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ChemoCentryx to Participate at the Piper Jaffray 29th Annual Healthcare Conference

On November 17, 2017 ChemoCentryx, Inc., (Nasdaq:CCXI), a biopharmaceutical company developing new medications targeted at inflammatory and autoimmune diseases and cancer, reported that Thomas J. Schall, Ph.D., President and Chief Executive Officer, will participate in a fireside chat at the Piper Jaffray 29th Annual Healthcare Conference on Tuesday, November 28, 2017 at 11:30am ET (Press release, ChemoCentryx, NOV 17, 2017, View Source [SID1234522130]). The conference will be held at the Lotte New York Palace Hotel in New York, NY.

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A live audio webcast of the fireside chat discussion can be accessed through the Investors section of the Company’s website at www.ChemoCentryx.com. A replay of the webcast will be available on the Company’s website for two weeks following the live discussion.

10-Q/A [Amend] – Quarterly report [Sections 13 or 15(d)]

Celgene Corporation and bluebird bio Announce bb2121 Anti-BCMA CAR-T Cell Therapy Has Been Granted Breakthrough Therapy Designation from FDA and Prime Eligibility from EMA for Relapsed and Refractory Multiple Myeloma

On November 16, 2017 Celgene Corporation (NASDAQ:CELG) and bluebird bio, Inc. (NASDAQ:BLUE) reported that bb2121, a chimeric antigen receptor T-cell (CAR-T) therapy targeting b-cell maturation antigen (BCMA) in previously treated patients with multiple myeloma, has been granted Breakthrough Therapy Designation (BTD) by the U.S. Food and Drug Administration (FDA) and PRIority MEdicines (PRIME) eligibility by the European Medicines Agency (EMA) (Press release, bluebird bio, NOV 16, 2017, View Source [SID1234522119]).

BTD designation and PRIME eligibility for bb2121 were based on preliminary clinical data from the ongoing phase 1 study CRB-401. Updated data from CRB-401 is scheduled to be presented at the 59th annual meeting of the American Society of Hematology (ASH) (Free ASH Whitepaper) in Atlanta during an oral presentation on Dec. 11.

"Receiving Breakthrough Therapy Designation and PRIME eligibility for bb2121 further underscores the potential of this novel cellular immunotherapy approach to multiple myeloma treatment," said Jay Backstrom, M.D., Chief Medical Officer and Head of Global Regulatory Affairs for Celgene. "We will work closely with these agencies as we accelerate development of bb2121, a novel technology and therapy for patients with multiple myeloma."

"Despite recent advances, multiple myeloma remains an incurable disease, and heavily pretreated patients have limited therapeutic options," said David Davidson, M.D., Chief Medical Officer for bluebird bio. "Early data suggest that treatment with bb2121 has the potential to induce durable responses in this patient population. It is encouraging for both the FDA and EMA to identify bb2121 as a candidate for accelerated development as we continue our work with Celgene to bring this therapy to patients in need of new options."

Breakthrough Therapy Designation is intended to expedite the development and review of drugs that are intended to treat serious or life-threatening conditions. The criteria for breakthrough therapy designation require preliminary clinical evidence that demonstrates the drug may have substantial improvement on at least one clinically significant endpoint over available therapy.

PRIME is a program launched by the EMA to enhance support for the development of medicines that target an unmet medical need. This voluntary program is based on enhanced interaction and early dialogue with developers of promising medicines, to optimize development plans and speed up evaluation so these medicines can reach patients earlier. The program focuses on medicines that may offer a major therapeutic advantage over existing treatments, or benefit patients without treatment options. These medicines are considered priority medicines by EMA. To be accepted for PRIME, a medicine must show its potential to benefit patients with unmet medical needs based on early clinical data.

Arbutus’ LNP Licensee Alnylam Initiates Rolling Submission of New Drug Application (NDA) to U.S. Food and Drug Administration (FDA) for Patisiran

On November 16, 2017 Arbutus Biopharma Corporation (Nasdaq:ABUS), an industry-leading Hepatitis B Virus (HBV) therapeutic solutions company, reported that the Company’s lipid nanoparticle (LNP) licensee Alnylam Pharmaceuticals, Inc. (Nasdaq:ALNY), initiated submission of a rolling New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for patisiran, an investigational RNAi therapeutic being developed for patients with hereditary ATTR amyloidosis with polyneuropathy (Press release, Arbutus Biopharma, NOV 16, 2017, View Source [SID1234522117]). This submission allows the FDA to review completed portions of the NDA on an ongoing basis. Alnylam expects to submit final clinical data by year end.

"Our LNP licensing partner continues to make great progress towards achieving final regulatory approval for patisiran. This is a testament to the value of our proprietary LNP platform, which is the most widely adopted RNAi delivery technology to date," said Dr. Mark J. Murray, Arbutus’ President and CEO. "Arbutus is entitled to receive single digit royalties on global sales of patisiran, pending final regulatory approvals."

FDA approves Roche’s Gazyva for previously untreated advanced follicular lymphoma

On November 17, 2017 Roche (SIX: RO, ROG; OTCQX: RHHBY) reported that the US Food and Drug Administration (FDA) approved Gazyva (obinutuzumab) in combination with chemotherapy, followed by Gazyva alone in those who responded, for people with previously untreated advanced follicular lymphoma (stage II bulky, III or IV) (Press release, Hoffmann-La Roche, NOV 16, 2017, View Source [SID1234522126]). The approval is based on results from the phase III GALLIUM study, which showed superior progression-free survival (PFS) for patients who received this Gazyva-based regimen compared with those who received a Rituxan (rituximab)-based regimen as an initial (first-line) therapy. Follicular lymphoma, the most common slow-growing (indolent) form of non-Hodgkin lymphoma (NHL), is incurable and becomes harder to treat each time it returns.

"Today’s Gazyva approval is an important advance for the thousands of people diagnosed each year with follicular lymphoma who hope to delay disease progression for as long as possible," said Sandra Horning, MD, Roche’s Chief Medical Officer and Head of Global Product Development. "We’re pleased we can now offer patients with this incurable blood cancer an initial treatment option shown to improve upon Rituxan, the standard of care in this setting for more than 10 years."

The GALLIUM study showed the Gazyva-based regimen significantly reduced the risk of disease worsening or death compared to a Rituxan-based regimen by 28% (PFS as assessed by independent review committee [IRC]; HR=0.72; 95% CI 0.56-0.93; p=0.0118). The most common Grade 3-5 side effects (occurring in at least 5% of patients) observed more frequently in the Gazyva arm were low white blood cell count, infusion reactions, low white blood cell count with fever and low platelet count. The most common side effects (occurring in at least 20% of patients) observed at least 2% more frequently in the Gazyva arm included infusion reactions, low white blood cell count, upper respiratory tract infection, cough, constipation and diarrhoea.

Gazyva’s supplemental Biologics License Application based on the GALLIUM data was granted Priority Review, a designation given to medicines that the FDA has determined to have the potential to provide significant improvements in the treatment, prevention or diagnosis of a disease. With this approval, Gazyva is available in the US for three different indications across two common types of blood cancer.

About the GALLIUM study
GALLIUM (NCT01332968) is a global phase III open-label, multicentre, randomised two-arm study examining the efficacy and safety of Gazyva plus chemotherapy followed by Gazyva alone for up to two years, as compared head-to-head against Rituxan plus chemotherapy followed by Rituxan alone for up to two years. Chemotherapies used (CHOP, CVP or bendamustine) were selected by each participating study site prior to beginning enrolment. GALLIUM included 1,385 patients with previously untreated non-Hodgkin lymphoma (NHL), of whom 1,202 patients had advanced follicular lymphoma (stage II bulky, III or IV). Efficacy results in follicular lymphoma with a median observation time of 38 months were the following:

Safety was evaluated based on 1,385 patients with previously untreated follicular lymphoma (86%) or marginal zone lymphoma (14%). The most common Grade 3-5 side effects that occurred more often with Gazyva plus chemotherapy followed by Gazyva alone compared to Rituxan plus chemotherapy followed by Rituxan alone were low white blood cell count, infusion reactions, low white blood cell count with fever and low platelet count.