KaloBios Pharmaceuticals, Inc. (KBIO) today announced the completion of the randomized, double-blind placebo-controlled Phase 2 study of KB003, its anti-GM-CSF monoclonal antibody (mAb), in patients with severe asthma. While the study showed that KB003 was generally safe and well tolerated, it did not meet its primary clinical endpoint of improvement in FEV1 (a measurement of pulmonary function) compared to placebo in the overall study population.
The company also evaluated improvement in FEV1 compared to placebo in a number of pre-specified subgroup analyses. Of those subgroups, eosinophilic patients (Baseline Blood Eosinophils ≥0.3 GI/L) and patients demonstrating high reversibility at baseline (>20%) showed a statistically significant improvement compared to placebo. Other subgroups such as atopic or non-atopic patients did not demonstrate a statistically significant improvement in FEV1 from baseline as compared to placebo. In the key secondary endpoints evaluated, there was no meaningful reduction in exacerbations observed in patients receiving KB003 in the overall study population or in any material subgroup as compared to those receiving placebo, and there was no statistically significant improvement in asthma control questionnaire (ACQ) scores in the overall patient population.
"The results of this study replicate the effects seen in our earlier Phase 1/2 study in eosinophilic and highly reversible patients. Unfortunately the drug effect did not include improvement in exacerbations and ACQ, nor was the FEV1 improvement seen in the broader patient population evaluated in this study," said Nestor A. Molfino, MD, MSc., KaloBios' Chief Medical Officer. "The KaloBios team is working to continue to analyze further the results of the study, and we plan to meet with thought leaders to better understand the outcomes in the various subgroups of the patient population included in the study. We expect to submit the full results to a scientific meeting or journal for future publication."
"We are obviously disappointed in this outcome given the unmet need for additional treatments for severe asthma," said David Pritchard, KaloBios' Chief Executive Officer. "Based on the initial data evaluation of this Phase 2 study, we are discontinuing clinical development of KB003 in severe asthma. Instead we will refocus our resources and efforts on advancing our other products in the clinic, especially KB001-A where we continue to enroll patients in our Phase 2 study evaluating treatment of cystic fibrosis patients with Pseudomonas aeruginosa (Pa) lung infections, and on KB004, our oncology program where we are commencing enrollment of the Phase 2 expansion trial evaluating patients with acute myeloid leukemia and myelodysplastic syndrome."
Conference Call with Management
Management will host a teleconference and webcast to provide an overview of the trial results later today , January 29, 2014, at 4:30PM Eastern Time (1:30PM Pacific Time).
Interested parties can listen to the live teleconference by dialing (800) 514-4861 from the U.S. and Canada or +1 (484) 756-4333 for international callers. Individuals may access the live audio webcast by visiting the event URL at: http://ir.kalobios.com/events.cfm. A replay of the webcast will be available on the Company's website for 30 days following the live event.
KB003 Trial Design
The Phase 2 clinical trial of KB003 in inadequately controlled severe asthma patients was a randomized, double-blind, placebo controlled study evaluating 160 patients randomized one to one between treatment with current standard of care plus either KB003 or placebo. Eligible patients were identified as severe asthma patients having had at least two exacerbations in the preceding 24 months. In addition, in order to participate in the study, eligible patients were required to be diagnosed as reversible at the time of enrollment, defined as patients whose FEV1 improved by 12% or more after receiving treatment via a bronchodilator. Patients enrolled in the 24 week study received, in addition to current standard of care, either placebo or a 400mg dose of KB003 every four weeks via intravenous administration, with one additional dose at week 2. The primary endpoint of the study was the change in FEV1 from baseline for patients in the KB003 arm as compared to those in the placebo arm. The study was powered at 80% to detect a 6.7% improvement in FEV1 when compared to placebo. FEV1 is a measurement of pulmonary function.
KaloBios Pharmaceuticals, Inc. is developing a portfolio of proprietary, patient-targeted, first-in-class monoclonal antibodies designed to treat severe life-threatening or debilitating diseases for which there is an unmet medical need, with a clinical focus on severe respiratory diseases and cancer.
Currently, KaloBios has three drug development programs:
- KB003 is an anti-GM-CSF mAb with potential to treat inflammatory diseases, being developed for the treatment of severe asthma. A Phase 2 clinical study in 160 patients with severe asthma has been completed in the United States, Europe and Australia, which did not meet its primary endpoint of average improvement in FEV1 from baseline as compared to placebo. KaloBios has discontinued development of this compound in severe asthma, and is continuing to analyze the Phase 2 data to review with thought leaders in order to determine next steps, if any, in the development of KB003.
- KB001-A is an anti-PcrV mAb fragment, partnered exclusively with Sanofi Pasteur, and is being developed for the prevention and treatment of Pa infection. KaloBios has retained rights for the cystic fibrosis (CF) indication and is conducting a 180 patient Phase 2 study in CF subjects with chronic Pseudomonas aeruginosa (Pa) lung infection in the United States. KaloBios has received Orphan Drug designation from both the U.S. FDA and the European Medicines Agency for KB001-A for the treatment of Pa lung infection in CF patients. Sanofi is pursuing a ventilator-associated pneumonia prevention indication in the intensive care setting, an indication which has received U.S. FDA Fast Track Designation.
- KB004 is an anti-EphA3 mAb, with potential in treating hematologic malignancies and solid tumors. KaloBios is currently testing this drug in a Phase 1 study in subjects with hematologic malignancies, and intends to initiate a Phase 2 expansion for acute myeloid leukemia and myelodysplastic syndrome in the first quarter of 2014.
All of the company's antibodies were generated using its proprietary Humaneered® technology, a method that converts nonhuman antibodies (typically mouse) into recombinant antibodies that have a high binding affinity to their target and are designed for chronic therapeutic use. The company believes that antibodies produced using its Humaneered® technology offer important clinical and economic advantages over antibodies generated by other methods in terms of high binding affinity, high manufacturing yields, and minimal to no immunogenicity (inappropriate immune response) upon repeat administration in humans.
For more information on KaloBios Pharmaceuticals, please visit our web site at http://www.kalobios.com.
Forward Looking Statements
This release contains forward-looking statements made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995, and statements regarding the company's clinical development of KB001-A, KB003 and KB004. Forward-looking statements reflect management's current knowledge, assumptions, judgment and expectations regarding future performance or events. Although management believes that the expectations reflected in such statements are reasonable, they give no assurance that such expectations will prove to be correct and you should be aware that actual results could differ materially from those contained in the forward-looking statements. Forward-looking statements are subject to a number of risks and uncertainties including, but not limited to, the analysis of summary data from clinical studies, including but not limited to KB003-04, the potential, if any, for future development of KB003, the company's limited cash reserves and its ability to obtain additional capital on acceptable terms, or at all, including the additional capital which will be necessary to complete the clinical trials that the company has initiated or plans to initiate; the company's dependence on Sanofi Pasteur for the development and commercialization of KB001-A; the company's ability to successfully progress or complete further development of its programs; the uncertainties inherent in clinical testing; the timing, cost and uncertainty of obtaining regulatory approvals; the company's ability to protect the company's intellectual property; competition; changes in the regulatory landscape or the imposition of regulations that affect the company's products; and other factors listed under "Risk Factors" in the company's Annual Report on Form 10-K filed with the Securities and Exchange Commission on April 1, 2013, the quarterly reports on Form 10-Q filed on May 14, August 19, and November 12, 2013, and the company's other filings with the Securities and Exchange Commission.
All forward-looking statements are expressly qualified in their entirety by this cautionary notice. You are cautioned not to place undue reliance on any forward-looking statements, which speak only as of the date of this release. The company has no obligation, and expressly disclaims any obligation to update, revise or correct any of the forward-looking statements, whether as a result of new information, future events or otherwise.
For more information, visit http://www.kalobios.com.