On December 2, 2016 PIQUR Therapeutics AG, a Swiss clinical-stage pharmaceutical company, reported that
the U.S. Food & Drug Administration (FDA) has granted orphan drug designation to PIQUR’s lead compound PQR309 for the treatment of primary central nervous system lymphoma (PCNSL) (Press release, PIQUR Therapeutics, DEC 2, 2016, View Source [SID1234527272]).

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PCNSL is a very rare and aggressive form of lymphoma involving brain and its linings, eyes or spinal cord. The disease affects less than 1 person in 100,000 in the USA with approximately 7,500 new cases reported annually in the USA, Europe and Japan. Treatment options available for PCNSL are limited, and the prognosis with current therapies is poor.

"We are very pleased to receive FDA orphan drug designation for PQR309 in PCNSL. This is an important regulatory milestone for the company and a significant step towards the clinical advancement of PQR309," said Dr. Ruggero Della Bitta, Chief Medical Officer of PIQUR. "This represents an important step towards addressing a high unmet medical need and bringing a potential treatment to those with this rare and life-threatening disease."

The Orphan Drug Designation Program, administered by the FDA’s Office of Orphan Products Development, is intended to encourage companies to develop therapeutics for diseases that affect fewer than 200,000 people in the USA. The designation provides the company several benefits and incentives, including assistance with clinical study design and drug development, tax credits for qualified clinical trials costs, exemptions from certain FDA application fees, as well as a seven-year period of market exclusivity upon regulatory product approval.

About PQR309
PIQUR’s lead compound, PQR309, is an oral, brain-penetrant, dual inhibitor of the PI3K/mTOR pathway, which is activated in 60 – 80% of human cancers. Unlike most of its competitors, PQR309 crosses the blood-brain barrier, expanding its use to malignant diseases involving the brain. Preclinical and Phase 1 studies have shown PQR309 to have a favorable safety, tolerability and pharmacokinetic profile. In addition, PQR309 has shown both preclinical
activity in various tumor models and clinical activity in Phase 1 and 2 studies.

PQR309 is currently being investigated in five Phase 1 and 2 clinical studies in advanced solid tumors (NCT02483858), relapsed or refractory lymphoma (NCT02249429), relapsed or refractory PCNSL (NCT02669511) and progressive glioblastoma multiforme (NCT02850744). In addition, the PIQHASSO Phase 1/2b study investigates PQR309 in combination with Eisai’s Eribulin in metastatic HER2-negative and triple-negative breast cancer (NCT02723877).

On December 1, 2016 Alligator presented the corporate presentation (Presentation, Alligator Bioscience, DEC 1, 2016, View Source [SID1234538696]).

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On December 1, 2019 Xenetic Biosciences, Inc. (NASDAQ: XBIO) ("Xenetic" or the "Company"), a clinical-stage biopharmaceutical company focused on the discovery, research and development of next-generation biologic drugs and novel orphan oncology therapeutics, reported that Scott Maguire, CEO of Xenetic Biosciences, will present at the LD Micro Main Event 2016 Annual Conference on Wednesday, December 7, 2016 at 4:30 p.m. PT at the Luxe Sunset Boulevard Hotel in Los Angeles (Press release, Xenetic Biosciences, DEC 1, 2016, View Source [SID1234537807]).

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During his presentation, Mr. Maguire will provide a corporate update and discuss the Company’s clinical and regulatory progress for its in-house product candidates, as well as those being developed with Xenetic’s partners. Xenetic’s current in-house product pipeline includes Virexxa (sodium cridanimod), which is being evaluated for the treatment of endometrial cancer and triple negative breast cancer, and ErepoXen, a polysialylated form of erythropoietin (EPO), a hormone created by the kidneys to maintain red blood cell production and address anemia. Xenetic is also currently evaluating OncoHist for the treatment of acute myeloid leukemia (AML) in refractory patients.

Mr. Maguire will also discuss the Company’s up to $100 million license deal with Shire, one of the Company’s largest shareholders, along with the clinical status of their product candidate.

A live webcast of the presentation will be available by accessing the IR Calendar in the Investors section of Xenetic’s website (www.xeneticbio.com). A replay of the webcast will be available for 90 days, starting approximately two hours after the presentation ends.

On December 1, 2016 Deciphera Pharmaceuticals reported initial clinical data from an ongoing Phase 1 study of DCC-2618, a pan-KIT and PDGFRα targeted tyrosine kinase inhibitor in development for the treatment of genetically-defined cancers, including gastrointestinal stromal tumors (GIST) as well as other KIT-driven diseases such as systemic mastocytosis (Press release, Deciphera Pharmaceuticals, DEC 1, 2016, View Source [SID1234516905]). These data support DCC-2618 as a potential treatment option for patients with these difficult-to-treat solid tumor cancers based on encouraging tumor responses and preliminary data showing decreases in circulating tumor DNA that codes for KIT mutations in heavily-pretreated GIST patients with multiple resistance mutations. The clinical results were described in a late-breaking oral presentation at the 28th EORTC-NCI-AACR (Free EORTC-NCI-AACR Whitepaper) Symposium on Molecular Targets and Cancer Therapeutics taking place November 29 to December 2, 2016 in Munich, Germany. A poster describing preclinical results with altiratinib (DCC-2701), another candidate in Deciphera’s pipeline that is a spectrum-selective MET and TRK-targeted kinase inhibitor for the treatment of solid tumors, was also presented at the meeting.

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"DCC-2618 is one of the most active compounds I have seen in the phase I setting in my career. While it is early, we observed signs of benefit in the GIST patients treated whose disease had progressed despite multiple previous treatments.," said Filip Janku, M.D., Ph.D., Assistant Professor, Department of Investigational Cancer Therapeutics, Division of Cancer Medicine, The University of Texas MD Anderson Cancer Center.

"Scientists at Deciphera are at the forefront of advancing kinase inhibitor research, and the development of a new generation of kinase inhibitors, such as DCC-2618, offers potential new therapies for patients with genetically-defined cancers and other diseases including GIST and systemic mastocytosis," said Oliver Rosen, M.D., Chief Medical Officer of Deciphera Pharmaceuticals. "We are very encouraged by the impressive early clinical results presented on DCC-2618. By inhibiting even difficult to treat drug resistant mutant kinases, DCC-2618 offers the potential for more durable responses in patients with cancer mutations that are resistant to other kinase inhibitor therapies and we look forward to providing further updates in the months to come."

In a late-breaking oral presentation, titled "DCC-2618, a pan KIT and PDGFR switch control inhibitor, achieves proof-of-concept in a first-in-human study," Dr. Janku of MD Anderson Cancer Center reported initial data from an ongoing Phase 1, dose escalation study of oral DCC-2618 in advanced solid tumor patients in which objective tumor responses and metabolic PET responses in GIST patients were observed. The data reported on the first 24 patients dosed in an ongoing Phase 1 dose-escalation study of DCC-2618 given orally twice-daily in 28-day cycles at doses ranging from 20-150 mg in advanced solid tumor patients. Highlights from the presentation of the Phase 1 data (as of November 11, 2016) include:

Partial Metabolic Responses (EORTC criteria) were observed in 14 of 15 patients with KIT-mutant GIST along with initial signs of decreases in circulating tumor DNA that codes for KIT, demonstrating broad spectrum inhibition of KIT mutants in heavily-pretreated GIST patients harboring multiple resistance mutations.
Two patients achieved partial response (RECIST criteria), including one patient with KIT, PDGFRα and VEGFR2 co-amplified glioblastoma multiforme and a patient with GIST and a KIT Exon 11/17 mutation.
DNA analyses at baseline revealed established resistance mutations in 9 of 13 patients with KIT-mutant GIST, with up to 5 secondary mutations and 3 established resistance mutations in a single patient confirming extensive tumor heterogeneity in these heavily-pretreated patients. Preliminary evidence of significant decreases in circulating tumor DNA encoding both primary and resistance mutations in KIT demonstrates the broad spectrum inhibition of mutant KIT kinases in heavily-pretreated GIST patients.
DCC-2618 was well tolerated with an encouraging safety profile. The most common treatment emergent adverse events (>25%) included: fatigue, dyspnea, anemia and decreased appetite. One dose limiting toxicity, a grade 3, asymptomatic lipase elevation in the 100 mg cohort, was reported.
The maximum tolerated dose for DCC-2618 has not yet been reached in this dose-escalation study.
In a poster presentation, titled "The type II switch control kinase inhibitor, DCC-2701 (altiratinib) effectively inhibits resistant NTRK kinase domain mutants," Deciphera collaborators at Memorial Sloan Kettering Cancer Center and Oregon Health and Sciences University described preclinical data in which altiratinib with its unique inhibitor binding mode maintained high affinity and inhibitory efficacy for mutant TRK kinase-fusions to circumvent emergent drug resistance.

About DCC-2618 and Altiratinib
DCC-2618 and altiratinib are both currently in Phase 1 clinical trials. DCC-2618 is a pan-KIT and PDGFRα kinase inhibitor in clinical development for the treatment of genetically-defined cancers, including gastrointestinal stromal tumors (GIST) and other KIT-driven diseases such as systemic mastocytosis. Altiratinib is a spectrum selective inhibitor of MET, TRK, TIE2 & VEGFR2 kinases in clinical development for the treatment of solid tumors.