Of note, we
have discussed in the past prior investigations into migraine and pain management. We still consider these areas attractive and ideally suited to our technology. At this time, we are reevaluating the clinical and commercial aspects of these potential programs.
We currently have three candidates in development; Treprostinil, Palonosetron and Epinephrine. The first is Treprostinil for pulmonary arterial hypertension, PAH.
PAH is a sizable market expected to reach $10 billion by 2020. Treprostinil, a prostacyclin vasodilator is currently available in several formulations. However, these often carry a significant treatment burden. This often leads to poor compliance and poor clinical outcomes.
Our Treprostinil program is in the early technical assessment phase.
Preclinical work is expected to begin in the second quarter of 2016 followed by clinical trials beginning in first quarter of 2017. We are
actively looking for a development partner for this program. Ideally, the partner would be companies that already are active in this space.
The second candidate I will speak about is Palonosetron for chemo-induced nausea and vomiting. The
CNIV market is forecasted to grow at 5.7% CAGR from 2013 to 2020 going from 1.2 billion to a market of 1.88 billion. Palonosetron is a 5-HT3 inhibitor indicated for the treatment of acute and delayed chemo-induced nausea and vomiting. Clinical data suggest that Palonosetron is superior from an efficacy and safety standpoint to any other 5-HT3 inhibitor with or without the addition of dexamethasone, when used in a moderately emetogenic regimen.
There are many advantages to the oral inhalation while it avoids the need for IV access or having to access the patient’s port, it can be used when oral agents are not practical, for example, with mucositis. It can be self-administered, for example, to prevent anticipatory nausea and vomiting. It has the potential to decrease the cost of care, nursing time, pharmacy costs, et cetera. And it can be used in multiday regimens.
Palonosetron is in the early technical assessment phase with
preclinical work expected to begin in the third quarter of 2016 followed by clinical trials beginning in second quarter of 2017. Again, we are
actively looking for a development partner for this program. Ideally, company or companies already active in the oncology supportive care space.
The last candidate I’d like to speak about is Epinephrine for the acute treatment of anaphylaxis.
In the U.S. alone, Epinephrine used in anaphylaxis represents a market over $1 billion. Epinephrine is used as a drug of choice with initial treatment of suspected anaphylactic reactions. Patients with known allergies are often asked to carry Epinephrine auto injectors. These drug device auto injectors tend to be large and inconvenient to carry around.
They also involve an invasive procedure that is to say an injection into the lateral thigh. This has led to episodes where patients have postponed this injection leading to an adverse clinical outcome. We believe that the oral inhalation route will provide more than adequate levels of Epinephrine. In addition, this noninvasive step has the potential to prevent untoward outcomes secondary to delaying treatment for fear of an injection.
Epinephrine is in the early technical assessment phase.
Preclinical work is expected to begin in the second quarter of this year followed by clinical trials beginning in the first quarter of 2017.
For those that may have questions whether an inhaled medication is suitable for use during the initial phase of an anaphylactic reaction,
patients typically know when they are having the reaction. This is well before the full physiologic effects of anaphylaxis become apparent. This is when they typically take an antihistamine, for example, Benadryl because they do not want to inject themselves thinking that the Benadryl will help. Well, it doesn’t. This product will now offer them a noninvasive option.
Clearly, there is a
very substantial U.S. and global market already for Epinephrine including millions of pediatric and young adult patients. The
market is dominated by one player [Mylan]. We will be
pursuing partners in which to penetrate this established market with a product that addresses a significant unmet medical need.
Mylan Confirms Availability of EpiPen® (epinephrine injection) Auto-Injector for U.S. Patients Affected by Sanofi's Auvi-Q® (epinephrine injection, USP) Recall
www.prnewswire.com/news-releases/mylan-confirms-availability-of-epipen-epinephrine-injection-auto-injector-for-us-patients-affected-by-sanofis-auvi-q-epinephrine-injection-usp-recall-300168925.html[
Imagine Mnkd partners with Mylan for inhaled Epinephrine to beat Sanofi]
HERTFORDSHIRE, England and PITTSBURGH, Oct. 29, 2015 /PRNewswire/ -- Mylan Specialty, a division of Mylan N.V. (Nasdaq: MYL) which markets and distributes EpiPen® (epinephrine injection) Auto-Injector, today commented on the voluntary nationwide recall of Sanofi's Auvi-Q® (epinephrine injection, USP) auto-injector.
Mylan confirms that EpiPen® and EpiPen Jr® (epinephrine injection) Auto-Injectors are not affected by the Auvi-Q® recall, and the company has sufficient supply of EpiPen® Auto-Injector to meet any anticipated demand.
Yesterday, Sanofi announced the recall of all Auvi-Q® auto-injectors currently on the market, including the 0.15 mg and 0.3 mg strengths for hospitals, retailers and consumers.
According to Sanofi, Auvi-Q® products have been found to potentially have inaccurate dosage delivery. If a patient experiencing a serious allergic reaction did not receive the intended dose, there could be significant health consequences. Given the life-threatening nature of anaphylaxis, those affected by the recall are encouraged to contact their healthcare provider as soon as possible to obtain a prescription for EpiPen® Auto-Injector. According to Sanofi, any out of pocket costs incurred for a new epinephrine auto-injector will be reimbursed. Additional information related to the Auvi-Q® recall can be found on Auvi-Q.com or by calling 1-866-726-6340 or emailing cs@sanofi.com.
"We are confident that the
widespread familiarity with EpiPen® Auto-Injector established over more than 25 years, coupled with our robust training resources, will provide access and support for those impacted by the Auvi-Q® recall. Anaphylaxis is unpredictable, and should an emergency occur, patient preparedness and immediate access to reliable treatment is critical. Mylan continues to be committed to help ensure access to epinephrine, the first-line treatment for anaphylaxis, and is working to provide a seamless transition for patients and their caregivers who may now be prescribed EpiPen® Auto-Injector," said Mylan CEO, Heather Bresch.
Training and support have been a primary focus since the launch of EpiPen® Auto-Injector. Epinephrine auto-injector devices look and function differently, so it is important that patients receive training on proper administration of a prescribed epinephrine auto-injector in a physician's office and practice between doctor office visits. EpiPen® Auto-Injector features include a flip-top carry case designed for single-handed opening and a never-see-needle design that offers needle protection before and after use. Each EpiPen 2-Pak® and EpiPen Jr 2-Pak® includes a needle-free, medication-free Trainer to practice administration technique.
Additional training resources available for patients and caregivers include:
A "How To" video that is available on epipen.com, our YouTube Channel and through the MyEpiPlan® App.
The Anaphylaxis: Know It. See It. Treat It. Training Video for school staff which is available on DVD or for download at EpiPen4Schools.com.
EpiPen® Trainers may be ordered on epipen.com.
Additionally, Mylan offers several patient programs and resources including the "$0 Co-Pay Offer" for eligible patients* and a patient assistance program for qualified individuals. Patients can download the co-pay card at epipen.com.
Indications
EpiPen® (epinephrine injection) 0.3 mg and EpiPen Jr® (epinephrine injection) 0.15 mg Auto-Injectors are for the emergency treatment of life-threatening allergic reactions (anaphylaxis) caused by allergens, exercise, or unknown triggers; and for people who are at increased risk for these reactions. EpiPen® and EpiPen Jr® are intended for immediate administration as emergency supportive therapy only. Seek immediate emergency medical help right away.
Pulmonary Arterial Hypertension (
PAH) Market to hit US$5.19 Bn by 2020
JANUARY 14TH, 2016 RELEASES
Pulmonary arterial hypertension (PAH) is a progressive disorder characterized by high blood pressure in pulmonary arteries that carry blood from the heart to the lungs. PAH is a very rare condition that occurs when pulmonary arteries become narrow in diameter or get blocked. Based on information gathered from various sources including WHO and CDC, the worldwide prevalence of PAH has been estimated to be between 100,000 – 200,000, approximately 15 – 50 cases per million people. Although, there is no cure for PAH, treatments are available to reduce symptoms, improve the quality of life, and slow down disease progression. Prostacyclin and prostacyclin analogs, endothelin receptor antagonists (ERAs), and phosphodiesterase-5 (PDE-5) inhibitors are the most-prescribed drug classes for the treatment of PAH. Recently, a new drug class, soluble guanylate cyclase (sGC) stimulators, has been identified to treat people suffering from PAH.
Get a Sample Report:
www.transparencymarketresearch.com/sample/sample.php?flag=S&rep_id=1629The recent approval of drugs and the anticipated approval of new PAH drugs in the coming years have been identified as the major growth drivers of the PAH market during the forecast period between 2014 and 2020. Opsumit (macitentan), Orenitram (treprostinil extended release tablet), and Adempas (riociguat) are the PAH drugs that received approval from various regulatory agencies across the world in the recent years (2013 and 2014). A new therapeutic agent Uptravi (selexipag) is anticipated to be introduced in the first half of 2016. In addition, governments of various countries providing incentives to companies engaged in the development and manufacturing of PAH drugs are also expected to contribute to the market growth.
On the other hand, the patent expiry of major PAH drugs such as Tracleer, Letairis, Adcirca, and Tyvaso is expected to affect the market revenue during the forecast period, as their generic versions would be sold at prices 70% to 80% lower than that of the branded ones. Development of breakthrough therapy for the treatment of PAH is expected to be a growth opportunity for drug manufacturers, as the currently available drugs are not enough to meet the medical needs of people suffering from PAH.
The global PAH market has been segmented into prostacyclin and prostacyclin analogs, ERAs, PDE-5 inhibitors, and sGC stimulators, based on the commercially-available drug classes, to treat PAH. Of these, ERAs represented the largest segment in 2013 with a revenue share of 55.8%. However, during the forecast period, the
prostacyclin and prostacyclin analogs segment is expected to record the highest growth, mainly due to the approval of Orenitram (treprostinil extended release tablet) in December 2013 and the anticipated approval of Uptravi (selexipag) in the first quarter of 2016. Adempas (riociguat) is the only approved sGC stimulator for the treatment of PAH. Adempas was first approved by the U.S. FDA in October 2013.
In terms of geography, the global pulmonary arterial hypertension (PAH) market has been segmented into four major regions: North America, Europe, Asia-Pacific, and Rest of the World (RoW). North America, with a share of 44.6%, represented the largest regional market for PAH in terms of revenue in 2013, followed by Europe. These two developed regions are expected to witness substantial growth during the forecast period, primarily due to the recently approved PAH drugs in the market and products that are anticipated to receive approval during the next few years. In the Asia Pacific region, Japan, China, India, Australia, and New Zealand exhibit immense opportunities for the companies operating in the PAH market. In RoW, the low level of awareness regarding the diagnosis and treatment of PAH is one of the prime reasons restraining the market growth. However, during the forecast period, growing awareness regarding PAH would drive growth of the market.
Actelion Pharmaceuticals, Ltd., Bayer HealthCare, Gilead Sciences, Inc., GlaxoSmithKline plc, Novartis International AG, Pfizer, Inc. and United Therapeutics Corporation are the key players operating
in the global PAH market.
Slide 11 of 2/3/16 CC:
Treprostinil
– Treprostinil is indicated for the treatment of pulmonary arterial
hypertension
– It is a
prostacyclin vasodilator– Currently available is several formulations
• However, these options carry a significant treatment burden
• This can lead to poor compliance and poor clinical outcomes
Actelion sees Uptravi price of $160,000-170,000/patient
www.reuters.com/article/us-actelion-pah-prices-idUSKBN0UJ1SQ20160105Tue Jan 5, 2016 12:30pm EST
Swiss drug company
Actelion expects an average annual per-patient price for its new pulmonary arterial hypertension medicine in the United States of $160,000 to $170,000, before rebates, Chief Operating Officer Otto Schwarz told analysts on Tuesday.
The drug, Uptravi, won approval from the U.S. Food and Drug Administration last month and went on sale this week, giving doctors another option to treat the deadly disease that kills victims within a few years.
Actelion declined to give forecasts for Uptravi's average price after rebates or say how many patients could be eligible annually for the treatment to open pulmonary arteries, easing pressure on the heart.
However, Schwarz said Uptravi will likely compete for treating a group of intermediate-stage disease sufferers who number around 3,800 people in the United States and now get inhaled treatments.
"There is an influx of new patients in this segment," Schwarz said, predicting
physicians will prescribe Uptravi for them before progressing to an inhaled treatment.
Analysts have said
Uptravi could reap more than $1 billion in global sales annually at its peak.
Even at $160,000 to $170,000 Schwarz said Uptravi is priced more cheaply than other inhalable pulmonary arterial hypertension treatments including United Therapeutics's Tyvaso and, consequently, unlikely to spawn significant payer resistance.
"Uptravi is priced - and this is why I think it is responsible pricing - below all inhaled prostacyclin therapies currently on the market," Schwarz said.
Chief Executive Officer Jean-Paul Clozel, on the same call, said the
addition of Uptravi to Actelion's stable of drugs following the 2015 patent expiry of its blockbuster Tracleer drug will be sufficient to fund expansion for the next decade, including into new treatment areas.
"The revenues generated will enable us to fuel our research and development efforts outside the pulmonary arterial hypertension franchise and to transform our company," he said.
Clozell is counting on internal growth after being beaten out last year by AstraZeneca on the purchase of U.S biotech company ZS Pharma for $2.7 billion.
Actelion shares last traded up 1.68 percent at 139 francs, giving it a market value of 15.6 billion Swiss francs ($15.42 billion), about a quarter of the value of AstraZeneca's stock.
Orenitram® (treprostinil) Extended-Release Tablets [Mnkd could partner with United Therapeutics for inhaled Treprostinil + PDE5 Inhibitor PAH]Drugmaker Names Pill After CEO Who Sought Daughter's Cure
www.npr.org/sections/health-shots/2013/12/24/256839330/orenitram-united-therapeutics-names-pill-after-ceo-who-sought-daughters-cureUpdated December 24, 20134:25 PM ET
It's not every day that the Food and Drug Administration approves a drug three months ahead of schedule. Or approves a pill that could take the place of injections. Or gives the OK to a medicine named for the CEO who started a company to help her sick daughter.
But all those things just happened.
United Therapeutics' Orenitram got the FDA's approval as a treatment for pulmonary arterial hypertension. The life-threatening illness is caused by constriction of the arteries that carry blood from the heart to the lungs. It's marked by fatigue, shortness of breath, chest pain and heart failure.
Drugs that relax the arteries can help. But a core treatment used to be available only by injection.
"When I started the company
17 years ago, the only option for treating pulmonary hypertension was intravenous prostacyclin," Martine Rothblatt, CEO of United Therapeutics told investors in a celebratory conference call Monday. "My young daughter looked at me and said, 'Can't there be a pill?' "
Now there is: Orenitram. The name comes from "Martine Ro," as in Martine Rothblatt, backward.
"What it has resulted in is something that as the parent of a lovely young lady with pulmonary hypertension, I cannot even put into words, the emotional meaning that it has to have a
pill form of prostacyclin," Rothblatt said.
A little later in the call, she tried. "When something takes a long time to bake, be it a turkey or a cake, the bad thing is that you're hungry and you're starving for a long time," Rothblatt said. "But the good thing is you usually have an awesome meal at the end. It's a very, very similar situation with Orenitram. This baby has taken a long time to bake — no doubt about it."
There are many drugs available for pulmonary hypertension, including pills containing the same active ingredients as Viagra and Cialis. But Orenitram marks a first in the U.S. — a prostacyclin that can be taken by mouth, the company said.
Orenitram won't be available for sale for about six months, the company said, and its exact price hasn't been determined either. But it will be
very expensive, about $150,000 a year, according to an estimate by Dr. Mark Schoenebaum, an industry analyst with ISI.
Insurers generally pay for pulmonary hypertension drugs, though there were questions from analysts about whether health plans would pay for Orenitram if it's used in combination with other drugs.
The FDA approved Orenitram on the basis of a study that gauged its safety and effectiveness as a standalone treatment. The drug helped people walk a little farther in a timed walking test. The most common side effects seen in the study were headache, nausea and diarrhea.
Rothblatt acknowledged that the cost of the drugs is high but manageable, because pulmonary hypertension remains pretty rare, though the exact prevalence is hard to pin down.
