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Plex Pharmaceuticals Announces Positive Ex Vivo Efficacy and Top Line In Vivo Ocular Safety Data of CAP1160

Plex Pharmaceuticals Announces Positive Ex Vivo Efficacy and Top Line In Vivo Ocular Safety Data of CAP1160 Plex is preparing to advance CAP1160 into in vivo animal studies for preclinical efficacy and proof of concept

PR Newswire

SAN DIEGO, Dec. 3, 2018

SAN DIEGO, Dec. 3, 2018 /PRNewswire/ -- Plex Pharmaceuticals, Inc. ("Plex" or "the Company"), a preclinical stage biopharmaceutical company focused on developing innovative small molecule therapeutics for protein misfolding and aggregation diseases, today announced positive efficacy results from the study of CAP1160 in an ex vivo organ culture cataractous model.  Along with the announcement came results from top line in vivo safety and pharmacokinetics (PK) studies conducted in New Zealand white rabbits with topically administered CAP1160.  These studies were partially funded through a Phase I SBIR grant by the National Eye Institute, National Institutes of Health.

CAP1160, a topically administered small molecule drug candidate, has been formulated for improved aqueous solubility and enhanced corneal permeation targeting the alpha A-crystallin (AAC) protein of the crystalline eye lens.  CAP1160 prevented cataract formation in porcine lenses in a preclinical ex vivo cataract model, which was developed and validated by Plex.  In this study CAP1160 demonstrated a dose-dependent prevention of cataract, with CAP1160 treated lenses reproducibly maintaining 54% higher clarity than control lenses (p=1.3x10-9).  Lens clarity was maintained over a period of three days without repeated dosing of CAP1160.  Consistent with these results CAP1160 treated lenses exhibited a 2-fold increase in soluble protein content compared to untreated controls.  In a second ex vivo experiment, CAP1160 demonstrated reversal of lens opacity, with, an improvement of 25%, which was maintained over a period of six days (p=0.006).

CAP1160 was also evaluated for in vivo safety and PK in New Zealand white rabbits.  Daily acute repeat topical treatment of low-dose and high-dose for 7 days showed CAP1160 to be safe.  All animals from both groups were subjected to detailed ophthalmic examination and showed no clinical signs of corneal tissue damage, toxicity, or limiting ocular irritation.  CAP1160 demonstrated permeation of the cornea and accumulation in the lens with no detectable systemic uptake.

"The positive preclinical ex vivo efficacy results and top line in vivo safety data strongly positions CAP1160 as a novel and disruptive drug candidate for the non-surgical treatment and prevention of cataracts to be advanced into in vivo efficacy, proof of concept and IND enabling studies," said Dr. Krishna Sharma, Beryl J. Ortwerth Distinguished Professor in Ophthalmology at the University of Missouri and advisor to Plex.  Cataracts are a leading cause of blindness with 65 million people globally suffering vision loss from cataracts.  "At Plex, we are committed to finding an affordable non-invasive alternative to prevent and treat cataracts," said Dr. G. Sridhar Prasad, Chief Scientific Officer of Plex.  "We are encouraged by the rapid progress of our R&D team and now have greater confidence in advancing CAP1160 into in vivo translational proof of concept and IND enabling studies and ultimately towards initiating our first-in-human clinical trials," continued Dr. Prasad.

About Cataracts

Cataracts, the clouding of the eye lens, is responsible for over 50% of blindness worldwide. Cataract surgery is the number one implanted medical device globally each year.  By age 75, approximately 50% of all Americans develop cataracts.  Although cataracts can be surgically removed, there are significant complications: (i) 30-50% of patients in the U.S. that undergo cataract surgery develop opacification of the posterior lens capsule within two years and require laser treatment; (ii) 0.8% suffer retinal detachments; (iii) 0.6%-1.3% are hospitalized for corneal edema or require corneal transplantation and (iv) about 1% present with endophthalmitis. Additionally, many people remain blind from cataracts due to inadequate patient access to surgery and in part to high costs, risk of complications and general aversion to surgery.  As life expectancy increases globally, the number of people suffering from cataracts is anticipated to grow.

About Plex Pharmaceuticals

Plex Pharmaceuticals, Inc., a subsidiary of Collidion, Inc., was founded in 2009.  Plex is focused on improving the lives of patients diagnosed with complex degenerative diseases caused by protein misfolding: cataracts and Parkinson's disease.  Plex Pharmaceuticals has been primarily funded through grants from the NIH and the Michael J. Fox Foundation.  Plex's drug discovery lab and operations are located in San Diego, California.  For more information, please visit or

Forward-Looking Statements

This release may contain certain forward-looking statements regarding our prospective performance and strategies within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. We intend such forward-looking statements to be covered by the safe harbor provisions for forward-looking statements contained in the Private Securities Litigation Reform Act of 1995 and are including this statement for purposes of said safe harbor provisions. Forward-looking statements, which are based on certain assumptions and describe future plans, strategies, and expectations of the Company, are generally identified by use of words "anticipate," "believe," "estimate," "expect," "intend," "plan," "project," "seek," "strive," "try," or future or conditional verbs such as "could," "may," "should," "will," "would," or similar expressions. Our ability to predict results or the actual effects of our plans or strategies is inherently uncertain. Accordingly, actual results may differ materially from anticipated results. Readers are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date of this release. Except as required by applicable law or regulation, Plex undertakes no obligation to update these forward-looking statements to reflect events or circumstances that occur after the date on which such statements were made.


William H. Watson, III
VP Business Development


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Last Updated: 03-Dec-2018