Search This Blog
Tuesday, September 4, 2018
Tabula Rasa HealthCare acquires Mediture and eClusive
Tabula Rasa HealthCare announced it has acquired Mediture and eClusive, an electronic health record solution and provider of third party administrator services in the PACE market. Mediture and eClusive also service several managed long-term care organizations in the State of New York. Mediture and eClusive will become part of TRHC’s new CareVention HealthCare technology and service division.
https://thefly.com/landingPageNews.php?id=2785129
Catalyst Biosciences announces publication of hemophilia Phase 1 data
Catalyst Biosciences announced the peer-reviewed publication of previously reported data from the Phase 1 trial of marzeptacog alfa in individuals with hemophilia A or B with or without inhibitors in the Journal of Thrombosis and Haemostasis.The paper, entitled: “Phase 1, single-dose escalating study of marzeptacog alfa, a recombinant factor VIIa variant, in patients with severe hemophilia,” details the safety, tolerability, pharmacokinetics and pharmacodynamics of single ascending intravenous bolus doses of MarzAA. Subjects in the international Phase 1 open-label study were assigned to single dose MarzAA cohorts. MarzAA showed linear dose-response PK across the 4.5-30 mug/kg dose range, with a terminal half-life of 3.5 hours. Dose-dependent shortening of activated partial thromboplastin time and prothrombin time, and an increase in peak thrombin, determined with a thrombin generation assay, was also observed. MarzAA was well tolerated at all dose levels and was not associated with dose-limiting toxicity. No treatment-emergent severe or serious adverse events occurred.”MarzAA showed favorable pharmacological data in this first-in-human study and no potential safety concerns were identified. Together, these results supported further examination of MarzAA for the treatment of hemophilia A or B with inhibitors, particularly via subcutaneous administration,” said Ralph Gruppo, M.D., lead study author.
https://thefly.com/landingPageNews.php?id=2785147
Varian Medical installs cyclotron for ProBeam Compact proton therapy system
Varian announced the installation of the cyclotron for the Varian ProBeam Compact single-room proton therapy system at the cancer treatment center on the campus of the Delray Medical Center in Delray Beach, Florida. The cyclotron is a core piece of equipment of the ProBeam proton therapy system, and its installation is a key milestone for every new proton therapy center. Varian is partnering with Proton International for the installation, and the first patient treatments are expected to start in the fall of 2019. The Varian ProBeam Compact system is the only single-room system equipped with a 360-degree gantry for intensity modulated proton therapy. The ProBeam system also consists of a superconducting cyclotron, and high-speed pencil-beam scanning. The system’s integrated cone beam CT provides accurate patient positioning based on high quality anatomical images with excellent soft tissue resolution. In combination with the ARIA oncology information system and Eclipse treatment planning system, the ProBeam system enables an efficient adaptive workflow.
https://thefly.com/landingPageNews.php?id=2785179
U.S. Physical Therapy acquires interest in physical therapy practice
U.S. Physical Therapy announced that it has acquired a majority interest in a four clinic physical therapy practice. The practice produced $4.3M in revenue in 2017. U.S. Physical Therapy acquired a 70% interest for $7.7M. The remaining 30% partnership stake is being retained by existing management.
Baird: Is This a Golden Age for Biotech?
What Breakthrough Innovations and the Debate over Drug Prices Mean for the Sector
While medical advances are helping people battling serious illnesses live longer and experience a better quality of life, the biotech and pharmaceutical sectors are facing growing pressure to lower drug prices. Some widely publicized price rollbacks have spooked many investors, yet others remain bullish on both sectors. We sat down with Senior Research Analyst Brian Skorney, CFA, who covers biotech, to separate the headline risk from the long-term potential of investing in breakthrough medicines and discuss what the future might hold.
Q: Over the past few years, there have been significant new developments in the way we treat disease. Are we now in a golden age for innovation? And what’s fueling this innovation?
Brian: I would say that we are. I can think of dozens of diseases over the past 20 years where we’ve seen just amazing leaps in terms of clinical benefit. This is because we have a better understanding of human biology and the biology of various diseases. That’s made it easier to target disease, and we have better and better modalities for being able to hit targets.
Q: How is treatment different today?
Brian: Fifty years ago we had no insight into diseases like cancer. Most of the treatments were ubiquitous, but there is not one cancer. There are thousands of different cancers defined by site of origin, pathological features or genetic features. In addition, very few diseases affect large populations in consistent ways. So now we can treat 1,000 different cancers 1,000 different ways, knowing which cancers and which patients are more likely to respond to drugs with certain characteristics. That’s the shift. More and more drugs are being developed for smaller patient populations.HIV is another great example. In the early 1980s, it was a death sentence. Today, patients who have clear access to the range of HIV therapies, and are willing and able to take them on a consistent basis, are essentially living with a chronic disease that will never kill them.
Q: Despite its track record for innovation, the industry is still facing harsh criticism from the Trump administration over drug pricing. What’s been the overall impact of this negative attention?
Brian: It’s affected how investors think about the sector, and how sector executives think about drug pricing. But mostly it’s noise. Trump appointees, such as Alex Azar, the Secretary of Health and Human Services and a former drug company executive, and Scott Gottlieb, the Commissioner of the Food and Drug Administration, overall have been pharma friendly. The FDA has been more lenient in its drug approvals and is allowing them to get through faster.
Q: Are these pressures affecting pricing strategies?
Brian: Payers and drug makers are more cautious when discussing price. The pharmaceutical companies, certainly the large cap ones, are also getting savvier. They are more careful about how they position pricing discussions and how much credit to give the Trump administration for any pricing changes.
Q: Can you give us some examples?
Brian: Amgen recently received approval for a new migraine drug called Aimovig. It was priced substantially lower than the Street was expecting. Amgen has acknowledged they were aware of Trump administration critiques when they made their pricing decision. However, it’s hard to tell how much is an effort to soften criticism because at the same time they are trying to head off expected competition in the migraine space. It’s possible Amgen took a lower price to enable quicker market penetration.Merck has also said publicly that it’s not going to increase drug prices this year. In fact, they’ve cut a number of drug prices. They took a 60 percent cut on a drug called Zepatier to treat hepatitis C. The cut appears very significant, but newer regimens from Gilead Sciences and AbbVie have surpassed Zepatier’s usefulness. Merck attributed the price cut to the Trump administration, but the drug is a meaningless part of Merck’s profit and loss statement. If they wanted to reduce costs in the system, they’d cut prices for a really big product like Keytruda or Januvia.
Q: Why is it so difficult to determine what constitutes a fair return on a new drug?
Brian: A lot of characteristics go into determining a drug’s total value. You can spend 5, 10, 15, 20 years developing a drug and sinking increasingly larger amounts of money into development before turning a profit. And the vast majority of times, the drug fails somewhere in development making the whole thing an expense.That’s where U.S. patent law comes in. Companies that bring a new drug to market are given a monopoly on its sale for a period of time. The standard is somewhere from 10 to 20 years of commercial life for small molecule chemistry depending on when a company files a patent. That’s how long the commercial life of a drug will be before a generic version can be introduced. Once a generic drug is introduced, we tend to see a 30 percent reduction in sales per quarter, so a very rapid erosion of essentially all profits associated with that drug.But there isn’t a clear pathway to a real generic replacement for biologics, so companies have a lot of latitude. They’ll have one patent on the base composition of matter of a chemical, but then five years later they’ll file something for some minor change in that chemical. That enables them to extend the patent for five years, and then they keep filing additional patents every five to extend their monopoly.
Q: Will all the developments in biotech and pharma ultimately lead to more M&A?
Brian: There was a view at the beginning of the year that repatriation of capital would allow for more M&A. While that money has been repatriated, it really hasn’t been gone toward M&A. Takeda Pharmaceutical’s deal for Shire has been the biggest one this year except Takeda’s shareholders seem to hate it. The next biggest one is probably Norvartis’ acquisition of AveXis. That’s the kind of deal the market was hoping for, but otherwise M&A has not really seen any pick up this year.
Q: What’s the business case for more M&A?
Brian: Big pharma companies are great at selling drugs but bad at coming up with new ideas and R&D. Biotech companies are really good at R&D and coming up with new ideas, but really bad at selling drugs. Therefore, there’s constant pressure on pharmaceutical companies to buy biotech companies.
Q: Sounds like a win-win. So what’s the problem?
Brian: There are two problems. Most large pharma or biotech companies believe valuations for the small or midcap names are stretched. Rather than overpay, they are choosing to buy back their own stock and issue dividends. In addition, it’s hard to absorb another company. There’s a limit to how much M&A you can do in a year just from a structural point of view, even if you have the cash to do it. For example, Amgen has about $50 billion in cash. Yet they couldn’t handle buying five $10 billion companies. It’s too distracting to absorb that much personnel, facilities and technology at one time.
Q: What kind of breakthroughs can we expect in the years ahead?
Brian: Neurological disease is kind of the next oncology. We understand more of the underlying biology of various central nervous system diseases, making it easier to develop treatments for smaller and smaller groups. Rather than just target a broad classification like “dementia,” we’re learning more about individual patients’ genetic relationships and the biology causing those diseases. It’s a fantastic investment area.I’m also bullish on gene therapy. I cover Sarepta Therapeutics, and it looks like they have a gene therapy that is essentially normalizing patients with Duchenne Muscular Dystrophy.
Q: It’s certainly an exciting time in this space. But there’s also a lot of hype. How can investors find quality opportunities and avoid story stocks like Theranos?
Brian: I’m always suspicious when a management team says they have some concept that is completely groundbreaking and so novel it’s going to take over the world. To me, that’s a red flag. If you look at the drugs that have been successful, they are not great leaps forward. They wind up being leaps forward in terms of how well they function, but they’re not conceptually a leap forward.It’s also a good idea to look at what smart investors in the space are doing, or even more specifically, not doing. If a company can’t convince any dedicated healthcare shops to participate, it’s probably because there’s a problem. In the case of Theranos, there was a paucity of savvy life science investors involved. Plus, the board was composed of people who had no healthcare experience. To do well in this sector, you need to select great companies and technologies, and be wary of stories that sound too good to be true.
Brian Skorney, CFA, is Baird’s senior analyst covering Biotechnology. Prior to joining Baird in 2012, he was a senior vice president in equity research at Brean Murray Carrett & Co, a vice president in equity research at ThinkEquity, LLC, and a research analyst at Susquehanna International Group, LLC, all in the biotech/pharmaceutical industry. He also worked as a coordinator and lab manager at the Howard Hughes Medical Institute. In 2016, he was ranked by Thomson Reuters Analyst Awards (StarMine) as the No. 3 stock picker in Biotechnology. Brian received a BS in biological sciences from the University of Notre Dame and an MBA in finance from New York University.
Zogenix announces published data from seizure med study
Zogenix announced that detailed results of the Phase 2, open-label study evaluating its investigational drug, ZX008, for the treatment of refractory patients with Lennox-Gastaut Syndrome, or LGS, were published in the September issue of Epilepsia. Consistent with the previously-reported data from this study, the results demonstrated that ZX008 provided sustained, clinically meaningful seizure reduction in the majority of patients and was generally well-tolerated. The single-center, Phase 2, open-label dose-finding trial was a 20-week core study and a long-term extension option for those patients who were responders in the core study. Results presented in the paper included up to 15 months of treatment for patients in the long-term extension. Patients initiated treatment of ZX008 twice-daily. Patients who were responders remained at their effective dose, while non-responders were considered for a dose increase. Patients enrolled in the study had refractory LGS and a baseline median seizure frequency of 61 per month with multiple seizure types. Patients failed a median of five anti-epileptic treatments, including vagus nerve stimulation and ketogenic diet. Patients achieved a 53% median reduction in convulsive seizure frequency during the 20-week treatment period of the core study. A reduction in convulsive seizure frequency of at least 50% was seen in 62% of patients, with a reduction of at least 75% being reported in 23% of patients. The median dose of ZX008 was 0.4 mg/kg/day.
The FDA holds a public meeting on biosimilars
Public Hearing on the FDA’s approach to enhancing competition and innovation in the biological products marketplace, including by facilitating greater availability of biosimilar and interchangeable products will be held at the FDA Silver Spring, MD offices on September 4 at 9 am. Webcast Link: https://collaboration.fda.gov/biosimilarspart15
Subscribe to:
Posts (Atom)