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Blueprint Medicines To Present Broad Range of Data Reinforcing Commitment to Advance Patient Care in Systemic Mastocytosis at 62nd ASH Annual Meeting and Exposition

On November 23, 2020 Blueprint Medicines Corporation (NASDAQ: BPMC), a precision therapy company focused on genomically defined cancers, rare diseases and cancer immunotherapy, reported plans to report data presentations highlighting the medical needs in systemic mastocytosis (SM) and the broad potential of AYVAKIT (avapritinib) across multiple SM patient populations at the 62nd American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition (Press release, Blueprint Medicines, NOV 23, 2020, View Source [SID1234571589]). AYVAKIT is designed to potently and selectively target D816V mutant KIT, the primary driver of SM, and is being developed for the treatment of advanced and non-advanced forms of the disease.

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"The breadth of our upcoming ASH (Free ASH Whitepaper) presentations highlights our commitment to fundamentally change the way systemic mastocytosis is diagnosed and treated across a broad population of patients impacted by this disease," said Andy Boral, M.D., Ph.D., Chief Medical Officer at Blueprint Medicines. "These presentations reflect our collaborative efforts with the systemic mastocytosis community to better understand the burden of disease, advance important strategies for facilitating diagnosis and establish new methods for measuring treatment response."

The accepted abstracts are listed below and available on the ASH (Free ASH Whitepaper) conference website: View Source

Oral Presentation

Presentation Title: Pure Pathologic Response Is Associated with Improved Overall Survival in Patients with Advanced Systemic Mastocytosis Receiving Avapritinib in the Phase I EXPLORER Study
Session Title: Myeloproliferative Syndromes: Clinical: Translational Science in MPN—Hitting the Mark
Session Date & Time: Sunday, December 6, 2020 at 10:15 a.m. PT (1:15 p.m. ET)
Abstract Number: 345

Poster Presentations

Presentation Title: Patient Reported Outcomes Among Systemic Mastocytosis (SM) Patients in Routine Clinical Practice: Results from the TouchStone Survey
Session Title: Outcomes Research—Non-Malignant Conditions: Poster I
Session Date & Time: Saturday, December 5, 2020 from 7:00 a.m. – 3:30 p.m. PT (10:00 a.m. – 6:30 p.m. ET)
Abstract Number: 1638

Presentation Title: Results from PIONEER: A Randomized, Double-Blind, Placebo-Controlled, Phase 2 Study of Avapritinib in Patients with Indolent Systemic Mastocytosis
Session Title: Myeloproliferative Syndromes: Clinical: Poster I
Session Date & Time: Saturday, December 5, 2020 from 7:00 a.m. – 3:30 p.m. PT (10:00 a.m. – 6:30 p.m. ET)
Abstract Number: 1248

Presentation Title: Increased Detection of KIT D816V Mutation in Peripheral Blood Samples from Patients with Indolent Systemic Mastocytosis (ISM) in the Phase 2 PIONEER Study Using a High Sensitivity Droplet Digital (dd) PCR Assay Compared with Next Generation Sequencing (NGS)
Session Title: Hodgkin Lymphoma and T/NK Cell Lymphoma—Clinical Studies: Poster III
Session Date & Time: Monday, December 7, 2020 from 7:00 a.m. – 3:30 p.m. PT (10:00 a.m. – 6:30 p.m. ET)
Abstract Number: 3004

Presentation Title: Perceptions of Patient Disease Burden and Management Approaches of Systemic Mastocytosis By Healthcare Providers: Results from the TouchStone SM Survey
Session Title: Health Services Research—Non-Malignant Conditions: Poster III
Session Date & Time: Monday, December 7, 2020 from 7:00 a.m. – 3:30 p.m. PT (10:00 a.m. – 6:30 p.m. ET)
Abstract Number: 3403

Presentation Title: PIONEER Part 2: A Randomized, Double-Blind, Placebo-Controlled, Phase 2 Study to Evaluate Safety and Efficacy of Avapritinib in Indolent Systemic Mastocytosis
Session Title: Hodgkin Lymphoma and T/NK Cell Lymphoma—Clinical Studies: Poster III
Session Date & Time: Monday, December 7, 2020 from 7:00 a.m. – 3:30 p.m. PT (10:00 a.m. – 6:30 p.m. ET)
Abstract Number: 2988

About AYVAKIT (avapritinib)

AYVAKIT (avapritinib) is a kinase inhibitor approved by the U.S. Food and Drug Administration (FDA) for the treatment of adults with unresectable or metastatic gastrointestinal stromal tumor (GIST) harboring a PDGFRA exon 18 mutation, including PDGFRA D842V mutations. For more information, visit www.AYVAKIT.com. This medicine is approved in Europe under the brand name AYVAKYT for the treatment of adults with unresectable or metastatic GIST harboring the PDGFRA D842V mutation.

AYVAKIT is not approved for the treatment of any other indication, including SM, in the U.S. by the FDA or in Europe by the European Commission, or for any indication in any other jurisdiction by any other health authority.

Blueprint Medicines is developing AYVAKIT globally for the treatment of advanced and indolent SM. The FDA granted breakthrough therapy designation to AYVAKIT for the treatment of advanced SM, including the subtypes of aggressive SM, SM with an associated hematologic neoplasm and mast cell leukemia.

Blueprint Medicines has an exclusive collaboration and license agreement with CStone Pharmaceuticals for the development and commercialization of AYVAKIT in Mainland China, Hong Kong, Macau and Taiwan. Blueprint Medicines retains development and commercial rights for AYVAKIT in the rest of the world.

Myovant Sciences to Present at Upcoming December Investor Conferences

On November 23, 2020 Myovant Sciences (NYSE: MYOV), a healthcare company reported on redefining care for women and for men, invites investors and the general public to listen to webcasts at the following investor conferences (Press release, Myovant Sciences, NOV 23, 2020, https://investors.myovant.com/news-releases/news-release-details/myovant-sciences-present-upcoming-december-investor-conferences [SID1234571588]):

Evercore ISI 3rd Annual HealthCONx Virtual Conference on December 1, 2020 at 4:45 p.m. Eastern time. Lynn Seely, M.D., chief executive officer of Myovant Sciences, Inc., and Frank Karbe, president and chief financial officer of Myovant Sciences, Inc., will participate in a webcast presentation. Management will also participate in virtual one-on-one investor meetings on December 1.

Piper Sandler 32nd Annual Virtual Healthcare Conference. A pre-recorded presentation by Dr. Seely will be available at 10 a.m. Eastern time today on the investor relations page of Myovant’s website, investors.myovant.com. Management will participate in virtual one-on-one investor meetings on December 3, 2020.
The presentations will be accessible on the Events page under the Investors & Media section of the Myovant website at www.myovant.com.

BeiGene to Present at the Piper Sandler 32nd Annual Virtual Healthcare Conference

On November 23, 2020 BeiGene, Ltd. (NASDAQ: BGNE; HKEX: 06160), a commercial-stage biotechnology company focused on developing and commercializing innovative medicines worldwide, reported that the Company will participate in the Piper Sandler 32nd Annual Virtual Healthcare Conference (Press release, BeiGene, NOV 23, 2020, View Source [SID1234571587]). Presentations will be available for registered attendees via the Piper Sandler conference site from November 23 to December 3, 2020.

An archived replay will be available for 90 days following the event in the investors section of BeiGene’s website at View Source or View Source

Actinium Highlights Foundational Patents Covering the Composition of Apamistamab Antibody and Iomab-B Antibody Radiation Conjugate for Targeted Conditioning Until 2037 and Recent EU Patent Activity

On November 23, 2020 Actinium Pharmaceuticals, Inc. (NYSE AMERICAN: ATNM) ("Actinium" or the "Company") reported that highlighted its intellectual property portfolio for apamistamab, a CD45 targeting antibody, and the Antibody Radiation Conjugate (ARC) comprised of apamistamab and the radioisotope iodine-131 used in the Company’s lead Phase 3 candidate, Iomab-B, and its Iomab-ACT programs (Press release, Actinium Pharmaceuticals, NOV 23, 2020, View Source [SID1234571585]). Actinium owns issued or pending patents within the United States and globally covering composition of matter, formulation, methods of use, and methods of administration with potential coverage for 19 years or longer. Importantly, Actinium owns an issued patent in the US covering composition of matter, for which the Company expects validity until 2037. In addition, the Company owns a second issued US patent that further covers composition of matter, methods of use, and methods of administration for Iomab-B. The company has also received a notice of allowance in Europe for this second patent and expects it to be in force until 2036.

Iomab-B is currently being investigated in the ongoing pivotal Phase 3 SIERRA trial, which is over 75% enrolled, for targeted conditioning prior to potentially curative bone marrow transplant (BMT) for patients with relapsed or refractory Acute Myeloid Leukemia ("R/R AML"). In addition, Actinium is utilizing apamistamab with lower doses of iodine-131, known as Iomab-ACT, for targeted conditioning prior to gene therapy and adoptive cell therapy ("ACT"), namely CAR-T, including in its recently announced collaboration with Memorial Sloan Kettering Cancer Center that is supported by NIH STTR Fast Track grant funding.

"The continued protection of our lead asset Iomab-B, our Iomab-ACT program and apamistamab by a strong patent position is an important component of our development efforts, particularly as we approach the conclusion of our pivotal Phase 3 SIERRA trial for BMT conditioning in R/R AML The growth of BMT, ACT and Gene Therapy has highlighted the importance of conditioning and the need to move beyond non-targeted chemotherapy to increase the number of patients that could benefit from these potentially curative therapies. CD45 is an ideal target for conditioning applications given its unique expression on blood cancer cells and blood forming stem and immune cells and with no expression outside the hematopoietic or blood system," said Dr. Dale Ludwig, Actinium’s Chief Scientific Officer. "Apamistamab is well characterized and its use in conditioning is supported by extensive clinical data across multiple clinical trials and indications. Our robust data shows that apamistamab has a favorable biodistribution profile that, together with our ARC technology, has significant advantages over other approaches such antibody drug conjugates that require payload internalization, making them impractical for targeting CD45. Further, our ARC approach allows us to use varying intensities of targeted radiation to achieve our desired conditioning outcome. With these important patents in place, and continued expansion of our patent portfolio in the US, EU and other select countries, we look forward to continuing to build out our targeted conditioning strategic business unit."

About Iomab-B

Iomab-B is Actinium’s lead product candidate that is currently being studied in a 150-patient, multicenter pivotal Phase 3 clinical trial in patients with relapsed or refractory acute myeloid leukemia who are age 55 and above. Upon approval, Iomab-B is intended to prepare and condition patients for a bone marrow transplant, also referred to as a hematopoietic stem cell transplant, which is often considered the only potential cure for patients with certain blood-borne cancers and blood disorders. Iomab-B targets cells that express CD45, a pan-leukocytic antigen widely expressed on white blood cells with the monoclonal antibody, apamistamab (formerly BC8), labeled with the radioisotope, iodine-131. By carrying iodine-131 directly to the bone marrow in a targeted manner, Actinium believes Iomab-B will avoid the side effects of radiation on most healthy tissues while effectively killing the patient’s cancer and marrow cells. In a Phase 1/2 clinical study in 68 patients with advanced AML or high-risk myelodysplastic syndrome (MDS) age 50 and older, Iomab-B produced enabled 100% of patients to proceed to transplant with all patients achieving transplant engraftment by day 28. Iomab-B was developed at the Fred Hutchinson Cancer Research Center where it has been studied in almost 300 patients in a number of blood cancer indications, including acute myeloid leukemia, chronic myeloid leukemia, acute lymphoblastic leukemia, chronic lymphocytic leukemia, Hodgkin’s disease, Non-Hodgkin lymphomas and multiple myeloma. Actinium obtained the worldwide, exclusive rights to apamistamab (BC8) and Iomab-B from the Fred Hutchinson Cancer Research Center. Iomab-B has been granted Orphan Drug Designation for relapsed or refractory AML in patients 55 and above by the U.S. Food and Drug Administration and the European Medicines Agency.

Theralase Granted FDA Fast Track Designation for NMIBC Phase II Clinical Study

On November 23, 2020 Theralase Technologies Inc. ("Theralase" or the "Company") (TSXV: TLT) (OTCQB: TLTFF), a clinical stage pharmaceutical company focused on the research and development of light activated Photo Dynamic Compounds ("PDC") and their associated drug formulations used to safely and effectively destroy various cancers, bacteria and viruses reported that the U.S. Food and Drug Administration ("FDA") has granted Theralase Fast Track Designation ("FTD") for its Phase II Bacillus Calmete Guérin ("BCG")–Unresponsive Non-Muscle Invasive Bladder Cancer ("NMIBC") Carcinoma In Situ ("CIS") clinical study ("Study II") (Press release, Theralase, NOV 23, 2020, View Source [SID1234571584]).

As a Fast Track designee, Theralase will have access to early and frequent communications with the FDA to discuss Theralase’s development plans and ensure timely collection of the appropriate clinical data to support the approval process. The accelerated communication with the FDA potentially allows, TLD-1433, in combination with the TLC-3200 medical laser system ("TLC-3200"), to be the first intravesical patient-specific Ruthenium-based PDC for the treatment of patients with BCG-Unresponsive NMIBC CIS, with or without papillary Ta or T1 tumours. FTD can lead to an Accelerated Approval and Priority Review, if certain criteria are met, which the FDA has previously defined to the Company to represent approximately 20 to 25 patients enrolled and treated, who demonstrate significant safety and efficacy clinical outcomes.

Michael Jewett MD, FRCSC, FACS, Inaugural Farquharson Clinical Research Chair in Oncology, Departments of Surgery (Urology) and Surgical Oncology, Princess Margaret Cancer Centre, University Health Network ("UHN"), stated "By awarding Fast Track Designation to the photosensitizer drug TLD-1433 activated by the laser TLC-3200, currently being assessed in a Phase II clinical study for the treatment of NMIBC, the FDA has recognized Theralase’s potential to meaningfully improve patient outcomes for this life-threatening disease. This is a significant accomplishment for the Company. This latest milestone complements the clinical development strategy to provide urologists, uro-oncologists and patients with the tools to combat BCG-Unresponsive NMIBC, safely and effectively."

Shawn Shirazi PhD, Chief Executive Officer, Theralase, stated, "FDA’s FTD for our lead drug candidate, TLD-1433, activated by the TLC-3200, is another important milestone for Theralase, as it can potentially speed the development of this drug-device combination for NMIBC patients. The TLD-1433 – TLC-3200 technology represents a paradigm shift in medical technology and an advanced approach to treat NMIBC. We are excited by the progress the Company has delivered in Study II, as we continue to enroll and successfully treat patients. The Company continues to work towards launching new clinical study sites in Canada and the US with a mandate to enroll and treat all patients for their first Study II treatment in 2021".

About Fast Track Designation

FTD is an FDA process designed to facilitate the development, and expedite the review of, medicines to treat serious conditions and fill unmet medical needs. Filling an unmet need is defined as providing a therapy where none exists or providing a therapy that may be potentially better than available therapies. The FDA created this process to help deliver important new drugs to patients earlier, and it covers a broad range of serious illnesses.

About Study II

Study II utilizes the Therapeutic Dose (0.70 mg/cm2) of TLD-1433, activated by the TLC-3200, and is focused on the enrollment and treatment of approximately 100 BCG-Unresponsive NMIBC CIS patients in up to 20 clinical study sites located in Canada and the US.

Study II has a:

Primary endpoint of efficacy (defined by Complete Response ("CR") at any point in time
Secondary endpoint of duration of CR at 360 days post-initial CR (approximately 450 days post initial Study treatment, assuming CR is achieved at the 90 day assessment)
Tertiary endpoint of safety measured by incidence and severity of Adverse Events ("AEs") grade 4 or higher that do not resolve within 450 days post-initial treatment
The FDA, in its 2018 guidance to industry, stated that, "For single-arm trials of patients with BCG-Unresponsive disease, CR is defined as at least one of the following:

Negative cystoscopy and negative (including atypical) urine cytology
Positive cystoscopy with biopsy-proven benign or low-grade NMIBC and negative cytology
For intravesical therapies without systemic toxicity, negative cystoscopy with malignant urine cytology, if cancer is found in the upper tract or prostatic urethra and random bladder biopsies are negative.
The FDA further states that, "Intravesical instillation does not deliver the investigational drug to the upper tract or prostatic urethra; therefore, the development of disease in these areas cannot be attributed to a lack of activity of the investigational drug. Thus, sponsors can consider patients with new malignant lesions of the upper tract or prostatic urethra, who have received intravesical therapy to have achieved a CR in the primary analysis; however, sponsors should record these lesions and conduct sensitivity analyses in which these patients are not considered to have achieved a CR."1