On January 6, 2020 BridgeBio Pharma, Inc. (Nasdaq: BBIO) subsidiary QED Therapeutics reported that it has secured both Fast Track Designation in adults with first-line advanced or metastatic cholangiocarcinoma and Orphan Drug Designation for infigratinib for treatment of cholangiocarcinoma (Press release, BridgeBio, JAN 6, 2020, View Source [SID1234576234]). In addition, the company announced that enrollment is ongoing and patient dosing has started in the PROOF trial, a Phase 3 clinical trial evaluating oral infigratinib, an investigational drug, in adults for first-line treatment of advanced cholangiocarcinoma with FGFR2 (fibroblast growth factor receptor 2) gene fusions or translocations.

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Cholangiocarcinoma, a cancer of the bile ducts of the liver, is a serious and often fatal disease which affects approximately 20,000 people in the United States and European Union each year. FGFR2 genetic aberrations are present in approximately 15% to 20% of people who have this disease. Currently, treatment options are limited, and the 5-year survival rate is only 9%.1

Infigratinib received Fast Track Designation for first-line treatment of adult patients with unresectable locally advanced or metastatic cholangiocarcinoma with FGFR2 gene fusions or translocations.

"We believe that Fast Track and Orphan Drug Designations for infigratinib for the treatment of cholangiocarcinoma underscores the need for new, targeted treatments for genetically-driven subsets of this cancer, particularly for adults with first-line advanced or metastatic cholangiocarcinoma," said Susan Moran, MD, MSCE, chief medical officer for QED. "Fast Track Designation will enhance our interaction with the FDA on our first-line advanced or metastatic cholangiocarcinoma program and may help us get this medicine to patients more quickly."

The PROOF trial will enroll approximately 384 patients with first-line cholangiocarcinoma with FGFR2 fusions or translocations, as determined by molecular profiling. The primary endpoint is progression-free survival compared to standard of care chemotherapy (gemcitabine and cisplatin). Patients will be randomized 2:1 to infigratinib versus standard of care.

"Importantly, in this trial, patients who are assigned to receive standard of care will be allowed to cross over and receive infigratinib if they do not respond to chemotherapy," said Stacie Lindsey, president of the Cholangiocarcinoma Foundation. "Having a crossover option is very significant to patients and including it in the design of this trial demonstrates that QED is listening to them."

For additional information on the PROOF trial, including eligibility, patients should ask their physician, visit clinicaltrials.gov, or email [email protected]

For more information on molecular profiling, patients can find resources from the Cholangiocarcinoma Foundation at View Source

About Orphan Drug Designation

Under the FDA’s Orphan Drug Designation program, orphan drug designation is granted by the FDA to drugs or biologics intended to treat rare diseases or conditions. The designation allows the drug developer to be eligible for a seven-year period of U.S. marketing exclusivity upon approval of the drug, if the drug receives the first FDA approval for the rare disease or condition, as well as tax credits for clinical research costs, the ability to apply for annual grant funding, clinical trial design assistance, and the waiver of Prescription Drug User Fee Act (PDUFA) filing fees.

About U.S. FDA’s Fast Track Designation Program

The FDA’s Fast Track program was established to facilitate the development and expedite the review of drugs with the potential to treat serious conditions and address an unmet medical need. Companies that receive Fast Track designation are provided the opportunity for more frequent interactions with FDA during clinical development and are eligible for accelerated approval and/or priority review, if relevant criteria are met. Additionally, companies that receive Fast Track designation are allowed to submit completed sections of their New Drug Application (NDA) or Biologics License Application (BLA) for the drug on a rolling basis, resulting in the potential for an expedited FDA review process.

On January 6, 2020 Black Diamond Therapeutics reported that its series A raise is going for a $100 million IPO (Press release, Black Diamond Therapeutics, JAN 6, 2020, View Source [SID1234553607]).

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It’s been a whirlwind 12 months, with two quick-fire raises coupled with a series of big-name execs coming to the biotech, which nabbed Yumanity Therapeutics, Merck KGaA and SR One veterans back in September to help head up its research work.

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This trio of new research leads are Christopher Roberts, Ph.D., chief scientific officer; Matt Lucas, Ph.D., vice president of chemistry; and Tai-An Lin, Ph.D., VP and head of biology.

Roberts oversees all of Black Diamond’s research and early development. Lucas will run its chemistry work in Cambridge, Massachusetts, while Lin is heading up its labs in New York.

The new CSO comes to the biotech from SR One, the corporate venture capital arm of GlaxoSmithKline, where he was an entrepreneur in residence. Lucas, meanwhile, was the senior director of chemistry at Parkinson’s disease biotech Yumanity.

Lin has been director of the immuno-oncology translational innovation platform and experimental medicine at Merck KGaA/EMD Serono, with stints before this on a series of Big Pharma drug discovery teams at Bristol-Myers Squibb, Roche and Janssen.

The Versant Ventures biotech raised $85 million in a series B at the start of the year, coming just weeks after Black Diamond exited stealth mode. It broke cover with a $20 million in series A and a platform designed to identify and target allosteric mutant oncogenes.

Together, these cash grabs teed up the allosteric oncogenic mutation specialist up to move two or three candidates into the clinic in the coming years: It now wants an extra $100 million from the public markets to help in this mission.

The biotech’s founders David Epstein and Elizabeth Buck designed the platform to unlock the therapeutic potential of oncogenes activated by allosteric mutations, which, unlike kinase domain mutations, are yet to make a mark on cancer care.

If the platform works as Black Diamond hopes, it will generate single molecules capable of treating baskets of mutations previously seen as unactionable from a therapeutic perspective.

The company is now focused on getting its lead program, BDTX-189, into the clinic to test this platform. According to its Securities and Exchange Commission filing, the biotech submitted its IND for BDTX-189 in November 2019 and got the FDA green light a month later.

Black Diamond is plotting a combined phase 1/2 clinical trial in the first half of 2020. "The Phase 1 portion of the trial will evaluate escalating doses of BDTX-189 and is designed to determine the recommended Phase 2 dose and to assess preliminary indications of anti-tumor activity," it said in the filing.

"The Phase 2 portion will determine the objective response rate, or ORR, and duration of response in patients with solid tumors that have an allosteric HER2 mutation or EGFR or HER2 exon 20 insertion mutation determined using next-generation sequencing, or NGS. Depending on results, we plan to pursue an accelerated approval of BDTX-189 for a tumor-agnostic indication in patients with mutations of the ErbB family."

It’s also working on a preclinical candidate it hopes can combat glioblastoma, a particularly aggressive form of brain cancer with few treatments and poor outcomes.

For this candidate, Black Diamond said: "Our lead molecules are designed to be potent, allosteric EGFR selective and to be brain penetrant. We have observed measurable brain exposure in animal models. We are completing preclinical characterization of our glioblastoma candidate leads and plan to select a development candidate in 2020."

The Cambridge, Massachusetts-based company plans to list on the Nasdaq under the symbol "BDTX."

On January 6, 2020 Merck (NYSE: MRK), known as MSD outside the United States and Canada, reported an exclusive worldwide research collaboration and license agreement with Taiho Pharmaceutical Co, Ltd., ("Taiho") and Astex Pharmaceuticals (UK), a wholly owned subsidiary of Otsuka Pharmaceutical Co., Ltd. ("Astex"), focused on the development of small molecule inhibitors against several drug targets, including the KRAS oncogene, which are currently being investigated for the treatment of cancer (Press release, Merck & Co, JAN 6, 2020, View Source [SID1234553268]).

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"At Merck we continue to pursue new regimens designed to extend the benefits of highly selective therapies to more patients with cancer," said Dr. Roger M. Perlmutter, president, Merck Research Laboratories. "This agreement with Taiho and Astex combines our respective small molecule assets and industry-leading expertise in cancer cell signaling to enable development of the most promising drug candidates."

Under the terms of the agreement, Merck, Taiho and Astex will combine preclinical candidates and their data with knowledge and expertise from their respective research programs. In exchange for providing Merck an exclusive global license to their small molecule inhibitor candidates, Taiho and Astex will receive an aggregate upfront payment of $50 million and will be eligible to receive approximately $2.5 billion contingent upon the achievement of preclinical, clinical, regulatory and sales milestones for multiple products arising from the agreement, as well as tiered royalties on sales. Merck will fund research and development and will be responsible for commercialization of products globally. Taiho has retained co-commercialization rights in Japan and an option to promote in specific areas of South East Asia.

"Taiho has used its unique and proprietary drug discovery platform to generate a number of small molecule inhibitors," said Teruhiro Utsugi, Ph.D., managing director at Taiho. "This alliance builds on our KRAS research up to now and together with Merck, allows us to combine our expertise to significantly accelerate the global research, development, and commercialization of a number of our mutant KRAS programs by accessing external talent and resources."

"Together with our Taiho colleagues we are delighted to be working with Merck, one of the global leaders in oncology drug development, on this strategic alliance. This collaboration is another testament to Astex’s position as the leader in fragment-based drug discovery," said Harren Jhoti, Ph.D., president and CEO of Astex.

KRAS is among the most frequently mutated oncogenes in cancer. It is estimated to occur in more than 90% of pancreatic cancers and approximately 20% of non-small cell lung cancers (NSCLC) and is associated with poorer outcomes.

Merck’s Focus on Cancer

Our goal is to translate breakthrough science into innovative oncology medicines to help people with cancer worldwide. At Merck, the potential to bring new hope to people with cancer drives our purpose and supporting accessibility to our cancer medicines is our commitment. As part of our focus on cancer, Merck is committed to exploring the potential of immuno-oncology with one of the largest development programs in the industry across more than 30 tumor types. We also continue to strengthen our portfolio through strategic acquisitions and are prioritizing the development of several promising oncology candidates with the potential to improve the treatment of advanced cancers. For more information about our oncology clinical trials, visit www.merck.com/clinicaltrials.

On January 6, 2020 Precigen, Inc., a biopharmaceutical company specializing in the development of innovative gene and cellular therapies to improve the lives of patients, reported that the US Food and Drug Administration (FDA) has granted orphan drug designation (ODD) to PRGN-3006, a first-in-class investigational therapy using Precigen’s non-viral UltraCAR-T therapeutic platform for patients with relapsed or refractory acute myeloid leukemia (AML) (clinical trial identifier: NCT03927261) (Press release, Precigen, JAN 6, 2020, View Source [SID1234552952]). Precigen announced in Q3 2019 that it had completed enrollment for the first cohort of this clinical trial and expects an initial data readout in the second half of 2020.

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The FDA grants ODD status to medicines intended for the treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the US. Medicines that receive the ODD designation may qualify for a number of incentives that help to expedite and reduce the cost of development, approval and commercialization.

"This regulatory designation underscores the critical medical need for new therapies to treat AML patients. AML is a progressive, debilitating and often fatal disease with limited treatment options," said Helen Sabzevari, PhD, President and CEO of Precigen*. "As the first regulatory designation for our proprietary UltraCAR-T platform, this orphan drug designation helps to advance the PRGN-3006 investigational therapy and provides important incentives and support to deliver this medicine as rapidly as possible for those patients suffering from this disease."

PRGN-3006 utilizes Precigen’s transformative UltraCAR-T therapeutic platform, which eliminates ex vivo expansion, reduces manufacturing time and provides the ability to administer CAR-T therapy to patients only one day after non-viral gene transfer at the cancer center. PRGN-3006 UltraCAR-T is a multigenic CAR-T cell treatment utilizing Precigen’s advanced non-viral gene delivery system to co-express a chimeric antigen receptor, membrane-bound interleukin‐15 (mbIL15), and a kill switch for better precision and control in targeting relapsed or refractory AML and higher risk MDS.

Preclinical data for PRGN-3006 UltraCAR-T evaluating non-viral, multigenic autologous CAR-T cells administered one day after gene transfer for the treatment of AML and MDS were presented at the 2019 American Society for Hematology (ASH) (Free ASH Whitepaper) annual meeting and exposition in Orlando, Florida.

About Acute Myeloid Leukemia (AML)
AML is a cancer that starts in the bone marrow, but most often moves into the blood1. Though considered rare, AML is among the most common types of leukemia in adults2. In 2019, it was estimated that 21,450 new cases of AML would be diagnosed in the US2. AML is uncommon before the age of 45 and the average age of diagnosis is about 682. The prognosis for patients with AML is poor with an average 5‐year survival rate of approximately 25 percent overall, and less than a 5 percent 5‐year survival rate for patients older than 653. Amongst elderly AML patients (≥ 65 years of age), median survival is short, ranging from 3.5 months for patients 65 to 74 years of age to 1.4 months for patients ≥ 85 years of age3.

About the FDA Orphan Drug Designation
FDA orphan drug designation is granted to drugs intended to treat rare diseases or disorders that affect fewer than 200,000 people in the US. Medicines that receive the ODD designation may qualify for a number of incentives that help to expedite and reduce the cost of development, approval and commercialization, such as grants, clinical design support, FDA fee waivers, tax incentives, and seven years of market exclusivity.

Precigen : Advancing Medicine with PrecisionTM
Precigen is a dedicated discovery and clinical stage biopharmaceutical company advancing the next generation of gene and cellular therapies using precision technology to target the most urgent and intractable diseases in immuno-oncology, autoimmune disorders, and infectious diseases. Precigen also follows the science opportunistically in pursuit of promising programs in emerging therapeutics. Our technologies enable us to find innovative solutions for affordable biotherapeutics in a controlled manner. Precigen operates as an innovation engine progressing a preclinical and clinical pipeline of well-differentiated unique therapies toward clinical proof-of-concept and commercialization. Precigen leverages a diverse portfolio of technology platforms to advance human health. For more information about Precigen, visit www.precigen.com or follow us on Twitter @Precigen and LinkedIn.

*Precigen’s parent company, Intrexon Corporation (Nasdaq: XON), announced on January 2, 2020 that it will refocus the company on healthcare, will change its name to Precigen, Inc., and has appointed Helen Sabzevari, PhD, as President and CEO. The parent company will continue to hold, among its several health assets, all of Precigen’s discovery and clinical stage technology and programs.

Precigen’s UltraCAR-TTM Therapeutic Platform
Precigen’s UltraCAR-T platform has the potential to disrupt the CAR-T treatment landscape by increasing patient access through shortening manufacturing time, decreasing manufacturing-related costs, and improving outcomes using advanced approaches for precise tumor targeting and control of the immune system. The platform brings several key advancements: 1) Non-viral gene transfer using multigenic vectors for expression of multiple effector genes leads to better precision and control of tumor targeting and eliminates the need for virus; 2) Sustained persistence and desired phenotype of infused UltraCAR-T helps address T-cell exhaustion, a common issue with current CAR-T therapies; 3) T-cell control by incorporation of kill switch technology to potentially improve the safety profile; and 4) Rapid manufacturing of UltraCAR-T cells using our proprietary non-viral gene transfer process, which eliminates the need for ex vivo propagation, thus dramatically reducing wait times for patients from weeks to fewer than two days.

On January 6, 2020 Aeglea BioTherapeutics, Inc. (NASDAQ:AGLE), a clinical-stage biotechnology company developing next-generation human enzyme therapeutics as solutions for diseases with high unmet medical need, reported it will present at the 38th Annual J.P. Morgan Healthcare Conference to be held January 13-16 in San Francisco, CA (Press release, Aeglea BioTherapeutics, JAN 6, 2020, View Source [SID1234552791]).

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Presentation Details

Highlights: Aeglea will present on its strategic focus in developing innovative human enzyme therapeutics with defined potential to address Arginase 1 Deficiency, Homocystinuria and Cystinuria.
Date: Wednesday, January 15, 2020
Time: 2:30 p.m. PST; 5:30 p.m. EST
Presenter: Anthony G. Quinn, M.B. Ch.B., Ph.D., Aeglea’s president and chief executive officer
Location: Westin St. Francis Hotel, San Francisco, California
Webcast: View Source

To access the live and archived audio webcast, visit the Presentations & Events section of the Aeglea BioTherapeutics investor relations website. Please connect to the website at least 15 minutes prior to the presentation to allow for any software download that may be necessary. Replays of the webcast will be available for 30 days thereafter.