Monday, December 2, 2019
Concert Pharma launches mid-stage study of CTP-692 in schizophrenia
Concert Pharmaceuticals (CNCE -3.4%) initiates a Phase 2 clinical trial evaluating CTP-692 for the adjunctive treatment of schizophrenia.
The primary endpoint is the change from baseline in a schizophrenia symptom scale called PANSS at week 12. Topline results should be available in about a year.
CTP-692 is a deuterium-modified form of an amino acid called D-serine. The company says it has the potential to restore NMDA receptor activity in key areas of the brain in schizophrenia sufferers, adding that it will be initially developed as an adjunctive therapy with standard antipsychotic medications.
Cortexyme up 27% ahead of Evercore event
Thinly traded Cortexyme (CRTX +27.3%) is up on triple normal volume, albeit on turnover of only 177K shares, ahead of tomorrow’s fireside chat by CEO Casey Lynch at Evercore ISI’s HealthCONx Conference in Boston at 2 pm ET.
The company’s lead candidate is COR388, a brain-penetrating small molecule gingipain inhibitor for the potential treatment of Alzheimer’s disease (AD).
Gingipains are toxic enzymes secreted by a certain bacterium present in 90% of the brains of AD sufferers.
A Phase 2/3 study, GAIN, is in process.
Freestanding EDs Can Increase Patient Spending
Freestanding emergency departments (FrEDs) increased emergency care spending in three of four states examined in a recent study.
Advocates of FrEDs claim they can ease overcrowding at hospital-based emergency rooms and provide prompt care in convenient locations. Opponents of FrEDs claim they increase spending on emergency room services because the care can often be provided in lower-cost settings, such as urgent care centers.
The study in Academic Emergency Medicine examined FrED data collected in Arizona, Florida, North Carolina, and Texas from January 2013 to December 2017. The researchers focused on total spending on emergency care, out‐of‐pocket spending, utilization, and price per visit.
The research generated three key observations, the lead author of the study said.
“Entry of an additional freestanding emergency department in a local market was associated with an increase in spending per capita in three of four states. Entry was generally associated with an increase in emergency visits per capita, as well as out-of-pocket spending,” said Vivian Ho, PhD, director of the Center for Health and Biosciences at Rice University’s Baker Institute for Public Policy in Houston.
The research features several data points:
- In local markets in Florida, North Carolina, and Texas, entry of an additional FrED resulted in a 3.6 percentage point increase in emergency provider reimbursement per insured beneficiary
- In local markets in Arizona, entry of an additional FrED resulted in no significant reimbursement change
- In local markets in Arizona, Florida, and Texas, entry of an additional FrED increased the number of emergency care visits by 0.18 per 100 insured beneficiaries
- In local markets in North Carolina, entry of an additional FrED did not significantly change the utilization rate
- In local markets in Arizona, Florida, and Texas, entry of an additional FrED increased the average estimated out-of-pocket payments for emergency care by 3.6 percentage points, but out-of-pocket payments decreased 15.3 percentage points in North Carolina
Interpreting the Data
The research is a cautionary tale about FrEDs, Ho and her co-authors wrote. “Rather than functioning as substitutes for hospital-based EDs, FrEDs have increased local market spending on emergency care in three of four states’ markets where they have entered. State policymakers and researchers should carefully track spending and utilization of emergency care as FrEDs disseminate to better understand their potential health benefits and cost implications for patients.”
The utilization findings provide weak support for proponents of FrEDs, Ho said.
“Some of the observed increase in utilization may have led to increased convenience for patients seeking emergency care. However, the overall increase suggests that FrEDs don’t serve as a substitute for hospital-based emergency care. And other studies in the literature have found that entry of FrEDs do not lower waiting times at nearby hospital emergency departments,” she said.
The spending findings support the claims of FrED critics, Ho said. “The results are consistent with critics’ concerns that FrEDs increase spending on emergency care. FrED operators have come to realize that, ‘If you build it, they will come.’ Other research suggests that much of the care that patients receive at FrEDs could be obtained at much lower costs at urgent care centers.”
Primary Source
Academic Emergency Medicine
FDA guidance finally opens door for insulin biosimilars
New draft guidance from the FDA has opened the door for the development of insulin biosimilars, potentially allowing for cheaper near-identical versions of these biologic drugs to be interchanged with the originator.
The new draft guidance is good news for patients in the US looking for cheaper diabetes medications, and for the biosimilar manufacturers who under current rules can’t compete directly with the more expensive branded drugs.
But it’s bad news for companies like Sanofi and Novo Nordisk, who are already under pressure to cut prices of their branded insulins in the US amid wider scrutiny about prescription drug prices.
It also brings the US in line with the European Medicines Agency, which introduced a biosimilar approval pathway first and has already approved biosimilar insulins.
For example, the FDA did not recognise Eli Lilly/Boehringer Ingelheim’s version of Sanofi’s Lantus (insulin glargine) as a biosimilar, approving it instead as a “follow-on” product that was filed as a new drug.
However the same product was approved as a biosimilar in Europe, allowing it to be interchanged with the originator drug.
The new guidance has other provisions that will help biosimilar manufacturers producing insulins – for instance the FDA had previously required clinical studies evaluating any immunogenicity risks from all insulin-based products.
This would have extended to any insulin biolosimilars under previous thinking, but in this updated guidance the FDA has offered a work-around.
If a comparative analytical assessment based on state-of-the-art technology demonstrates that products are ‘highly similar’, the FDA reasoned that in most cases this would remove almost all uncertainty regarding immunogenicity.
Therefore a clinical immunogenicity study would be unnecessary in most situations, save where candidate products contain certain impurities or novel excipients, for example.
Under the new guidance the FDA recommends a filing for a biosimilar or interchangeable insulin product contains adequate chemistry, manufacturing, and control (CMC) information and a comprehensive and robust comparative analytical assessment between the insulin biosimilar and the reference biological.
Filings must also contain a comparative clinical pharmacology study and an immunogenicity assessment justifying why a comparative clinical study to assess immunogenicity is not necessary.
The FDA is consulting on the draft until 27 January 2020.
FDA OKs new tube system for child ear infections designed for doctor’s visit
The FDA has approved a new system that will make it easier for physicians to treat recurring, childhood ear infections during a visit to the doctor’s office by placing tubes in the eardrum using only a local anesthetic.
Developed by Tusker Medical, the system includes the company’s tympanostomy tubes, its painkiller Tymbion and separate devices used in the delivery of the treatment. The FDA described it as the first designed for young children in this care setting.
“This approval has the potential to expand patient access to a treatment that can be administered in a physician’s office with local anesthesia and minimal discomfort,” said the FDA’s device center director, Jeff Shuren, in an agency statement. “As millions of children suffer from ear infections every year, it is important to have safe and effective treatments available to this susceptible patient population.”
Also known as otitis media, inflammatory ear infections are common in children, with the NIH’s National Institute of Deafness and Other Communication Disorders estimating that five in every six children will have at least one ear infection before their third birthday.
If antibiotics fail in the face of repeated infections, small tubes can be used to drain and help prevent fluid and pressure from building up behind the eardrum. In young children, these tubes are typically placed at a hospital or surgery center under general anesthesia.
By comparison, Tusker Medical’s system uses a small electric current to deliver a local numbing agent directly into the eardrum, needle-free, just before a tube is inserted. The FDA approved the procedure for adults and children as young as six months old.
A clinical study of 222 pediatric patients showed the device was 86% successful in children under age five and demonstrated a rate of 89% between ages five and 12.
It is not intended for people with preexisting eardrum issues, such as a perforation. Dubbed the Tula System, for tubes under local anesthesia, the product previously received a breakthrough device designation from the FDA.
Baird: Biogen’s aducanumab likely is not getting approved
Baird analyst Brian Skorney has dismissed the chances of Biogen winning approval for its Alzheimer’s disease drug aducanumab. Skorney’s skeptical reading comes days before Biogen is due to discuss data from its phase 3 program at the Clinical Trials on Alzheimer’s Disease (CTAB) annual congress.
The presentation of the full data on aducanumab at CTAB gives Biogen an opportunity to show why it is pushing ahead with a filing for approval of the anti-amyloid beta antibody despite it failing a futility analysis in phase 3. However, with Biogen’s resurrection of aducanumab sending the stock up 30%, Skorney thinks there is also scope for the presentation to spook investors.
“We think it is underappreciated that CTAB could serve as a meaningful negative catalyst for [Biogen] shares, as we think the hype around [Alzheimer’s] will descend into a broader critique of the full data set and Biogen’s decision to file on it,” Skorney wrote in a note to investors.
If Skorney is right, that will only be the start of Biogen’s problems. The analyst expects criticism of Biogen’s decision to file for approval of aducanumab to exacerbate concerns about the prospect of Roche’s risdiplam winning market share from Spinraza and a legal decision leading Tecfidera to face generic competition in 2021, rather than 2028.
With competition from risdiplam, the Tecfidera intellectual property ruling and the FDA’s decision on whether to approve aducanumab all scheduled for 2020, there is the potential for Biogen to suffer a triple whammy of bad news. Skorney thinks at least one of those potential pieces of bad news is likely to come to pass.
“The preponderance of the data indicates aducanumab doesn’t provide a clinical benefit. If Biogen said based on this data it was running another study, the stock would be down because the data doesn’t justify investing in another study. The regulatory threshold is higher than that. The bottom line is, the FDA standard of approval is substantial evidence of efficacy and the cumulative data for aducanumab falls really far of this standard,” Skorney wrote.
Skorney downgraded Biogen to underperform and kept his price target at $250. Biogen currently trades at close to $300 a share.
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