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2007 Rare Diseases ORPHAN DRUGS IN DEVELOPMENT FOR Report PRESENTED BY AMERICA ' SBIOPHARMACEUTICAL COMPANIES , GENETIC ALLIANCE , AND NATIONAL ORGANIZATION FOR RARE DISEASES More Than 300 Medicines Are in Development to Treat or Prevent Rare Diseases E very patient faces unique health challenges. And, for millions of Americans who have been diagnosed with a rare disease—defined as a condition affecting fewer than 200,000 patients in the United States—treatment options can be limited. But over the last few years, great progress has been made with the approval of several new medicines for rare diseases such as Pompe disease, myelodysplastic syndromes, enzyme deficiencies, and rare cancers. America's biopharmaceutical research companies are continuing that progress with 303 medicines currently in human clinical trials or awaiting approval by the U.S. Food and Drug Administration (FDA) for many rare diseases. This compares to 133 medicines in development in 1989 and 189 in 1992. Since 1995, more than 160 medicines were approved to treat rare diseases, compared to 108 in the decade before and fewer than 10 in the 1970s. Advances in science, such as a better understanding of molecular and genetic causes of disease, have given researchers new tools to explore rare diseases, which are often more complex than more common diseases. In addition, the Orphan Drug Act of 1983 provided tax relief and some marketing exclusivity for companies that develop an orphan drug. That legislation is credited with the explosion in drug approvals for rare diseases after 1983. Under the Orphan Drug Act, 1,679 medicines have been designated orphan drugs (not all are approved) as of January 10, 2007. TheNational Institutes of Health estimates there are 6,000 rare diseases affecting 25 million Americans. A major area of research in rare diseases is cancer. Rare cancers, such as solid tumors of the liver and thyroid, cancer of the blood, and melanoma account for more than one-third of all rare disease research, with 139 medicines in development. Other important areas of research include: neurologic disorders, such as multiple sclerosis and muscular dystrophy, with 35 medicines in development; infectious diseases, such as anthrax and West Nile virus, with 28 medicines in development; and genetic disorders, such as cystic fibrosis, with 26 medicines in development. Some examples of medicines in development for rare diseases include: •Amonoclonal antibody for chronic sarcoidosis, an immune system disorder. •Amedicine for Lennox-Gastaut syndrome, a severe form of epilepsy. •Gene therapy for cystic fibrosis. •Amedicine for epidermolysis bullosa, a group of inherited disorders where skin blisters develop in response to minor trauma. •Amedicine for Friedreich's ataxia, a genetic disorder. Biopharmaceutical research is entering an exciting new era with our growing understanding of the genome and powerful scientific research tools. Experts predict the number of orphan drugs will rise in the coming years as more new medicines are developed that target specific genetic disorders. The 303 medicines in this report represent the enormous commitment by America's research companies to find treatments, and perhaps cures, for many of the 6,000 known rare diseases. Without question, the Orphan Drug Act of 1983 has worked exceedingly well in achieving the important purpose for which it was enacted—to provide incentives that would encourage orphan drug research and help the 25 million Americans afflicted with a rare disease. Sincerely, Billy Tauzin President and CEO PhRMA ORPHAN DRUGS IN DEVELOPMENT * Neurological Disorders Infectious Diseases Genetic Disorders Cardiovascular Diseases Cancer Blood Disorders3 Autoimmune Disorders 16 81 Cancer, Blood 42 Cancer, Skin 19 Cancer-Related Conditions 9 7 26 28 Transplantation Respiratory Disorders 35 13 Other 7 37 *Some medicines are listed in more than one category.
Hemoxin ™ Xechem International treatment of sickle cell disease Phase II New Brunswick, NJ (732) 247-3300[/B]ICA 17043 ICAgen treatment of sickle cell disease Phase III bis (4-fluorophenyl) Durham, NC (919) 941-5206 phenylacetamide idebenone (INN) Santhera Pharmaceuticalstreatment of cardiomyopathy Phase II Liestal, Switzerland associated with Friedreich's ataxia www.santhera.com -------------------------------------------------------------------------------------- treatment of Leber's hereditary Phase II optic neuropathy www.santhera.com INS 316 Inspire Pharmaceuticals treatment of cystic fibrosis Phase II Durham, NC (see also respiratory) (919) 941-9777 isofagomine Amicus Therapeutics treatment of Gaucher disease Phase I tartrate (AT-2101) Cranbury, NJ (609) 662-2000 Liazal® Barrier Therapeutics treatment of congenital Phase II liarozole Princeton, NJ ichthyosis (609) 945-1200 mannitol Pharmaxis to facilitate clearance of mucus in Phase III Frenchs Forest, Australia patients with bronchiectasis and www.pharmaxis.com in patients with cystic fibrosis at risk for bronchiectasis methylbicyclone Sucampo Pharmaceuticalstreatment of cystic fibrosis Phase II (SPI-8811) Bethesda, MD (301) 961-3400 oxalobacter OxThera treatment of primary hyperoxaluria Phase I formigenes (OC3) Alachua, FL (386) 418-1428 PTC 124 PTC Therapeutics for use in the treatment of cystic Phase II South Plainfield, NJ fibrosis resulting from a nonsense (908) 222-7000 (premature stopcodon) mutation in the cystic fibrosis transmembrane conductance regulatory gene -------------------------------------------------------------------------------------- treatment of muscular dystrophy Phase II resulting from premature stop (908) 222-7000 mutations in the dystrophic gene recombinant Applied Genetic treatment of alpha1-antitrypsin Phase I adeno-associated Technologies deficiency (386) 462-2204 virus alpha 1- Alachua, FL antitrypsin vector
Has this been posted?
Minister tasks scientists on commercial research
• Monday, Aug 13, 2007
The Minister of Science and Technology, Mrs. Grace Ekpiwhre, in Abuja urged scientists to focus on research with commercial value.
Ekpiwhre said during a facility tour of the Sheda Science and Technology Complex (SHESTCO) in Abuja that the “shelve was not the best place for research results”.
She said a lot of researches “were gathering dust” on the shelve because of lack of commercialisation.
Ekpiwhre therefore, called on the agencies under the ministry to intensify campaign on popularising such researches.
“That is the only way the average Nigerian can benefit from the contributions of the science and technology sector, “ she said. Ekpiwhre commended the management of the agency for the anti-sickling drug“‘NICOSAN’ ‘it developed.
“We must create awareness on the efficacy of the drug and ensure that, sufferers in the rural areas are inform about its availability”, she said.
“SHESTCO should ensure that those who cannot afford the drug have access to it by introducing favourable price regimes.”
Earlier, SHESTCO’s Director-General, Ayodele Coker, said the complex was equipped with three strategic advanced laboratories that would pioneer Nigeria’s quest to add value to local raw materials.
Coker said the advanced biotechnology laboratory was designed to serve as a centre of excellence for research and training with a view to addressing the country’s socio-economic progress.
He said the laboratory would also assist the country in strengthening its scientific and technological capabilities.
The Minister toured the Gamma Irradiation Facility, the Chemistry, Physics and Biotechnology Laboratories and the Xechem Pharmaceuticals, all located within the complex.
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For every seller there is a buyer, is someone buying your shares at this ridiculous price?
Makin...you da man!!!
What is NITE's deal with the 100 block the last few days???
Gotta love this action....Go XKEM!!!!!
correction..BusinessWeek ...eom
Welcome aboard Titan..eom
Everyone is dumping so they can buy XKEM.....
Raven..very well said!!eom
Good morning all, hope everyone had a great 4th!!
It means you will be on cloud nine before to long!!!
.0011/.0012 2x4 everyone hit the .0012 and we are off to the races.
take out .0012 and were gone
Xechem's Top Competitors;
Amgen Inc.
Biogen Idec, Inc.
Bristol-Myers Squibb Company
MedImmune, Inc.
Gilead Sciences, Inc.
Heska Corporation
Integra Life Sciences Holdings Corp
Serologicals Corporation
Nabi Biopharmaceuticals
Embrex, Inc.
Tapestry Pharmaceuticals, Inc.
TECHNE Corporation
VI Technologies, Inc. (VITEX)
Caraco Pharmaceutical Laboratories
Check the PPS of some of these guys!!
Nadine B Hack
President, beCause Global Consulting
Nadine B. Hack is President of beCause Global Consulting, which provides advisory services on cause-related strategies and philanthropic initiatives – as integral components of core business – for corporations, governments, foundations and individuals. She has expertise in concept development, policy analysis, problem solving and politically sensitive negotiations. She’s provided strategic guidance to or created crucial partnerships for clients internationally and has imparted creative direction for their campaigns and special projects. She links private resources with public needs benefiting women, children, minorities, human rights, health, the environment and economic development.
She has extensive experience with the UN system globally and served as NYC Commissioner for the United Nations, Consular Corps and International Business, the city’s senior official liaison with the world’s largest diplomatic and business communities. A longtime Africanist, she has served as an advisor to heads of state and other senior government officials, including the African National Congress executive committee in exile in Lusaka Zambia and South Africa’s subsequent ANC government.
She was president of the Sister City Program of the City of New York, formulating and managing financial, technical, educational, cultural and sports exchanges with cities globally. Other non- and for-profit boards on which she serves include Desmond Tutu Peace Foundation (chair), Nigerian-based Xechem International, Inc., Africa Division Human Rights Watch, Amnesty International USA, United Nations Association of New York, International League for Human Rights and Synergos Institute. Numerous honors include International Outstanding Achievement, NYWA Star, Woman of the Year, Distinguished Women of New York, SAAO Leadership, New American Leader, CDO Madame C.J. Walker and AJC Louise Waterman awards.
She has Master Degrees from the John F. Kennedy School of Government at Harvard University and the Graduate Faculty of Political and Social Science at the New School. She is adjunct professor of a course she created for the International Program at the Wagner Graduate School of Public Service at New York University, Global Development Strategies and Initiatives: Internal and External Capacity-Building through Creating and Sustaining Multi-Sector Partnerships.
Dated March 13th 2007;
Xechem International, Inc. (XKEM) just announced that its subsidiary Xechem Pharmaceuticals Nigeria Ltd. expects at least a significant increase in its pilot scale production of NICOSAN(TM) with the arrival of a long-awaited and very important piece of equipment in Nigeria. The device, a second spray dryer, will be joined by a third spray dryer currently in transit and scheduled to arrive in a few weeks. Installation of the second spray dryer just received is already underway; it should be operational within the next ten days.
Dr. Ramesh C. Pandey, the Chairman and CEO, stated, "With this acquisition, we can increase our pilot scale production dramatically, allowing us to bring NICOSAN(TM) to as many as 30,000 patients per month within the next 90 days, which would result in sales of approximately US$500,000 per month, assuming we are successful in scaling up and marketing and distributing the product. Upon our ability to raise sufficient funds to complete construction of our full scale facility on a timely basis, the Company believes it could produce sufficient product to serve the needs of approximately 50,000 to 100,000 patients by year end. Our current price of the product could in such event generate approximately $1 million to $2 million per month
Teague.....If I recall correctly...eom
hinch...be sure and tout Xechem while your there.jajajaja
Happy Birthday JoChef!!!!!!!!!!
Thanks DDog!!
Nlll "WHO ARE THOSE GUYS" don't know, but starting to like them!!
From NAN (Nigeria agency news)
March 13th, 2007
US firm to build sickle cell drug plant in Abuja
A Xechem International Inc., a company based in US is to build a $7-million (about N889-million) plant in Abuja to produce sickle cell drug.
The plant will be engaged in commercial production of NICOSAN, a herbal medication for the management of sickle cell disease.
The drug, developed as NIPRISAN by scientists at the National Institute for Pharmaceutical Research and Development (NIPRD) at Idu, near Abuja, is a non-toxic natural herbal drug with extracts from plants derived in Nigeria.
"We have already secured a $1.2-million (about 152-million) loan from the Nigeria Export and Import Bank (NEXIM) for investment in the construction of the plant," Ramesh Pandey, chairman of Xechem International Inc., told the North America correspondent of the News Agency of Nigeria (NAN) in New York at the weekend.
"We are again talking with NEXIM bank because they promised us another $5-million (about 635-million), while the US-NEXIM bank is also working on a facility for us to ensure the immediate take-off of the project.’’
He said the facility, to be located at SHESTCO Technology Park in Gwagwalada, Abuja, would be one of the most modern and state-of-the-art research and development drug plant in Africa.
Pandey said the plant would produce the sickle cell drug, formulated by NIPRD, and patent it to his company on commercial basis.
"We are working hard to bring NICOSAN to markets in Nigeria and subsequently to the US and other countries,’’ he said.
On how he acquired the production rights, the company executive stated: "In 2002, I signed an agreement for the transfer of exclusive ownership and rights for the research and development, production and worldwide sales and marketing of NIPRISAN to Xechem International Inc.
"Thereafter, the board of directors of Xechem International Inc., agreed to register Xechem Pharmaceuticals (Nigeria) Limited in 2002 to develop NIPRISAN as NICOSAN for marketing in Nigeria and worldwide.’’
He said the plant, due to its closeness to raw materials, would run on full capacity and provide "plenty of opportunities for Nigerian farmers and so it will create employment for a great number of unemployed persons."
"As soon as the drug plant starts rolling out, it will earn Nigeria foreign exchange,’’ he said. "Meanwhile, we are already producing this drug on a smaller scale as we are supplying the ones we are reproducing now to sickle cell patients.’’
On the potency of the drug, he says NICOSAN is not a curative drug.
"Sickle cell anaemia is a genetic disease. When a man with AS genotype marries a woman with AS, one of the four kids will be a sickler. But, what this drug does is very interesting. It disturbs sickling,’’ he explained.
"So the blood is still like a normal blood and the patient does not have any crisis. So when there is no crisis, the patient lives like a normal healthy person.’’
Pandey also disclosed that "as long as the patient takes his or her dose daily, there will not be crisis.
As a matter of fact, consistent use of NICOSAN reduces significant incident of crisis’’.
NAN learnt that the drug has been granted Orphan Drug status by the U.S. Food and Drug Administration and the EU’s European Medical Evaluation Agency, which gives the Xechem between seven to 10 years of exclusive production and marketing privileges.
.
© BusinessDAY Media Ltd. All Rights Reserved
Very well stated mpd68....eom
Hi makin.........nice in ft worth as well!!! Go Trans and Xechem, what a great team!!!!
just put a sell order in for .20 than they cant borrow your shares is what i heard dont know if its true or not.
Rotweiler..Thanks for all you are doing, incredible!!!!
I am willing to bet it will be a lot more than .26 a year from now! more like 1.26 or better on anticipation alone!!!
Been incognito Burnshorty had to go undercover, flying below the radar if you get my drift.......be back soon, with a vengance and when funds clear..
Yea.....
hey burnshorty......
How's it hangin' my friend.........
We miss you Jochef...................
Hey hinch hows it going? Good day today and plenty more to come, can't hardly wait till the factory is churning out 2-4 million a day at.70 cents per pill. That my friend is some serious income. eom
1,703,000 @ .0241
Ditto that!!!
Dr. Pandey..........
Dr. Pandey has had a distinguished career in the biomedical sciences spanning over 40 years, dedicated in bringing life saving drugs to the marketplace. He has served numerous universities, government agencies and pharmaceutical companies worldwide, including: The University of Illinois; The National Cancer Institute; LyphoMed, Inc.; Rutgers, The State University of New Jersey; National Chemical Laboratory (NCL); Abbott Laboratories; and others. Dr. Pandey has made over one hundred presentations at National and International conferences and seminars, has authored (or co-authored) 95 peer reviewed scientific papers and contributed to several books treating chemistry or biochemistry topics of major importance. He also holds several U.S. and International patents for biotechnology analysis and rare drug production processes, including paclitaxel, a plant product used for the treatment of ovarian, breast, small cell lung cancers, AIDS related Kaposi's sarcoma and stomach cancer. Developer of the first generic Vancomycin for Lyphomed (now owned by Fujisawa), Dr. Pandey also holds over 20 patents for the cancer drug, Paclitaxel, and next generation Paclitaxel. Dr. Pandey is presently leading and guiding Xechem in developing a phyto-pharmaceutical product NICOSAN™/ HEMOXIN™ for the treatment of the Sickle Cell Disease (SCD) for which there is no non-toxic drug in the market. Dr. Pandey is establishing a state-of-the-art research and production facility in Abuja, Nigeria. SCD is a genetic blood disorder found in Africans, Turks, Greeks, Saudi Arabians, Egyptians, Iranians, Italians, Latin Americans and Asiatic Indians. The United States Food and Drug Administration (US-FDA) and the European Evaluation Agency (EMEA) for European Union (EU) have assigned this product an Orphan Drug status, which means seven years exclusivity in the US and ten years in the EU countries. For his on going work in bringing quality control, standardization and setting specifications for the "Natural Herbal Drugs," also called "Ayurvedic Medicines" in India, Dr. Pandey was honored "Vaidya Ratna" at the Fourth International Healthcare & Herbal Expo and Seminar, held in New Delhi, India in April 2004. Dr. Pandey was also recognized in the 2nd International Conference on Appropriate Technology held in July 2006 in Bulawayo, Zimbabwe and by the State of Georgia in November 2006 for his outstanding achievements and community service in the medical field, especially in the field of Sickle Cell Anemia. Being a Paul Harris Fellow, member of the New Brunswick Rotary Club since 1996 and the president of the club for the term 1999-2000, he has been instrumental in helping children and down trodden in South Africa, Nigeria and India. Hailing originally from Ranikhet (District Almora, Uttarakhand), located in the foothills of the Himalayas in North India, Dr. Pandey has generations of association with Ayurveda since his father was the personal physician to the royal family of Kumaon.
Thats the cheapest shares I've bought since .014, got 100k @ .018
gotta love this buying opp if nothing else!
Makes no since at all!!
Yes, I think they are overwhelmed at this point in time!