February 26, 2014
When Dara BioSciences received the“orphan drug” designation Tuesday, it was as though the dealer stocked their bargaining kitty with a fresh stack of strong chips.
The U.S. Food and Drug Administration reviews drugs before companies may sell them. In certain cases, it awards a potential new drug the orphan drug designation, meaning that drug would treat a disease found in fewer than 200,000 people.
This is huge for Dara (Nasdaq: DARA) because it means this drug will have seven years of market exclusivity, a very strong bargaining chip when discussing a deal with potential partners.
Big Pharma likes owning orphan drugs because no other company may compete for those exact patients. Without competition, companies can keep prices high. In some cases, the price of a drug can drop as much as 90 percent after patent expirations.
Small companies like Dara typically work on potential new drugs in smaller trials, but can’t afford larger studies or later development because costs can run into the hundreds of millions. So, they often will look to partner with a larger drug company. In those deals, the large company takes on responsibility of developing, testing and filing a new drug application with the FDA, but agrees to pay the small company certain milestones and royalties if that drug reaches the market.
The orphan drug designation not only makes the Dara product more appealing for a deal in the first place, but when big drug companies compete against each other for a deal, it can mean higher milestone or royalty payments for Dara.
“For U.S. partners, this creates a lot of interest,” says Dara CEO Dr. David Drutz. “Some have told us they might be particularly interested if we get the (orphan drug) designation.”
The drug doesn’t have a name yet, and is simply titled KRN5500. Some studies on the drug have shown that it reduces nerve pain in patients undergoing chemotherapy, something for which there is not good treatment currently available, and for which traditional pain medication does not seem to work.
Thursday, February 27, 2014
11:30 a.m. - 1:00 p.m.
KPMG, Two Financial Center, Boston MA
What are the qualities that distinguish successful financial executives?
Join us as we explore the technical requirements, character essentials and skill sets necessary of high achieving financial executives.
Donna LaVoie, President & CEO, LaVoie Group
Donna has over 20 years in business strategy, corporate communications, public and investor relations. Since starting the agency in 2001, Donna's efforts combine building the agency's pharmaceutical and life science practices, strategic direction and core competencies. Under Donna’s leadership, LaVoie Group has become the acknowledged leader in life science and pharmaceutical communications and recognized as a top 25 independent healthcare agencies in the US. For the past four years, LaVoie Group has been recognized by the League of American Communications Professionals for agency awards. Over the last three years, the firm has received 16+ prestigious PR and marketing awards for their efforts on behalf of their clients.
Donna is a frequent speaker and has been published on a variety of subjects including integrated and crisis communications, investor and public relations best practices. She holds a Bachelor of Science in Management from Lesley College and has completed a strategic marketing program at The Wharton School. Donna is also a member of the Senior Roundtable at the National Investor Relations Institute, is a part co-Chair of MassBio’s communications and marketing committee and currently serves Advamed’s host and welcome committee for the 2013 conference.
Jackie Barry Hamilton, CFO, Intronis
As CFO, Jackie manages Intronis’ finance function and partners with our operating managers to help deliver our world-class backup solution. She joins the company from Monster Worldwide, where she was divisional CFO of Monster Worldwide Technologies and, prior to that, a corporate finance vice president. A veteran of the IT services market, Jackie was CFO of Corporate Software, where she led financial operations and managed its successful acquisition by Level 3 Communications. Following the purchase, Hamilton served as senior vice president of the Information Services Group of Level 3 Communication.
A graduate of Simmons College, Jackie received her master’s degree in finance from Boston College.
Jennifer Gridley, Director, Enterprise Market Intelligence, athenaHealth
Jennifer is Director, Enterprise Market Intelligence for athenaHealth, a provider of cloud-based services for electronic health records (EHR), practice management and care coordination with a market cap of $5billion.
Jennifer has been with athenaHealth since 2008, and in her current role is focused on equipping leadership, sales, client development and marketing teams with actionable, world-class insight that drives new revenue opportunity and builds athenaHealth's awareness, influence and loyalty among enterprise clients and sales targets. Previously she served as Chief of Staff and Director of Special Projects for CEO Jonathan Bush, and launched the company's award-winning Investor Relations program.
Jennifer holds an MBA from Babson College, and a BS from Bentley University.
This panel will be moderated by Beth Kurth, President, Boston Women Finance.
Update 2--U.S. FDA Okays Chelsea Therapeutics Drug for Low Blood Pressure
February 18, 2014
Reuters reporter Toni Clarke reported, “the FDA followed the recommendation and approved the drug on an "accelerated" basis. This allows for approval a drug to treat a serious disease based on an intermediate measure while the company conducts more trials.”
Chelsea Therapeutics Blood Press Drug Approved
February 18, 2014
The Associated Press noted in positive approval coverage that this significant milestone "marks a major comeback for the company and its drug."
Chelsea Wins U.S. Approval for Fainting Medicine Northera
February 18, 2014
Anna Edney of Bloomberg reported that Chelsea Therapeutics won U.S. approval for its drug to prevent sudden drops in blood pressure, the company’s first product to reach the market. Edney included a quote by Norman Stockbridge, director of the Division of Cardiovascular and Renal Drugs in the FDA’s Center for Drug Evaluation and Research noting that “People with neurogenic orthostatic hypotension are often severely limited in their ability to perform routine daily activities that require walking or standing.
The Wall Street Journal
Chelsea Therapeutics' Northera Receives Accelerated FDA Approval
February 18, 2014
The Wall Street Journal reported that “the U.S. Food and Drug Administration granted accelerated approval of Northera, its treatment of symptomatic neurogenic orthostatic hypotension.”
Chelsea Therapeutics Wins FDA Approval for Northera
February 18, 2014
Adam Feuerstein of TheStreet.com interviewed Chelsea president and CEO Joe Oliveto following the FDA approval announcement. "I love the [Northera] label…We think it is a doctors' normal standard practice to assess the efficacy of any drug periodically." Feuestein also noted that “Chelsea avoids a worst-case scenario under which FDA might have restricted the drug's use to no more than two weeks.”
Chelsea Wins FDA Nod for Once-Spurned Northera, But Success Isn't Guaranteed
February 18, 2014
In its second trip through the FDA, Chelsea Therapeutics' ($CHTP) antidizziness drug came out a winner, securing approval to fight faintness in patients with a rare disorder, but not without a daunting label and some strings attached.
Northera is now approved to treat lightheadedness associated with neurogenic orthostatic hypotension (NOH), a blood-pressure-lowering disorder tied to neurodegenerative ailments like Parkinson's disease. Chelsea intends to launch the oral drug in the second half of the year, targeting a U.S. patient population the company said is as large as 150,000. Analysts figure the drug could top out between $300 million and $450 million a year, although Chelsea has yet to discuss its pricing.
But the company may not be able to pull off a speedy launch of the once-rejected drug thanks to Northera's label. The treatment will carry a black-box warning due to its risks of increasing patients' blood pressure while they're lying down, and the FDA is cautioning those taking Northera to sleep with their heads elevated to cut back on the drug's stroke dangers. Furthermore, because the agency approved the drug through its accelerated orphan pathway, regulators looked at only two-week efficacy data and thus consider Northera's long-term benefits undemonstrated.
This 3-part webinar series is specifically geared toward the non-science professional who needs to better understand industry terminology, science, techniques and issues. This series provides an overview of the science and technology used to enable discovery and the processes scientists use to discover new therapeutics.
Investors seemed to struggle with what to make of the strongly worded label, as Chelsea's shares initially dipped on the postmarket announcement before soaring more than 30%. The Northera saga has made for some years-long bipolarity for Chelsea's stock price, but, hovering around $6.70 on Tuesday evening, the company's postapproval value is its highest since January 2011.
No matter Wall Street's reaction, the approval is a net win for Chelsea and Dainippon Sumitomo, which holds Asian rights to the drug. Northera is the first new NOH treatment in nearly 20 years, according to the company. Chelsea has a game plan for expanding its market share, preparing for a placebo-controlled study on about 1,400 patients with hopes of "definitively" proving Northera's lasting clinical benefit, the company said.
Safety concerns are hardly new for Northera, which endured an FDA rejection in 2012 due in part to agency worries about the hypertension risks now spelled out on its label. After piling on more efficacy data, Chelsea still faced some serious skepticism from FDA staff in the second go-round, but a panel of advisers voted in favor of approval in part because of the lack of viable treatments for NOH sufferers.
FDA OKs Northera for Hypotension
February 18, 2014
The FDA has approved droxidopa (Northera) for the treatment of neurogenic orthostatic hypotension, a rare disease that results in a drop in blood pressure when patients try to stand up.
The drug was passed up for approval 2 years ago when the FDA asked for more data from an additional clinical trial.
Approval stems from two clinical trials, each 2 weeks long, in which patients on droxidopa reported less dizziness, lightheadedness, feeling faint, or feeling as if they might black out than those on placebo.
The FDA noted, however, that the drug was OK'd under its accelerated approval program, which allows it to go on-market for demonstrating an effect on an "intermediate clinical measure" -- in this case, short-term relief of dizziness.
Drugmaker Chelsea Therapeutics, based in Charlotte, N.C., will have to demonstrate the drug's long-term benefit of extended dizziness relief in post-approval trials.
In its press release, the FDA emphasized that the "durability of the improvement in patient symptoms beyond 2 weeks has not been demonstrated."
Droxidopa will come with a boxed warning on the risk of increased blood pressure while lying down, which can cause stroke. FDA also warned it's essential that clinicians remind patients to sleep with their head and upper body elevated, and that supine blood pressure needs to be monitored before and during treatment, and more frequently when increasing doses. The drug is available in 100 mg, 200 mg, and 300 mg capsules.
In the two trials, the most common adverse events were headache, dizziness, nausea, high blood pressure, and fatigue.
Last month an FDA panel voted overwhelmingly in favor of approval (16 to 1) for droxidopa, but 2 years earlier nearly that same panel had been split on approval.
Following that advisory committee meeting, the FDA did not approve the drug, telling Chelsea Therapeutics it needed to submit more data from an additional trial.
Many panel members voting in favor of approval at the most recent advisory committee meeting said FDA should limit the drug to short-term use only, given the lack of long-term data.
Droxidopa is a pro-drug for norepinephrine, converted both peripherally and centrally, as it crosses the blood-brain barrier. It acts as a vasoconstrictor which, in theory, should help patients retain adequate blood pressure when they stand up from sitting or supine positions.
Chelsea’s Northera Wins FDA Approval in NOH; Black Box Warning
February 19, 2014
The long, hazard-fraught road for Chelsea Therapeutics International Ltd. with Northera (droxidopa) – including unfavorable briefing documents, followed by a positive advisory committee (adcom) vote – came to a happy end with approval of the drug by the FDA for neurogenic orthostatic hypotension (NOH), or a sudden drop in blood pressure when the patient stands up.
Ahead of the news Tuesday afternoon, Chelsea’s shares (NASDAQ:CHTP) halted trading at $4.95.
Northera bears a boxed warning about the risk of increased blood pressure while lying down. Patients are directed to sleep with their head and upper body elevated. Supine blood pressure should be monitored before and during treatment and more frequently when increasing doses, the warning says.
In January, an FDA panel voted 16-1 in favor of the approval of Charlotte, N.C.-based Chelsea’s orally active synthetic precursor of norepinephrine, though the panel was first skeptical, as was the agency itself, with regard to the durability of positive data gleaned from shorter-term studies. (See BioWorld Today, Jan. 13, 2014.)
It’s almost a reversal of what happened two years ago, when negative briefing documents appeared ahead of Northera’s first adcom, followed by a positive panel vote, followed by – instead of approval – a complete response letter from the unconvinced FDA, which wanted more trial data. (See BioWorld Today, Feb. 14, 2012, Feb. 24, 2012, and March 30, 2012.)
Deutsche Bank analyst Alethia Young predicted Northera could sell as much as $550 million worldwide, with $450 million of that in the U.S.
FDA Approves Chelsea's Northera
February 18, 2014
FDA granted accelerated approval to Northera droxidopa from Chelsea Therapetuics International Ltd. (NASDAQ:CHTP) to treat neurogenic orthostatic hypotension (NOH). The label includes a boxed warning on the risk of increased blood pressure while lying down. Northera plans to launch the orally available synthetic precursor of norepinephrine next half but said it has not yet determined the price. The company said it was seeking accelerated approval of Northera and as part of the post-marketing commitments will conduct a confirmatory placebo-controlled trial to establish the start date for the trial, which is expected to enroll about 1,400 patients.
The approval came after market close on Tuesday. Chelsea was up $0.22 to $4.95 on the day. The company jumped $1.76 (36%) to $6.71 in early after-hours trading.
Chelsea Therapeutics earned top headlines following the FDA’s Cardiovascular and Renal Drug Advisory Committee 16-1 vote to recommend approval of Northera™ (droxidopa) for neurogenic Orthostatic Hypotension (nOH), a rare condition associated with Parkinson’s disease and other underlying neurological diseases.
In addition to covering the 16-1 vote and reaction from panelists during the ad comm hearing, Reuters’ reporter Toni Clarke detailed the plight of Boynton Beach, Fla., resident Gail Hershkowitz, 65. Clarke interviewed the retired music teacher the week before the panel about how Gail’s illness made her afraid to leave her house after fainting and vomiting multiple times in public. She took Northera as part of a clinical trial and says it has enabled her to lead a relatively normal life.
The Wall Street Journal covered a positive Wedbush report following ad comm: "the panel came to appreciate the vagaries of clinical trials for neurological disorders especially in orphan diseases like symptomatic neurogenic orthostatic hypotension and the lack of good alternative treatments."
Medpage Today advanced the advisory committee hearing by interviewing Paola Sandroni, MD, PhD, a neurologist at the Mayo Clinic in Rochester, Minn., who said, "We have VERY limited options for orthostatic hypotension.” Dr. Sandroni added, “New drugs are needed." Medpage Today followed up with as story on the panel vote, FDA Panel Likes Northera on Second Go-Round.
TheStreet.com opined: Two positive panels following a very thorough presentation and discussion of all the issues with the Northera clinical data justifies an FDA approval. See Adam Feuerstein’s: A Few Minutes With Chelsea's CEO on Tuesday's Northera Panel and Future Plans
Charlotte Business Journal reports, “Chelsea has been reviewing its plans for commercialization in anticipation of the decision, Oliveto says. That includes hiring up to 120 people to support sales, management and branding once the drug is approved.”
Additional headlines and coverage include: