|FDA Approval of Pharmacyclics, J&J's Janssen's IMBRUVICA; RXi Pharma Initiates First Phase 2 Study with RXI-109|
|By Staff and Wire Reports|
|Wednesday, 13 November 2013 20:05|
Pharmacyclics, Inc. (NASDAQ: PCYC) announced that the U.S. FDA has approved IMBRUVICA™ (ibrutinib) as a single agent for the treatment of patients with mantle cell lymphoma (MCL) who have received at least one prior therapy.1 This indication is based on overall response rate (ORR). An improvement in survival or disease-related symptoms has not been established. IMBRUVICA is a new agent that inhibits the function of Bruton's tyrosine kinase (BTK).1 BTK is a key signaling molecule of the B-cell receptor signaling complex that plays an important role in the survival of malignant B cells.2,3,4 IMBRUVICA blocks signals that stimulate malignant B cells to grow and divide uncontrollably.1,5
The FDA granted IMBRUVICA Breakthrough Therapy Designation due to the ORR and duration of response (DOR) seen in the Phase II study, PCYC-1104, and the serious and life-threatening nature of MCL.
The approval was based on the results of a multi-center, international, single-arm trial of 111 patients with previously treated mantle cell lymphoma. Tumor response was assessed according to the revised International Working Group (IWG) for non-Hodgkin lymphoma (NHL) criteria. The efficacy results demonstrated a 65.8% overall response rate (95% CI: 56.2, 74.5); 17% of patients achieved a complete response and 49% of patients achieved a partial response. The median duration of response was 17.5 months (95% CI: 15.8, not reached).
Safety was evaluated in the same 111 patients. The most common Grade 3 or 4 non-hematological adverse reactions (greater than or equal to 5%) were: pneumonia (7%), abdominal pain (5%), atrial fibrillation, diarrhea (5%), fatigue (5%), and skin infections (5%). Five percent of patients had Grade 3 or higher bleeding events (subdural hematoma, gastrointestinal bleeding, and hematuria). Treatment-emergent Grade 3 or 4 cytopenias (adverse reactions and laboratory abnormalities combined) were reported in 41% of patients. The Warnings and Precautions listed in the Prescribing Information include hemorrhage, infections, myelosuppression, renal toxicity, second primary malignancies and embryo-fetal toxicity.
Ten patients (9%) discontinued treatment due to adverse reactions in the trial. Adverse reactions leading to dose reduction occurred in 14% of patients. The recommended dose in this patient population is 560 mg (four 140 mg capsules) orally once daily.
IMBRUVICA is one of the first treatments to receive FDA approval via the Breakthrough Therapy Designation pathway. Pharmacyclics completed its New Drug Application submission to the FDA in late June 2013 and received approval just over four months later.
"This is a meaningful day for previously treated mantle cell lymphoma patients, who are in need of new treatment options," said Michael Wang, M.D., Department of Lymphoma/Myeloma, The University of Texas MD Anderson Cancer Center and lead investigator for the pivotal registration trial PCYC-1104. "With IMBRUVICA, we now have an important new medicine that is a once-daily oral therapy with a favorable risk-benefit profile."
IMBRUVICA is commercially available immediately -- "The approval of IMBRUVICA marks significant progress toward Pharmacyclics' goal of serving as an ally to patients. After observing early signs of efficacy and tolerability of IMBRUVICA four years ago, we single-mindedly focused our attention on fully developing this medicine. We continue to explore IMBRUVICA's potential to treat cancer patients in need. Presently we are in the midst of investigating this medicine in numerous additional B-cell malignancies with 37 clinical studies ongoing," said Bob Duggan, CEO and Chairman of the Board of Pharmacyclics. "Today we celebrate the first approval of IMBRUVICA. I would like to thank the patients and physicians for their trust and participation in our clinical trials. We are very grateful to the FDA for their collaboration and support, and a very big 'thank you' to the entire Pharmacyclics & Janssen team who have tirelessly forwarded our mutual vision and mission to serve as allies for patients and physicians in the fight against cancer."
"Breakthrough Therapy Designation is intended to speed up the development and review of treatments to help address serious or life-threatening diseases. It is gratifying to see this early example of the new pathway meeting its intention – getting a promising treatment to patients who are waiting for new options," said Dr. Ellen Sigal, chair and founder of Friends of Cancer Research, a think tank and advocacy organization based in Washington, DC.
RXi Pharmaceuticals Corporation (OTCQX: RXII), a biotechnology company focused on discovering, developing and commercializing innovative therapies addressing major unmet medical needs using RNA-targeted technologies, announced the first patient has been enrolled in a Phase 2 study with the anti-scarring product RXI-109. In this study (RXI-109-1301), patients with a long hypertrophic scar in the lower abdominal area are eligible to receive scar revision surgery and subsequent treatment with RXI-109 in one of two treatment regimens. Patients will receive RXI-109 or placebo on a blinded basis at the distal ends of their revised scar, leaving a central untreated section of the scar. Each patient's revised scar area will provide the opportunity to compare the appearance of the revised areas after treatment with RXI-109 or placebo or when left untreated. This design allows for intra-subject comparison of the three revised scar segments, as such increasing the power of the study.
"The start of the first Phase 2 clinical study with RXI-109 is a very important step in bringing our first self-delivering (sd-rxRNA®) RNAi closer to the market," said Dr. Geert Cauwenbergh, President and CEO of RXi Pharmaceuticals. He added that, "Not only can the outcome of this first Phase 2 study create a novel treatment approach in an area where no FDA approved drugs exist; it should also confirm the clinical relevance of our proprietary sd-rxRNA approach for human diseases in a broader sense, creating numerous business development opportunities that could add substantial value to our company."
In its public disclosures, the Company has indicated that two additional Phase 2 studies will be initiated in the near future. The first of these will evaluate the effect of RXI-109 on the recurrence of keloids after keloid revision surgery. The second study will evaluate the effect of RXI-109 on suppressing recurrence of hypertrophic scars after bilateral scar revision surgery in the breast area.
About Scarring -- Skin scarring after surgery, trauma, or burns can cause debilitating aesthetic, functional and psychological effects. There are no FDA-approved therapeutics for treatment of post-operative scars. RXI‑109 has been shown in preclinical models to reduce CTGF, a growth factor essential in the wound healing cascade. Elevated levels of CTGF‑dependent signaling can prolong the tissue repair process and lead to pathological scarring.
About RXI-109 -- RXi Pharmaceuticals' first clinical program involves RXI‑109, a self-delivering RNAi compound (sd‑rxRNA®) developed for the reduction of dermal scarring. RXI‑109 is designed to reduce the expression of CTGF, a critical regulator of biological pathways involved in fibrosis, including scar formation in the skin. The first clinical trials of RXI‑109 showed excellent safety and tolerability with ascending single or multiple doses.
RXi's sd‑rxRNA oligonucleotides are designed for therapeutic use and have drug-like properties, such as high potency, target specificity, serum stability, reduced immune response activation, and efficient cellular uptake. These hybrid oligonucleotide molecules combine the beneficial properties of conventional RNAi and antisense technologies. This allows sd‑rxRNAs to achieve efficient cellular uptake and potent, long-lasting intracellular activity.
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Burcon NutraScience Corporation (TSX:BU) (Nasdaq:BUR), a leader in functional, renewable plant proteins, reported results for the fiscal second quarter ended September 30, 2013.
Cancer Genetics, Inc. (Nasdaq:CGIX), a DNA-based diagnostics company focused on developing genomic-based oncology tests and services, reported financial results for the third quarter ended September 30, 2013.
Chelsea Therapeutics International, Ltd. (Nasdaq:CHTP), a development stage pharmaceutical company focused on the acquisition, development and commercialization of innovative pharmaceutical products, announced today that it has priced an underwritten public offering of 6,666,667 shares of its common stock at an offering price of $3.00 per share, for total gross proceeds (before underwriting discount and commissions and estimated expenses) of approximately $20 million.
CollabRx, Inc. (Nasdaq:CLRX) today announced financial results for the second quarter of fiscal year 2014, which ended September 30, 2013.
Dresner Partners, a leading FINRA-registered, middle-market investment bank and IMAP member firm, announced today that it advised Novia CareClinics, LLC in its sale to QuadMed, LLC, a subsidiary of Quad/Graphics, Inc. (NYSE: QUAD).
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Fate Therapeutics, Inc. (Nasdaq:FATE), a biopharmaceutical company engaged in the discovery and development of adult stem cell modulators to treat orphan diseases, today reported financial results for the quarter ended September 30, 2013 and provided a summary of recent business highlights.
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Novavax, Inc. (Nasdaq:NVAX) today announced that positive clinical data for the company's virus-like particle (VLP) vaccine candidate against A(H7N9) influenza were published online in the Correspondence section of The New England Journal of Medicine.
Ocera Therapeutics, Inc. (Nasdaq:OCRX), a biopharmaceutical company focused on innovative therapeutics for orphan liver disease, announced today financial results for the three and nine months ended September 30, 2013.
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