Chelsea Therapeutics International Ltd. (CHTP)

Chelsea Therapeutics Receives Complete Response Letter from Food and Drug Administration for Northera(TM) (droxidopa) Capsules Call

March 29, 2012 08:00 am ET


Kate McNeil - Director, IR

Dr. Simon Pedder - President & CEO

Bill Schwieterman - CMO

Nick Riehle - VP, Administration and CFO

Keith Schmidt - VP, Marketing and Sales


Robyn Karnauskas - Deutsche Bank

Juan Sanchez - Ladenburg

Jonathon Eckard - Leerink Swann

Scott Henry - Roth Capital

Liana Moussatos - Wedbush Securities



Good day ladies and gentlemen and an welcome to the Chelsea Therapeutics March 29th conference call. At this time all participant lines are in a listen-only mode. Later we will conduct the question-and-answer session and instructions will be given at that time. (Operator Instructions). As a reminder this conference is being recorded. I would now like to turn the conference over to your host, Ms. Kate McNeil, please go ahead.

Kate McNeil

Thank you, operator. Good morning and thank you all for joining us to discuss our Northera new drug application for the treatment of symptomatic neurogenic orthostatic hypotension and the complete response letter received yesterday from the US Food and Drug Administration. Our press release regarding the FDA’s decision can be found on our website Joining me from Chelsea is Dr. Simon Pedder, President and Chief Executive Officer; Chief Financial Officer Mr. Nick Riehle; Chief Medical Officer Dr. Bill Schwieterman; Chief Scientific Officer Arthur Hewitt; VP of marketing and sales Mr. Keith Schmidt; and our Chief Operating Officer, Joe Oliveto.

Following formal remarks by Dr. Pedder, the conference call will be open for questions. Now before I turn the call over to Dr. Pedder, let me note that some of the remarks that you will hear today may contain forward-looking statements about the company's performance, actual future results might differ materially from those projected in the forward-looking statements. Additional information concerning these factors that could cause actual results to materially differ from those in these forward-looking statements contained in our SEC filings and periodic reports under the Securities and Exchange Act of 1934 as amended, copies of which are available on our website or may be requested directly from the company. And with that said I'll turn the call over to Dr. Pedder.

Dr. Simon Pedder

Thank you Kate and thanks to everybody for joining our call today. As we announced late yesterday, the US Food and Drug Administration has issued a complete response letter related to our new drug application for Northera also known as droxidopa for the treatment of symptomatic neurogenic orthostatic hypotension or NOH. This morning we would like to take you through each of these issues identified in last night's press release and answer questions you may have regarding the complete response letter or our path forward. Before we get into detail of the agency's response I would like to remind everyone that while we have had the opportunity to review the letter in detail, we of course have not had the opportunity to follow up or speak to anyone at the FDA.

In that respect some of our discussion will involve speculation until we consult with the agency on their recommendations. While the complete response letter identified several requests and recommendations, the principle request and the one with the greatest impact for Chelsea was the request for an additional efficacy data to support the findings of our pivotal study, Study 301 and to demonstrate the durability of effect the over a two to three-month period. The complete response letter detailed the agency's guidelines for an approval based on a single persuasive study and highlighted that Study 301 was positive and achieved a typically persuasive outcome on the OHQ, Orthostatic Hypotension Questionnaire.

They further acknowledged that that their guidance is open to the possibility of a single study approval in indications which there is an unmet serious medical need. And that NOH as a condition which is still [awaiting] symptoms with limited treatment options could be a condition for which they will consider an approval based on the results of one study. The letter also states that 301 has many of the characteristics of a single adequate well-controlled study that could make it adequate to support effectiveness.

However the agency's review of the data concluded that there were some subgroups namely women, older patients, US patients and those with Parkinson demonstrated less persuasive results following treatment with Northera and I will note separately that a majority of 301 was conducted in Ex US and that a majority of the US patients participating in the study were through consultation.

They further indicated that these inconsistencies, the results of Study 303 and 303 undercuts the persuasiveness of 301. Finally the letter raised some concerns regarding what they determined to be a disproportionate large favorable effect of one site on the outcome of Study 301. While we would agree that these results impacted the outcome of the 301 our analysis shows that consistent treatment effect is seen across the positive majority of the participating sites and that adjusted for the overall number of participants, results of Study 301 excluding the site would have been persuasive.

The letter also emphasizes the agency's interest in data that adequately demonstrates the durability of the effect. As you'll recall this was also a significant discussion point, both in the agency's briefing document and subsequent advisory committee meeting where the panel ultimately concluded the potential treatment benefits coupled with a significant unmet medical need warranted approval of Northera and outweighed the need for additional study concerning durability prior to the approval ultimately recommending approval of our NDA.

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