Search This Blog

Sunday, September 8, 2019

Biotech week ahead, Sept. 9

The holiday-shortened week saw a few huge moves by individual stocks.
Abeona Therapeutics Inc ABEO 1% was among the biggest gainers after the biopharma announced plans to explore strategic alternatives. On the other hand, Mallinckrodt PLC MNK 18.24% came under intense selling following reports of a potential bankruptcy filing.
Here are they key events for the upcoming week.

Conferences

  • International Association for the Study of Lung Cancer (IASLC) 2019 World Conference – Sept. 7-10, in Barcelona, Spain
  • 32nd European College of Neuropsychopharmacology (ECNP) Congress – Sept. 7-10, in Copenhagen, Denmark
  • 19th Congress of the International Headache Society – Sept. 5-8, in Dublin, Ireland
  • World Congress on Pain Medicine and Management – Sept. 9-10, in Singapore City, Singapore
  • 31st International Conference on Cancer Research and Therapy – Sept. 9-10, in Zurich, Switzerland
  • H.C. Wainwright Annual Healthcare Conference 2019 – Sept. 9-10, in Lotte New York Palace Hotel, New York
  • Morgan Stanley 17th Annual Healthcare Conference – Sept. 9-11, in Grand Hyatt, New York City, New York
  • 35th Congress of the European Committee For Treatment and Research In Multiple Sclerosis (ECTRIMS) – Sept. 11-13, in Stockholm, Sweden
  • Society for Research in Nicotine and Tobacco Europe 10th Annual Conference – Sept. 12-14, in Oslo, Norway
  • 17th International Myeloma Workshop – Sept. 12-15, in Boston, Massachusetts
  • 12th World Congress on Cell & Tissue Science – Sept. 13-14, in Singapore City, Singapore
  • Heart Failure Society of America 23rd Annual Scientific Meeting – Sept. 13-15, in Philadelphia

PDUFA Dates

Xeris Pharmaceuticals Inc XERS 0.56% awaits FDA verdict on its NDA for Gvoke – a ready-to-use glucagon injection – for treating severe hypoglycaemia. The PDUFA action date is scheduled for Tuesday.
The FDA is set to rule on Ardelyx Inc ARDX 2.79%‘s NDA for tenapanor in treating irritable bowel syndrome with constipation. The decision date is set for Thursday.
FDA’s Allergenic Products Advisory Committee is scheduled to meet Tuesday to discuss and make recommendations on the safety and efficacy of Aimmune Therapeutics Inc AIMT 2.37%‘s Allergen Powder indicated for the treatment to reduce the risk of anaphylaxis after accidental exposure to peanut in patients aged 4 to 17 years with a confirmed diagnosis of peanut allergy.

Clinical Trial Readouts

Adverum Biotechnologies Inc ADVM 0.66% is due on Thursday to release 24-week data from the first cohort of a Phase 1 study evaluating its ADVM-022 in wet age-related macular degeneration.

IASLC World Conference Presentations

Beyondspring Inc BYSI 19.15% – presentation of already-released Phase 3 data for plinabulin in chemotherapy-induced neutropenia (Sept. 8)
Seattle Genetics, Inc. SGEN 1.25% & GENMAB A/S/S ADR GMAB 0.9% – initial Phase 1/2 data for enapotamab vedotin in solid tumors (Sept. 8)
Eli Lilly And Co LLY 0.46% – updated Phase 1/2 data for LOXO-292 in RET-fusion non-small cell lung cancer (Sept. 8)
Amgen, Inc. AMGN 0.04% – Updated Phase 1 data for AMG 510 in solid tumors (Sept. 8)
AstraZeneca plc AZN 0.04% – already-released interim Phase 3 data for imfinzi and tremelimumab in small cell lung cancer (Sept. 9)

IHC Congress Presentations

Zosano Pharma Corp ZSAN 3.4% – long-term safety data from a Phase 3 study of Qtrypta in migraine (Sept. 8)

ECNP Congress Presentations

Concert Pharmaceuticals Inc CNCE 2.01% – Phase 1 data for CTP-692 in schizophrenia (Sept. 10)

ECTRIMS Congress Presentations

Atara Biotherapeutics Inc ATRA 0.43% – initial Phase 1 data for ATA188 in multiple sclerosis (Sept. 11)
Roche Holdings AG Basel ADR RHHBY 0.9% – Phase 3 data for satralizumab in neuromyelitis optica spectrum disorder (Sept. 11)
TG Therapeutics Inc TGTX 3.66% – Phase 2 long term follow up data for TG-1101 in relapsing form of multiple sclerosis.
Novartis AG NVS 0.19% and Genmab – already released Phase 3 data for OMB158 in relapsing multiple sclerosis.

Other Conference Presentations

Achieve Life Sciences Inc ACHV 2.7% – already-released Phase 2b data for Cytisine in smoking cessation at the SRNT conference (Sept. 13)
Cytokinetics, Inc. CYTK 1.12% – Phase 1 data from healthy volunteers for CK-3773274 in hypertrophic cardiomyopathy
Cellectar Biosciences Inc CLRB 1.86% – Poster presentation of Phase 1 data for CLR 131 in multiple myeloma at the International Myeloma Workshop (Sept. 14)

IPOs

10x Genomics, a provider of single-cell sequencing platform for biomedical research, is planning to offer 9 million shares in an IPO at an estimated price range of $31-$35. The company is seeking to list its shares on the Nasdaq under the ticker symbol TXG.
Satsuma Pharmaceuticals, which is developing, a fast-acting dry powder nasal spray for migraines, is planning a 5-million share IPO to be priced between $14 and $16. The shares are to be listed on the Nasdaq under the ticker symbol STSA.
SpringWorks Therapeutics, a developer of small-molecule therapies for rare cancers, is planning to offer 7.353 million shares in an IPO. The company expects to price the offering in the $16-$18 range, with the shares to be listed on the Nasdaq under the ticker symbol SWTX.
Posted by at No comments:

Pain Week: Can Cannabis Replace Opioids for Pain Treatment?

Can concurrent cannabinoid and opioid use enhance analgesic effects, or should their combined use be avoided? Presenters at the 2019 PAINWeek conference took on this question, agreeing that many of the effects associated with cannabinoids remain unknown.
“I feel like I’m speaking to the choir here — this is something we’re all dealing with already,” said Chris Herndon, PharmD, of Southern Illinois University in Carbondale, Illinois, at a talk entitled “Cannabis and Opioid Together: Syn or Synergy?”
The proportion of patients in primary care clinics that are on chronic opioid therapy and using cannabis has been reported to be anywhere from 16% to 25%, Herndon said. As such, the potential harms and benefits are certainly something that “need to be discussed.”
There are as many as 400 constituents of the cannabis plant, 66 of which are of the cannabinoid structure, including tetrahydrocannabinol (THC) and cannabidiol (CBD).
They each have a number of different pharmacological modalities, many of which support the idea of using these drugs to treat pain, including calcium channel antagonism, downstream effects on lysergic glutamatergic, and potential N-methyl-D-aspartate receptor (NMDA) activity, Herndon said.
He cited one recent study that found adding medical cannabis to opioid therapy among fibromyalgia patients was associated with significant improvements in pain, measured through the visual analog scale, fibromyalgia pain severity scales, and disability measures.
However, cannabis may affect pain tolerance in a bell or j-shaped curve, in which stimulating pain receptors at certain levels can actually produce a hyperalgesic effect, countered Bradlee Rea, PharmD, of Kaweah Delta Health Care in Visalia, California.
“THC may actually reduce a patient’s tolerance or ability to tolerate pain,” Rea said.
Herndon argued that at the right ratio, CBD and THC together have been shown to reduce pain. For example, one study that tested four different cannabis varieties in patients with fibromyalgia found much greater improvements in pain with Bediol — which contains 13.4 mg THC and 17.8 mg CBD — than with Bedrocan, which contains mostly THC (22.4 mg THC and <1 mg CBD).
But the problem is that in practice, medical marijuana can be a “potpourri surprise,” Rea said, and the majority of studies examining analgesic effects of cannabidiol are not standardized in terms of strain, species, or dosing.
“We have agonists, we have antagonists, we have inverse agonists,” Rea said. “How do we figure out what is going on when we have so many different products in the flower of cannabis? How do we know what product is doing what without more data looking at least in vitro at some of these constituents found in marijuana?”
Rea added that cannabidiol could be functioning by changing how patients perceive their pain, reducing pain catastrophizing, or decreasing the amount of other medications they use.
Herndon said in a clinical environment that is increasingly encouraging opioid alternatives, this could be one advantage to cannabinoids. In a 2019 study, nearly three-quarters of patients who completed a survey said they were able to completely terminate their opioid use after they started using cannabis for chronic pain, he noted. Another survey found just over one-third of patients authorized to use medical cannabis in Canada were able to substitute cannabis for prescription opioids.
“There are those who would get up and argue with me, and say they’re just substituting one drug for another, or potentially they were using prescribed opioids for non-therapeutic intent,” Herndon said. “But I think showing the change in non-opioid analgesics like some of these studies do supports my premise.”
However, cannabis is not without its own side effects, including associations with increased anxiety, depression of the central nervous system, and decreased testosterone production. But all of those have also been linked with opioid use, Rea said.
Patients with underlying depression and anxiety may be even more vulnerable to some comorbidities associated with both drugs, Rea said. For example, in one study in Israel examining patients on concomitant medical cannabis and opioids, patients with depression and anxiety were significantly more likely to show signs of misuse for both drugs.
“Chronic pain isn’t just chronic pain — it can be coupled with stress, anxiety, sleep disturbances, and PTSD [post-traumatic stress disorder],” Rea said. “All of these do have an effect on how people perceive their pain. It’s important to look at the whole picture and treat the whole picture versus just treating the chronic pain.”
Currently, 22 states permit the use of medical marijuana, each with different regulations and products. In New York, Colorado, and Illinois, for example, clinicians can now prescribe medical cannabis as a substitute for opioids. Ultimately, there are many factors that go into the decision, which should jointly be made by the provider and patient, Herndon said.
“We’re trying to balance what’s working for the patient, and trying to provide compassionate care with what little we know about this substance, and the different constituents, and how it might impact the drugs they’re already on,” Herndon said. “Then also making sure you protect yourself as a prescriber because you’re providing care for these patients.”
Herndon and Rea disclosed no relevant relationships with industry.
LAST UPDATED 
Posted by at No comments:

Pain Week: Teva Ajovy Effective for Tough-to-Treat Migraine

Fremanezumab (Ajovy) received FDA approval in 2018 for the prevention of episodic and chronic migraine prevention. But the trials supporting approval of the agent, which functions by targeting calcitonin gene-related peptide (CGRP), excluded patients who had failed two or more preventive medications, and those who had common comorbidities.
Two studies presented at the 2019 PAINWeek conference explored fremanezumab effects in these subgroups.
FOCUS Trial
Fremanezumab reduced headache among patients who had failed to respond to two to four other classes of migraine preventive treatments at 12 weeks versus placebo, according to results from the FOCUS study.
Among 837 participants, fremanezumab showed significantly reduced mean monthly migraine days versus placebo when administered quarterly (95% CI −3.8 to −2.4, P<0.0001) and monthly (95% CI −4.2 to −2.8, P<0.0001), reported Ladislav Pazdera, MD, of Vestra Clinics in Rychnov nad Kněžnou, Czech Republic.
And it worked fast, achieving at least a 50% reduction in the number of monthly migraines compared with placebo by as early as 4 weeks, though less than half of patients achieved this endpoint, she reported in a poster. The findings were published in 2019 in the Lancet.
“Placebo responses were low and decreased with increasing number of classes of prior preventive treatments failed, while significant improvements in efficacy were observed with fremanezumab compared with placebo, even in patients who had previously experienced an inadequate response to 4 different classes of migraine preventive treatments,” Pazdera’s group wrote.
Elizabeth Loder, MD, MPH, of Harvard Medical School in Boston said the follow-up in this study was “relatively short” and its not known whether the “quite modest” improvements in migraine days will be sustained over time.
Loder, who was not involved in the study, also noted that, as with many trials evaluating preventive treatments for migraine, this study struggled to reach the benchmark 50% reduction in migraine days in even half of patients, which perhaps reflects the heterogeneity of the condition.
“The question of whether medications work in patients who have not benefited from other preventive medications is an important one, since those patients are difficult to treat,” Loder told MedPage Today. “It makes sense that prior treatment failures may indicate the presence of more severe disease.”
She also noted the overall number of headache days are not shown, meaning some participants might be classified as achieving the endpoint because they have a reduction in severity of headache.
The trial enrolled adult patients who had failed to respond to preventative migraine medications within the past 10 years across 104 international sites. Participants were then randomly assigned to either the quarterly fremanezumab arm — in which they got 675 mg at month 1 and placebo at months 2 and 3 — or the monthly arm, in which episodic and chronic migraine patients got 225 mg and 675 mg of fremanezumab in month 1, respectively, and then 225 mg at months 2 and 3. A third arm was administered monthly placebo for 12 weeks.
Adverse events (AEs) were similar across all arms; the most common were injection-site erythema, injection-site induration, and nasopharyngitis. Serious adverse events occurred in six patients but were not treatment-related.
HALO Trial
Fremanezumab was also effective among chronic migraine patients with acute medication overuse, according to a post-hoc analysis of the HALO trials, which were used to support the drug’s approval.
Among 477 chronic migraine patients with acute medication overuse, fremanezumab reduced the number of migraine days by at least half in 37% and 48% of patients on the quarterly and monthly regimen, respectively, reported Stephen Silberstein, MD, of Thomas Jefferson University in Philadelphia.
Also, roughly 60% no longer met the criteria for acute medication overuse at 6 months in both the quarterly and monthly arms, which was maintained through the 1-year mark, he reported.
The “medication overuse” diagnosis is controversial and it remains unclear whether frequent use of medication is causing headaches to be worse, or rather that individuals with more severe headache tend to require more medication, Loder said.
“Some people — not me — feel that overuse of symptom relieving medication might render preventive treatment less effective,” Loder said. “Thus, it is reasonable to do a post-hoc analysis of trial participants who had medication overuse to see how they fare.”
For this study, adult patients with confirmed chronic migraine were rolled over from the phase III HALO trials and also enrolled from the community. New enrollees were allowed to continue stable use of two concomitant migraine preventive medications and rollover participants were permitted to continue using one. Rollover patients were not enrolled if using onabotulinumtoxinA, opioids, or barbiturates in the time preceding the study, though these exclusions were not applied to new patients.
In this study, acute medication overuse was defined as acute headache medication use on ≥15 days, migraine-specific acute medication use on ≥10 days, or use of combination medications for headache on ≥10 days.
Loder emphasized that the post-hoc nature of this analysis and the lack of a placebo comparator make this study “hypothesis-generating” at most.
“It is also the case that the method of enrolling participants in these studies may have favored positive findings,” Loder said, adding that rollover patients were the ones who did not drop out of the study due to adverse events or lack of efficacy.
Participants on the quarterly schedule received 675 mg at baseline and placebo at weeks 4 and 8, while those in the monthly arm were administered 675 mg at baseline and 225 mg at weeks 4 and 8.
Those included were a mean age of about 46, who had been diagnosed with migraine 23 years prior. About a quarter were on current preventive medication at the time of the trial as well.
Fremanezumab also reduced the number of days patients required medication by about 8 days at 1 year in both the quarterly and monthly arm, Silberstein reported. It also reduced headache-related disability, measured through Headache Impact Test (HIT-6) scores at 1 year in the quarterly (-6.9 points) and monthly groups (-8.1).
The most common AEs were injection-site reactions in both groups, with <10% of patients in both the quarterly and monthly arms experiencing a serious AE. However, 12% and 11% of patients developed an upper respiratory tract infection in the quarterly and monthly arms, respectively.
FOCUS and HALO were funded by Teva Pharmaceutical. Some co-authors are company employees.
Pazdera disclosed no relevant relationships with industry.
Silberstein disclosed multiple relevant relationships with industry.
Posted by at No comments:

Greater support needed to improve the treatment of knee osteoarthritis

A  new Curtin University-led paper has outlined that many people living with knee osteoarthritis may be needlessly suffering or receiving the wrong treatments for their symptoms, recommending a major change in how osteoarthritis is understood and treated.
The paper, published in the British Journal of Sports Medicine, concluded that people with osteoarthritis are often provided potentially risky treatments with limited benefit such as opioid medication, injections, and arthroscopic surgery to manage their pain.
Lead author Dr. JP Caneiro, from Curtin’s School of Physiotherapy and Exercise Science, said most people with osteoarthritis around the globe are not receiving the best practice care, including education, strengthening exercises,  and weight management, that they require. Greater support from the health system is needed.
“This editorial is a call to action by  in the field, from physiotherapy, rheumatology and orthopaedic surgery, including an outline of three key steps that are needed to change the way  understand and manage knee osteoarthritis,” Dr. Caneiro said.
“This includes changing the way clinicians understand and explain the condition to patients, how they treat the condition and by coaching patients to put them in charge of managing their own condition. Together, this helps to reduce the disability burden and suffering associated with knee osteoarthritis.”
Dr. Caneiro said the proposed steps would enable healthcare professionals to promote a new and better understanding of knee osteoarthritis that is aligned with contemporary evidence.
“Knee osteoarthritis can be painful and debilitating, and the pain is often blamed on structural damage, with people frequently given scans and told that their knees are ‘bone on bone,’ leading them to believe that a knee replacement is inevitable,” Dr. Caneiro said.
“Because of this, treatment often aims to ‘fix’ the structure, leading to many people receiving knee arthroscopic surgery or a knee replacement. In contrast, research tells us that non-surgical treatments targeting physical, lifestyle and psychological factors can reduce pain, disability, reliance on medication and need for surgery. However, effective non-surgical approaches are often not prescribed, and there is very little support within the health system to fund them.”
Co-author John Curtin Distinguished Professor Peter O’Sullivan, also from Curtin’s School of Physiotherapy and Exercise Science, said it was critical to spread the message to international and national healthcare professionals and the general public to change society’s misconceptions about .
“It is important for clinicians to shift away from labeling this condition as ‘structural damage’ and instead focus on knee health, encouraging  therapy, physical activity and weight loss, as well as reassuring patients that it is safe to exercise and strengthen their ,” Professor O’Sullivan said.
“People with osteoarthritis need health professionals to coach them to develop a positive mindset, engage with exercise, and learn how to manage possible flare-ups. To facilitate a shift in the treatment of osteoarthritis, important changes need to occur at the health system level such as funding, and better reimbursement for exercise, weight loss and education programs for  care.”
Explore further
More information: JP Caneiro et al. Three steps to changing the narrative about knee osteoarthritis care: a call to action, British Journal of Sports Medicine (2019). DOI: 10.1136/bjsports-2019-101328
Posted by at No comments:

Drugmakers file second court challenge to Canada’s new drug price rules

Canada’s main pharmaceutical industry lobby group, along with 16 of its member companies, filed a lawsuit on Friday to block new regulations meant to lower patented drug prices, the second legal challenge to a new regime that could eventually reduce prices in the United States as well.
Canada published the final regulations in August, despite heavy lobbying from drug companies, which stand to lose revenue as prices drop. The federal government estimates the new rules will save Canadian patients, employers and insurers, including governments, C$13.2 billion ($10 billion) over a decade.
Friday’s lawsuit was filed in federal court and led by Innovative Medicines Canada (IMC), which represents major drugmakers in Canada. It is separate from a lawsuit filed last month and focuses on federal patent law, arguing that Canada cannot use regulations to “fundamentally alter” the role of its federal drug price regulator.
IMC was not a plaintiff in a Quebec Superior Court challenge filed in August, which argued that price regulation falls within provincial jurisdiction.
“We would not enter into this lightly. The industry lives and breathes saving lives, but it does require a viable business model to do so,” IMC President Pamela Fralick said in an interview. “Canada is not creating a sustainable environment for innovative medicines.”

Fralick said industry had been trying to work with Health Canada to find policy alternatives to the proposal for nearly two years.
IMC has argued that new drugs may launch late or not at all in Canada if prices fall, and that the policy will discourage investment in Canada. The government says other countries with lower drug prices have investment and drug access that are as good as or better than Canada’s.

Lower prices in Canada could spill into the United States, the world’s largest pharmaceutical market, since the Trump administration said in July it would allow U.S. states and other groups to start pilot programs importing drugs from Canada. The administration is also considering linking what it pays for drugs under Medicare, which provides federal health insurance for Americans 65 or older, to prices abroad, including in Canada.
The new regulations, which go into force July 1, 2020, change the list of countries with which Canada’s federal drug price regulator, the Patented Medicine Prices Review Board, compares domestic prices, dropping the United States and Switzerland, where prices are highest. It will also let the agency consider the cost-effectiveness of new medicines for the first time.
Plaintiffs in the new case include Canadian subsidiaries of AbbVie Inc, Astellas Pharma Inc, AstraZeneca PLC, Bristol-Myers Squibb Co, Eli Lilly and Co, Novartis Pharmaceuticals, and Pfizer Inc.
Posted by at No comments:

Amgen drug shrinks lung cancer tumors in half of patients

An experimental Amgen Inc drug that targets a specific genetic mutation reduced tumor size in around half of advanced lung cancer patients given the highest dose in a small, early-stage trial, the company said on Sunday.
Out of 13 lung cancer patients taking a 960-milligram dose of AMG510, seven had tumors shrink by at least 30%, according to data presented in Barcelona at the World Conference on Lung Cancer. Cancer was stabilized in the other six high-dose patients.
Nine of the 13, or 69%, are still taking the daily pill. Two of the partial responders have died, and another left the study due to cancer progression. One patient with stable disease also died.
Patients who responded to the drug have been treated for a median of 15 weeks, but the median duration of response has not been reached, according to Greg Friberg, head of oncology development at Amgen.
He said one patient previously reported as free of lung cancer is being counted as a partial responder since cancer in lymph nodes or other tissue cannot be ruled out.
Thirty-four lung cancer patients are enrolled in the Phase 1 dose-ranging trial, but Amgen has moved only the highest dose into a mid-stage study. The trial enrolled patients with tumors carrying the targeted mutation whose cancer had worsened despite several previous treatments – including chemotherapy and checkpoint inhibitors.
Of the 23 lung cancer patients so far evaluated – at various doses of AMG510 – 11 had tumor shrinkage of at least 30%.
About a third of trial patients reported mild side effects. More serious side effects, diarrhea and anemia, were seen in 9%of patients and no one left the study due to toxicity, the company said.

Wall Street has taken a keen interest in the Amgen drug that could become the first approved medicine targeting a mutated form of a gene known as KRAS – one of the most common mutations found in non-small cell lung cancer (NSCLC).
NSCLC accounts for up to 85% of lung cancers, by far the leading cause of cancer death among men and women, according to the American Cancer Society.
AMG510 is part of a growing trend of precision medicines that target gene mutations driving cancer regardless of the organ in which the disease originated.
The targeted mutation, KRASG12C, occurs in around 13% of NSCLC cases, as well as 3-5% of colorectal cancers and up to 2% of other solid tumor cancers, including pancreatic.
Amgen plans to present Phase 1 AMG510 trial results for patients with colorectal cancer later this month at a separate meeting to be held in Barcelona, this one by the European Society for Medical Oncology.

The company is also enrolling patients in an early-stage study of AMG510 in combination with Keytruda, Merck & Co’s checkpoint inhibitor designed to unleash the body’s immune system to attack cancer cells.
Amgen said the U.S. Food and Drug Administration has granted “fast track” status to AMG510 for previously treated NSCLC with KRASG12C mutation.
Rivals such as Mirati Therapeutics Inc are also developing drugs that target KRAS mutations, but Amgen’s could be first to market.
Posted by at No comments:

More deaths linked to vaping; Michigan 1st with ban

Three more deaths linked to vaping have been reported in Indiana, California and Minnesota, bringing the total number of such U.S. deaths to five.
Some type of chemical irritation is likely associated with the illnesses, but more information is needed to determine the exact cause.
“While this investigation is ongoing, people should consider not using e-cigarette products,” the CDC wrote in a news release.
U.S. health officials are now investigating more than 450 cases of possible vaping-related illnesses in 33 states, while last week, the governor of Michigan announced the state will become the first to ban flavored e-cigarettes.
Related tickers: MO, BTI, PM, JUUL, OTCPK:VAPE, OTCPK:HPNN, OTCPK:MCIG, OTCQB:NTRR, OTCPK:VAPR, OTC:AHII, OTCQX:IMBBY, OTCPK:GLLA
Posted by at No comments:
Subscribe to: Posts (Atom)