|AcelRX Selling Off On FDA Reexamination; U.S. FDA Grants Approval for IMBRUVICA®|
|By Staff and Wire Reports|
|Monday, 28 July 2014 18:56|
Shares of AcelRx Pharmaceuticals (NASDAQ: ACRX) are selling off sharply monday morning after a blow from the FDA to Zalviso. AcelRx made the announcement at an unusual time: 9:59 p.m. ET Friday.
The FDA is looking for more information on how to use Zalviso, bench data to show a reduction in optical errors and information on the reported shelf life, as stated in a complete response letter. AcelRx disclosed that additional bench testing will be required to move forward and human factors testing may be required.
The silver lining is that amendments to the new drug application have been filed, but not yet reviewed by the FDA. This information may satisfy some FDA requests. CEO Richard King stated, “We are confident in the Zalviso development program and will work closely with the FDA to address the Agency's concerns as outlined in the CRL to ensure that healthcare professionals and patient communities will have access to Zalviso."
Shares of AcelRX were last trading 34.4 percent lower at $7.10.
Pharmacyclics (NASDAQ: PCYC) announced the U.S. FDA has granted IMBRUVICA® (ibrutinib) regular (full) approval for the treatment of patients with chronic lymphocytic leukemia (CLL) who have received at least one prior therapy, and for the treatment of CLL patients with deletion of the short arm of chromosome 17 (del 17p CLL), including treatment naive and previously treated del 17p CLL patients.
This is the first full FDA approval for IMBRUVICA, and was granted within six months after the accelerated approval for patients with previously treated CLL in February 2014. IMBRUVICA had received the Breakthrough Therapy Designation for patients with del 17p CLL in April 2013. IMBRUVICA is being jointly developed and commercialized by Pharmacyclics, Inc. and Janssen Biotech, Inc.
This full approval is based on data from the Phase III RESONATE™ study (PCYC-1112-CA), a randomized, multi-center, international head-to-head comparison of single-agent, orally-administered IMBRUVICA versus the intravenous, monoclonal antibody ofatumumab targeting the CD 20 antigen. This study enrolled 373 patients with CLL and 18 patients with small lymphocytic lymphoma (SLL), who received at least one prior therapy. The median number of prior treatments was 2 (range, 1 to 13 treatments). At baseline, the median age of these patients was 67 years, 58% of whom had at least one tumor > 5 cm, and 32% of whom had the del 17p mutation. Patients receiving IMBRUVICA demonstrated a statistically significant improvement in progression-free survival (PFS), overall survival (OS) and overall response rate (ORR) as compared to patients treated with ofatumumab. The median PFS and OS has not been reached on the IMBRUVICA arm. There was a 78% statistically significant reduction in the risk of progression or death as assessed by an independent review committee (IRC) according to the modified IWCLL criteria (HR 0.22, 95% CI, 0.15 to 0.32). In addition, the analysis of overall survival demonstrated a 57% statistically significant reduction in the risk of death for patients in the IMBRUVICA arm (HR 0.43; 95 CI, 0.24 to 0.79). This was observed despite a total of 57 patients who were initially randomized to ofatumumab crossing over to receive IMBRUVICA prior to the analysis. For previously treated del 17p CLL patients, there was a 75% reduction in the risk of progression or death as assessed by an IRC (HR 0.25, 95% CI, 0.14 to 0.45).
"IMBRUVICA demonstrated substantial evidence of its superiority over ofatumumab and significant benefit for previously treated CLL patients, while maintaining a favorable safety profile. This FDA approval for IMBRUVICA is a major step toward chemo-free treatment in CLL," said John Byrd, M.D.,* Director, Division of Hematology, The Ohio State University Comprehensive Cancer Center - Arthur G. James Cancer Hospital & Richard J. Solove Research Institute and lead investigator for RESONATE. "Patients with deletion 17p CLL are at particularly high risk for poor outcomes. Today's approval of IMBRUVICA provides these patients with the only FDA-approved treatment, regardless of whether their disease is treatment naïve or previously treated. I continue to be awed by the duration of my patients' responses to IMBRUVICA and am grateful IMBRUVICA now is available to a broader group of CLL patients."
CLL is a slow-growing blood cancer of the white blood cells. CLL is the most common adult leukemia in the Western world and predominately a disease of the elderly with a median age at diagnosis of 72 years.
"We are delighted IMBRUVICA has received full approval by demonstrating its ability to improve progression-free survival and, importantly, overall survival as compared to an approved standard of care, and that IMBRUVICA is now available to all patients with del 17p CLL," said Danelle James, M.D., Vice President, Clinical Development, Pharmacyclics. "Our goal is to provide patients with clinically meaningful treatments. Thanks to the physicians and patients who helped us complete this trial in near record time, today, we have delivered on that goal by bringing IMBRUVICA to an even broader group of patients."
Within CLL, the most commonly occurring adverse reactions (> 20%) were thrombocytopenia, neutropenia, diarrhea, anemia, upper respiratory tract infection, musculoskeletal pain, bruising, rash, fatigue, nausea, and pyrexia. Approximately 5% of patients with CLL receiving IMBRUVICA discontinued treatment due to adverse events. These included infections (2%), subdural hematoma (2%) and diarrhea (1%). Adverse events leading to dose reduction occurred in approximately 6% of patients. The Warnings and Precautions include: hemorrhage, infections, cytopenias, atrial fibrillation, secondary primary malignancies, embryo-fetal toxicities.
This approval for IMBRUVICA triggers $60 million in milestone payments to Pharmacyclics under its collaboration agreement with Janssen Biotech, Inc.
Advaxis, Inc. (Nasdaq:ADXS), a biotechnology company developing the next generation of cancer immunotherapies, announced that it has received a notice of allowance from the Japan Patent Office for a patent application related to the Advaxis proprietary Lm-LLO cancer immunotherapy platform technology.
Agios Pharmaceuticals, Inc. (Nasdaq:AGIO), a leader in the fields of cancer metabolism and inborn errors of metabolism, today announced that it will host a conference call and live webcast on Thursday, August 7, 2014 at 8:30 a.m. EDT to report its second quarter 2014 financial results and discuss recent business activities.
Amarantus Bioscience Holdings, Inc. (OTCQB:AMBS), a biotechnology company focused on the discovery and development of novel diagnostics and therapeutics related to endoplasmic reticulum stress, cell cycle dysregulation, neurodegeneration and apoptosis, today announced the appointment of Donald D. Huffman to the Company's Board of Directors, where he will chair the newly-formed audit committee.
Brewer Sports International, a multi-faceted global sports advisory firm, and Amarantus BioScience Holdings, Inc. (OTCQB:AMBS), a biotechnology company focused on the discovery and development of novel diagnostics and therapeutics related to cell cycle dysregulation, endoplasmic reticulum stress, neurodegeneration and apoptosis, are pleased to provide an update surrounding the 3rd Alzheimer's focused #C4CT (Coalition for Concussion Treatment) Concussion Awareness Summit, powered by MDM Worldwide on Thursday, July 31, 2014 at the United Nations in New York City, NY.
CareDx, Inc. (Nasdaq:CDNA), a molecular diagnostics company focused on the development and commercialization of clinically differentiated, high value, non-invasive surveillance solutions for transplant recipients today announced the results of its cohort studies of blood samples from the multicenter CARGO and CARGO II heart transplant observational studies, and from the KARGO kidney transplant study during a presentation today at The World Transplant Congress in San Francisco, CA.
Codexis, Inc. (Nasdaq:CDXS), a leading developer of biocatalysts for the pharmaceutical and fine chemical industries, today announced that Gordon Sangster has agreed to join Codexis as Senior Vice President & Chief Financial Officer.
Egalet Corporation (Nasdaq:EGLT) announced the appointment of Jeffrey M. Dayno, M.D. as chief medical officer (CMO).
ERBA Diagnostics, Inc. (NYSE MKT:ERB), a fully integrated in vitro diagnostics company, is pleased to announce that it will be exhibiting its full range of products at the world's largest show for Clinical Chemistry – American Association for Clinical Chemistry (AACC) in Chicago, Illinois, U.S.A., from July 28-31, 2014.
FEI (Nasdaq:FEIC) announced two new products for cryo-electron microscopy applications: a new phase plate solution and the Titan Halo™ transmission electron microscope (TEM).
Flexion Therapeutics, Inc. (Nasdaq:FLXN) today announced that it has executed an exclusive worldwide licensing agreement with SwRI to utilize proprietary microsphere manufacturing technologies for production of Flexion's sustained-release drug candidates, including leading candidate FX006.
Merge Healthcare Incorporated (Nasdaq:MRGE), a leading provider of innovative enterprise imaging, interoperability and clinical systems that seek to advance healthcare, today announced Comanche County Memorial Hospital, a non-profit, acute care facility and the largest hospital in western Oklahoma, has selected Merge's vendor-neutral archive (VNA) and image viewing and sharing solutions for improved disaster recovery and operational workflow.
The Board of Directors of Phibro Animal Health Corporation (Nasdaq:PAHC), today declared a quarterly cash dividend of $0.10 per share on its Class A common stock and Class B common stock, payable on September 24, 2014, to stockholders of record at the close of business on September 3, 2014.
Repligen Corporation (Nasdaq:RGEN) today announced that it will report second quarter 2014 financial results on Monday, August 11, 2014.
Stemline Therapeutics, Inc. (Nasdaq:STML) announced today the opening of its SL-401 Investigational New Drug (IND) and initiation of a broad clinical development program including trials in blastic plasmacytoid dendritic cell neoplasm (BPDCN) and acute myeloid leukemia (AML).
Stereotaxis, Inc. (Nasdaq:STXS) today announced that it will release financial results for its 2014 second quarter ended June 30, 2014 on Monday, August 4, 2014 after the close of the U.S. financial markets.
Surgical Care Affiliates, Inc. (Nasdaq:SCAI) today announced that it will release its second quarter 2014 financial results on Wednesday, August 13, 2014, after the market closes.
Variation Biotechnologies (US) Inc., a Cambridge-based vaccine development company announced today the completion of a merger on July 25, 2014 with Paulson Capital (Delaware) Corp. (Nasdaq:PLCC).
Vital Therapies, Inc. (Nasdaq:VTL), a biotherapeutic company developing ELAD®, a cell-based therapy targeting treatment of acute liver failure, announces that Dr. Lewis W. Teperman, Chief of the Division of Transplant Surgery at NYU Langone Medical Center, presented a poster on Sunday evening at the 2014 World Transplant Congress entitled "Protein Content Assessment During Treatment with the ELAD Bioartificial Liver Support System."
Zealand Pharma (NASDAQ OMX Copenhagen: ZEAL), a company specialized in the discovery, design and development of peptide medicines, and Boehringer Ingelheim, one of the world's leading pharmaceutical companies, today jointly announced a new global exclusive licence, research and development collaboration.