Business Wire

Knopp Biosciences Reports Positive Results From the Phase 2 EXHALE Trial of Oral Dexpramipexole in Moderate-to-Severe Eosinophilic Asthma at ATS 2021

Share

Knopp Biosciences LLC today announced detailed results of its positive Phase 2 dose-ranging EXHALE trial of the novel oral eosinophil-lowering drug dexpramipexole in patients with moderate-to-severe asthma. The data were presented in a virtual poster session at the ATS 2021 International Conference.

The EXHALE trial was a randomized, double-blind, placebo-controlled study of dexpramipexole in patients with moderate-to-severe asthma and blood absolute eosinophil count (AEC) ≥300/µL. Dexpramipexole study drug at oral doses of 75 mg/day, 150 mg/day, or 300 mg/day was added to standard of care. The primary endpoint was change in AEC from Baseline to Week 12 compared to placebo. Eosinophils, a type of white-blood cell, are validated as a therapeutic target in asthma as evidenced by the regulatory approval of multiple eosinophil-lowering biologics. The EXHALE trial enrolled 103 patients from 21 U.S. study centers. The majority of patients had severe asthma as classified by the Global Initiative for Asthma. Minority populations represented 23% of the patients enrolled.

Following are highlights of the ATS 2021 poster presentation by Calman Prussin, M.D., Knopp’s Vice President of Clinical and Translational Medicine:

  • Treatment with dexpramipexole resulted in significant reductions in AEC from Baseline to Week 12 at all doses tested (75 mg/day, p=0.019; 150 mg/day, p=0.001, and 300 mg/day, p<0.001). Dexpramipexole demonstrated a dose-dependent effect on eosinophil lowering that was significant by log-linear testing (p<0.001).
  • Patients receiving dexpramipexole 300 mg/day achieved an 80% reduction in their AEC from Baseline to Week 12 (p<0.001).
  • While the study was not powered to assess lung function, patients receiving dexpramipexole 300 mg/day had an increase in placebo-corrected pre-bronchodilator peak forced expiratory volume in one second (FEV1) of 182 mL at Week 12 (p=0.099). Notably, this FEV1 improvement was maintained through the Week 16/18 visit (∆FEV1 225 mL, p=0.036), despite patients having been off drug for 4-6 weeks.
  • A post-hoc analysis of all dexpramipexole doses across all study visits demonstrated a placebo-corrected improvement in prebronchodilator FEV1 of 172 mL (p=0.015).

Dexpramipexole was well tolerated in the trial, with no serious adverse events and no adverse events leading to discontinuation. Seventy-four of 76 dexpramipexole-treated patients and 25 of 27 placebo-treated patients completed the primary assessment phase.

“Dexpramipexole produced a significant reduction in eosinophil count, accompanied by clinically important improvements in lung function,” said Salman Siddiqui, M.D., Professor of Airway Disease at Leicester University, UK, and a co-author of the ATS 2021 poster. “We look forward to studying dexpramipexole as a potential pre-biologic alternative through the UK BEAT Severe Asthma Consortium Trials Program, funded by the National Institute for Health Research.”

Treatments approved to date for eosinophilic asthma are monoclonal antibodies requiring injection or infusion, while dexpramipexole is administered orally.

“We are pleased that EXHALE confirmed the significant eosinophil-lowering effects of dexpramipexole previously demonstrated in Phase 2 and Phase 3 clinical trials of more than 1,000 patients with other diseases,” said Michael Bozik, M.D., President and CEO of Knopp. “Even more encouraging are the clinical improvements in lung function that accompanied the targeted eosinophil depletion seen in asthma patients receiving dexpramipexole in the EXHALE trial.”

“Patients with eosinophilic asthma will welcome the addition of an oral alternative in treatment, providing the drug is safe and effective in Phase 3,” said Mary Jo Strobel, executive director of the American Partnership for Eosinophilic Disorders (APFED). “We look forward to future studies of dexpramipexole in additional eosinophil-associated diseases.”

Knopp expects to present additional analyses, including the effects of dexpramipexole on biomarkers of tissue eosinophilic inflammation and on additional measures of lung function, at a future scientific conference. In previous trials in hypereosinophilic syndrome and chronic rhinosinusitis with nasal polyps, dexpramipexole showed robust eosinophil reductions in skin, gastrointestinal, and sinus tissues.1,2

ABOUT KNOPP BIOSCIENCES LLC

Knopp Biosciences is a privately held drug discovery and development company focused on delivering breakthrough treatments for immunological and neurological diseases with high unmet need. Knopp’s clinical-stage oral small molecule, dexpramipexole, is in development for moderate-to-severe eosinophilic asthma. Knopp’s preclinical Kv7 platform is directed to small-molecule treatments for developmental and epileptic encephalopathies, other epilepsies, neuropathic pain, and tinnitus. Please visit www.knoppbio.com.

ABOUT DEXPRAMIPEXOLE

Dexpramipexole, a selective inhibitor of eosinophil maturation, is an oral small molecule drug in development by Knopp for asthma and other eosinophil-associated diseases. In hypereosinophilic syndrome, dexpramipexole has previously been shown in a Phase 2 trial to significantly reduce requirements for oral corticosteroids and in a subset of patients to produce durable disease remission. In its earlier development in amyotrophic lateral sclerosis, dexpramipexole was shown to be well tolerated in Phase 1, Phase 2, and Phase 3 trials comprising approximately 1,200 patients.

ABOUT APFED

Founded in 2001, the American Partnership for Eosinophilic Disorders (APFED) is a 501(c)(3) nonprofit organization that assists and supports patients and families affected by eosinophil-associated disorders by providing education, creating awareness, supporting research, and promoting advocacy. To learn more, visit apfed.org.

This press release contains "forward-looking statements," including statements relating to planned regulatory filings and clinical development programs. All forward-looking statements are based on management’s current assumptions and expectations and involve risks, uncertainties and other important factors, specifically including the uncertainties inherent in clinical trials and product development programs, the availability of funding to support continued research and studies, the availability or potential availability of alternative therapies or treatments, the availability of patent protection for the discoveries and strategic alliances, as well as additional factors that may cause Knopp’s actual results to differ from our expectations. There can be no assurance that any investigational drug product will be successfully developed or manufactured or that final results of clinical studies will be supportive of regulatory approvals required to market a product. Knopp undertakes no obligation to update or revise any such forward-looking statements, whether as a result of new information, future events, or otherwise.

Knopp’s pipeline consists of investigational drug products that have not been approved by the U.S. Food and Drug Administration. These investigational drug products are still undergoing pre-clinical or clinical study to verify their safety and effectiveness.

1 Panch SR et al. Blood. 2018 Aug 2; 132(5): 501–509.
2 Laidlaw TM et al. Laryngoscope. 2019 Feb;129(2):E61-E66.

To view this piece of content from cts.businesswire.com, please give your consent at the top of this page.

Contact information

Media inquiries:
Tom Petzinger
tom@knoppbio.com
412-488-1776

About Business Wire

For more than 50 years, Business Wire has been the global leader in press release distribution and regulatory disclosure.

Subscribe to releases from Business Wire

Subscribe to all the latest releases from Business Wire by registering your e-mail address below. You can unsubscribe at any time.

Latest releases from Business Wire

Esri’s Dr. Este Geraghty Receives HIMSS21 Most Influential Women in Health IT Award From HIMSS14.6.2021 19:10:00 EEST | Press release

HIMSS has announced Dr. Este Geraghty, the Chief Medical Officer for location intelligence world leader Esri, as a recipient of the Most Influential Women in Health IT Award. The award recognizes and celebrates influential women who harness the power of information and technology to transform health and healthcare. The winners are unique among power lists and award programs as it is inclusive of all women of influence, no matter where they may be in the health information and technology field. Dr. Este Geraghty, MD, MS, MPH, CPH, GISP, leads strategy and messaging for Esri’s Health and Human Services sector. Dr. Geraghty has been with the company since 2014 and has led business development and solution development in the market. During her time at Esri, Dr. Geraghty has helped organizations around the world use location intelligence to combat Zika virus, finish the fight against polio, grapple with the opioid crisis, combat homelessness, enhance health preparedness and response, inform

Verimatrix COO Asaf Ashkenazi To Present at Questex’s 2021 StreamTV Show14.6.2021 18:45:00 EEST | Press release

Regulatory News: Verimatrix, (Paris:VMX), the leader in powering the modern connected world with people-centered security, today announced that COO Asaf Ashkenazi is slated to present a partner keynote session during the virtual StreamTV Show. The event is free and registration is available here. Scheduled to take place at 4 p.m. Eastern Time on Tuesday, June 15, Ashkenazi’s session is titled, “Security That Enables Amazing.” Aiming to educate viewers on the latest threats to streaming services, he will also outline the specific approaches to combating them while enabling new types of events and spawning new products. The keynote will introduce how to disrupt the pirates’ business model and dissuade them from their efforts in the first place. “While video consumption has evolved, so have piracy and other threats – making it more important than ever for streaming services to fight fire with fire,” Ashkenazi said. “We’re proud to serve as a diamond sponsor of this year’s StreamTV Show an

GE Aviation and Safran Launch Advanced Technology Demonstration Program for Sustainable Engines; Extend CFM Partnership to 205014.6.2021 17:33:00 EEST | Press release

GE Aviation and Safran today launched a bold technology development program targeting more than 20 percent lower fuel consumption and CO2 emissions compared to today’s engines. The CFM RISE (Revolutionary Innovation for Sustainable Engines) program will demonstrate and mature a range of new, disruptive technologies for future engines that could enter service by the mid-2030s. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20210614005533/en/ John Slattery, President and CEO of GE Aviation and Olivier Andriès, CEO of Safran sign agreement extending the CFM International 50/50 partnership to the year 2050, declaring their intent to lead the way for more sustainable aviation in line with the industry’s commitment to halve CO2 emissions by 2050. (Photo: Business Wire) The companies today also signed an agreement extending the CFM International 50/50 partnership to the year 2050, declaring their intent to lead the way for more susta

Wipro Enables Zero Cost Transformation with Oracle Cloud Infrastructure14.6.2021 16:45:00 EEST | Press release

Wipro Limited (NYSE: WIT, BSE: 507685, NSE: WIPRO), a leading global information technology, consulting and business process services company, today announced it is collaborating with Oracle to launch Wipro Zero Cost Transformation, a new offering that helps organizations migrate to the cloud. Wipro is a member of Oracle PartnerNetwork (OPN). As enterprises seek to generate value from the cloud, their journey is often hindered by excessive costs, ineffective implementations and slow processes. With Wipro’s Zero Cost Transformation, companies can migrate their workloads to Oracle Cloud Infrastructure (OCI) with lower cost, shift from a Capital expenditures (CapEx) to Operating expenses (OpEx) model, and benefit from application and infrastructure support provided under a managed-services framework. Zero Cost Transformation eliminates the challenge of dealing with multiple service providers for cloud migration and support. It also provides a seamless and risk-mitigated migration, as Wipr

Wejo to Participate in Inaugural Credit Suisse Mobility Start-up Forum14.6.2021 16:00:00 EEST | Press release

Wejo Limited (“Wejo”), a global leader in connected vehicle data, announced today that it will participate in the Inaugural Credit Suisse Mobility Start-up Forum on Tuesday, June 22, 2021. Wejo Founder and CEO Richard Barlow and CFO John Maxwell will hold one-on-one conversations with investors to discuss the company’s mission to revolutionize the way we live, work and travel through connected vehicle data. For additional information for investors, please visit https://www.wejo.com/investor-relations. About Wejo Wejo is the leader in connected vehicle data, revolutionizing the way we live, work and travel by transforming and interpreting historic and near-real-time vehicle data. The company enables smarter mobility by organizing trillions of data points from over 11 million vehicles and more than 44.4 billion of journeys globally, across multiple brands, makes and models, and then standardizing and enhancing those streams of data on a vast scale. Wejo partners with ethical, like-minded

QCT and Robin.io Announce Strategic Partnership to Modernize 5G Solutions14.6.2021 16:00:00 EEST | Press release

Robin.io, the 5G and application automation platform company, and Quanta Cloud Technology (QCT), a global data center solution provider, today announced a strategic partnership through which the companies will jointly offer solutions that significantly reduce the costs, complexities and deployment times for mobile network operators (MNOs) and communication service providers (CSPs) rolling out next-gen networks and services. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20210614005152/en/ The Robin.io core technologies — Multi Cluster Automation Platform (MDCAP), Robin Cloud Native Platform (CNP), Robin Cloud Native Storage (CNS) — hyper-automation that orchestrates and manages the lifecycles of bare-metal infrastructures, 3rd party appliances, Virtual Network Functions (NVFs), Cloud-native Network Functions (CNFs) and services, with built-in logging, monitoring and policy engines, for closed loop automation, managed through a

Quest Appoints Brian Brugman as Director of Research14.6.2021 15:00:00 EEST | Press release

Quest Partners LLC (“Quest”), a quantitative investment firm managing nearly $1.8 billion of assets on behalf of some of the world’s largest institutions, today announced that Brian Brugman joined the firm as Director of Research. In the role, Mr. Brugman will spearhead Quest’s research and investment strategy development alongside Quest’s Founder and Chief Investment Officer, Nigol Koulajian. He will also join Quest’s management committee. “Brian’s breadth of experience in designing quantitative strategies with considerations around convexity risk bodes well with Quest’s differentiated approach to systematic macro trading,” said Mr. Koulajian. “He is joining as we are nearing the completion of a multi-year process to build out a state-of-the-art investment infrastructure to dramatically increase the efficiency and scalability of our research process. Brian will play a critical role in leveraging that technology and our research team to pursue highly valuable, negatively correlated ret

In our pressroom you can read all our latest releases, find our press contacts, images, documents and other relevant information about us.

Visit our pressroom