ProQR, Insmed to Present CF Therapy Progress at Analyst-Led Leerink Partners’ 4th Annual Rare Disease Roundtable

Patrícia Silva, PhD avatar

by Patrícia Silva, PhD |

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Two clinical-stage companies with investigational products for the cystic fibrosis treatment market have just announced their attendance and participation at the upcoming Leerink Partners’ 4th Annual Rare Disease Roundtable, taking place on Wednesday, September 30, 2015 at the Le Parker Meridien in New York, NY. The conference’s attendees will watch presentations on product and investment-related updates from ProQR Therapeutics N.V. and Insmed Incorporated, among others in the rare disease drug development industry.

Chief Executive Officer, Daniel de Boer of ProQR Therapeutics N.V., a company specializing in developing transformative RNA medicines to address severe diseases such as cystic fibrosis and Leber’s congenital amaurosis, will participate in an analyst-led fireside talk at 1:20pm ET. Those interested in viewing the live or archived webcast can access it through the ‘Investor Relations’ tab of ProQR’s website, under ‘Events and Presentations.’ Once archived, the webcast will be available for viewing 30 days after the presentation date.

Insmed CFO Andy Drechsler is scheduled to take part in a separate analyst-led talk at 11:30 AM ET. The webcast will also be available on the company’s website under the Investor Relations tab, and will be archived for 90 days after the presentation as well.

Both companies continue to make headlines in the CF community regarding their work into developing novel cystic fibrosis therapies. ProQR Therapeutics recently announced that its PQ-010-002 clinical trial is now open for patient enrollment. The study’s primary endpoint is the estimation of the effect of topical administration of QR-010 on the nasal mucosa in the restoration of CFTR function, as measured by Nasal Potential Difference (NPD).

QR-010 is an experimental therapy  designed to treat people with cystic fibrosis that have at least one copy of the ΔF508 mutation. In seventy percent of cystic fibrosis patients, the disease is caused by the ΔF508 mutation in the CFTR gene. QR-010 is unique in that it is designed to treat the underlying defect in CF, restoring normal function of the CFTR protein and halting progression of the disease.

Insmed recently reported new nontuberculous mycobacteria (NTM) lung disease information and a pharmacokinetic analysis of CF therapy ARIKAYCE (liposomal amikacin for inhalation) at the 2015 joint meeting of the Interscience Conference of Antimicrobial Agents and Chemotherapy (ICAAC) and the International Congress of Chemotherapy and Infection(ICC). The ICAAC/ICC 2015 was held in San Diego, California from September 17-21.