Scholar Rock, Takeda Pharmaceuticals, and others.

In 2019, Adimab also entered into a new Platform Transfer agreement with Takeda Pharmaceuticals.

"We are very happy with the number of new 2019 partnerships," said Guy Van Meter, Chief Business Officer of Adimab. "That said, our most important success metric from a deal perspective is partners coming back for expansions. More than 70% of our current partners, once they have seen our capabilities, have expanded their relationships with us."

"Historically, Adimab has positioned itself as the industry leader for generating the highest quality therapeutic IgGs with exquisite specificity and excellent developability profiles," said Tillman Gerngross, Chief Executive Officer and Co-Founder of Adimab. "We are increasingly expanding into adjacent areas; for example, we are engineering more and more IgGs and non-IgGs derived from other technologies. We are working with many types of bispecific molecules, and our new proprietary CD3 binders have been used in more than ten partnerships."

In the last six years, Adimab has worked with an increasing number of smaller companies backed by leading venture capital firms. Following successful antibody discovery campaigns, many of these companies have gone on to partner their programs with larger pharmaceutical companies. To date, more than 15 programs developed by venture-backed partners have been out-licensed to larger pharma companies. Transactions publicly announced in 2019 include: a Black Belt Therapeutics program out-licensed to CASI Pharmaceuticals, a Dragonfly program out-licensed to Abbvie, Iconic granted an option to Exelixis, Iconic granted an option to Novartis, among others.

On January 10, 2010 SurvivorNet has reported just closed a new round of funding, bringing its total outside investment to $15-million (Press release, SurvivorNet, JAN 10, 2020, View Source [SID1234553044]). The latest $10-million investment was led by London-based asset manager Gatemore Ventures, and will be used to accelerate SurvivorNet’s growth and expand its coverage across various types of cancer.

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!

1.5 million unique visitors per month are now coming to SurvivorNet to access information from some of the world’s top oncologists, representing a powerful presence in the cancer market which sees 1.8 million new diagnoses per year in the U.S.

"If something was really wrong with someone you loved, you wouldn’t want them to turn to Dr. Google," says SurvivorNet CEO & Co-Founder Steve Alperin. "SurvivorNet started as a personal mission to give families like mine a better source for cancer information. With the help of our extraordinary partners in the cancer community, we are now helping millions of people get better information that can help save their lives or make their journey a little easier. With this new support from our investors we are going to continue expanding our coverage across more cancer types and bring more of the country’s leading experts to the people who need them."

"We believe that SurvivorNet could change the landscape for all participants in the healthcare industry, and we are excited about our involvement in the company’s growth," says George Cadbury, Partner at Gatemore.

70% of Americans are not treated at a major cancer center, and the data is clear that these patients don’t respond as well to treatment. There is an astounding 10% higher death rate for many types of cancer patients treated outside academic centers. SurvivorNet is addressing this gap by collaborating with many of the country’s top cancer centers to make their experts available to the millions of people who can’t get to them but desperately need the information. Dozens of the leading comprehensive cancer centers in the country have contributed to SurvivorNet, including MD Anderson, Memorial Sloan Kettering, Cleveland Clinic, The Mayo Clinic, Stanford, and Cedars Sinai.

"Health information is the one problem in healthcare that we all suffer from, and nobody has really addressed it. It’s a dramatically underinvested space," says Alperin. "By building a deeper, higher quality product in this space, we are helping patients ask better questions. With so much incredible progress in cancer right now, doctors have trouble keeping up. So patients need to go in armed and ready. A simple example is ovarian cancer. 80% of women are still not offered a genetic test that could make them eligible for medication to greatly extend their lives. We think this information gap is unacceptable and we are doing something about it."

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).

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!

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.