Its an issue of process under 2106. But the exon skipping proceeds by different processes - the materials (PMO) are different. All BMRN can do is litigate and delay, but if they do that, the patients and mothers will chain themselves to Biomarin's doors. You can't patent uranium fission and then claim rights over plutonium fission by claiming the idea of fission.
Step 1: Is the claim directed to one of the four patent-eligible subject matter categories: process, machine, manufacture, or composition of matter? The subject matter of the claim must be directed to one of the four subject matter categories. If it is not, the claim is not eligible for patent protection and should be rejected under 35 U.S.C. 101, for at least this reason. A summary of the four categories of invention, as they have been defined by the courts, are:
i. Process – an act, or a series of acts or steps. See Gottschalk v. Benson, 409 U.S. 63, 70, 175 USPQ 673, 676 (1972) ("A process is a mode of treatment of certain materials to produce a given result. It is an act, or a series of acts, performed upon the subject-matter to be transformed and reduced to a different state or thing." (emphasis added) (quoting Cochrane v. Deener, 94 U.S. 780, 788, 24 L. Ed. 139, 1877 Dec. Comm'r Pat. 242 (1876))); NTP, Inc. v. Research in Motion, Ltd., 418 F.3d 1282, 1316, 75 USPQ2d 1763, 1791 (Fed. Cir. 2005) ("[A] process is a series of acts." (quoting Minton v. Natl. #$%$’n. of Securities Dealers, 336 F.3d 1373, 336 F.3d 1373, 1378, 67 USPQ2d 1614, 1681 (Fed. Cir. 2003))). See also 35 U.S.C. 100(b); Bilski v. Kappos, 561 U.S. ___, 130 S. Ct. 3218, 95 USPQ2d 1001 (2010).
Responses to RFI for tamiflu and relenza were back in Feb
The United States Department of Homeland Security (DHS), Office of Health Affairs (OHA), requires antiviral medications for a highly pathogenic outbreak that could lead to a pandemic. DHS was appropriated funding in accordance with PL 109-148 for the preparation, planning and mitigation of a pandemic influenza. DHS has been closely monitoring the effectiveness of pharmaceutical interventions as a part of its overall pandemic preparedness plan. Additionally, DHS has been working closely with the Department of Health and Human Services (HHS) with regards to antivirals, and the pre-positioning of antivirals in the Strategic National Stockpile. DHS OHA has oversight and planning responsibility for all DHS components with regards to the DHS Antiviral Stockpile.
1. Use well specified protocols for tissue site selection (muscle loci), sample prep, freezing, etc. (like Sarzani)
2. Multiple locations for imaging, density/fiber counting.
3. Confirm IHC with Western Blot. (use complementaryl forms of chemical analysis)
4. Have pre-treatment/placebo control tissue samples to compare post treatment dystrophin levels v. negligible DMD
If you use good well-defined procedures, and you comparator tissue before treatment or placebo/delayed treatment, you should have "adequate" confidence, ie reliable results. If FDA asks for 10 digit precision, you're just fooling yourself and delaying availability of a therapy that could save a generation of DMD patients. Hell, 6MWT has about 10 factors, effort, location, initial strength, etc that can affect results. Dystrophin measurement should not require more precision
Thinking of stay at Hyatt Denver Tech Center, anybody else going. Waiting to see what 3/26 results are before deciding how to vote on this R/S proxy, and interested in seeing Mgt in action.
Responses to this RFI were due Feb 26, presumably BCRX was an interested supplier
The EID Tx program is seeking to develop an antiviral therapeutic medical countermeasure (MCM) effective against naturally occurring or biologically engineered Filoviridae virus. Specifically, EID Tx is interested in an antiviral therapeutic that addresses one or more Filoviridae (Ebola Zaire, Ebola Sudan, Marburg) with the potential of addressing additional Biological Warfare Agents (BWAs) beyond the Filoviridae indication targeting the following virus families: Arenaviridae (Lassa Fever, Junin), Bunyaviridae (Hantaan), Filoviridae (Tai Forest, Bundibugyo), Togaviridae (Eastern Equine Encephalitis, Western Equine Encephalitis, Venezuelan Equine Encephalitis).
Why Mar 20 is important: Understanding MOA, dystrophin, and measurement as surrogate endpoints
Hamburg testimony Mar 10
FDA believes that accelerating the development of reliable biomarkers is essential to advancing important new therapies. FDA already accepts the use of hundreds of biomarkers for a variety of purposes throughout drug development, such as proof-of-concept, diagnosis of disease, enrichment of trials with patients most likely to respond, and as surrogate endpoints that can support accelerated or traditional drug approval. For example, 45 percent of drugs were approved by FDA on the basis of a surrogate endpoint between 2010 and 2012. There remain, however, many diseases such as Alzheimer’s disease for which disease-specific biomarkers have not yet been developed, or shown to be reliable for use in the regulatory review process. When we do not understand the disease pathways, biomarkers appearing to be linked to disease progression can fail because they are not, in fact, in the causal pathway for the disease. A wide range of stakeholders is necessary to achieve meaningful progress in developing additional biomarkers that can be used by the scientific community. Important work is already underway through the National Institutes of Health (NIH), the Biomarkers Consortium in which FDA participates, and the Critical Path Institute.
The principal barrier to biomarker development is the lack of scientific understanding about the causes and biochemical pathways of many diseases
Investing in Domestic and International Public Health Preparedness. The health of people overseas directly affects America’s safety and prosperity, with far-reaching implications for economic security, trade, the stability of foreign governments, and the well-being of U.S. citizens abroad and at home. The Budget includes $975 million for domestic and international public health preparedness infrastructure, including an increase of $12 million for Global Health Security Agenda implementation to build the capacity for countries to detect and respond to potential disease outbreaks or public health emergencies and prevent the spread of disease across borders.
As new infectious diseases and public health threats emerge, HHS continues to invest in efforts to bolster the Nation’s preparedness against chemical, biological, nuclear, and radiological threats. This includes a $391 million increase for Project BioShield to support procurements and replenishments of new and existing countermeasures and to advance final stage development of new products, and to replace expiring countermeasures and maintain current preparedness levels in the Strategic National Stockpile. In addition, the Budget will support CDC’s critical infrastructure and cross-cutting research to facilitate rapid response to public health emergencies and other public health threats, such as the ongoing measles outbreak. Among other activities, CDC directs public health response efforts; detects sources of disease outbreaks; and develops tests to rapidly detect biological, chemical, and radiological agents.
Roth conf said HSV fully enrolled Dec 2014, 60 days of pre-vaccine shedding recording from swabs,
results by mid year 2015.
Objective: Reduce shedding 30%, reduce infection of others - using full rather than truncated genome and Vaxfectin adjuvant. Meanwhile market cap is only twice year end 2014 cash.
After listening to Plug presentation, their interest is in green hydrogen as a byproduct of Gevo biofuel production. Most hydrogen is from NG but customers interested in renewable sources.
Gevo could not be found on the list of presenting companies at the Roth agenda website, at least as far as I could find it. However, curiously enough, on the PLUG board there is a discussion of "Andy's" presentation which says
Gevo 21:50 mark, bio fuels for hydrogen production
IS this an indication of a possible takeout of Gevo by Plug - why else would they discuss Gevo? Go to Roth and listen to Plug webcast to check this info.
Why does Company Profile for Roth still say Biocryst has 2 late stage clinical programs peramivir and BCX4208 for gout. Nothing has been said about gout for months. Why is it still in theprofile?
That's why, although the workshop is supposedly about measurement, its an opportunity for a full explanation of the gene, the mutation skip causing the dystrophin shortage, and the role of repairing by supplying skipped exon to produce truncated dystrophin, as a means of moving toward Becker MD, a life prolonging, more manageable condition. It will reinforce the consensus of Muntoni, Mendell, McDonald, and Wilton that exon skipping which produces increased dystrophin of some form is a clinical benefit avoiding loss of muscle. So FDA will have a) scientific consensus about the therapy MOA and b) statistical confidence in measurement of the surrogate endpoint. (which patients and families already have, of course.)
Sole source procurement announcements were posted Jan 7 on FBO site,
HSHQDC-10-R-0015 for Oseltamivir
HSHQDC-10-R-0016 for Relenza
12 month buys plus 4 more years of options,
Contract shows amounts and cost redacted.
But now that DHS funding completed, further contracts can be implemented.
Webinar on dystrophin was informative, shows role of dystrophin well understood. Dystrophin essential for muscle maintenance, but mutation gaps prevent formation of adequate amounts. Exon skipping partially fixes, moving no Dystrophin toward Beckers, and without dystrophin production no drug works. March 20 FDA workshop while nominally about measurement is really about FDA understanding this in depth, ie if a drug is safe, and produces measurable increases toward normal dystrophin levels, it will have a positive clinical effect on muscles for walking, breathing, and so measurable dystrophin is an adequate surrogate endpoint for drug approval. Agenda due March 18 should be interesting. Boys who underwent. 4th biopsies will help get eteplirsen approved if results show measurable dystrophin increases. Hope FDA doesn't get lost in data precision when fundamental issue is - was dystrophin increased by treatment, whether its 45% or 52%.
Dept of Army RFI Feb 12 responses Feb 26 natural or engineered filovirus
A--Request for Information for antiviral therapeutic medical countermeasures against Filoviridae viruses.
Solicitation Number: W911QY-15-S-BDTX
1Q15 results: May 7, 2015
2Q15 results: August 6, 2015
next qtr will show rapivab sales
Who reports 4 primates, and a lab worker, may have been infected with melioidosis in accidental release from unknown source at Tulane Primate lab - tests underway to see if any other areas contaminated. Topic of drug in Soligenix pipeline.
Mendell also talked about eteplirsen at congressional briefing, McDonald knows natural history of progressive disease abnd Muntoni know complexity of dystrophin expression - more new info for sarepta coming this month w Feb 3 Webinar and Feb20 FDA workshop
March 3, 2015 from 1pm to 2pm ET
This webinar will review the basic biology of dystrophin (the gene, structure-function of the protein, its role in the dystrophin-glycoprotein complex, characteristics of different forms of dystrophin (both naturally occurring and drug-induced truncated forms), discuss how dystrophin is measured in both preclinical studies and clinical trials, and discuss the need for and role that dystrophin analysis play in clinical trials of novel agents designed to exert their therapeutic effects through an increase in dystrophin levels. We will also discuss what is required in order to have dystrophin considered as a surrogate