Positive Clinical Trial Results of Topical Botulinum Toxin for Overactive Bladder

PITTSBURGH--(BUSINESS WIRE)--A physician-initiated, double-blind, randomized, placebo-controlled clinical trial comparing the intravesical instillation of a topical botulinum toxin type A formulation (LP-09) to placebo in 24 subjects diagnosed with overactive bladder (OAB) showed significant reductions in both urinary urgency and urinary frequency. This result, published in European Urology, indicates the potential for reducing the need for cystoscopic needle injections when using botulinum toxin for treating OAB patients that are refractory to anti-muscarinic therapy. The clinical trial was conducted at Tzu Chi University, Hualien, Taiwan.

“The possibility of urinary retention and urinary tract infection are the primary drawbacks of the currently available botulinum toxins when treating OAB”

 

 

 

This elimination of the need for cystoscopic injections, if proven effective in larger clinical studies, could significantly impact the market adoption of botulinum toxin for the treatment of OAB. It is important to note that this study showed no treatment-associated urinary retention or increase in the patients’ post-void residual volume, which is a common adverse effect when using cystoscopic needle injection. “The possibility of urinary retention and urinary tract infection are the primary drawbacks of the currently available botulinum toxins when treating OAB,” according to Michael Chancellor MD, Chief Medical Officer of Lipella Pharmaceuticals. In an accompanying editorial, Professor Martin Michel from Johannes Gutenberg University, Mainz, Germany, who was not part of the study, noted that the possibility that botulinum toxin exerts its beneficial effects, at least in part, at the levels of the urothelium and/or afferent nerves raises the possibility that more direct targeting of these sites may maintain efficacy but have improved tolerability.

Lipella Pharmaceuticals Inc., the developer of LP-09, is a specialty biopharmaceutical company focused on the development of topical liposomal formulations for multiple therapeutic applications, including Interstitial Cystitis, Hemorrhagic Cystitis, and Overactive Bladder. Lipella’s initial pre-clinical development of LP-09 in the United States received funding from the National Institutes of Health Small Business Innovation and Research program. Further company details are available at www.lipella.com.

Caution: LP-09 is not currently approved for commercial use in any indication in the United States.

Citations

Kuo H-C, et al. Pilot Study of Liposome-encapsulated OnabotulinumtoxinA for Patients with Overactive Bladder: A Single-center Study.European Urology (2014), http://dx.doi.org/10.1016/j.eururo.2014.01.036

Michel MC. OnabotulinumtoxinA: How Deep Will It Go? European Urology (2014), http://dx.doi.org/10.1016/j.eururo.2014.02.046

Lipella’s Liposome Formulation of Botulinum Toxin (LP-09) Superior to Placebo for Overactive Bladder Symptoms in Double-Blind Trial

PITTSBURGH--(BUSINESS WIRE)--Pittsburgh based Lipella Pharmaceuticals Inc. today announced newly published results of a multi-site, double-blind, placebo-controlled clinical trial comparing the bladder instillation of a botulinum toxin in liposome formulation (LP-09) to placebo in 62 subjects diagnosed with overactive bladder (OAB). In the study, instillation of LP-09 showed significant clinical benefit without an increased risk of side effects. These results are published here in The Journal of Urology and are consistent with an earlier LP-09 clinical study previously reported here. The successful trial indicates the growing potential of LP-09 as a promising approach for the treatment of OAB.

“We are excited to continue the development LP-09 for not only OAB, but also for interstitial cystitis.”

 

 

OAB is a urological condition associated with urinary frequency and urgency that affects millions of Americans. While there are approved oral pharmacotherapies for OAB, there remains an unmet medical need for patients who stop oral therapy for lack of efficacy, adverse side-effects, or both. These patients increasingly turn to alternative therapies including neuromodulation and botulinum toxin needle injections during cystoscopy into the urinary bladder. LP-09 is a proprietary topical-liposome-botulinum-toxin formulation that does not require needle injections or cystoscopy.

The LP-09 clinical trial was physician sponsored and conducted at Buddhist Tzu Chi General Hospital and Kaohsiung Chang Gung Memorial Hospital in Taiwan. In the study, a single intravesical instillation of LP-09 was associated with statistically significant improvement in urinary frequency, and urgency severity score for LP-09 vs. placebo four weeks post instillation. These results were not accompanied by an increased post void residual urine volume.

“This is the second controlled clinical study of LP-09 where a dose of LP-09 significantly improved OAB symptoms without increasing residual urine volume,” said Dr. Michael Chancellor, Chief Medical Officer of Lipella. “We are excited to continue the development LP-09 for not only OAB, but also for interstitial cystitis.”

About Lipella Pharmaceuticals Inc.

Lipella Pharmaceuticals Inc., the developer of LP-09, is a specialty biopharmaceutical company focused on the development of topical liposomal formulations for multiple therapeutic applications, including Interstitial Cystitis, Hemorrhagic Cystitis, and Overactive Bladder. LP-09 is not currently approved for commercial use in any indication in the United States. Further company details are available at www.lipella.com.

Citation

Chaung YC, et al. Bladder Instillation of Liposome Encapsulated OnabotulinumtoxinA Improves Overactive Bladder Symptoms- A Prospective Multi-center Double Blind Randomized Trial. J Urol. 2014 Dec;192(6):1743-9

Lipella Pharmaceuticals Receives National Institutes of Health Funding for Its Orphan Drug Program

PITTSBURGH--(BUSINESS WIRE)--Lipella Pharmaceuticals Inc., today announced the award of a grant by the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) to support the pre-clinical development of LP-10, a topical-formulation of liposomal tacrolimus for the treatment of hemorrhagic cystitis.

“If LP-10 proves successful in the clinic, that could have a significant improvement in the lives of those with complications resulting from pelvic radiation therapy, which is often employed for the treatment of prostate cancer”

Hemorrhagic cystitis is a rare and serious complication of either pelvic radiation therapy or certain types of chemotherapies commonly used for treating pelvic area cancers. Hemorrhagic cystitis causes significant urinary bleeding that can ultimately result in cystectomy (surgical removal of the urinary bladder) if not adequately managed. There are currently no approved drugs for hemorrhagic cystitis, and existing management strategies (such as hyperbaric therapy) are extremely costly with limited availability.

To date, pre-clinical studies using LP-10 have demonstrated in-vivo efficacy in an animal model. Tacrolimus (the active ingredient in LP-10) is a powerful immunosuppressant primarily indicated for suppressing transplant rejection. Lipella recently received orphan designation from the FDA for the use of tacrolimus for hemorrhagic cystitis. “If LP-10 proves successful in the clinic, that could have a significant improvement in the lives of those with complications resulting from pelvic radiation therapy, which is often employed for the treatment of prostate cancer,” according to George Harriston, President of the Radiation Cystitis Foundation.

“This funding provides financial support for our remaining requirements to obtain investigational new drug approval from the FDA, which is the primary regulatory requirement for starting clinical trials,” stated Jonathan Kaufman, Chief Executive Officer of Lipella.

About Lipella Pharmaceuticals Inc.

Lipella Pharmaceuticals Inc. is a specialty biopharmaceutical company focused on the development and commercialization of novel liposome formulations for multiple therapeutic applications. Lipella’s research and development assets include experimental and clinical stage products for interstitial cystitis, overactive bladder, and hemorrhagic cystitis. Lipella is a privately held company based in Pittsburgh, PA. For further company details, visit www.lipella.com.

Caution

LP-10 is currently not approved for commercial use in any indication in the United States and is limited by U.S. Federal Law to investigational use only.

The project described is supported by Award Number R44DK102247 from the NIDDK. The content is solely the responsibility of the authors and does not necessarily represent the official views of the NIDDK or the National Institutes of Health.

Lipella Pharmaceuticals Inc. Names David Lucchino to Board of Directors

Lipella Pharmaceuticals Inc. today announced the appointment of David L. Lucchino, co-founder and former Chief Executive Officer of Semprus BioSciences, to the company’s Board of Directors. Semprus BioSciences was acquired in May 2012 for $80M.

Mr. Lucchino co-founded Semprus BioSciences with Massachusetts Institute of Technology’s Institute Professor, Robert S. Langer, Sc.D., while earning his MBA as an Alfred P. Sloan Fellow. There, he successfully secured $28.5 million in venture financing and $5.4 million federal funding, led his team to obtain FDA approval and CE Mark to market the company’s flagship technology and grew the organization from two to 40 employees.

Prior to co-founding Semprus, Mr. Lucchino was a Senior Associate at Polaris Venture Partners, a $3 billion venture capital fund based in Boston. Additionally, Mr. Lucchino co-founded and served as Managing Director of LaunchCyte, an investment firm that specializes in developing biomedical intellectual property. LaunchCyte successfully co-founded six portfolio companies.

“David’s industry expertise and insight add a unique dimension to Lipella as we prepare to enter a new phase of growth,” said Jonathan Kaufman, President and Chief Executive Officer of Lipella. “We are delighted to welcome him to our board, and look forward to his many contributions."

Mr. Lucchino currently serves on the board of the Massachusetts Biotechnology Council and Multiple Myeloma Research Foundation. He is a Trustee of Mt. Auburn Hospital, a Harvard Medical School facility, where he serves on the hospital's Audit/Compliance and Nomination committees. He also serves on the Audit Committee for CareGroup, the parent organization of Mt. Auburn, Beth Israel Deaconess Medical Center and The New England Baptist Hospital. He most recently completed a 3-year directorship on the board of AdvaMed, the Advanced Medical Technology Association, the national trade association representing the U.S. medical device industry.

About Lipella Pharmaceuticals Inc.

Lipella Pharmaceuticals Inc. is a privately held pharmaceutical company dedicated to the development of experimental therapeutic products that are designed to be delivered directly to the bladder via a urinary catheter. This local delivery option limits the risk of systemic side effects. Lipella’s R&D assets include experimental products for interstitial cystitis, overactive bladder and hemorrhagic cystitis. For further company details, visit www.lipella.com

  • 1
  • 2