Reports Q1 revenue $117.8M, consensus $140.42M
Thursday, May 3, 2018
FDA moves Alnylam lumasiran into Phase 3 short track, sets up shot at OK in ’20
Alnylam $ALNY may be going solo on the development of rare disease drug lumasiran after Sanofi took a pass on its option, but the FDA has come through with a major assist. The company announced today that regulators have agreed on a Phase III study design that would accelerate — should all go well — its journey to approval.
For the trial designed to test lumasiran in the ultra-rare primary hyperoxaluria type 1 (PH1), the newly reached alignment allows Alnylam to adopt a primary endpoint based on a biomarker: the reduction of urinary oxalate at six months. The FDA has also sanctioned a study size of around 25 patients with PH1.
The use of a biomarker spotlights the FDA’s willingness to let drugmakers shoot for faster OKs, rather than forcing them to wait for data on disease pathology (Alnylam notes the biomarker is “directly linked to the pathophysiology of PH1 and known to be well correlated with disease progression”).
The trial design pact with the agency also marks an advance for the late-stage pipeline at Alnylam as it waits for an expected approval of patisiran, which will launch their first commercial operations in RNAi. This biotech has been through all the ups and downs often seen with any new approach to drug development. And it’s poised for some major changes.
With the study design hammered out, Alnylam expects to start the PhIII study in mid-2018, report topline results in 2019 and, if positive, submit an NDA in early 2020.
“We are very pleased with the FDA’s shared sense of urgency to evaluate the efficacy and safety of lumasiran as a potential therapeutic option for patients as rapidly as possible,” said Pritesh Gandhi, general manager of the lumasiran program, in a statement.
Alnylam is currently conducting a Phase I/II Part B study in 20 patients. Preliminary results, announced last year, suggest that lumasiran led to a mean maximal reduction in urinary oxalate of 66% in an unblinded group of four patients, and all of them achieved urinary oxalate levels at or near the normal range. Lumasiran, an RNAi therapeutic, is designed to “reduce the hepatic levels of the [glycolate oxidase] enzyme, thereby depleting the substrate necessary for oxalate production.” It was granted a breakthrough therapy designation in March.
Sarepta Duchenne med spurned by European regulators
Sarepta’s approval for eteplirsen — or Exondys51 — in the US remains one of the most controversial OKs in FDA history, earning a rare label that says the biotech has yet to produce evidence the drug works.
But lightning won’t strike twice on the regulatory pathway for this biotech.
The biotech reported Thursday afternoon that the CHMP is prepping a negative vote, barring the drug from the continent. And Sarepta shares immediately tumbled 5% in after-market trading.
The drug, which has a solid set of supporters in the DMD community, is getting snubbed despite the Europeans’ acceptance of PTC Therapeutics’ rival drug, which has failed repeated pivotal studies. But in DMD, regulators have a tendency to make things up as they go along, frequently bending the rules to accommodate a passionate group of patients and parents — or not. PTC has been repeatedly slapped down by the FDA, even after Janet Woodcock overruled a host of agency insiders with her insistence on approving eteplirsen.
Sarepta CEO Doug Ingram had this to say:
“Unfortunately, the CHMP’s trend vote was negative. Based on discussions with CHMP representatives, it is our understanding that the CHMP did not conclude that eteplirsen is ineffective for exon 51 amenable patients, but rather that Sarepta has not yet met the regulatory threshold for conditional approval, in part due to the use of external controls as comparators in the studies. Sarepta plans to file for re-examination and will request that a Scientific Advisory Group (SAG), which is made up of DMD and neuromuscular specialists, be convened to provide expert guidance and insight into, among other things, the validity of the external controls used and the importance of slowing pulmonary decline in patients with DMD.”
Hoping to take some of the sting out of the news, while disappointing some analysts with its Q1 performance, Sarepta also announced a deal to partner with Myonexus Therapeutics on its work developing gene therapies for Limb-girdle muscular dystrophies. Sarepta paid $60 million upfront and offered $45 million more in milestones for the deal, which also provides the biotech with a buyout option at proof-of-concept.
“Myonexus and its focus on gene therapy using the AAVrh.74 vector to treat forms of LGMD aligns brilliantly with our vision to emerge as one of the most meaningful global precision genetic medicine companies by focusing on the use of genetic medicine to improve the lives of those with rare fatal diseases,” said Ingram in a prepared statement.
Why DexCom Is Surging
Investors in DexCom (NASDAQ: DXCM) are having a pleasant start to the day. Shares of the diabetes-focused medical device company jumped as much as 14% in early morning trading on Thursday after the company reported strong first-quarter results. The stock was up about 8% as of 10:22 a.m. EDT.
Here’s a review of the key numbers from Dexcom’s first quarter:
- Revenue jumped 30% to $184.4 million. That was far higher than the $172.1 million in revenue that Wall Street had expected.
- Non-GAAP net loss was $28.3 million, or $0.32 per share. That was a penny better than what analysts had projected.
- Cash balance at quarter end was $534 million.
The strong start to the year caused management to raise its revenue guidance by $20 million for the full year. The company now expects revenue to land between $850 million to $860 million, which implies a growth rate of 19% at the midpoint.
Given the better-than-expected results and guidance boost, it is easy to understand why traders are feeling giddy.
Dexcom’s stock has now fully recovered from the drubbing that it took in late 2017 after the FDA approved Abbott‘s new FreeStyle Libre System. Anyone who was smart enoughto buy shares after the huge plunge has made a killing in a short period of time.
Can Dexom keep the momentum up? I’d argue that the answer is yes. The company just won FDA approval for its G6 system which boasts a number of improvements over its current system. In addition, the diabetes market is so huge that Dexcom should be able to continue growing quickly even if Abbott or Senseonics Holdings have successful product launches.
Sauna bathing reduces risk of stroke
Frequent sauna bathing is associated with a reduced risk of stroke, according to a new international study. In a 15-year follow-up study, people taking a sauna four to seven times a week were 61 per cent less likely to suffer a stroke than those taking a sauna once a week. This is the first prospective large-scale study on this topic, and the findings were reported in Neurology.
Stroke is one of the leading causes of disability worldwide, placing a heavy human and economic burden on societies. The reduced risk associated with sauna bathingwas found by a team of scientists from the Universities of Eastern Finland, Bristol, Leicester, Emory, Cambridge and Innsbruck.
The findings are based on the population-based Kuopio Ischaemic Heart Disease Risk Factor (KIHD) study and involved 1,628 men and women aged 53 to 74 years living in the eastern part of Finland. Based on their frequency of taking traditional Finnish sauna baths (relative humidity 10-20 per cent), the study participants were divided into three groups: those taking a sauna once a week, those taking a sauna two to three times a week, and those taking a sauna four to seven times a week.
The more frequently saunas were taken, the lower was the risk of stroke. Compared to people taking one sauna session per week, the risk was decreased by 14 per cent among those with two to three sessions and 61 per cent among those with four to seven sessions. The association persisted even when taking into account conventional stroke risk factors, such as age, sex, diabetes, body mass index, blood lipids, alcohol consumption, physical activity and socio-economic status. The strength of association was similar in men and women.
Lead researcher Dr Setor Kunutsor, from Bristol Medical School: Translational Health Sciences at the University of Bristol’s Musculoskeletal Research Unit, said “The findings are very significant and highlight the multiple health benefits of taking frequent sauna baths.”
Previous results from the KIHD study at the University of Eastern Finland have shown that frequent sauna bathing also significantly reduces the risk of cardiovascular and all-cause mortality. According to the researchers, mechanisms driving the association of sauna bathing with reduced stroke may include a reduction in blood pressure, stimulation of immune system, a positive impact on the autonomic nervous system, and an improved cardiovascular function. In a recent experimental study, the same group of scientists also showed that sauna bathing has acute effects on the stiffness of the arterial wall, hence influencing blood pressure and cardiac function parameters.
Kunutsor noted that the study is observational, and does not show a cause-and-effect relationship between sauna use and lower stroke risk. It only shows an association. A limitation of the study was that the study was based on traditional Finnish saunas and the results cannot be applied to other types of heat therapy such as infrared heat exposure, steam rooms and hot tubs. Kunutsor also said that since only a few people in the study never took saunas, the researchers could not compare people who used saunas to people who never used saunas.
Evidence suggests some people should not use saunas, including people who recently had a heart attack and those with unstable angina, or chest pain. Elderly people with low blood pressure should use caution when taking a sauna.
More information: ‘Sauna bathing reduces the risk of stroke in Finnish men and women: A prospective cohort study’ by Kunutsor, Setor; Khan, Hassan; Zaccardi, Francesco ; Laukkanen, Tanjaniina; Willeit, Peter; Laukkanen, Jari A. Neurology, 2018.
Cochlear implant recipients empowered through home care service
People with cochlear implants who receive postoperative care via a remote, telemedicine service trialled by the University of Southampton have shown a significant increase in their empowerment coupled with real improvements in their hearing tests after using the online care tools.
The University’s Auditory Implant Service, which designed, implemented and evaluated the long-term follow-up pathway for people with cochlear implants hopes that the results lead to an even more patient-centred and efficient service.
The 60 patients taking part in the randomised trial over a six-month period were either offered their usual appointment schedule, which requires visiting the University-based Service from wherever they live, or given access to the innovative online service. This gave them remote access to self-adjustment of devices and a personalised online intervention package for testing their own hearing with access to rehabilitation, troubleshooting and training at home.
“We found that only the remote care group had a significant increase in their cochlear implant empowerment after using the remote care tools,” said Project Lead Dr. Helen Cullington, Clinical Scientist at the University of Southampton Auditory Implant Service and lead author of a paper exploring the findings published in the journal BMJ Open.
“Quality of life remained stable in the two groups during the randomised trial,” Dr. Cullington continued. “The hearing test result in clinic, however, had improved in the remote care group; this may suggest that the remote care group were more able to take action to keep their hearing stable during the trial. Patients and clinicians were generally keen to continue remote care, with the most popular aspect being the home hearing test.
“We would like to spread these benefits to many more people using cochlear implants,” said Dr. Cullington. “At the University of Southampton Auditory Implant Service, we are working to make the cochlear implant care pathway person-centred and provide a more efficient service to allow quicker identification of problems. We believe that using a remote care person-centred model instead of the usual clinic-centred model will provide better long-term outcomes to people with cochlear implants: higher empowerment, and more stable hearing. We would like to move to a model where patients, clinicians and families decide together when an appointment is needed, rather than the clinic-centred routine appointment schedule.”
Around 1400 people receive a cochlear implant in the UK each year. Patients require lifetime annual follow-up at one of 18 specialist centres like the Service in Southampton which may be several hours away from their home.
All essential National Health Service (NHS) cochlear implant care is provided free of charge at the point of delivery. However, there are other costs associated with attending appointments, e.g. travel costs, lost income, childcare, accommodation and parking. Some deaf people may not feel confident to travel by public transport alone, so there may be additional costs for an accompanying person. In addition to financial costs, there are also time constraints, social inconvenience and psychological barriers associated with a journey to hospital.
Currently the cochlear implant centres provide lifelong care, following a schedule of appointments with little opportunity for personalisation; this may result in appointments occurring which do not provide benefit. Conversely, when some patients attend a routine appointment, an audiologist can diagnose that there has been hearing deterioration which the patient had not noticed. This is often remedied by replacing equipment that the patient could have done at home. Empowering the patient to self-care at home would enable more stable hearing and a more convenient and accessible service.
“There is much evidence to show that people who are activated and empowered – possessing the knowledge, skills and confidence to manage their condition effectively – have better health outcomes and care experiences,” Dr. Cullington concluded.
More information: Feasibility of personalised remote long-term follow-up of people with cochlear implants: a randomised controlled trial BMJ Open 2018;8:e019640. DOI: 10.1136/bmjopen-2017-019640
Allscripts (MDRX) to Buy HealthGrid
Allscripts (NASDAQ: MDRX), a global leader in healthcare technology, today announced that it signed a definitive agreement to acquire HealthGrid Holding Company, a mobile enterprise patient engagement platform business. The agreement represents a significant expansion of the Allscripts FollowMyHealth® platform portfolio, the company’s patient engagement solution focused on connecting patients with providers. Allscripts expects to close the acquisition in the second quarter of calendar 2018, subject to the satisfaction of customary closing conditions, including the expiration or termination of the waiting period under U.S. antitrust laws.
HealthGrid is a leading mobile, enterprise patient engagement solution that has helped independent providers, hospitals and health systems to dramatically improve patient interactions and satisfaction. The growing adoption of value-based care combined with the modest level of usage of patient portals across the healthcare industry (as low as 30% in hospital-owned practices, and 10-15% in physician-owned practices) has made it critical for health IT to bring an enhanced approach to patient-engagement solution design.
Upon closing of the strategic acquisition, Allscripts expects to tightly integrate the HealthGrid capabilities into its FollowMyHealth platform, adding functionality that would enable providers to reach 100% of their patient populations without requiring patients to sign up for a portal. Instead, the new functionality will leverage existing patients’ contact information. By continuing to grow usage of FollowMyHealth and helping connect providers with patients outside the portal as well, Allscripts will help providers greatly increase their outreach to patients and boost patients’ engagement in their own health.
This expected advancement of the FollowMyHealth platform further drives Allscripts strategy to offer the most comprehensive patient engagement solutions and enable providers to stay connected with their patients in pre-care, point-of-care and post-care settings, helping care teams to collaborate with patients in real time across the continuum. Driven by a complex rules engine based on clinical protocols, integrated functionality would enable intelligent outreach by providers to connect to patients at the precise, optimal time in the care process, keeping patients engaged, informed and supported throughout their care experience.
Additional, enhanced capabilities Allscripts plans to introduce to FollowMyHealth upon closing of the acquisition include appointment confirmation and waitlist notification functionality, post-discharge surveys, and more robust tools to assist providers in meeting critical regulatory requirements.
“Adding these significant capabilities to our portfolio, further growing our FollowMyHealth platform, will benefit our clients and their patients, as our robust offering will create opportunities to reach new heights of patient outreach and engagement,” said Allscripts Solutions Development Executive Vice President Jim Hewitt. “Additionally, it advances our EHR-agnostic approach. You don’t have to have an Allscripts EHR to make this work. Engaging consumers in their own health is critical to achieving a healthier tomorrow, and enabling meaningful connections between patients and providers is a crucial piece of fostering successful healthcare delivery. It’s a role Allscripts is proud to play—to improve the way patients engage with their own health and their clinicians is to fuel the progress of healthcare itself.”
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