You would think if it shows up on July 4 as an orphan drug for an antibacterial for systemic uses that somewhere prior to July 4, a drug company would have applied for an orphan drug status and had that drug validated in order to proceed further. At this point, the only company that we are aware of is Insmed. Is it possible that Insmed is thinking there are more hoops to jump thru for NTM and CF, and maybe a quicker way to market Arikayce is as an antibacterial with a different delivery system. Amikacin is already a proven drug. What is the difference if amikacin is delivered as a liposomal or the current method? and is it safer?
Who knows what company has filed, as I was under the impression that Insmed was not going to reapply until the P3 study was completed.
4 July 2016 EMA/462942/2016
Information Management Division Applications for new human medicines under evaluation by the Committee for Medicinal Products for Human Use July 2016
This document lists information on applications for centralised marketing authorisation for human medicines that the European Medicines Agency has received for evaluation. It includes the international non-proprietary names (INN) and therapeutic areas for all new innovative medicines under evaluation by the Committee for Medicinal Products for Human Use (CHMP). For generic and biosimilar medicines, it includes the INN (active moiety only, with no information on salt, ester or derivative) and therapeutic area. This list only includes information for medicines whose applications have been validated at the time the report was compiled. The information in this report was compiled on 4 July 2016. Information on designated orphan medicines that are being assessed for marketing authorisation is also available in the monthly reports of the Committee for Orphan Medicinal Products (COMP). Information in bold corresponds to new entries in the monthly list. Entries are removed from this list once the medicine has received a positive or negative opinion from the CHMP or when the applicant has withdrawn the application. The Agency publishes information on these opinions and withdrawn applications on its website. Information on CHMP opinions is also published in the monthly CHMP highlights.
Orphan medicinal products
International non-proprietary name (salt, ester, derivative, etc.) / Common Name Therapeutic area3
Aceneuramic acid Other medicines for disorders of the musculoskeletal system
Amikacin Antibacterials for systemic use
I just noticed on the EMA website under the CHMP heading that new orphan drugs had submitted applications as of July 4. I tried to post the information but was unable to do so. Amikacin was one of them. Not sure what this means, but the information said that this could include medicines that had been withdrawn, which Insmed did in June..
Sure Terry. I sold quite a bit at the top, and bought more when its lower. Dont know where its going, but its IP is worth more than where it is now. You are the phony on this board!!
If that was the case, kvnmcdd, and many others would have your postings deleted all day long. You are FOS, and cannot back it up with facts!!
There is no PROOF. and they know it. They are just spewing BS. If there are no facts it is either speculation or BS, and we know Terry is full of BS, and Cj thinks like Terry.
We have different viewpoints. It is already being used as part of a multi drug regiment, and it has better results via the liposomal delivery. The present treatments do not always "permanently" clear the bacteria. I would imagine that if you had NTM you might want the liposomal vs. the present system.
Makes you wonder if the CHMP committee members are sane. This is already an approved drug, just a different delivery system that appears more efficient in fighting the disease. Where's their compassion and common sense?
However, Piper Jaffray analyst Joshua Schimmer remains positive on the stock, reiterating an Overweight rating and price target of $24, which represents a potential upside of 111.5% from where the stock is currently trading.
Schimmer commented, “Though the company responded to the 180-day list of questions in Q1 and participated in an oral explanation meeting this quarter, the CHMP was not inclined to approve the product at this time. Similar to the FDA, the European regulators wish to see the results of the full Phase 3 212/CONVERT study, which will report in 2017. This is not a big surprise and does little to change the outlook for the company since success in the P3 trial likely would have been needed to stay on the market anyways. We had already pushed our modeled EU launch back to 2018 in January of this year recognizing the uncertain outlook for the CHMP decision.”
It was from the EMA website on what transpired at the CHMP meeting this week. My guess is that the CHMP committee members had a different viewpoint than the other co-rapporteurs or the PRAC committee. Also, Insmed probably feels snakebitten after EMA dangled the carrot. So much for compassionate use!
Too bad members of the CHMP committee can't experience what the people suffering from NTM have for a week, and see if they would have the same opinion. Makes me wonder how influential the large pharma are with these committees.
What did the company present to support its application?
The company submitted results of an early (phase 2) study in 89 adult patients with lung infections
caused by MAC or similar bacteria. This study compared Arikayce with placebo (a dummy treatment)
and looked mainly at how effectively the medicine cleared bacteria from patients’ sputum (phlegm)
after about 3 months of treatment.
How far into the evaluation was the application when it was withdrawn?
The application was withdrawn after the CHMP had evaluated the documentation provided by the
company and formulated lists of questions. After the CHMP had assessed the company’s responses to
the last round of questions, there were still some unresolved issues.
What was the recommendation of the CHMP at that time?
Based on the review of the data and the company’s response to the CHMP lists of questions, the CHMP
was of the provisional opinion that Arikayce could not be approved for the treatment of MAC lung
infection. The Committee’s main concern was that the study submitted in the application did not
provide enough evidence that Arikayce can permanently clear the bacteria from patients’ sputum.
Therefore, at the time of the withdrawal, the CHMP was of the opinion that the benefits of Arikayce did
not outweigh its risks.
What were the reasons given by the company for withdrawing the
In its letter notifying the Agency of the withdrawal, the company stated that its decision is based on
the understanding that CHMP was not going to recommend approval of the medicine on the basis of
current data. The withdrawal letter is available here.
What consequences does this withdrawal have for patients in clinical trials?
The company informed the CHMP that there are no consequences for patients currently included in
clinical trials for Arikayce. If you are in a clinical trial and need more information about your treatment,
contact the doctor who is treating you.
Historian, i read it as they were investigating the claim for a class action lawsuit, but they were not too specific as to the reason. I guess we will see if any of what's left of the retail shares think there might be a case.
Consider the following facts as you decide whom to elect at this year’s Annual Meeting:
Five of the seven current directors joined the Board in 2012 or earlier. During the period from December 31, 2012 to June 10, 2016, RiceBran’s common stock has experienced an absolute stock price decline of 85.4%, from $10.80 per share to $1.58 per share.
Revenue has remained stagnant for the past five years in the $40 million range.
From 2012 to 2015, the Company accumulated $55.8 million in net losses attributable to RiceBran shareholders. Despite these losses, aggregate total compensation of the Company’s Named Executive Officers was more than $7 million during such period. In addition, the total cash compensation of the Company’s non-employee directors was $249,708, $320,000, $441,783 and $427,750 in 2012, 2013, 2014 and 2015, as disclosed in the Company’s filings with the SEC, despite the Company’s net losses noted above.
Our Board nominees are highly-qualified director candidates with considerable credibility and decades of relevant business and financial experience. Several of our director candidates have extensive food and consumer products industry expertise. Our director candidates will bring fresh perspective into the boardroom and work to preserve and enhance the value of our investments. Moreover, Michael Goose – our CEO candidate – has over 13 years of operational and branding experience at the Hain Celestial Group, Inc., a leading publicly-held organic and natural products company with operations in North America, Europe and India and approximately $2.2 Billion in net sales during the last year of his tenure,1 and its affiliates. Mr. Goose has strong experience in building brands in the healthy food space.
Our goal is simply to restore good governance and effective strategy with a view to the Company’s long-term profitability and value creation. Contrary to the Board’s assertions, we are trying to gain representation on the Board to implement strategies and make changes for the sole purpose of creating shareholder value; we are not seeking to “acquire” the Company. The current members of the Board did not pay any “control premium” when they were elected.
The current board is entrenched and has a miserable track record of overseeing the destruction of shareholder value
Despite our sincere efforts, the current Board has rebuffed meaningful attempts for us to engage in a constructive dialogue. We believe the interests and attention of the current Board are not properly focused on the interests of the Company’s shareholders.
RiceBran’s directors have demonstrated inadequate action in response to current management’s weak strategic execution, ineffective financial stewardship and mediocre performance. For example, during the current Board’s tenure, the Company generated net losses attributable to RiceBran shareholders of $8,268,000, $23,029,000, $15,021,000 and $9,509,000 in 2015, 2014, 2013 and 2012, respectively, and annual revenues for 2015, 2014, 2013 and 2012 have stagnated at around the $40 million level.
What we believe to be Robert Schweitzer’s inadequate leadership and failure to guide the Board to help create shareholder value have shown that he is not performing successfully in the role of Chairman—the Company’s shareholders deserve a more effective Chairman.
The PR spokesperson said Insmed needed 261 out of the 300 recruited and enrolled, for the final numbers after the 6 months in the trial in order to analyze the data.