$PFE / FDA Approves Pfizer's Leukemia Drug Date : 09/01/2017 @ 5:04PM Source : Dow Jones News Stock : Pfizer, Inc. (PFE) Quote : 33.96 0.04 (0.12%) @ 4:00PM FDA Approves Pfizer's Leukemia Drug
https://ih.advfn.com/p.php?pid=nmona&article=75566657 Pfizer Inc. is bringing back a cancer drug it pulled from the U.S. market over safety concerns in 2010 -- but the second iteration carries a higher cost, in the latest sign of the industry's pricing power.
Mylotarg, which treats a form of leukemia, will cost $24,600 per patient for an average course of treatment, Pfizer said Friday after the Food and Drug Administration approved the product's comeback. Pfizer says most patients will receive only one course. The new price is 58% higher than the $15,522 cost when the drug was pulled from the market in 2010.
Drugmakers have taken heat from politicians, patients and health insurers for charging high prices for new drugs and raising prices of older drugs in recent years. This week, the FDA approved a pioneering new Novartis AG leukemia treatment that costs $475,000 per patient, which some critics said was excessive but which the company defended as a responsible price in light of the treatment's benefits.
Pfizer says the new price for the resurrected Mylotarg doesn't look as steep once inflation is factored in. Accounting for average annual U.S. medical-price inflation of about 3%, the 2010 price would be about $19,128 in today's dollars, Pfizer said. Applying a higher rate of average drug-price inflation means the 2010 price would be about $30,252 today, Pfizer spokeswoman Sally Beatty said.
Ms. Beatty said the company arrived at the new price after consulting with doctors, insurers and patients to assess the drug's clinical and economic benefits. "Based on clinical data and real-world evidence, and support from oncologists, we believe Mylotarg has the potential to provide meaningful improvement to the majority of patients with AML," she said.
The FDA first approved Mylotarg in 2000 to treat patients age 60 and older with a type of acute myeloid leukemia, a particularly aggressive form of blood cancer. The American Cancer Society estimates there are about 21,380 new cases of AML annually in the U.S., and about 10,590 deaths from the disease.
After the drug's 2000 approval, additional patient studies failed to confirm its efficacy, and revealed an elevated risk of death of patients early in treatment. The FDA requested that Pfizer stop selling the drug in 2010.
After the withdrawal, independent researchers tested different doses and other ways to make Mylotarg safer and more effective, with Pfizer providing the drug for testing purposes, the company said. The results, which showed the drug improved certain measures of survival and induced remission in some patients, led to Pfizer's application to bring the drug back to market.
The FDA on Friday approved a lower dose than the first version of Mylotarg, a different dosing schedule and in a slightly different patient population that includes pediatric patients.
One study of the new Mylotarg showed that adding it to chemotherapy helped newly diagnosed AML patients live an average of 17.3 months without complications, relapse or death from start of treatment, versus 9.5 months for chemotherapy alone, the FDA said. Another study found that Mylotarg, given alone, prolonged median overall survival to 4.9 months versus 3.6 months for certain older treatments, the FDA said.
FDA Approves First Gene Therapy in U.S. -- Update Date : 08/30/2017 @ 12:50PM Source : Dow Jones News Stock : Gilead Sciences, Inc. (MM) (GILD) Quote : 83.75 0.04 (0.05%) @ 8:00PM FDA Approves First Gene Therapy in U.S. -- Update
The Food and Drug Administration has approved the first gene therapy in the U.S., a long-heralded move for a promising method to combat cancer.
The FDA said Wednesday it approved Novartis AG's Kymriah for certain children and young adults who suffer from a form of leukemia.
"We're entering a new frontier in medical innovation with the ability to reprogram a patient's own cells to attack a deadly cancer," FDA Commissioner Scott Gottlieb said.
Numerous pharmaceutical companies are investing in gene-therapy projects. This week, Gilead Sciences Inc. agreed to pay about $11 billion for Kite Pharma Inc., in an ambitious bet on the approach.
Kymriah is a type of personalized immunotherapy known as CAR-T, or chimeric antigen receptor T-cell therapy. T-cells, which are a type of white blood cell, are removed from an individual patient and sent to a manufacturing center where they are genetically programmed to target leukemia cells that have a specific marker. The cells are then infused back into the patient to kill cancer cells.
The specific form of cancer, acute lymphoblastic leukemia, impacts bone marrow and blood and is the most common childhood cancer in the U.S. There are about 3,100 patients ages 20 and younger diagnosed with the disease each year, according to the National Cancer Institute. Kymriah is approved for use in the most common form of the disease and is only for patients whose cancer hasn't responded to initial treatments.
While promising, CAR-T treatments won't be like other drugs that win FDA approval, which can quickly wind up on pharmacy shelves and hospitals. The rollout of this new breed of treatments is currently complicated by unresolved questions.
Manufacturing and delivery are more complex in CAR-T therapies than for a typical drug. In the U.S., only a few dozen specialized hospitals are currently qualified to provide CAR-T treatments, which require retrieving, processing and then returning immune cells to the patient. Novartis said it expects between 30 and 35 centers to be certified to offer the treatment by the end of the year.
Treatment with Kymriah also has the potential for severe side effects, including cytokine release syndrome, which leads to high fever, flu-like symptoms, and other neurological events. The FDA also approved expanded use of Roche Holding AG's Actemra, which treats cytokine release syndrome.
New Worry in 'Repo': Just One Bank for $3.5 Trillion Market Date : 08/30/2017 @ 12:40PM Source : Dow Jones News Stock : JP Morgan Chase & Co. (JPM) Quote : 91.7 0.81 (0.89%) @ 7:59PM New Worry in 'Repo': Just One Bank for $3.5 Trillion Market
https://ih.advfn.com/p.php?pid=nmona&article=75545445 When ED&F Man Capital Markets in June opened a settlement account for government bonds at Bank of New York Mellon Corp., it was a watershed moment in the world of "repos."
London-based ED&F became the first bond broker to change clearing banks in this obscure but vital corner of the global financial system in nearly a decade.
The move signaled that a market whose resistance to change has long vexed regulators is now shifting in a way that intensifies many traders' concerns about repo safety.
Bank of New York's onetime sole rival in the business of clearing U.S. Treasurys and repos backed by them, J.P. Morgan Chase & Co., is exiting the business, prompting more than two dozen brokers to move to Bank of New York.
Individual brokers' transitions have by all accounts been smooth. Yet many traders fret over the risks of having a single bank handle all clearing and settlement -- the process of completing trades and distributing funds according to contract -- in a short-term lending market estimated by the Treasury's Office of Financial Research at $3.5 trillion.
Many worry that having all those transactions handled by just one clearing bank potentially exposes the world's safest bond market to threats ranging from mundane power outages to cyberattacks and terrorism.
"This clearing function is only in one bank now and is so systemically important," said Scott Skyrm, head of repo at Wedbush Securities, a Los Angeles broker dealer.
In repos, or "repurchase agreements," lenders such as money-market funds make short-term loans to bond brokers, often using government bonds as collateral.
The market has been targeted by the Federal Reserve for reform for nearly a decade. Those efforts picked up after shortcomings in repos were exposed in 2008, when lenders' retreat from Bear Stearns Cos. and Lehman Brothers Holdings Inc. played a role in accelerating the financial crisis.
Troubles at those firms and others, driven in part by their exposure to subprime-lending losses and reliance on short-term loans to fund longer-term investments, helped pave the way for an updated repo market.
A decade ago, many financial firms funded themselves "wholesale" by borrowing in the market overnight. Today, repo borrowings tend to be longer-term and backed by stronger collateral, such as Treasury securities rather than privately issued mortgage bonds.
"When bad things happened in 2008, we saw that there were virtually no bids in the market for anything other than the risk-off sovereign market, " said Mark Robinson, a former managing director at Bank of New York and most recently a business development executive at fintech company Broadridge Financial Solutions.
Regulators have been trying on and off for years to resolve concerns about problems in repo spilling over to broader financial markets.
In April, Federal Reserve Bank of New York President William Dudley wrote that repo markets pose risks to market functioning and "are not settled yet," in part because participants can still choose to raise cash in a hurry by selling assets in a so-called "fire sale."
With J.P. Morgan's 2016 decision to retreat from clearing government securities, concerns about market stability began falling squarely on Bank of New York, which already controlled 85% of the market. The company this past May formed a new unit with a separate governance team to oversee the repo business, acknowledging its unique role and responsibility.
Bank of New York this summer began transitioning some clients of J.P. Morgan. Besides ED&F, it has also added as new clearing clients INTL FCStone and Landesbank Baden-Württemberg. In all, about 30 are expected to move.
"We were nervous at first," said Bruce Fields, group treasurer at INTL FCStone. But he said his fears have been allayed since his firm's conversion on July 10, which he said has provided access to a wider array of repo lenders than at J.P. Morgan.
Beyond Bank of New York, efforts to make repo safer continue. In May a unit of Depository Trust & Clearing Corp., a financial plumbing firm controlled by large banks, got permission from the Securities and Exchange Commission to expand a repo safety net already in use for bond brokers, covering institutional investors too. Some view this as potentially mitigating fire sales. Hedge fund firm Citadel LLC joined in June.
Trading volumes have shrunk, owing to new rules that have levied extra capital charges on banks. Other rules have meant more repos are locked in for longer terms, reducing the incentives for firms to borrow short term while lending long term and creating an unsound condition known as an asset-liability mismatch.
Perhaps most important, the hundreds of billions of dollars in intraday loans that J.P. Morgan and Bank of New York once made every morning to bond brokers have been reduced by 97%, according to Fed estimates, virtually eliminating the exposure the two clearing banks had precrisis to a broker default.
MannKind: Afrezza Is Blooming With Scientific Chatter Jun. 4, 2018 5:06 PM ET|Includes: MannKind Corporation (MNKD) Summary Catalysts and Capital.
Scientific Chatter Reprise.
The Plan.
Afrezza is the Scientific Chatter
Heretofore the clinical significance of Afrezza among endocrinologists and the diabetes community was largely unappreciated. As it may be, things have taken a turn for the better for MannKind and Afrezza—thanks to world-renowned diabetes expert and Chief Medical Officer for MannKind Corporation, Dr. David Kendall. Dr. David Kendall and his team of global diabetes experts have set out on a mission to not only see that Afrezza becomes the Standard of Care, but to be a solution for the global diabetes epidemic.
Afrezza was the, "shot heard around the world" after Dr. Kendall's scientific revelations with Afrezza. The preliminary data regarding the 65 Afrezza studies was so compelling that Dr. Kendall could only describe it as, "veins of gold". Which to me, simply means scientific data with colossal value.
The veins of gold within the 65 Afrezza studies also reflects a rather large body of previously unpublished clinical information. Dr. Kendall indicated in MannKind's recent Q1 2018 Earnings Call that he and the global diabetes experts, the MannKind Scientific Advisory Board, were closely and carefully examining this new and exciting scientific information as they march forward towards making Afrezza the Standard of Care.
Despite the persistent, wide-spread attempts to curtail Afrezza, MannKind science has prevailed. Soon the ancient, barbaric subcutaneous prandial insulin formulations will just become a ripple of the past.
Potential 2018 MannKind Non-Dilutive Sources of Capital and Catalysts
1. MannKind will receive a $2.2 million upfront payment from Cipla within 30 days of entering the agreement, with the potential to receive additional regulatory milestone payments, minimum purchase commitment revenue and royalties on Afrezza sales in India.
2. There have been almost 200 patients referrals to MannKind Care's since April 1 and many of these have not yet converted into prescriptions. (5/9/18)
—In 2018, approximately eight out 10 patients who are referred to MannKind Care get their prescriptions filled, so we're certainly going to watch this closely in the future but we wanted to share some early trending with you today.
3. The TreT, a Treprostinil Phase 1 single ascending dose study has started, and we are excited about that. As you may have seen last week, the United Therapeutics did a deal for $200 million for a pump. We believe this asset is a significant opportunity for us. We can put a Phase 1 readout that will be Phase 3-ready as soon as we get those results. We believe it's a three-player market with intense competition for differentiation. And we remain committed that Treprostinil will demonstrate that we believe this will show differences in potential dosing and convenience in this category.
—Readout Program on track to for this year ~ Q3 - Q4
4. TreP Phase 1 "SAD" Clinical Trial
—Estimated Primary Completion Date: September 2018
—Estimated Study Completion Date: December 2018
5. Levin (initiating mealtime Afrezza in uncontrolled T2D) Clinical Trial
—Estimated Primary Completion Date: September 15, 2018
—Estimated Study Completion Date: September 15, 2018
6. A-One (T2D) (Afrezza One Drop) Clinical Trial
—Estimated Primary Completion Date: September 30, 2018
7. Pediatric PK Study Part 3 is on track to start this year ~ Q3 - Q4
The Plan
I'm really going to be excited about the people, the culture and the foundation that we will be building at MannKind to ensure that Afrezza becomes the standard of care the meal-time insulin.
—Pat McCauley
So we are focused on continuing to make Afrezza a standard of care.
—Mike Castagna
Another question I often get is what's holding doctors back, why have they not used it. The reality is we've had 65 trials done in the history of the company to get to the point of approval in subsequent last couple of years. The majority of those trials were never published. And so when payers and database searches people look for PubMed to say what is the clinical data behind the product, they don't find a lot of our information. And what we've discovered is when we share our information with the doctors and why we believe the drug should be the standard of care, they're amazed at how data we have. They're amazed at the lung function data, and they really are a little bit surprised.
—Mike Castagna
One of the challenges we've had since we got the product back from Sanofi is really planning for the long-term. And with the capital raised last year and the recent capital raise, we've been able to continue to lay out our plan for the next 18 to 24 months, and execute against that plan. And you will see some of that in the upcoming slides. What that means is we can have evening symposiums, we can have CME events that we can sponsor, we can have booths at all the key conferences, and really [indiscernible] on that scientific chatter around the brand that's been missing since day of launch. So that will give the air coverage to the sales force. We'll continue to bring thought leaders onboard, and David will share the direction we're going in that environment.
—Mike Castagna
We are pleased to convene this board and will be working with them not only looking at the existing data from the 65 trials that have been completed, but looking very carefully and judiciously at any future trials that may be necessary, and most useful in assuring the safe, effective, timely, and appropriate use of Afrezza. An example of some of the outcomes of the work that was started, both before my arrival here at MannKind and our understanding of Afrezza, as a clinical aspect as Mike referred to earlier, is the scientific chatter.
—Dr. David Kendall
We have scientific information being presented at the ADA. We hope to have additional scientific information presented at each of these meetings, and many of the abstracts that have been developed by our medical team are currently under review, and we hope will be accepted for presentation at these upcoming meetings. So the scientific chatter is growing, and we believe making this scientific information available to clinicians to understand dosing, the effectiveness, safety and appropriate use can do nothing but support the commercial efforts that Pat and his team have put forth to better serve the diabetes population will require more effective meal time insulin therapy.
—Dr. David Kendall
We had previously announced the acceptance of both an oral presentation and a poster presentation from the so-called STAT study, a study that was first developed in collaboration with Satish Garg and colleagues at the Barbara Davis Diabetes Center looking at continuous glucose monitoring. And these results will be available in the poster sessions beginning on Saturday of the scientific sessions with an oral presentation being presented on Monday of the scientific sessions. I'm also very pleased to announce that a team here within our medical group took the opportunity to look at data performed from a trial in Type 1 diabetes and assess not just the rates of hypoglycemia, but adjusted total and severe hypoglycemia rates comparing Technosphere Insulin with Insulin Aspart. And these data will be presented as one of approximately 100 late-breaking poster presentations at the American Diabetes Association.
—Dr. David Kendall
Conclusion
For life repays what you put in.
—Al Mann
Sources:
Press Releases | MannKind Corporation
Webcasts | MannKind Corporation
Pharmacokinetics, Safety and Tolerability of Treprostinil Inhalation Powder in Healthy Normal Volunteers - Full Text View - ClinicalTrials.gov
Afrezza Safety and Pharmacokinetics Study in Pediatric Patients - Full Text View - ClinicalTrials.gov
Initiating Mealtime Ultra-Rapid Acting Insulin (Afrezza) in Uncontrolled Type 2 Diabetes Patients - Full Text View - ClinicalTrials.gov
A Randomized Controlled Trial Evaluating One Drop | Premium With Afrezza vs. One Drop | Premium Alone - Full Text View - ClinicalTrials.gov
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