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Monday, December 16, 2019

Acadia Pharma gets a new bull

Nuplazid is on track to become the established standard of care in Parkinson’s disease psychosis (PDP), says Guggenheim’s Yatin Suneja, initiating Acadia Pharmaceuticals (NASDAQ:ACAD) with a Buy rating and $60 price target (36% upside from last week’s close).
Then there’s the drug’s approval and launch for dementia-related psychosis (DRP), which is 10x the size of PDP.
Suneja is also a fan of the company’s pipeline beyond those two treatments.
The average Sell-Side rating on ACAD is bullish, and the average SA Author rating is very bullish.
Shares are up 1.95% premarket to $45.20.

Wave craters 59% premarket on exit from Duchenne med development

Wave Life Sciences (NASDAQ:WVE) plummets 59% premarket on light volume in reaction to its decision to stop development of suvodirsen for the treatment of Duchenne muscular dystrophy (DMD) in patients amenable to exon 51 skipping.
The company made its decision after interim results from a Phase 1 study failed to show a change from baseline in dystrophin expression.
Two other suvodirsen trials, on open-label extension study and Phase 2/3 DYSTANCE 51, will be terminated as well.
The company is also axing development of WVE-N531 for the potential treatment of DMD patients amenable to exon 53 skipping, representing a complete exit from DMD.
DMD leader Sarepta Therapeutics (NASDAQ:SRPT) is up 2% premarket.

Lilly to use DexCom CGMs in automated insulin dosing system

Eli Lilly (NYSE:LLY) inks a non-exclusive agreement with DexCom (NASDAQ:DXCM) for the integration of the latter’s continuous glucose monitoring (CGM) devices in both pen- and pump-based platforms in its personalized diabetes management system.
Lilly is designing its system to integrate personalized CGM data with data from glucose-sensing technologies into a compatible software application, called a hybrid-closed loop system, aimed at automating insulin dosing.
Financial terms are not disclosed.

New autism guidelines focus in early diagnosis, treatment

The American Academy of Pediatrics (AAP) on Monday issued its first new autism treatment guidelines in 12 years aimed at helping doctors identify at-risk children and getting them the care they need as early as possible.
Since developmental delays are often present in very young children with autism, the report, published in the journal Pediatrics, urges doctors to check for issues during all well-baby visits and refer children for treatment at the first sign of an issue, rather than wait for a formal autism evaluation.
More than five million Americans are living with autism, a spectrum of disorders marked by deficits in social communication and interaction and repetitive behaviors of widely varying severity.
“The benefit of identifying children as early as possible is they can then be referred for treatment,” Dr. Susan Levy, a developmental and behavioral pediatrician at Children’s Hospital of Philadelphia and co-author of the report, said in a phone interview.
Early treatment, and particularly behavioral interventions, do make a difference, Levy said.

Since 2007, when AAP published its last two guidance documents, the number of children in the United States diagnosed with autism has risen sharply. Autism now affects 1 in 59 children in the United States, up from 1 in 155 in 2007.
In that time, scientists have developed a better understanding of the potential risk factors and genes that contribute to autism, the related medical and behavioral conditions that commonly occur in children with the condition, and have detailed evidence on which interventions work best.
“This report is really focused on educating pediatricians and other healthcare providers about all the options and issues, and working to empower them since they’re in the front lines to make the early referrals,” Levy said.
The report urges doctors to steer families toward interventions backed by research and away from those with flimsy evidence. It specifically calls out many nutritional interventions that “do not have evidence to support their use.”
It also focuses on the need to screen for and treat other conditions that commonly occur in children with autism.

Some 40% of individuals with autism have intellectual disability, the report said, and about 40% to 60% of school-aged children and adults with autism have anxiety disorders. Others include attention-deficit/hyperactivity disorder (ADHD), language disorders, sleep and feeding disorders, gastrointestinal symptoms and seizures.
The report encourages doctors to share decision making with families and help them plan for when a child transitions to adolescence and adulthood.

Merck’s last-resort antibiotic Recarbrio nears EU approval

Merck & Co/MSD’s three-drug antibiotic Recarbrio has been recommended for approval in Europe for Gram-negative infections, but should be reserved for use in patients with limited treatment options.
The European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) backed approval of Recarbrio at its meeting last week, a few months after the drug was approved by the FDA for complicated urinary tract and intra-abdominal infections (cIAI).
The new drug combines the active ingredients in MSD’s broad-spectrum antibiotic Primaxin (imipenem/cilastatin) with a new beta lactamase inhibitor called relebactam that is designed to restore susceptibility to imipenem in resistant strains.
Drugs in the carbapenem class like imipenem have become last-line treatments for a number of multidrug resistant (MDR) infections, so Recarbrio could offer a fallback option in people with serious infections.
The CHMP notes that Recarbrio should only be prescribed “after consultation with a physician with appropriate experience in the management of infectious diseases” and with consideration to “official guidance on the appropriate use of antibacterial agents.”
The drug targets Gram-negative pathogens like Enterobacter cloacea, Escherichia coli, Klebsiella aerogenes, Klebsiella pneumoniae and Pseudomonas aeruginosa.
Recarbrio’s reserved status means that it won’t be a big seller for MSD, with Cowen & Co analysts expecting sales to reach around $150 million in 2024, but it will be an important new option for patients.
The low returns on new antibiotic research have driven a lot of the multinational drugmakers out of the category, raising fears that MDR will become rampant and society could enter a post-antibiotic age where once-trivial infections can be life-threatening.
That disincentive is one of the factors behind a new UK proposal for a new payment system for antibiotics that will see companies paid upfront for access to new antibiotics based on their value to the NHS, rather than the volume of medicines sold.
Recarbrio was one of five new medicines recommended for approval by the CHMP at its latest meeting, along with Novartis’ wet age-related macular degeneration (AMD) therapy Beovu, Accord Healthcare’s generic versions of azacitidine for blood cancers and sedative dexmedetomidine, and Pfizer’s Amsparity – a biosimilar of AbbVie’s immunology blockbuster Humira (adalimumab).
There were also several new positive opinions on already-marketed drugs, including:
  • Janssen-Cilag’s Darzalex (daratumumab) for use in combination with Takeda’s Velcade (bortezomib), thalidomide and dexamethasone for adult patients with newly diagnosed multiple myeloma who are eligible for an autologous stem cell transplant;
  • A new 61 mg soft capsule formulation of Pfizer’s Vyndaqel (tafamidis) for the treatment of wild‑type or hereditary transthyretin amyloidosis in adult patients with transthyretin amyloid cardiomyopathy (ATTR-CM);
  • Extending the age-range for children with plaque psoriasis eligible for treatment with Janssen-Cilag’s Stelara (ustekinumab) to six years and over from 12 and over;
  • Extending the age range for treatment with Janssen-Cilag’s pulmonary multidrug‑resistant tuberculosis (MDR‑TB) therapy Sirturo (bedaquiline) to include adolescents aged over 12 as well as adults;
  • New oral and intravenous infusion formulations of Helsinn Birex Pharma’s Akynzeo (fosnetupitant/palonosetron) for prevention of chemotherapy-induced nausea and vomiting (CINV);
  • Eli Lilly’s Cyramza (ramucirumab) in combination with Roche’s Tarceva (erlotinib) for the first-line treatment of adult patients with metastatic non-small cell lung cancer with activating EGFR mutations;
  • A new film-coated formulation of Astellas’ Dificlir (fidaxomicin) that extends the use of the Clostridioides difficile infection (CDI) drug to include children weighing over 12.5 kg as well as adults.
  • https://pharmaphorum.com/news/mercks-last-resort-antibiotic-recarbrio-nears-eu-approval/

Humana to acquire Enclara Healthcare

Humana (NYSE:HUM) has agreed to acquire privately held Enclara Healthcare, a hospice pharmacy and benefit management provider, for an undisclosed sum. The transaction should close in H1 2020 and is not expected to impact Humana’s 2020 earnings.

Axsome +56% premarket on positive AXS-05 data in major depression

Axsome Therapeutics (NASDAQ:AXSM) is up 56% premarket on the heels of AXS-05, an oral, investigational NMDA receptor antagonist with multimodal activity, meeting primary endpoint in the GEMINI Phase 3 trial in major depressive disorder (MDD).
The study enrolled 327 patients. AXS-05 met the primary endpoint by demonstrating a highly statistically significant reduction in the Montgomery-Ã…sberg Depression Rating Scale (MADRS) total score compared to placebo at Week 6, with mean reductions from baseline of 16.6 points for AXS-05 and 11.9 points for placebo (p=0.002).
AXS-05 rapidly and durably improved depressive symptoms as compared to placebo with statistical significance on the MADRS total score demonstrated at Week 1 key secondary endpoint, p=0.007).
Statistically significant improvement for AXS-05 was observed versus placebo on all secondary endpoints at week 6, including remission (p<0.001), disease severity (p=0.002), functional impairment (p=0.002), and quality of life (p=0.011).
Axsome plans to file NDA for AXS-05 in H2 2020.
AXS-05 is covered by 41 issued U.S. and international patents providing protection out to 2034, and maintains worldwide rights.