BioWorld Today Contributing Writer

Reformulations aren't exactly a novel proposition in the biotech world, but Madeira Therapeutics LLC is taking that strategy one step further. The company is one of the few biotechs focused on repositioning adult compounds for the pediatric market.

More broadly, Madeira specializes in drugs developed under the 505(b)(2) new drug application pathway – a popular route for biotechs and pharmas, enabling modification and repositioning of existing drugs for expeditious marketing clearance.

"We want to take existing small-molecule medications and find new uses for them," explained Pete Joiner, a retired Sanofi SA veteran who now serves as Madeira's CEO.

Although plenty of drugs are approved for conditions that traditionally affect children – asthma and allergy, attention deficit hyperactivity disorder and head lice, to name a few – along with childhood vaccines, a dearth of compounds exists for diseases that traditionally affect adults, Joiner said. Those indications include lipid control, hypertension and diabetes – diseases that are filtering down into the pediatric population, largely driven by obesity.

"As far as I'm aware, Madeira is the only company that's focusing on the obesity area, including cardiovascular issues, in children," Joiner told BioWorld Today.

The need is great, he added, since the National Institutes of Health has identified as many as 400 compounds for pediatric drug trials. And the market for such products is likely to grow since the American Academy of Pediatrics updated its guidelines on lipid screening and cardiovascular health in 2008, advising physicians to screen children after age 2 if one or both parents have a history of elevated lipids and to treat children pharmacologically beginning at age 8 if they show signs of LDL greater than or equal to 190 mg/dL – even lower in the presence of other risk factors.

Although pediatrics is a sweet spot for the company, "eventually we will have as many sales in the geriatric area as we do in the pediatric market," Joiner predicted. "If we can take an existing compound and teach an old dog new tricks, we certainly will."

Joiner had quickly tired of retirement when, in 2005, he joined several former Sanofi colleagues at Alliant Pharmaceuticals Inc., an Alpharetta, Ga.-based pediatric specialty pharma that was acquired for $109 million in 2007 by Sciele Pharma Inc. (now Shionogi Pharma Inc.), of Atlanta.

Although Joiner was now twice retired, he was smitten by drug development. He approached Ken Phelps, president and CEO of Cincinnati-based Camargo Pharmaceutical Services LLC and an industry expert in 505(b)(2) FDA submissions. In 2008, Phelps helped co-found Leawood, Kan.-based Madeira and currently serves as chief scientific officer while retaining his post at Camargo.

Initially, Madeira – which alludes to the grape-like flavor prized for pediatric syrup compounds – is developing liquid statin and pain products. Both will be easily titrated and lend themselves to the pediatric and geriatric markets.

"We think we have a very nice niche there," Joiner said, adding that the company will have intellectual property protection around its delivery systems.

Madeira, which operates with just two full-time employees, has completed its pre-investigational new drug application (NDA) for the liquid statin and is following guidance from the FDA on the next steps in the process, including additional formulation work. The company expects to file the NDA this year and launch a stability trial by the first quarter of 2012, which will run concurrently with a clinical trial. "We expect to have FDA approval for the statin product by late fourth quarter of 2012 and to launch the product in the first quarter of 2013," Joiner said.

Although development will take longer, a new pain management compound is equally desired in pediatrics, he added. Tylenol with codeine is the current drug of choice but, in many cases, a child's liver is too immature to metabolize the active ingredient.

"We plan to bring to market a more potent analgesic in a liquid format with a synthetic opioid as our active ingredient," Joiner said. "We feel very confident about the safety profile."

Starting a company at the height of the U.S. recession has made fundraising "a chore," Joiner admitted, "but we've made some progress." To date, the company has raised $2.2 million from angel investors and grants, with the Maverick Angels representing the firm's largest investor group. Last year, Madeira received $400,000 through the federal Qualifying Therapeutic Discovery Project, which funded its applications both for the liquid statin and pain products.

Earlier this year, SC Launch, an affiliate of Columbia, S.C.-based research and commercialization organization SCRA, provided the company with a $200,000 convertible note.

The process of applying for the funding opened additional doors for Madeira, which subsequently added a research site in Charleston, where it can work closely with the Medical University of South Carolina Children's Hospital.

"We need to raise another $1.5 million to gain FDA approval of our first product," Joiner said, adding that the company expects to secure those funds this year, but will not build infrastructure.

The pain management compound will come down the road, once revenues start to flow.

"We've started our pre-IND work, and we expect to submit that to the FDA by August," Joiner said. "We'll then go out for a Series B to finance the additional dosing trials needed for that product."

Ultimately, the company is open to partnering opportunities as well as the prospect of building a sales force.

"As we get closer to market, we'll make a final decision as to whether we want to team with somebody or go out on our own," Joiner said.