Wednesday, January 9, 2019
Pfizer shutting down two manufacturing plants in India
Pfizer is shuttering two manufacturing plants in India that make generic injectables like penicillin as a response to declining demand, Reuters reports. The factories employ nearly 6% of the company’s global workforce. In an emailed statement, Pfizer said “Pfizer has conducted a thorough evaluation of the … sites in India and concluded that due to the very significant long term loss of product demand, manufacturing at these sites is not viable.”
https://thefly.com/landingPageNews.php?id=2846319
Protagonist Initiates Phase 2 Trial for Treatment of Beta Thalassemia
Protagonist Therapeutics, Inc. (Nasdaq:PTGX) today announced that the first patient has been dosed in the Phase 2 study of PTG-300, an injectable hepcidin mimetic peptide in development for the treatment of patients with beta thalassemia, a rare disease characterized by chronic anemia and iron overload. The study is designed to evaluate the safety and preliminary efficacy of PTG-300 in patients with transfusion-dependent or non-transfusion-dependent beta thalassemia. In a completed Phase 1 study in healthy volunteers, administration of PTG-300 was well tolerated and demonstrated a dose-related and sustained reduction in serum iron levels.
“We are encouraged to move forward with clinical development of PTG-300 in patients with beta thalassemia,” commented Dinesh V. Patel, Ph.D., Protagonist President and Chief Executive Officer. “In addition to beta thalassemia, PTG-300 has broad potential in the treatment of other disorders, including hereditary hemochromatosis and the myeloproliferative neoplasms polycythemia vera and myelodysplastic syndrome. The Phase 2 trial incorporates an open label trial design and we expect to report initial results in the second half of 2019. We are actively evaluating additional disease indications for development of PTG-300 and plan to commence on a second indication in the second half of 2019.”
The global Phase 2 study is a single-arm, open label, multiple-ascending dose design which will evaluate safety, proof-of-concept and dose finding in approximately 84 adolescent and adult patients with anemia associated with non-transfusion-dependent or transfusion-dependent beta thalassemia. Non-transfusion-dependent patients will receive 12 weeks treatment with PTG-300 in escalating dose cohorts. The primary efficacy endpoint in non-transfusion-dependent patients will be change in hemoglobin from baseline. Transfusion-dependent patients will receive 16 weeks treatment with PTG-300 in escalating dose cohorts. The primary efficacy endpoint in transfusion-dependent patients will be a change in transfusion burden from baseline.
Likely Approvals in View, Bluebird Offers up Pricing Strategy for Gene Therapy
For a moment, simply contemplate the possible price: $2.1 million for a drug.
It’s not just any drug, however. Cambridge, Mass.-based bluebird bio’s LentiGlobin is a gene therapy being evaluated for transfusion-dependent ß-thalassemia (TDT). TDT is an inherited blood disorder caused by a mutation in the ß-globin gene. This causes ineffective red blood cell production, which leads to severe anemia. Patients with TDT require regular transfusions to maintain hemoglobin (Hb) to stay alive. Unfortunately, chronic transfusions create the risk of iron overload, which can cause multiple organ damage and shortened life expectancy.
On December 3, 2018, the company released new data from the Phase III Northstar-2 and Northstar-3 clinical trials of Lentiglobin in TDT. In both trials, LentiGlobin allowed patients to stop chronic blood transfusions.
So with the gene therapy headed for likely regulatory approval, bluebird bio has turned some of its resources to developing a price for the therapy. The price floated so far is up to a staggering $2.1 million, an amount that will probably be lower, but what the company’s chief executive officer Nick Leschly told The Wall Street Journal reflected the drug’s “intrinsic value,” including improvements in quality and length of life. “Whether it’s just below or considerably below [$2.1 million] is something we need to sort through.”
It is possible the therapy will be approved in Europe this year and in the U.S. by 2020. About 300,000 people worldwide have TDT, with only about 10,000 in the U.S.
There’s no doubt the $2.1 million is staggeringly high. Gene therapies, in general, are coming with sky-high prices. Spark Therapeutics’ Luxturna for a hereditary eye disease is running about $850,000—per eye! Novartis’ cancer cell therapy, Kymriah, runs $475,000 and Gilead’s competitive drug, Yescarta, is priced at $373,000.
Bluebird’s proposal, which was outlined Tuesday at the JP Morgan Healthcare Conference in San Francisco, is a payment plan spread out over five years. It wouldn’t be the first company to consider that kind of plan, although it would be one of the first to adopt it if it does. Other companies have taken on plans where the pricey drugs are paid for if they work.
Spark, for example, offers rebates to some insurers, such as Harvard Pilgrim Health Care in Wellesley, mass., if the treatment doesn’t work. Spark has also proposed a payment-plan scheme to the U.S. Centers for Medicare and Medicaid Services (CMS).
Steve Miller, chief clinical officer at Cigna Corp., told The Wall Street Journal, “With gene therapy, you’re going to have drugs routinely priced at over $1 million, and it’s really going to push all of us to think in a different way.”
As described, the payment plan would run for five years, with each annual payment from health plans to bluebird bio being contingent on the gene therapy’s continued effectiveness.
Leschly, who was introduced by JP Morgan analyst Cory Kasimov as the “chief bluebird,” at the healthcare conference, said, “Our goal is to recode for life. It’s a big statement, a big dream, a big vision. And one we take very seriously.”
The company hopes to have four products approved by 2022 with a deep pipeline behind it. But on the topic of LentiGlobin pricing, Leschly suggested recoding for the how they bring things to market was also part of the plan.
“How do we deliver this to patients across a very complex supply chain in Europe and the U.S., and eventually, globally?” he noted, describing the build-out of his patient treatment centers and viral and therapy manufacturing facilities, with long-term goals in mind.
“It also includes the payers, the plumbing—how do we make sure everyone gets paid in the process to make sure there’s a fluid system,” Leschly said. The emphasis is clearly on paying attention to value instead of price, but with awareness, no one is going to be fooled, the therapy is likely to be expensive, but the goal is to get the treatment to the patients who need it. And if all goes according to plan, the treatment will cure the patients for life.
Leschly emphasizes it has to be affordable for the system as well as for patients. He noted four buckets.
First, he said, “Fair value. We’re going to look at this over lifetime effectiveness.” Secondly, “Real value, actual value to the patient and to the system. That’s quality of life and life extension.”
Third, he went on, “We’re going to share risk. We pay only full boat if it works. That means we’re suggesting putting up 80 percent of our price at risk based on success.” That means five years.
Leschly noted they were committing to no price increases above CPI. “It’s an unusual statement to say when you don’t even have a price. But we’re committing to it because we think it’s important. And an acknowledgment that we’re sharing risk but sharing value with the totality of the stakeholders involved.”
And finally, Leschly and bluebird bio notes, “no cost after the payment period.” This ties into health system affordability. “Don’t think about the price, think about the totality over time and how you consider that as you think about the value of your treatments.”
Tilray initiated at Piper Jaffray
Tilray initiated with an Overweight at Piper Jaffray. Piper Jaffray analyst Michael Lavery initiated coverage of Tilray with an Overweight rating and $90 price target. The analyst expects “strong” industry growth long-term, and believes Tilray is well positioned to be one of several likely winners given its relationships in medical, in the U.S., and in beverages. Lavery consider the company’s initial public offering lock-up expiry to be a near-term risk, especially with 77% insider ownership, but his 12-month Overweight view is based on its fundamental outlook. He pegs Tilray’s market opportunity at $15B-$50B in the near-term, and potentially $250B-$500B over the longer term.
Takeda looks to divest $10B of assets after Shire deal
Takeda (TKPYY) is seeking to divest $10B of assets after completing a GBP46B deal to buy Shire (SHPG), The Financial Times reports. CEO Christophe Weber says Nycomed is among the company’s “non-core assets” that it could consider selling. Looking ahead, Takeda will focus on five core areas: oncology, gastrointestinal conditions, neuroscience, rare diseases and plasma-derived therapies
Phone companies sell customers’ data in real time, Motherboard reports
A Motherboard investigation found U.S. phone companies T-Mobile (TMUS), Sprint (S) and AT&T (T) sell customers’ data in real time, allowing bad actors to potentially track their location to within a few hundred meters. The investigation found data sold to firms like credit checkers can end up on the black market.
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