The R&D revolution wants you

Posted under Blog, Clinical Trials, Companies, Diagnostics, Funding, Medical Devices, Medical Supply, Pharmaceuticals, Startups, Universities, Videos by John Carroll

Over the last few years, the whole drug R&D ecosystem has been undergoing a sea change. Productivity in R&D on the Big Pharma side of the equation has been abysmal. And on the biotech side the emphasis has been on capital efficiency and cutting-edge innovation--which don't necessarily go hand in hand.

As a result of these forces, we're seeing pharma breaking down many of the old barriers that had been established to guard the research castle. Pharma scientists are being reorganized into smaller groups and redeployed to get closer to the top development hubs, while companies restructure internal research divisions from soup to nuts. And biotechs have been springing up in the richest fields aiming to advance disruptive ideas, increasingly looking to partner up with pharma at a very early stage of development. 

Getting to mid-stage proof of concept data on a careful budget is now one of the most demanding mandates in the industry. And I'll be moderating a breakfast panel on June 19 with a group of experts who will explore just how this trend is reshaping the way drugs are discovered and developed. 

Joining us at the Westin Waterfront in Boston will be Jason Gardner, head of the Center of Excellence in External Drug Discovery for GlaxoSmithKline; "Mene" Pangalos, executive vice president, innovative medicine, at AstraZeneca; Jim Burns, head of the Sanofi Boston R&D hub; biotech veteran Jill Milne, who helped found the virtual biotech company Catabasis Pharmaceuticals; and Bernard Munos, the founder of the InnoThink Center for Research in Biomedical Innovation, one of the most influential industry voices calling for change in R&D.

We're hosting this discussion as an affiliated event to the big BIO annual confab. So if you're already in Boston, or planning to attend BIO, I hope to see you there. It's one of the most important discussions now under way in biopharma. You won't want to miss it. And if there's something you'd like to tell me about, be sure to come by after the breakfast. I'm staying until the last guest leaves. Register for the breakfast here. -- John Carroll, Editor-in-Chief. Follow me on Twitter and LinkedIn.

Newron soars on positive PhIII results, $26M pact for Parkinson’s drug

Posted under BioTie Therapies, Blog, Chutes and Ladders, Clinical Trials, Companies, Diagnostics, Funding, Medical Devices, Medical Supply, Merck KgAA, Newron, Newron Pharmaceuticals, Parkinson's disease, Pharmaceuticals, Startups, Universities, Videos by john

Despite being left at the M&A altar last fall after a key partnership deal collapsed, Newron says it has successfully completed two late-stage studies of its experimental Parkinson's drug. And the Italian biotech used the opportunity to announce that it has garnered a 20 million euro licensing deal with Zambon for the drug. At the same time, Newron's founding CEO is exiting after 13 years at the biotech.

Newron didn't release details on the data from the two Phase III studies of safinamide. But investigators assert that the drug achieved positive numbers for maintaining motor functions and improved "on" times for patients who suffer from the disease. And the news fueled a spike in Newron's shares, which soared 60% as news of the results spread.

"The positive results from these global trials provide substantial evidence for the efficacy of safinamide as an add-on treatment to the most commonly prescribed drugs in PD patients," said CMO Ravi Anand. "These results together with the previously reported positive results from Phase II and Phase III studies indicate that safinamide will be a unique addition to the therapeutic armamentarium in the treatment of PD."

Founder Luca Benatti, meanwhile, is out as CEO, replaced by CFO Stefan Weber.

Newron has been scrambling to recover since last fall, when Merck KGaA walked away from its partnership on safinamide, complaining that the commercial potential of the treatment had flagged considerably since it first signed on. That retreat in turn prompted Finland's Biotie Therapies to drop its $63 million offer to buy the company, leaving its share price in tatters. Zambon initially stepped in last April to fund the remaining R&D work and preparations to file for regulatory approval, which is slated for 2013. Meiji Seika Pharma earlier gained rights to the drug in Japan and other Asian markets.

- get the press release on Phase III results
- here's the release on the Zambon deal
- read the press release on the executive switch
- here's the story from Reuters

Related Articles:
Biotie backs out of $63M Newron Pharma buyout
Merck Serono returns rights to Parkinson's drug to Newron

Lundbeck soars on PhIII success for blockbuster hopeful

Posted under Blog, Clinical Trials, Companies, depression, Diagnostics, Funding, Lexapro, Lundbeck, Medical Devices, Medical Supply, Pharmaceuticals, Pipeline, Regulatory, Startups, Takeda, Universities, Videos by john

Lundbeck shares spiked 9% this morning on the news that three late-stage studies of its new depression drug had delivered the data needed to support upcoming regulatory filings. The developer badly needs new therapeutics to replace Lexapro, an antidepressant that now faces generic competition.

"Lu AA21004 statistically significantly reduced depression symptoms in patients with (major depression) compared to placebo as measured by the Montgomery-Asberg Depression Rating Scale," the company reported. "In total, out of the ten large, placebo-controlled studies of Lu AA21004 completed in patients with MDD, eight have shown effect of Lu AA21004. Further analysis of the data is ongoing and data are expected to be presented at upcoming medical conferences." 

Lundbeck and its partner, Takeda, now plan to jointly file for an approval in the U.S. in the second half of the year, while Lundbeck will push ahead on its own in Europe and Canada. Bloomberg notes that Lundbeck has estimated potential annual revenue from the drug at about $2 billion.

It's standard operating procedure to mount a series of clinical studies for any new depression drug, as high placebo responses have been known to torpedo studies. Takeda and Lundbeck plan to jointly continue studies examining the drug's impact on cognitive dysfunction in depression.

- here's the press release
- read the Bloomberg piece

Related Articles:
Lundbeck banks on pipeline as antidepressant sales skid
Forest profits reel as Lexapro faces first copycats

Download our eBook ‘Supplying global clinical trials: Keys to avoiding costly delays’

Posted under Blog, Catalent Pharma Solutions, clinical trial, clinical trial management, Clinical Trials, Companies, Diagnostics, Funding, Medical Devices, Medical Supply, Pharmaceuticals, Startups, Universities, Videos by selvidge

Getting clinical trials right, and right the first time, is crucial for biopharma companies, especially as the cost of getting a drug to market is skyrocketing. Matthew Herper estimates in "The Truly Staggering Cost Of Inventing New Drugs"--which ran in Forbes in February--that drug development costs have reached the eye-watering level of somewhere between $4 billion and $11 billion. R&D returns have therefore fallen drastically and are around half what they were 10 years ago, explains Ian Shott, managing director and principal consultant at Shott Consulting: "Attrition is the overriding driver of cost, with a preclinical success rate of 0.01% and a success rate in the clinic of 10% or less."

The larger part of the cost of clinical-stage drug development comes from clinical trials. According to Timothy Scott, president of the U.S.-based pharmaceutical chemistry development organization, Pharmatek Laboratories, Phase II costs can range from $4,000-$20,000 per patient. So, the last thing a drug developer wants is any kind of interruption of a trial, or to have to start a trial all over again, and this makes the reliable supply of clinical trial material (CTM), whether manufactured in-house or outsourced to a contract manufacturing organization (CMO), absolutely critical.

"An interruption in the provision of the clinical trial material … could require that trial to be conducted over again," Scott says. "And … there is the cost of missed opportunity in the marketplace. If the drug being developed has the potential of being a billion-dollar drug, that is nearly $3 million in lost revenue every day the drug is not on the market."

Clinical trial supply isn't just an issue for investigational drugs. Some clinical trials compare the study drug against a placebo; however, others, particularly those for serious diseases for which it would be unethical to withdraw active treatment, compare with a marketed drug or another investigational drug. Other trials combine an investigational drug with another drug in a combination therapy. It's vital for these trials that there is a constant supply of the comparator or combination drug as well as the investigational drug. Maintaining this kind of reliable clinical trials supply needs a robust supply chain. "This requires complex planning to orchestrate the various elements that need to be brought together to have the right supplies ready in the right place at the right time," says Gerry Hepburn, chief operating officer, vice president and general manager, clinical supply services, Catalent Pharma Solutions. Click here to download the full ebook>> -- Suzanne Elvidge (email)