|Novartis Announces NEJM Publication Of Secukinumab Phase III Data; Infinity Announces Enrollment Of 120th Patient In Phase 2 DYNAMO™ Study; Jazz Pharma Announces FDA Acceptance for Filing for Defibrotide|
|By Josh Gee|
|Wednesday, 30 September 2015 19:52|
Below is a look at some of the headlines for companies that made news in the healthcare sector on September 30, 2015.
Novartis announced results from the pivotal Phase III FUTURE 1 study for secukinumab in psoriatic arthritis (PsA) were published online in the New England Journal of Medicine (NEJM). Secukinumab is the first interleukin-17A (IL-17A) inhibitor to demonstrate efficacy in a Phase III study in patients with active PsA, a painful, debilitating condition causing inflammation of joints and skin1,7. PsA is part of a family of long-term diseases impacting joints, known as spondylorarthritis8.
In this study, secukinumab met the primary endpoint with a 20% reduction in the American College of Rheumatology response criteria (ACR 20) at Week 24 showing rapid and significant clinical improvements versus placebo. ACR is a standard tool used to assess improvement of PsA signs and symptoms9. In addition, secukinumab met all secondary endpoints, including improvements in skin and joint diseases and joint structural damage progression1.
Results showed that half of patients (50.0% and 50.5%) in both secukinumab-treated dose groups (150 mg and 75 mg; p<0.001) achieved ACR 20 response compared with only 17.3% of placebo patients1. Clinically significant improvements with secukinumab were observed as early as Week 1 and sustained throughout 52 weeks of treatment.
"Secukinumab is the first IL-17A inhibitor with detailed positive results for the treatment of PsA, further validating the importance of the role IL-17A plays in spondyloarthritis,” said Vasant Narasimhan, Global Head of Development, Novartis Pharmaceuticals. “Novartis looks forward to advancing this important therapy to address the unmet need for patients living with PsA."
PsA is a debilitating, long-lasting inflammatory disease associated with joint pain and stiffness, skin and nail psoriasis, swollen toes and fingers, persistent painful tendonitis and irreversible joint damage. These all lead to significant disability, poor quality of life and reduced life expectancy. Importantly, in FUTURE 1, clinical benefits with secukinumab were observed regardless of prior exposure to anti-tumor-necrosis-factor (anti-TNF) medicines, the current standard of care. Many patients do not respond to, or tolerate these therapies and approximately 45% of people are dissatisfied with current treatments. There is therefore, a high unmet need for patients with PsA.
Secukinumab was well tolerated in the study, with a safety profile that was consistent with that observed in the large psoriasis clinical trial program involving nearly 4,000 patients. The most common adverse events (AEs) were the common cold, headache and upper respiratory tract infections.
Infinity Pharmaceuticals, Inc. (NASDAQ: INFI) announced the 120th patient has been enrolled in DYNAMO™, a Phase 2 study in patients with refractory indolent non-Hodgkin lymphoma (iNHL) evaluating the safety and efficacy of duvelisib an oral, dual inhibitor of phosphoinositide-3-kinase (PI3K)-delta and PI3K-gamma. This enrollment achievement triggers a $130 million milestone payment from AbbVie Inc., Infinity's global development and commercialization partner for duvelisib in oncology. Infinity expects to report topline data from DYNAMO in the third quarter of 2016.
"The completion of patient enrollment in DYNAMO represents a significant milestone for Infinity," said Julian Adams, Ph.D., president, research and development at Infinity. "We are grateful for the support of the DYNAMO investigators, and most importantly the patients and their families, for their participation in this study. In addition, we would like to thank the Infinity and AbbVie teams for their hard work in ensuring completion of enrollment in this trial. We look forward to sharing topline DYNAMO data next year."
"The duvelisib development program underscores our commitment to developing innovative treatment options for patients with hematologic malignancies, and completing patient enrollment in DYNAMO represents an important step toward advancing the duvelisib program," stated Adelene Perkins, Infinity's president and chief executive officer. "Additionally, the milestone payment provides Infinity with important financial resources as we continue to execute on our strategic development plans as we work with AbbVie to bring duvelisib to patients. Very few therapeutic options exist for patients with relapsed/refractory indolent non-Hodgkin lymphoma, and more oral therapies are needed."
DYNAMO is a global, Phase 2 open-label, single-arm, monotherapy study of duvelisib (25 mg BID) in 120 patients with iNHL whose disease is refractory to rituximab and to either chemotherapy or radioimmunotherapy. The primary endpoint is overall response rate.
Jazz Pharmaceuticals plc (NASDAQ: JAZZ) announced the FDA has accepted for filing with Priority Review its recently submitted New Drug Application (NDA) for defibrotide. Defibrotide is an investigational agent proposed for the treatment of patients with hepatic veno-occlusive disease (VOD), also known as sinusoidal obstruction syndrome (SOS), with evidence of multi-organ dysfunction (MOD) following hematopoietic stem-cell transplantation (HSCT).
Priority Review status is designated for drugs that may offer major advances in treatment or provide a treatment where no adequate therapy exists. Based on timelines established by the Prescription Drug User Fee Act (PDUFA), FDA review of the NDA is expected to be completed by March 31, 2016.
"The FDA's acceptance for filing and Priority Review status of the NDA for defibrotide is an important milestone for Jazz and reflects our commitment to bringing meaningful medicines to patients who have significant unmet needs," said Karen Smith, M.D., Ph.D., Global Head of Research and Development and Chief Medical Officer of Jazz Pharmaceuticals. "We look forward to continuing to work closely with the FDA to obtain approval for defibrotide for patients with hepatic VOD with evidence of MOD in the U.S. as quickly as possible, as there are no other approved therapies for treating this rare, often fatal complication of HSCT."
The NDA includes safety and efficacy data from three clinical studies of defibrotide for the treatment of hepatic VOD with MOD following HSCT, as well as a retrospective review of registry data from the Center for International Blood and Marrow Transplant Research. The safety database includes over 900 patients exposed to defibrotide in the clinical development program for the treatment of hepatic VOD.
"We applaud the FDA for working with Jazz in accepting this application for a timely review as bone marrow transplant patients who develop VOD with MOD currently have no effective options for this potentially life-threatening syndrome," said Susan K. Stewart, Executive Director, BMT InfoNet (Blood & Marrow Transplant Information Network).
Applied Genetic Technologies Corporation (Nasdaq:AGTC), a biotechnology company conducting human clinical trials of adeno-associated virus (AAV)-based gene therapies for the treatment of rare eye diseases, today announced two studies supporting further clinical investigation of the Company's novel investigational gene therapy candidate for treating XLRS were published in Human Gene Therapy Clinical Development. "These preclinical data are encouraging and support clinical studies of AAV-based gene therapies for use in vision-robbing retinal diseases," said Stephen M. Rose, Ph.D., Chief Research Officer, Foundation Fighting Blindness. "We are looking forward to seeing results from AGTC's ongoing clinical study and watching the Company's progress in bringing ocular gene therapies to market." One study evaluated the safety and biodistribution profiles of rAAV2tYF-CB-hRS1, a recombinant AAV vector expressing retinoschisin (RS1), in RS1-deficient mice. Three groups of 20 mice each received an intravitreal injection in one eye of either vehicle or a higher or lower dose of vector. Results demonstrated that intravitreal injection of rAAV2tYF-CB-hRS1 was well tolerated in all groups with no test article-related changes in ophthalmic examinations. Microscopic pathology results demonstrated minimal to slight mononuclear cell infiltrates in 80 percent of vector-injected eyes at day 30, decreasing to 20 percent at day 90, with minimal ocular inflammatory cells detected by histopathology. RS1 expression, measured by immunohistochemistry, was demonstrated in all vector-injected eyes and was associated with decreased severity of splitting and disorganization of the inner nuclear layer of the retina at the higher dose level. Biodistribution studies demonstrated vector DNA in the injected eye and no vector DNA in any other tissue.
BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) announced today that the company will present research results at the 20th International Congress of the World Muscle Society, including one oral presentation in Pompe disease and two posters each in Duchenne muscular dystrophy and Pompe disease programs. The meeting will be held in Brighton, England from September 30 to October 4. During an oral session, preliminary clinical efficacy and safety data from an existing extension study of reveglucosidase alfa (BMN 701) in patients with late onset Pompe disease will be presented. Pompe disease is an inherited condition caused by the deficiency in the enzyme acid alpha-glucosidase, which leads to glycogen accumulation and progressive muscle weakening, including muscles essential for breathing. Reveglucosidase alfa is a novel fusion protein of insulin-like growth factor 2 and acid alpha-glucosidase, designed to target delivery to cell structures called lysosomes where the enzyme is most needed. Results will also be presented on Duchenne muscular dystrophy, including those from a prospective natural history study to measure progression of physical impairment, activity limitation and quality of life in the condition. Duchenne muscular dystrophy is a progressive muscle disorder caused by mutations in dystrophin, a protein which plays an important structural role in muscle cells. Boys living with Duchenne experience progressive muscle weakness, resulting in serious medical complications and death.
Capnia, Inc. (NASDAQ:CAPN), focused on the development of novel products based on its proprietary technologies for precision metering of gas flow, today announced its participation in two upcoming investor conferences: The Aegis 2015 Growth Conference on Thursday, October 8, 2015 at 3:00 PM PT at the Encore at Wynn Las Vegas in Las Vegas, NV --- Dawson James Securities 2015 Growth Stock Conference on Thursday, October 15, 2015 at 12:15 PM ET at the Wyndham Grand Hotel in Jupiter, FL.
Dermira, Inc. (NASDAQ:DERM), a specialty biopharmaceutical company focused on bringing innovative and differentiated products to dermatologists and their patients, today announced the election of Kathleen Sebelius, the 21st U.S. Secretary of Health and Human Services, to its board of directors. "We are honored and excited to welcome Kathleen Sebelius to Dermira's board of directors," said Tom Wiggans, chairman and chief executive officer of Dermira. "Kathleen is an exceptional individual of immense personal and public achievement and I am confident we will benefit from her wealth of experience and wise counsel in the coming months and years." Ms. Sebelius was Secretary of Health and Human Services from 2009 to 2014. As Secretary, she presided over 11 operating divisions, including the Centers for Disease Control and Prevention (CDC), Food and Drug Administration (FDA) and National Institutes of Health (NIH), and an annual budget approaching $1 trillion. She also oversaw the passage and implementation of the Affordable Care Act, the most significant health reform in half a century. Previously, Ms. Sebelius was Governor of Kansas, having been elected in 2002 and winning re-election in 2006. As Governor, she was credited with reducing government waste, paying down state debt and being a strong supporter of public education, all of which were accomplished without a tax increase. For the preceding eight years, Ms. Sebelius held the position of Kansas Insurance Commissioner, and from 1987-1995, she served four terms in the Kansas House of Representatives. Ms. Sebelius holds a BA in political science from Trinity Washington University and a Master of Public Administration from the University of Kansas. "I am delighted to join Dermira's board at this very exciting time in the company's evolution," said Kathleen Sebelius. "Dermira is uniquely positioned in medical dermatology with a development pipeline of innovative product candidates that have the potential to improve patients' lives through new and differentiated treatment options."
Edge Therapeutics, Inc. (Nasdaq:EDGE) today announced the pricing of its initial public offering of 7,315,151 shares of its common stock at a public offering price of $11.00 per share, before underwriting discounts and commissions. All of the shares of common stock are being offered by Edge Therapeutics. In addition, Edge Therapeutics granted the underwriters a 30-day option to purchase up to an additional 1,097,272 shares of common stock at the same price, to cover over-allotments, if any. The shares are scheduled to begin trading on The NASDAQ Global Select Market on October 1, 2015 under the ticker symbol "EDGE." Leerink Partners and Credit Suisse are acting as joint book-running managers for the offering. Guggenheim Securities and JMP Securities are acting as co-managers. The offering is being made only by means of a prospectus. A copy of the final prospectus related to this offering will be filed with the Securities and Exchange Commission and may be obtained, when available, from Leerink Partners LLC, Attention: Syndicate Department, One Federal Street, 37th Floor, Boston, MA 02110, or by email at [email protected], or by phone at (800) 808-7525, ext. 6124, or Credit Suisse Securities (USA) LLC, Attention: Prospectus Department, One Madison Avenue, New York, NY 10010, or by telephone at (800) 221-1037, or by email at [email protected] A registration statement relating to the securities being sold in this offering was declared effective by the Securities and Exchange Commission on September 30, 2015.
Entra Health today announced the launch of a new video consult application integrated with the company’s MyHealthPoint platform. The new feature allows a clinician, clinical researcher or care provider to engage in direct video consultation with patients through a computer or mobile device. MyHealthPoint is an FDA 510K Class II approved real-time online data collection network. “Our new video consult capability brings clinicians and patients face-to-face, no matter where they are,” said Richard C. Strobridge, CEO of Entra Health. “This capability has been critically lacking in the FDA-approved remote patient monitoring space. Using video consults, clinicians and care providers can communicate directly with patients to better understand and evaluate the data they are seeing from the Cloud.” Video Consult is an expansion of Entra360 platform, providing easy-to-implement, turnkey solutions for cloud-based analytics and remote patient monitoring. Researchers, clinicians and other care providers can easily schedule immediate or future video consultations with patients from within the MyHealthPoint Clinical Portal using a drag and drop appointment scheduler. Both the patient and provider receive a notification on a tablet app, by email or SMS message that a video consult has been scheduled. A reminder is then sent 15 minutes before the session begins. During any video consult, other care providers or remote family members can be invited to participate in a multipoint, multi-person live videoconference. Additional functions include screen-sharing, screen captures and image uploads. The entire video consultation can be recorded and saved as part of the patient electronic health record (EHR) and used for billing. Together with Entra360 technology, patients can be almost anywhere in the world, without physical connection to clinicians that are monitoring their progress.
Medgenics, Inc. (NYSE MKT:MDGN) today announced that it intends to offer and sell shares of its common stock in an underwritten public offering. The offering is subject to market conditions, and there can be no assurance as to whether or when the offering may be completed, or as to the actual size or terms of the offering. As part of this proposed offering, Medgenics intends to grant the underwriters a 30-day option to purchase up to an additional 15% of the shares of common stock offered to the public. Piper Jaffray & Co. is acting as sole book-running manager for the proposed offering. JMP Securities LLC is acting as lead manager and Needham & Company, LLC is acting as co-manager. All of the shares to be sold in the proposed offering are being sold by Medgenics. Medgenics expects that the net proceeds will be used for product development activities, including (a) the development of technologies acquired in the previously announced acquisition of neuroFix, LLC, (b) patent maintenance fees and intellectual property support, (c) licensing and research collaborations and (d) general corporate purposes and working capital, which may include the acquisitions or licensing of complementary technologies, products or businesses and making certain payments in connection with the acquisition of neuroFix, LLC, including an expected $2.8 million payment to satisfy a specific corporate milestone payment to the former stockholders of neuroFix, LLC.
Pacific Biosciences of California, Inc., (NASDAQ:PACB) a pioneer and leader in long-read sequencing using its Single Molecule, Real-Time (SMRT®) Technology, today announced it has launched a new nucleic acid sequencing platform. The Sequel™ System provides higher throughput, more scalability, a reduced footprint and lower sequencing project costs compared to the PacBio® RS II System, while maintaining the existing benefits of the company's SMRT Technology. Pacific Biosciences will showcase the new product at the American Society of Human Genetics annual meeting taking place in Baltimore, Maryland beginning October 6, 2015. The core of the Sequel System is the capacity of its redesigned SMRT Cells, which contain one million zero-mode waveguides (ZMWs) at launch, compared to 150,000 ZMWs in the PacBio RS II. Active individual polymerases are immobilized within the ZMWs, providing windows to observe and record DNA sequencing in real time. With about seven times as many reads per SMRT Cell as the PacBio RS II, customers should be able to realize lower costs and shorter timelines for sequencing projects, with approximately half the up-front capital investment compared to previous technology. The Sequel System occupies a smaller footprint — less than one-third the size and weight — compared to the PacBio RS II. Since the new system is built on the company's established SMRT Technology, most aspects of the sequencing workflow are unchanged. Michael Hunkapiller, Ph.D., CEO of Pacific Biosciences, commented: "We are extremely proud to introduce the Sequel System, which provides access to the existing benefits of SMRT Sequencing, including long reads, high consensus accuracy, uniform coverage, and integrated methylation information – a set of core attributes first pioneered with the PacBio RS. The system's lower price and smaller footprint represent our continued commitment to leveraging the scalability of our technology and the unique characteristics of SMRT Sequencing. Moreover, with its lower cost of goods (approximately a quarter of that of the PacBio RS II) we expect to be able to achieve substantial gross margin improvement and move more quickly toward profitability."
Pharmaceuticals, Inc. (NASDAQ:VTAE), a clinical-stage biotechnology company, today announced the appointment of Karen Bernstein, Ph.D., to its board of directors, effective immediately. Dr. Bernstein is best known for co-founding and shaping BioCentury Publications Inc. into one of the biotechnology sector's most highly regarded sources of global industry intelligence. Dr. Bernstein stepped down from her role as Editor-in-Chief in August, but continues to serve as BioCentury's Chairman. "Karen's vast professional network, industry insight, and strategic perspective will be of great value to the Vitae team as we continue to advance our wholly owned product candidates through the clinic," said Don Hayden, Chairman of Vitae's board of directors. Dr. Bernstein co-founded BioCentury in 1992. During her tenure as Chairman and Editor-in-Chief, she was instrumental in growing the company into a key source of independent intelligence and analysis for a global biopharmaceutical audience. "Vitae's first-in-class RORγt inhibitor is a fascinating oral target in the dynamic field of immunology," said Dr. Bernstein. "The Company's clinical pipeline of novel drug candidates has the potential to vastly improve the quality of life for patients afflicted with serious autoimmune disorders and inflammatory skin conditions. I look forward to working closely with Vitae's management team."
Surgery Partners, Inc., a leading healthcare services company, today announced the pricing of its initial public offering ("IPO") of 14,285,000 shares of common stock at a public offering price of $19.00 per share. The underwriters have also been granted an option to purchase up to 2,142,750 secondary shares from certain of the Company's stockholders at the public offering price less underwriting discounts and commissions. The Company's common stock is expected to begin trading on the NASDAQ Global Market under the ticker symbol "SGRY" on October 1, 2015. The offering is expected to close on October 6, 2015, subject to customary closing conditions. BofA Merrill Lynch, Goldman, Sachs & Co. and Jefferies LLC will be acting as joint book-running managers and representatives of the underwriters for the offering. Citigroup, Morgan Stanley, Credit Suisse Securities (USA) LLC, Raymond James & Associates, Inc. and RBC Capital Markets will be acting as joint book-runners, and Stifel will be acting as co-manager. A registration statement relating to these securities has been declared effective by the U.S. Securities and Exchange Commission (the "SEC") on September 30, 2015. The offering is being made only by means of a prospectus. A copy of the final prospectus related to the offering will be filed with the SEC and copies may be obtained, when available, by contacting (i) BofA Merrill Lynch, 222 Broadway, New York, NY 10038, Attn: Prospectus Department, or by email at [email protected], (ii) Goldman, Sachs & Co., 200 West Street, New York, NY 10282, Attn: Prospectus Department, or by telephone at (866) 471-2526, or by email at [email protected], or (iii) Jefferies LLC, Equity Syndicate Prospectus Department, 520 Madison Avenue, 2nd Floor, New York, NY 10022, or by telephone at (877) 821-7388, or by email at [email protected]
Vitae Pharmaceuticals, Inc. (NASDAQ:VTAE), a clinical-stage biotechnology company, today announced the appointment of Karen Bernstein, Ph.D., to its board of directors, effective immediately. Dr. Bernstein is best known for co-founding and shaping BioCentury Publications Inc. into one of the biotechnology sector's most highly regarded sources of global industry intelligence. Dr. Bernstein stepped down from her role as Editor-in-Chief in August, but continues to serve as BioCentury's Chairman. "Karen's vast professional network, industry insight, and strategic perspective will be of great value to the Vitae team as we continue to advance our wholly owned product candidates through the clinic," said Don Hayden, Chairman of Vitae's board of directors. Dr. Bernstein co-founded BioCentury in 1992. During her tenure as Chairman and Editor-in-Chief, she was instrumental in growing the company into a key source of independent intelligence and analysis for a global biopharmaceutical audience. "Vitae's first-in-class RORγt inhibitor is a fascinating oral target in the dynamic field of immunology," said Dr. Bernstein. "The Company's clinical pipeline of novel drug candidates has the potential to vastly improve the quality of life for patients afflicted with serious autoimmune disorders and inflammatory skin conditions. I look forward to working closely with Vitae's management team."