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.

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"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."

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.

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"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.

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]).

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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.

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On November 16, 2017 X4 Pharmaceuticals, a clinical stage biotechnology company developing novel CXCR4 inhibitor drugs to improve immune cell trafficking to treat cancer and rare disease, reported that Dr. David C. Dale, MD will present the first clinical data from an on-going study of X4P-001-RD in patients with WHIM Syndrome, a sub-type of a primary immunodeficiency disease, at the 59th Annual Meeting of the American Society for Hematology (ASH) (Free ASH Whitepaper) (Press release, X4 Pharmaceuticals, NOV 16, 2017, View Source [SID1234522146]). Preliminary data from these abstracts are available on the ASH (Free ASH Whitepaper) conference website. The details of the poster presentation is as follows:

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X4P-001: A Novel Molecularly-Targeted Oral Therapy for WHIM Syndrome

Saturday, December 9, 2017, Abstract # 995, Presenter: David C. Dale, MD

"We are very pleased to share preliminary data showing that X4P-001-RD has a meaningful impact on the levels of circulating white blood cells in this severely immunodeficient patient population," said Paula Ragan, PhD, President and CEO of X4. "These data demonstrate the potential of X4P-001-RD to benefit patients with WHIM who otherwise have no approved treatment options and support our goal of advancing toward a pivotal study."

About WHIM Syndrome

WHIM syndrome is a primary immunodeficiency disease ("PID") caused by genetic mutations in the CXCR4 receptor gene resulting in susceptibility to certain types of infections. WHIM is an abbreviation for the characteristic clinical symptoms of the syndrome: Warts, Hypogammaglobulinemia, Infections, and Myelokathexis. Within the overall category of primary immunodeficiencies, there are between 15,000 and 100,000 patients in the US that are classified with PID of unknown origin — of which WHIM is one.1,2,3 WHIM syndrome is a rare disorder and the precise prevalence or incidence of patients that have the genetic mutation responsible for WHIM syndrome is unknown. Because patients are highly susceptible to infections, WHIM syndrome is associated with significant morbidity beginning in early childhood and continuing throughout life. Current therapy is limited to treatment of acute infections with antibiotics or prevention through the use of intravenous immunoglobulin or G-CSF. There is no approved therapy for the treatment of WHIM syndrome.

About X4P-001-RD for Primary Genetic Immunodeficiency Disease

X4P-001-RD, an oral, small molecule inhibitor of CXCR4, or C-X-C receptor type 4, is being developed for use as a life-long treatment for patients with WHIM syndrome and other primary genetic immunodeficiencies. X4P-001-RD is currently being studied in a Phase 2/3 trial in patients with WHIM syndrome. Within the bone marrow, a normally functioning CXCR4 receptor controls the release of neutrophils and leukocytes into the blood stream, thereby ensuring normal immune surveillance functions throughout the body. In patients with WHIM syndrome, mutations to the CXCR4 receptor cause aberrant signaling leading to retention of neutrophils and leukocytes in the bone marrow and inadequate immune surveillance and function.4,5 X4P-001-RD is designed to normalize the signaling for the mutant CXCR4 receptor to promote the release of neutrophils and leukocytes, thereby restoring healthy immunity.