A breakthrough therapy is a drug:
intended alone or in combination with one or more other drugs to treat a serious or life threatening disease or condition and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over existing therapies on one or more clinically significant endpoints, such as substantial treatment effects observed early in clinical development.
If a drug is designated as breakthrough therapy, FDA will expedite the development and review of such drug. All requests for breakthrough therapy designation will be reviewed within 60 days of receipt, and FDA will either grant or deny the request.
Just to review what we applied for:
A. Serious Condition
1. Whether a Condition Is Serious
FDA intends to interpret the term serious as it has done in the past for the purposes of accelerated approval and expanded access to investigational drugs for treatment use.6 A serious disease or condition is defined in the expanded access regulations as follows:
. . . a disease or condition associated with morbidity that has substantial impact on day-to-day functioning. Short-lived and self-limiting morbidity will usually not be sufficient, but the morbidity need not be irreversible if it is persistent or recurrent. Whether a disease or condition is serious is a matter of clinical judgment, based on its impact on such factors as survival, day-to-day functioning, or the likelihood that the disease, if left untreated, will progress from a less severe condition to a more serious one.
2. Whether the Drug Is Intended to Treat a Serious Condition
As referenced in section IV, the statutory and regulatory eligibility criteria for expedited programs require that a drug be intended to treat a serious condition. To satisfy this criterion, a drug must be intended to have an effect on a serious condition or a serious aspect of a condition, such as a direct effect on a serious manifestation or symptom of a condition or other intended effects, including the following:
• A diagnostic product intended to improve diagnosis or detection of a serious condition in a way that would lead to improved outcomes
• A product intended to mitigate or prevent a serious treatment-related side effect (e.g., serious infections in patients receiving immunosuppressive therapy)
• A product intended to avoid or diminish a serious adverse event associated with available therapy for a serious condition (e.g., product that is less cardiotoxic than available cancer therapy)8
• A product intended to prevent a serious condition or reduce the likelihood that the condition will progress to a more serious condition...
From "Diamonds and Dogs":
June 18, 2014
Insmed Incorporated (INSM)
The diamond for June 18th is the biopharmaceutical company, Insmed Incorporated. The company was granted the Breakthrough Therapy Designation, meaning the FDA is willing to expedite the development and review of the drug. Arikayce, the treatment drug for adults with nontuberculous mycobacterial (NTM) that received the grant, had positive Phase II clinical trial results. NTM is a disease in extreme need of treatment, which may be part of the reason why the FDA is looking to push the drug through to approval. Insmed's stock price soared 47% up to $17.50, which contrasted the downward movement of the stock price a few months prior to the grant. Now, the company has price targets ranging from $30 to $50, making this security one to watch.
Actually the PTs are $25 to $51 but who is counting.
B. Available Therapy
For purposes of this guidance, FDA generally considers available therapy (and the terms existing treatment and existing therapy) as a therapy that:
• Is approved or licensed in the United States for the same indication being considered for the new drug9 and
• Is relevant to current U.S. standard of care (SOC) for the indication
FDA’s available therapy determination generally focuses on treatment options that reflect the current SOC for the specific indication (including the disease stage) for which a product is being developed. In evaluating the current SOC, FDA considers recommendations by authoritative scientific bodies (e.g., National Comprehensive Cancer Network, American Academy of Neurology) based on clinical evidence and other reliable information that reflects current clinical practice. When a drug development program targets a subset of a broader disease population (e.g., a subset identified by a genetic mutation), the SOC for the broader population, if there is one, generally is considered available therapy for the subset, unless there is evidence that the SOC is less effective in the subset.
When determining whether a drug granted accelerated approval or approved with a risk evaluation and mitigation strategy (REMS) that includes elements to assure safe use (ETASU) under section 505-1 of the Federal Food, Drug, and Cosmetic Act (FD&C Act) (21 U.S.C. 355-1), is considered available therapy, the following principles will be applied:
• A drug would not be considered available therapy if the drug is granted accelerated approval based on a surrogate endpoint or an intermediate clinical endpoint and clinical benefit has not been verified by post approval studies.
• A drug would be considered available therapy if the drug is granted accelerated approval because of restricted distribution and the study population for the new drug under development is eligible to receive the approved drug under the restricted distribution program.
Mean Recommendation (this week): 1.7
Mean Recommendation (last week): 2.1
* (Strong Buy) 1.0 - 5.0 (Sell)
Price Target Summary
Mean Target: 32.83
Median Target: 30.00
High Target: 51.00
Low Target: 25.00
No. of Brokers: 6
Wedbush has been removed hmmmmmm
Piper Jaffray $51
UBS AG $31
HC Wainwright $30
JMP Secuirties $25
REHDVM - I am "hoping" you are not suggesting all current patients diagnosed would not warrant Arikayce once approved. I also suspect that you are "jumping" ahead and suggest (which I would agree) that Arikayce is the first line of therapy when new NTM patients are diagnosed.
If Arikayce is able to eliminate or minimize NTM effects in any patient, WHY would you not choose to do so?
The more serious patients are the best gauge to insure applicability and measure improvement (or not) but the newly diagnosed patients will become the best way to eradicate NTM altogether (optimistically speaking).
NASDAQ has been slaughtered these past few days and Insmed has been driven down with it. This never makes any sense to me as there is nothing substantiating the suppression.
"Sustained Improvement in Lung function"
SAN DIEGO -- A once-daily inhaled antibiotic appeared to offer long-term benefits in cystic fibrosis (CF) patients with Pseudomonas aeruginosa infection, researchers said here.
In an extension of a trial that compared investigational liposomal amikacin for inhalation (Arikace) with twice daily tobramycin, the positive gains in forced expiratory volume in 1 second (FEV1) for patients on the once-a-day antibiotic were still seen a year later, reported Diana Bilton, MD, of Imperial College London, and colleagues.
"In the patients who were on liposomal amikacin for 18 months -- 6 months in the original trial and 12 months in the extension -- we are seeing a sustained improvement in lung function," she said at the American Thoracic Society annual meeting.
In addition, patients being treated long-term with amikacin continued to show reductions in P. aeruginosa colonies, although the reduction appeared to lose some steam through the course of treatment.
"There was a half-log reduction after 6 months, and we see a gradual wane of effectiveness out to 1 year, but certainly no increase in colonies from baseline," Bilton said.
P. aeruginosa can cause chronic lung infection that can result in severe lung damage over time. In CF patients, the bacteria is traditionally treated with antibiotics administered through a nebulizer.
Terry - you ALWAYS try and find the negative spin here -
"Once a drug receives Fast Track designation, early and frequent communication between the FDA and a drug company is encouraged throughout the entire drug development and review process. The frequency of communication assures that questions and issues are resolved quickly, often leading to earlier drug approval and access by patients." and "Breakthrough Therapy is a new program at FDA that will complement these existing programs and facilitate and expedite drug development and review for serious conditions. FDA has been vigilant in assuring that reducing the time necessary for drug development has not compromised the safety and effectiveness of drugs for patients with serious conditions."
Arikace has been granted orphan drug status by the FDA and the EMA for the treatment of P. aeruginosa infections in CF patients, according to its maker, Insmed.
In the original trial (CLEAR-108), 129 patients were assigned to treatment with amikacin and 137 were assigned to treatment with tobramycin. The mean age of the patients was 21 and the participants were split fairly evenly by sex. The vast majority were white.
For the ongoing extension trial (CLEAR-110), 80 of the cystic fibrosis patients continued on amikacin and 88 switched from tobramycin to amikacin.
Eligible patients who completed CLEAR-108 were given liposomal amikacin for inhalation (590 mg) once daily via an investigational nebulizer system. Patients are scheduled to receive up to 12 cycles of treatment, comprising 28 days on-treatment followed by 28 days off-treatment.
Bilton noted that sputum density reductions were maintained through the 1-year extension without regard to the baseline medication, and that, in general, patients who switched from tobramycin to amikacin had similar results.
Serious adverse events were experienced by 28.2% of patients, usually due to hospitalization because of pulmonary exacerbations.
"We are not seeing anything in this report that is unexpected," commented Denis Hadjiliadis, MD, from the University of Pennsylvania Health System in Pennsylvania. "The main benefit that liposomal amikacin has when compared to tobramycin is that the liposomal formula can be administered once a day."
However, "where amikacin will fit in the treatment of CF is uncertain," he said. "I think it will depend on the cost of the drug and whether the convenience of once-daily treatment will outweigh increased costs. However, all these drugs are expensive, so I don't think that amikacin -- if it is approved by the FDA -- will be that much more expensive than tobramycin."
Nonetheless, clinicians would like to have a new drug in their CF treatment armamentarium, he said
AMSTERDAM, Netherlands, June 12, 2014 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leader in human gene therapy, today announced that Will Lewis has joined uniQure's Supervisory Board as of June 11th, 2014. Mr. Lewis has been serving as President and Chief Executive Officer and as a member of the Board of Directors of Insmed, a biopharmaceutical company specialized in inhalation therapies for orphan lung diseases, since 2012.
"Will has a tremendous track record in achieving success for companies much like ours," said Jörn Aldag, CEO of uniQure. "His exceptional experience in the field of orphan diseases as well as his broad knowledge of the US and European financial markets will be of high value to the company."
"uniQure is at the forefront of gene therapy, not only aiming at curative treatments for orphan diseases but seeking to have a broader impact on human health by developing therapies to treat diseases such as Parkinson's," commented Will Lewis, MBA/JD. "Having been involved in both the scientific and financial aspects of management in the past, I am committed to bring my expertise in both areas to uniQure."
Mr. Lewis has more than 20 years of executive experience in the pharmaceutical and finance industries both in the US and internationally. He is the former Co-Founder, President and Chief Financial Officer of Aegerion Pharmaceuticals, Inc. During his tenure at Aegerion, Mr. Lewis played a pivotal role in re-orienting the company's strategy to focus on orphan disease indications. Prior to Aegerion, Mr. Lewis spent 10 years working in the U.S. and Europe in investment banking for JP Morgan, Robertson Stephens and Wells Fargo. During his time in banking, he was involved in a broad range of domestic and international capital raises and advisory work valued at more than $20 billion.
Someone put down $100K in the pre=market (5 K at $19.28)so yes something is up!
Is it really $13.75 after everything (the facts) has been reported/revealed this week? The market is discounting the primary STILL? The PH 3 Cystic Fibrosis study (now 18 months) is ignored? NTM culture conversions (36% of the tougher or more seriously ill patients) is minimized? ETC. And when almost 100K shares traded earlier today the PPS barely moved? Something nefarious is truly going on here. Still miffed!!!
Working on the conservative side assume $50K x 50,000 and that is $2.5B and that is just NTM. Because they have the "market" cornered with their patents, there will be many more indications beyond NTM. They are looking at partnership(s) for Asia which may bring in some upfront cash. Within the next 2 years, we should see revenue coming in. Now, if JMP does not raise their 52 week PT to $30 or more then that would be a bit disappointing.
This could also explain the shelf offering (mainly form 4's) from last week. June has not been good to us here at Insmed....this year it changes??
One on Sunday and two on Tuesday. This is a very busy week (finally).
all for dropping the PPS by .01? - they kept raising the ask from 1K to 5K and 10K to keep this under $13.30 and then kept the pressure down from $13.25 until the close. As we have said many times, seems orchestrated and NASADQ was up over 51 pts.