3 months ago during the 4th qtr CC, Rob introduced 4 new Triferic indications for the 1st time.
One was a new orphan indication, or one that treats a small population of people with a rare disease with few or no other alternatives. If approved by the FDA, Rockwell will get a 7-year market exclusivity to market Triferic with little or no competition.
Rob initially said this:
"We also are pursuing potential new drugs and drug products for development. Additionally, we are initiating clinical programs for delivering iron via Triferic and TPN total parenteral nutrition solution, PD, parenteral dialysis solution and an orphan indication."
Then this in reply to a Stifel Q:
"Well, no. The money we will spend on the clinical part for Triferic, for TPN and PD and this orphan indication that I mentioned. That will be strictly for Triferic."
Then Rob said this on 5/7/15 CC
" We're moving forward on clinical programs for Triferic delivery via TPN solution and PD solution, as well as an orphan indication for patients who are iron resistant."
A new orphan indication for patients who are iron resistant. Hmmmm
Triferic is going to be transformative in treating anemia in dialysis patients.
Triferic is the most significant advancement in dialysis in 25 years
$83.6 million in cash. No debt. Strong financial position to launch 2 drugs
“…both drugs on track for commercial launch in the July/August timeframe.”
“For Triferic, we continue to work diligently on numerous pre-launch initiatives. Our activities at the moment are focused on the manufacturing and marketing aspects of the launch, which involves the making of API and the scale-up and CMO redundancy required, as well as our communications with customers and our efforts to educate and to establish brand awareness around Triferic.“
‘…we have spent time interacting with and educating physicians and providers, and their feedback continues to be very positive.”
“Securing at least two suppliers to produce the estimated demand for the market is a must; this redundancy is critical and we have achieved it with both drugs.”
New Business Development
“..Multiple potential licensing partners..”
“..not only Triferic for iron delivery in hemodialysis patients outside the US, but also indications beyond the hemodialysis setting, both in the US and globally.”
Our goal is to:
1) Secure the highest value partner
2) Most favorable economics
3) In each partnered territory
4) For each indication.
New Triferic Markets
“We are moving forward on clinical programs for Triferic delivery via TPN (total parenteral nutrition) solution and PD (Peritoneal Dialysis) solution, as well as an orphan indication for patients who are iron resistant.”
“We also intend to use the Triferic technology in other indications, such as cancer and oral delivery.”
“We are also pursuing regulatory approval for Triferic in several countries outside the US.”
According to American Society of Hematology [Source: Kassebaum N J et al. Blood 2014; 615-624] est. global anemia population is 68.92 million:
* Mild 5.75m
* Moderate 51.8m
* Severe 11.4m
Global ranking of all causes of anemia:
1) Iron-deficiency anemia
2) Hookworm disease
3) Sickle cell disorders
6) Chronic Kidney Disease
8) Uterine fibroids
9) Other tropical diseases
10) Other infectious diseases
11) Other hemoglobinopathies
12) Other endocrine
13) Chronic Kidney Disease (due to diabetes)
14) Chronis Kidney Disease (due to hypertension)
15) Other gynecological diseases
16) Gastritis and duodenitis
17) G6PD deficiency
18) Maternal hemorrhage
19) Peptic ulcer disease
About Iron Deficiency Anemia
Another reliable source: There are over 7.5 million people in the U.S. with iron deficiency anemia (IDA), a condition that occurs when body iron stores are inadequate for normal red blood cell production. Fatigue, difficulty concentrating, shortness of breath, and dizziness are common symptoms, significantly impacting patients' quality of life.
IDA is a common complication of many diseases and conditions, including:
2. chronic kidney disease
3. gastrointestinal conditions
4. obstetric and gynecological conditions
5. congestive heart failure.
It affects up to one-third of inflammatory bowel disease patients and nearly one-quarter of patients who have undergone gastric bypass surgery. IDA is prevalent in women, affecting over 3 million U.S. women of childbearing age due to conditions such as heavy uterine bleeding, postpartum anemia, and pregnancy.
No wonder "Rockwell is already conducting studies with total parenteral nutrition patients and is also looking at the cancer market, as cancer patients exhibit the same functional iron deficiency as dialysis patients." Baxter is the clear leader in this space.
Flash forward 20 years from those early days in his dad's commercial space, and Chioini is now the head of a company that is a world leader in therapeutic dialysis products with over 300 employees and $50 million in revenue. Now his company is about to introduce an innovative new therapeutic dialysis drug to the market, Triferic, with $150 million in funding to back it.
Triferic offers iron replacement and maintenance of hemoglobin in hemodialysis patients through concentrates. In 2002 Rockwell licensed the technology for delivering iron through concentrates, and in 2004 they made the decision to rebrand as a biopharmaceutical company and take the drug to the market themselves in order to get the full value. They raised money and went all the way through the clinical process including clinical trials, eventually receiving FDA approval for the drug more than 10 years later (a pretty standard amount of time for a drug to go through the full clinical process). Now Rockwell is just a few months away from the proprietary drug being released into the market, where there is no other competing product currently in existence.
"There is no other iron delivery method that can do what this drug can do," Chioini says. "Every [dialysis patient] gets [iron replacements] through an IV in the bloodline, but IV iron isn't approved by the FDA as a maintenance therapy, it's a depletion therapy. It's being used that way because there is nothing else available."
Rockwell Medical's innovative drug Triferic will impact iron deficiency therapy around the world
Nicole Rupersburg | Friday, April 03, 2015 Prosper Magazine
Wixom-based Rockwell Medical is preparing to release its industry-changing iron maintenance and replacement therapeutic drug treatment Triferic to the market later this year, positioning itself as a global leader in its field.
Rockwell Medical got its start in Founder, Chairman, and CEO Rob Chioini's garage in 1995.
Admittedly that's a bit of hyperbole – it wasn't technically a garage, though it might as well have been.
After graduating from Michigan State University, Chioini went to work as a salesman in the medical industry, selling different products to hospitals that primarily included sterile instrument tray kits. The kits were a more efficient way for hospitals to utilize instrument trays that would otherwise get sent back to a central supply department for sterilization and re-distributed to various wards like the ER and ICU. Chioini would also work with different doctors and nurses to customize the trays to best suit their needs.
After that he worked in the field of dialysis selling hemodialysis concentrates (solutions and powders), where he also introduced his kit concept. Shortly thereafter he went off on his own, taking over a 10'x10' room in his dad's commercial cleaning space. Chioini would go out and sell the kits, come back and build them with help from his mother and younger brother, and then his dad would then deliver them in his spare time.
Within a year customers were looking for concentrates as well, so in 1996 Rockwell Medical partnered with two doctors, moved to Wixom, and started manufacturing. Later Chioini was able to buy out his partners and take company public, grow from one to three manufacturing plants, and made the strategic decision to become a drug company.
Baxter has been assisting clinicians in treating patients' diverse nutrient needs since the 1940s, when the company first introduced liquid proteins in the form of amino acids. Since then, Baxter has continued to advance intravenous (IV) nutrition. As an example, Baxter pioneered the world's first "triple-chamber system" for IV nutrition, which provides many of the essential ingredients of balanced nutrition - protein, carbohydrates, lipids and electrolytes in a single container - simplifying the preparation of parenteral nutrition for patients. Today, Baxter provides one of the broadest PN portfolios globally, which includes premix IV solutions, vitamins and lipids, as well as pharmacy workflow management, labeling and compounding technology.
Baxter and Laboratoire Aguettant Announce Global Collaboration for Parenteral Nutrition Trace Elements
Exclusive Agreement Supports Patients Requiring Access to a Comprehensive, Balanced IV Nutritional Therapy
DEERFIELD, Ill., April 2 - Baxter International Inc. (NYSE:BAX) and Laboratoire Aguettant SAS, Lyon, France, today announced they have signed an exclusive global licensing and distribution agreement for trace elements, which are essential micronutrients used in parenteral nutrition (PN) therapy. The collaboration allows Baxter to augment its leading PN portfolio, currently available in more than 90 countries, with essential trace elements.
Parenteral nutrition is an intravenous (IV) therapy, which may include a balance of protein, carbohydrates, lipids (fats), electrolytes, vitamins and trace elements for patients who cannot ingest food orally or enterally (tube-fed).
"Providing access to a comprehensive range of parenteral nutrition products is critical for patients who rely on IV administration of these nutrients," said Brik Eyre, president of Baxter's Hospital Products business. "This agreement complements Baxter's existing parenteral nutrition portfolio and provides clinicians with options to meet the ...
Dig deeper into Triferic science. Pyrophosphate shell binds to transferrin cause it has 2 binding sites attract each other cause charges off by 1. Also Triferic molecule has extremely high stability constant so it doesn't split apart in blood circulation or solution. Triferic does not need dailysate for delivery. IV. Pill. Injection. Powder. All are probable.