12:21 uur 14-01-2021

Knopp Biosciences maakt positieve topresultaten bekend van de fase 2 EXHALE-studie van oraal dexpramipexol bij matig tot ernstig eosinofiel astma

Het primaire eindpunt werd bereikt, waarbij dexpramipexol een statistisch significante afname van het aantal eosinofielen in het bloed vertoonde gedurende 12 weken

Afname van het aantal eosinofielen in het bloed correleerde met een klinisch belangrijke verbetering van de longfunctie

PITTSBURGH– (BUSINESS WIRE) – Knopp Biosciences LLC heeft vandaag positieve topresultaten bekendgemaakt in een fase 2-dosisbereikonderzoek van het nieuwe orale geneesmiddel dexpramipexol bij patiënten met matig tot ernstig eosinofiel astma.

Het EXHALE-onderzoek bereikte het primaire eindpunt, waarbij dexpramipexol een statistisch significante, dosisafhankelijke verlaging van het absolute aantal eosinofielen in het bloed (AEC) van baseline tot week 12 aantoonde in vergelijking met placebo wanneer het werd toegevoegd aan de standaardbehandeling. De omvang van de verandering in de AEC in het bloed was consistent met de eosinofieldepletie die werd waargenomen in eerdere klinische onderzoeken met dexpramipexol bij andere indicaties.

Knopp Biosciences Announces Positive Top-Line Results from the Phase 2 EXHALE Trial of Oral Dexpramipexole in Moderate-to-Severe Eosinophilic Asthma

Primary endpoint was achieved, with dexpramipexole demonstrating a statistically significant reduction in blood eosinophil count over 12 weeks

Reduction in blood eosinophil count correlated with clinically important improvement in lung function

PITTSBURGH–(BUSINESS WIRE)– Knopp Biosciences LLC today announced positive top-line results in a Phase 2 dose-ranging trial of the novel oral drug dexpramipexole in patients with moderate-to-severe eosinophilic asthma.

The EXHALE study met its primary endpoint, with dexpramipexole demonstrating a statistically significant, dose-dependent reduction in blood absolute eosinophil count (AEC) from Baseline to Week 12 compared to placebo when added to standard of care. The magnitude of change in blood AEC was consistent with the eosinophil depletion seen in previous clinical trials of dexpramipexole in other indications.

In the EXHALE trial, eosinophil depletion in blood by oral dexpramipexole correlated with clinically important improvement in lung function as measured by pre-bronchodilator FEV1.

Dexpramipexole was well tolerated by patients at all doses tested. Preliminary analyses showed no clinically meaningful differences in safety results between the dexpramipexole and placebo groups, with no serious adverse events and 96% of subjects completing the primary assessment phase. Background standard of care was inhaled corticosteroids in combination with a long-acting β2 agonist.

“These Phase 2 results strongly support the continued development of dexpramipexole as a potential first-in-class oral agent for the treatment of eosinophilic asthma,” said Michael Bozik, M.D., President and CEO of Knopp. “We expect future studies to determine the drug’s effects on severe asthma exacerbations, and to further assess its effects on lung function.”

EXHALE was a randomized, double-blind, Phase 2 trial assessing the efficacy and safety of three dose levels of oral dexpramipexole vs. placebo in reducing blood eosinophil counts over 12 weeks in 103 adult subjects with moderate-to-severe eosinophilic asthma. The trial was conducted at 28 U.S. study centers. Knopp expects to present results of the trial at a forthcoming medical meeting.

Clinical trials for approved eosinophil-depleting drugs have shown that drugs that reduce eosinophil counts provide significant benefit for reduction in asthma exacerbations in patients with moderate-to-severe eosinophilic asthma.1 Treatments approved to date for eosinophilic asthma are monoclonal antibodies requiring injection or infusion, while dexpramipexole is administered orally.


Knopp Biosciences is a privately held drug discovery and development company focused on delivering breakthrough treatments for immunological and neurological diseases of high unmet need. Knopp’s clinical-stage oral small molecule, dexpramipexole, is in Phase 2 clinical trials in 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.


Dexpramipexole, a selective inhibitor of eosinophil maturation, is an oral small molecule 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.2 Dexpramipexole has been well tolerated in clinical trials comprising approximately 1,200 patients.


EXHALE was a randomized, double-blind, Phase 2 trial assessing the efficacy and safety of 75 mg/day, 150 mg/day, and 300 mg/day of oral dexpramipexole vs. placebo in reducing blood eosinophil counts over 12 weeks in 103 adult subjects with moderate-to-severe eosinophilic asthma. The study was conducted at 28 U.S. study centers.

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 Nair P and O’Byrne P. Measuring Eosinophils to Make Treatment Decisions in Asthma. Chest. 2016 Sep;150(3):485-7.

2 Panch R, Bozik M et al. Dexpramipexole as an oral steroid-sparing agent in hypereosinophilic syndromes. Blood. 2018 Aug 2;132(5):501-509.


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