On January 4, 2019 Blueprint Medicines Corporation (NASDAQ: BPMC), a precision therapy company focused on genomically defined cancers, rare diseases and cancer immunotherapy, reported "2020 Blueprint," a two-year strategy to launch Blueprint Medicines’ global commercial business (Press release, Blueprint Medicines, JAN 4, 2019, View Source [SID1234532460]). Under this strategy, by the end of 2020, the company expects to have two marketed products and at least four additional marketing applications pending in the United States and Europe. In addition, Blueprint Medicines reported recent milestones and key goals through 2020 across its differentiated portfolio of investigational precision therapies.

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"Our vision is to rapidly evolve Blueprint Medicines into the leading global precision therapy company, with a robust scientific platform reproducibly designing innovative compounds and an effective and nimble commercial organization delivering a portfolio of important medicines to patients worldwide," said Jeff Albers, Chief Executive Officer of Blueprint Medicines. "As we enter the new year, we are at the precipice of this transformation with the planned submission of our first new drug application for our lead therapeutic candidate avapritinib and growing momentum in building our commercial capabilities. Combined with seven ongoing or planned registration-enabling clinical trials for avapritinib and BLU-667, we believe Blueprint Medicines is well-positioned to quickly capitalize on this first potential regulatory approval and realize our goal of becoming a portfolio-based commercial-stage company."

"2020 Blueprint" Global Business Strategy

The "2020 Blueprint" strategy establishes a path to transform Blueprint Medicines into a global commercial enterprise focused on delivering a portfolio of precision therapies to patients with cancer and rare diseases. Under this strategy, Blueprint Medicines anticipates achieving the following by the end of 2020:

2 marketed products in the United States and 1 marketed product in Europe
4 additional marketing applications pending in the United States and Europe
6 therapeutic candidates in global clinical development
8 research programs that leverage strategic areas of focus
"Our ‘2020 Blueprint’ strategy extends to our ongoing research efforts, where we are advancing multiple programs designed to complement our scientific and clinical expertise and planned commercial profile," said Marion Dorsch, Ph.D., Chief Scientific Officer of Blueprint Medicines. "Under this strategy, we have three research areas of focus: franchise opportunities in gastrointestinal stromal tumors, systemic mastocytosis and lung cancer where we believe our understanding of mutational drivers gives us an advantage; cancer immunotherapy under our ongoing collaboration with Roche; and novel genetic drivers that we characterize and target with our differentiated scientific platform."

Recent Portfolio Milestones and Key Goals through 2020

RECENT MILESTONES:

Blueprint Medicines today announced:

Top-line results from the Phase 1 NAVIGATOR clinical trial of avapritinib in patients with advanced gastrointestinal stromal tumors (GIST), as of a data cutoff date of November 16, 2018. These data will be used to support the submission of a planned new drug application (NDA) to the U.S. Food and Drug Administration (FDA) in the first half of 2019 for the treatment of patients with PDGFRA Exon 18 mutant GIST, which primarily includes PDGFRα D842V GIST, and fourth-line GIST. The primary endpoints for registration are objective response rate (ORR) and duration of response (DOR) based on central radiology and modified Response Evaluation Criteria in Solid Tumors version 1.1 (mRECIST 1.1) criteria.
In 43 patients with PDGFRA Exon 18 mutant GIST treated with a starting dose of 300 or 400 mg once daily (QD), the ORR was 86 percent (one response pending confirmation). Median DOR was not reached.
In 111 patients with fourth-line GIST treated with a starting dose of 300 or 400 mg QD, the ORR was 22 percent (one response pending confirmation). Median DOR was 10.2 months.
Top-line safety results were consistent with those previously reported. Avapritinib was well-tolerated, and most adverse events (AEs) reported by investigators were Grade 1 or 2. Across all doses (n=237), only 23 patients (9.7 percent) discontinued treatment with avapritinib due to treatment-related AEs.
Two registration-enabling clinical trials for avapritinib in systemic mastocytosis (SM) are now underway. The first clinical site recently opened for the Phase 2 PIONEER clinical trial for patients with indolent and smoldering SM, and initial patient screening for this trial is anticipated in January 2019. In addition, patient dosing has been initiated in the Phase 2 PATHFINDER clinical trial for patients with advanced SM.
As part of the collaboration with CStone Pharmaceuticals, the China National Medicinal Products Administration has approved an investigational new drug (IND) application for the ongoing global Phase 1 trial of BLU-554 in advanced hepatocellular carcinoma. The companies expect to initiate patient enrollment in the trial by the middle of 2019.
The FDA has cleared an IND application for BLU-782, a selective ALK2 inhibitor in development for patients with fibrodysplasia ossificans progressiva (FOP), and the company plans to initiate a Phase 1 clinical trial in healthy volunteers in the first quarter of 2019.
KEY GOALS:

Blueprint Medicines reported the following key goals and anticipated milestones through 2020:

Avapritinib: advanced gastrointestinal stromal tumors

Submit an NDA to the FDA for PDGFRA Exon 18 mutant GIST and fourth-line GIST in the first half of 2019.
Present additional data from the Phase 1 NAVIGATOR trial in the first half of 2019.
Complete enrollment of the Phase 3 VOYAGER trial in third- or fourth-line GIST in the second half of 2019.
Initiate the Phase 3 COMPASS-2L precision medicine trial in second-line GIST in the second half of 2019.
Submit an NDA to the FDA for third-line GIST in 2020.
Avapritinib: systemic mastocytosis

Present updated data from the Phase 1 EXPLORER trial in advanced SM in the first half of 2019.
Present initial data from the Phase 2 PIONEER trial in indolent and smoldering SM in the second half of 2019.
Complete enrollment of the Phase 2 PATHFINDER trial in advanced SM in the second half of 2019.
Submit an NDA to the FDA for advanced SM in 2020.
BLU-667: RET-altered non-small cell lung cancer (NSCLC), medullary thyroid cancer (MTC) and other solid tumors

Present updated data from the Phase 1 ARROW trial in RET-altered cancers in the first half of 2019.
Complete enrollment of previously treated NSCLC and MTC cohorts in the Phase 1 ARROW trial in the first half of 2019.
Initiate a Phase 3 trial in first-line RET-fusion NSCLC in the second half of 2019.
Initiate a Phase 2 combination trial of BLU-667 and osimertinib in treatment-resistant, EGFR-mutant NSCLC harboring an acquired RET alteration in the second half of 2019.
Submit an NDA to the FDA for second-line RET-fusion NSCLC and second-line RET-mutant MTC in the first half of 2020.
BLU-554: advanced hepatocellular carcinoma

Enroll the first patient in China in the ongoing global Phase 1 trial of BLU-554 under the collaboration with CStone Pharmaceuticals by the middle of 2019.
Initiate a Phase 1 combination trial of BLU-554 and CS-1001, CStone Pharmaceuticals’ anti-PD-L1 inhibitor, in China in the second half of 2019.
BLU-782: fibrodysplasia ossificans progressiva

Initiate a Phase 1 trial in healthy volunteers in the first quarter of 2019.
Initiate a Phase 2 trial in patients with FOP in the first half of 2020.
Research portfolio

Provide an update on the company’s robust and diverse research portfolio, including disclosure of up to two new targets, at a Blueprint Medicines Research and Development day in 2019.
Nominate at least one new wholly-owned discovery program in 2019.

On January 4, 2019 MabVax Therapeutics Holdings, Inc. (OTC Pink: MBVX), a clinical-stage immuno-oncology drug development company with a fully human antibody discovery platform focused on the clinical development of products to address unmet medical needs in the treatment of cancer and pancreatitis, reported that it has entered into discussions to merge with Oncotelic, Inc., a privately held cancer immunotherapy company (Press release, MabVax, JAN 4, 2019, View Source [SID1234532459]). Oncotelic is developing a unique TGF-b antisense therapy which has demonstrated the ability to break immune tolerance in mid-stage clinical trials for the treatment of glioblastoma and pancreatic cancer.

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Based on terms of a non-binding letter of intent signed by both companies on January 3, 2019, MabVax and Oncotelic are entering discussions to combine the companies to form a publicly traded company focused on the development of proprietary immunotherapy-based products of both companies to diagnose and treat cancer. Under the terms of the letter of intent, Oncotelic will merge with a wholly-owned subsidiary of MabVax in an all-stock transaction and will become a wholly-owned subsidiary of MabVax. The merger is subject to the approval of the MabVax board of directors and achieving certain financing objectives and other customary conditions. Upon closing of the transaction, MabVax will be re-named Oncotelic, Inc. and will operate under the leadership of the combined Oncotelic and MabVax management teams, with Vuong Trieu, founder of Oncotelic, an experienced and successful biotechnology entrepreneur, as executive chairman. Certain current senior management team members at MabVax will remain with the merged companies to fill key operational roles.

On a pro forma basis, calculated at the close of the merger, the current MabVax stockholders will own 25% and Oncotelic stockholders will own 75% of the combined company, respectively. The merger agreement contemplates securing financing of at least $10 million simultaneous with execution of the merger to support the clinical development of Trabedersen, Oncotelic’s TGF-b antisense therapy, in both glioblastoma and pancreatic cancer trials.

Vuong Trieu, Ph.D., who will be executive chairman of the company post-merger, said, "Our goal has been to grow a strong RNA therapeutic company that leverages innate immunity to achieve durable and effective immunotherapies for solid tumors and the merger with MabVax will allow us to complement that effort with key programs and capabilities from MabVax that will significantly strengthen the effort."

David Hansen, President and CEO of MabVax, said, "Through this transaction we hope to leverage our clinical and operational expertise in the post-merger company to advance the clinical development of Trabedersen while also integrating the development of key assets discovered by MabVax. This will allow us to continue the process of realizing the maximum value of assets we have developed through a process already initiated with Objective Capital while simultaneously maximizing the value of our lead technologies for stockholders."

On December 13, 2018, MabVax announced that it had engaged Objective Capital Partners, LLC to serve as a financial advisor to assist MabVax in exploring the sale of clinical and preclinical assets of the Company with the goal of maximizing the value of these assets within the near term. MabVax’s lead monoclonal antibody product, 5B1 for the treatment of pancreatic cancer, could be one of the assets that potentially could be developed into later stage clinical trials by the combined company.

On January 4, 2019 Idera Pharmaceuticals, Inc. (NASDAQ: IDRA) reported that the company will present at the 37th Annual J.P. Morgan Healthcare Conference on Wednesday, January 9, 2019 at 8:30 a.m. Pacific Time (11:30 a.m. Eastern Time) at the Westin St. Francis Hotel in San Francisco (Press release, Idera Pharmaceuticals, JAN 4, 2019, View Source [SID1234532458]). Idera’s Chief Executive Officer, Vincent Milano, will present a corporate overview and outlook for 2019 during the presentation.

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A live audio webcast of Idera’s presentation will be accessible in the Investors and Media section of Idera’s website at View Source An archived version will also be available on the company’s website after the event for 90 days. As a convenience to investors, a copy of the company’s presentation will be posted on the Idera corporate website at 7:30 a.m. Eastern Time on Monday, January 7, 2019.

On January 4, 2019 Aura Biosciences, a leader in the development of novel targeted therapies in ocular oncology, reported that it has received written confirmation from the U.S. Food and Drug Administration (FDA) regarding agreement on the design of its Phase 3 registration trials designed to evaluate light-activated AU-011 for the treatment of patients with choroidal melanoma (Press release, Aura Biosciences, JAN 4, 2019, View Source [SID1234532454]). This written confirmation is the result of successful outcome of an "End of Phase 2" meeting with the FDA.

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The STARBRIGHT registration program will consist of two small identical clinical trials designed to assess the safety and efficacy of AU-011 versus sham control for the treatment of patients with small choroidal melanoma and high risk indeterminate lesions. Both trials, which will be titled STARBRIGHT1 and STARBRIGHT3, will be global, multicenter, randomized and masked, and will be conducted in parallel. The primary endpoint will be comprised of a combination of tumor control and vision preservation.

In addition to the design of the Phase 3 trials, the FDA agreed with Aura’s proposed safety database. The FDA also agreed that no further non-clinical studies are needed.

"We are pleased to have received such clear guidance from the FDA with respect to the Phase 3 STARBRIGHT program to be able to meet the scientific and regulatory requirements for marketing approval in the U.S." said Elisabet de los Pinos, Ph.D., Chief Executive Officer of Aura.

The currently available treatments for choroidal melanoma come with the risk of severe vision loss, especially for patients with melanomas located close to the fovea or optic disk. The ongoing Phase 1b/2 study with light-activated AU-011 has shown that the drug was well-tolerated, with clear evidence of tumor control and preservation of visual acuity at long term follow up.

"We believe that a minimally invasive, non-radiation-based treatment option that enables early intervention while preserving vision has the potential to transform the therapeutic landscape for this difficult to treat, often deadly form of melanoma," said Cadmus Rich, M.D., Chief Medical Officer of Aura. "Overall, the meeting removed any remaining uncertainty on the regulatory path to approval and highlighted FDA’s commitment to guide Aura toward a potential first drug approved for patients with this highly unmet medical need."

About Choroidal Melanoma

Choroidal melanoma is a rare and aggressive type of eye cancer. Choroidal melanoma is the most common primary ocular tumor and develops in the uveal tract of the eye. No targeted therapies are available at present, and current radiotherapy treatments can be associated with severe visual loss and other long-term sequelae such as dry eye, glaucoma, cataracts and radiation retinopathy. The most common current treatment is plaque radiotherapy, which involves surgical placement of a radiation device on the exterior of the eye over the tumor. The alternative is enucleation, or total surgical removal of the eye. Choroidal melanoma metastasizes to the liver in about 40-50 percent of cases in the long term (source: OMF), and only 15 percent of patients whose melanoma has metastasized survive beyond five years after diagnosis (source: ACS).

About Light-Activated AU-011

AU-011 is a first-in-class targeted therapy in development for the primary treatment of choroidal melanoma. The therapy consists of proprietary viral-like particle bioconjugates (VPB) that are activated with an ophthalmic laser. The VPBs bind selectively to unique receptors on cancer cells in the eye and are derived from technology originally pioneered by Dr. John Schiller of the Center for Cancer Research at the National Cancer Institute (NCI), recipient of the 2017 Lasker-DeBakey Award. Upon activation with an ophthalmic laser, the drug rapidly and specifically disrupts the cell membrane of tumor cells while sparing key eye structures, which may allow for the potential of preserving patients’ vision and reducing other long-term complications of radiation treatment. AU-011 can be delivered using equipment commonly found in an ophthalmologist’s office and does not require a surgical procedure, pointing to a potentially less invasive, more convenient therapy for patients and physicians. AU-011 for the treatment of choroidal melanoma has been granted orphan drug and fast track designations by the U.S. Food and Drug Administration and is currently in clinical development.

On January 4, 2019 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 that Isaac Ciechanover, M.D., the Company’s President and Chief Executive Officer, will present at the 37th Annual J.P. Morgan Healthcare Conference on Tuesday, January 8, 2019 at 10:30 a.m. Pacific Standard Time (Press release, Atara Biotherapeutics, JAN 4, 2019, View Source [SID1234532447]). The conference will be held at the Westin St. Francis in San Francisco, California.

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A live audio webcast of the 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.