Eli Lilly announced that the FDA has granted priority review for its supplemental biologics license application, or sBLA, for Emgality injection for the preventive treatment of episodic cluster headache in adults. Priority review aims to expedite the review of applications for drugs that, if approved, could potentially represent a significant advancement in treatment for a serious condition. The sBLA is based on data from a Phase 3 study that evaluated the safety and efficacy of Emgality injection 300mg in 106 adult patients with episodic cluster headache. Emgality was previously granted breakthrough therapy designation by the FDA in September 2018 for the preventive treatment of episodic cluster headache, indicating the significant unmet need for this debilitating condition for which there are currently no approved preventive medications.
https://thefly.com/landingPageNews.php?id=2874191
Tuesday, March 5, 2019
Acceleron receives FDA Orphan Drug Designation for ACE-083
Acceleron Pharm announced that the FDA has granted Orphan Drug designation to ACE-083, the company’s locally-acting “Myostatin+” muscle agent, for the treatment of patients with Charcot-Marie-Tooth disease. ACE-083 is currently being evaluated in two Phase 2 trials: one in CMT and one in facioscapulohumeral muscular dystrophy, or FSHD. Preliminary results from Part 2 of the trials are expected by year end 2019 for CMT and in the second half of 2019 for FSHD. During 2018, the FDA granted Fast Track designation for ACE-083 in CMT as well as Fast Track and Orphan Drug designations for ACE-083 for the treatment of patients with FSHD.
https://thefly.com/landingPageNews.php?id=2874221
https://thefly.com/landingPageNews.php?id=2874221
Pacira to acquire MyoScience, add FDA-approved iovera system for pain control
Pacira Pharmaceuticals announced the signing of a definitive agreement for the acquisition of MyoScience by Pacira. MyoScience currently markets the iovera system, a novel, FDA-approved non-opioid treatment that alleviates pain through a mechanism known as cryoanalgesia, which applies intensely focused cold therapy to a specific nerve to interrupt its ability to transmit a pain signal. Results can be felt immediately after iovera treatment with pain relief that can last three months, and in some cases longer, as the nerve regenerates over time. “We are delighted to announce this strategic acquisition, which is highly complementary to EXPAREL and underscores our corporate mission to provide an opioid alternative to as many patients as possible,” said Dave Stack, chairman and CEO of Pacira. “We believe the iovera system has significant growth opportunity given the key role it can play in the management of pain associated with both orthopedic surgery and persistent orthopedic conditions such as osteoarthritis. We will be working to leverage our strong commercial infrastructure, partnership network, including our substantial commercial collaboration with Johnson & Johnson, and deep domain expertise to drive widespread adoption of this exciting treatment. By combining iovera with EXPAREL, we are offering healthcare providers an effective, non-opioid multimodal regimen that can help mitigate or even eliminate the use of opioids for managing pain before, during and after surgery.” Under the terms of the agreement, Pacira will make an initial payment of $120M. MyoScience shareholders will be eligible to receive up to an additional $100M in contingent payments upon achievement of certain regulatory and commercial milestones. Pacira expects the acquisition to be accretive to net income beginning in the second half of 2020 and increasingly accretive thereafter. The transaction is expected to close in April 2019 subject to customary closing conditions, including the expiration or termination of the waiting period under the Hart-Scott-Rodino Antitrust Improvements Act of 1976, as amended. Upon the closing of the acquisition, Pacira intends to change its corporate name to Pacira BioSciences, Inc., in order to better reflect a broadening portfolio of innovative non-opioid pain management and regenerative health solutions. Pacira will continue to trade under the symbol “PCRX.” Upon the closing of the transaction, MyoScience will become Pacira CryoTech, Inc., a wholly owned subsidiary of Pacira.
Morgan Stanley ups Alnylam to Overweight on Onpattro, pipeline
As previously reported, Morgan Stanley analyst David Lebowitz upgraded Alnylam to Overweight from Equal Weight, stating that he expects Onpattro to ultimately exceed the Street’s expectations for 2019 given early metrics about the drug’s launch. Even modest sequential adds as the year progresses to the 200 patients on the drug at year-end 2018 should put it on track to beat his 2019 sales estimate of $105M, as well as consensus of $94M, said Lebowitz. He also expects positive news flow from the company’s late stage pipeline, particularly Givosiran and Lumasiran. Lebowitz raised his price target on Alnylam shares to $122 from $81.
Mirati Therapeutics initiated at Cantor Fitzgerald
Mirati Therapeutics initiated with a Neutral at Cantor Fitzgerald. Cantor Fitzgerald analyst Varun Kumar last night started Mirati Therapeutics with a Neutral rating and $66 price target. Current consensus estimates reflect high expectations around the novel KRASi asset, and do not fully appreciate potential clinical, regulatory and commercial risks associated with the company’s lead asset sitravatinib, Kumar tells investors in a research note.
https://thefly.com/landingPageNews.php?id=2874251
https://thefly.com/landingPageNews.php?id=2874251
FDA grants Priority Review for Celgene’s NDA for fedratinib
Celgene Corporation announced the U.S. Food and Drug Administration has accepted the company’s New Drug Application for fedratinib and granted a Priority Review. Fedratinib is a highly selective JAK2 inhibitor intended for the treatment of patients with myelofibrosis, a serious bone marrow disorder that disrupts the body’s normal production of blood cells.1 Under the Prescription Drug User Fee Act, the FDA has set its action date as Sept. 3, 2019. The NDA for fedratinib is based on results from a randomized, placebo-controlled, phase 3 trial in patients with primary or secondary myelofibrosis, as well as a single-arm, open-label phase 2 trial in patients with primary or secondary myelofibrosis previously exposed to ruxolitinib, the only FDA-approved treatment for the disease. Results of these two trials have been previously published in peer-reviewed journals. The FDA has also provided fedratinib Orphan Drug designation for the treatment of secondary and primary myelofibrosis. Celgene also plans to evaluate fedratinib in combination with luspatercept. Fedratinib is an investigational compound that is not approved for any use in any country.
https://thefly.com/landingPageNews.php?id=2874259
https://thefly.com/landingPageNews.php?id=2874259
Monday, March 4, 2019
Ultrasound scans could boost screenings for osteoporosis
A new study that aimed to advance methods of assessing bone health found that data from ultrasound scans were equal to those gathered using X-ray.
The way we live shapes our bones. There are a lot of things we can do to help keep our bones healthy.
Bone mineral density (BMD) gauges the amount of bone mineral in bone tissue, and doctors use it to determine bone quality.
Genetics, nutrition, and lifestyle all contribute to BMD.
Following a balanced diet rich in calcium and vitamin D and doing physical activity both contribute to improving bone quality. They also reduce the risk of developing bone diseases.
The body establishes peak BMD by the time a person is in their mid- to late 20s, and it decreases naturally as people get older. If bones are not sufficiently strong during adult life, the risk of conditions such as osteoporosis increases.
The global burden of osteoporosis
Osteoporosis reduces bone quality; they become more fragile and their fracture risk increases. The most common fractures occur in the hips, spine, and wrist.
Vertebral fractures can have serious consequences, including loss of height and intense back pain.
Bone loss has a serious impact on a person’s health and quality of life. In some cases, it can lead to long-term disability and death. Also, osteoporosis represents a socioeconomic burden, resulting in high medical costs and loss of work days.
According to the International Osteoporosis Foundation (IOF), the condition causes almost 9 million fractures annually, worldwide.
Osteoporosis affects about 200 million women. Women tend to have thinner bones than men, and chances of developing the condition increase during menopause, when the hormone estrogendecreases.
Despite its global impact, this condition remains underdiagnosed and undertreated. The IOF claim that doctors neither identify nor treat about 80 percent of people who are at high risk and have already had one fracture.
Advancing methods of assessing bone health
One recent study in The Journal of the American Osteopathic Association found that data obtained from ultrasound scans were equal to those gathered using dual-energy X-ray absorptiometry (DEXA). Experts consider this to be one of the best methods to assess bone quality.
While DEXA remains an excellent option to gather comprehensive information on bone health, the equipment is expensive and many people cannot afford this procedure.
DEXA uses a very small dose of ionizing radiation to create pictures of spine and hips to measure bone loss.
Ultrasound could lead to increased screenings for osteoporosis. The machine works by measuring how sound waves move through the bone. It is portable, inexpensive, and involves no radiation.
“Prior research,” explains Prof. Andrea Nazar, of West Virginia School of Osteopathic Medicine in Lewisburg, “has demonstrated strong correlations between education level and socioeconomic status and bone quality.”
Prof. Nazar goes on to say that young adults should try to pay special attention to lifestyle, nutrition, and exercise to avoid bone diseases later in life. She recommends that people follow a balanced diet combined with weight-bearing exercises that protect and strengthen bones.
“Because of its low cost, mobility, and safety, ultrasound is a promising tool for assessing more people, across multiple demographics.”Prof. Andrea Nazar
Study co-author Carolyn Komar, Ph.D. — also of West Virginia School of Osteopathic Medicine — believes that using ultrasound will not give all the information that a DEXA scan could gather.
However, it can help doctors decide whether they should initiate treatment for the person.
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