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Ra Pharmaceuticals and Camurus Announce Exclusive License Agreement for FluidCrystal® Extended Release Formulation of Zilucoplan

FluidCrystal® extended release formulation of zilucoplan achieved rapid and sustained pharmacodynamic inhibition of complement C5 in non-human primates, supporting at least once weekly dosing

CAMBRIDGE, Mass. & LUND, Sweden--(BUSINESS WIRE)--Jul. 16, 2019-- Ra Pharmaceuticals, Inc. (Nasdaq:RARX) and Camurus AB (Nasdaq STO:CAMX) today announced an exclusive worldwide license agreement for the use of Camurus’s proprietary FluidCrystal® (FC) technology to develop, manufacture, and commercialize a long-acting formulation of zilucoplan, Ra Pharma’s complement component 5 (C5) inhibitor in development for the treatment of multiple complement-mediated disorders.

”Ra is committed to delivering convenient and accessible products for managing C5-mediated diseases. Building on the strength of our daily formulation, which offers a quick, low volume injection and room temperature storage, the FluidCrystal® extended release (XR) formulation of zilucoplan has the potential to control disease for at least seven days from a single subcutaneous dose without the need for intravenous loading, on-body infusion devices, tissue-degrading enzymes, or permeation enhancers. The promising data from our pre-clinical studies conducted with Camurus, the potential for cost-effective manufacturing, and Camurus’s proven late-stage regulatory experience with FluidCrystal® were compelling reasons to add the FluidCrystal® technology into our zilucoplan XR life-cycle extension program,” said Doug Treco, Ph.D., President and Chief Executive Officer of Ra Pharma.

In pre-clinical testing, a single dose of the FC XR formulation of zilucoplan in non-human primates rapidly achieved and maintained target levels of complement inhibition for at least seven days without the need for an intravenous loading regimen (see Figure 1 below).

“The partnership with Ra Pharma follows the successful completion of a feasibility study of the FluidCrystal® extended release zilucoplan injection, which met formulation, pharmacokinetic, and tolerability target specifications,” said Fredrik Tiberg, President & CEO of Camurus. “We look forward to the next phase of our collaboration with Ra Pharma and initiating clinical development of a new promising product candidate based on our unique FluidCrystal® technology.”

Under the agreement, Camurus will receive an upfront payment of $2 million and is eligible to receive up to $14.5 million in development milestones and other license payments, up to $55 million in sales milestones, and tiered single digit royalty payments on product sales related to the FC XR formulation of zilucoplan.

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Figure 1: Inhibition of ex-vivo sheep red blood cell hemolysis assay following a single subcutaneous dose of FC XR formulation of zilucoplan in cynomolgus monkeys (mean+sem, n=4). (Graphic: Business Wire)

Figure 1: Inhibition of ex-vivo sheep red blood cell hemolysis assay following a single subcutaneous dose of FC XR formulation of zilucoplan in cynomolgus monkeys (mean±sem, n=4). (Graphic: Business Wire)

About Zilucoplan

Ra Pharma is developing zilucoplan and zilucoplan extended release (XR) for generalized myasthenia gravis (gMG), immune-mediated necrotizing myopathy (IMNM), and other tissue-based, complement-mediated disorders with high unmet medical need. The product candidates are designed for convenient subcutaneous (SC) self-administration. Zilucoplan is an investigational, synthetic, macrocyclic peptide discovered using Ra Pharma's powerful proprietary drug discovery technology. The peptide is designed to bind complement component 5 (C5) with sub-nanomolar affinity and allosterically inhibit its cleavage into C5a and C5b upon activation of the classical, alternative, or lectin pathways.

About FluidCrystal® Injection Depot

The FluidCrystal® injection depot delivers therapeutic levels of drug substance over selected extended periods – from days to months – from a single injection. The FluidCrystal® injection depot offers a liquid solution that transforms into a controlled release, biodegradable liquid crystal gel matrix in situ on contact with minute quantities of aqueous fluid at the injection site. Medicines based on the FluidCrystal® injection depot can be administered by the patients themselves or by healthcare professionals, without time-consuming and complicated reconstitution procedures. The technology is validated by approvals of Buvidal® in the EU and Australia and by the Brixadi™ tentative approval in the US and has been studied in more than 20 completed clinical trials. FluidCrystal® is a registered trademark of Camurus AB.

About Ra Pharmaceuticals

Ra Pharmaceuticals is a clinical-stage biopharmaceutical company focused on leading the field of complement biology to bring innovative and accessible therapies to patients with rare diseases. The Company discovers and develops peptides and small molecules to target key components of the complement cascade. For more information, please visit: www.rapharma.com.

About Camurus

Camurus is a Swedish science-led biopharmaceutical company committed to developing and commercialising innovative and differentiated medicines for the treatment of severe and chronic conditions. New drug products with best-in-class potential are conceived based on the company’s proprietary FluidCrystal® drug delivery technologies and its extensive R&D expertise. Camurus’s clinical pipeline includes products for the treatment of cancer, endocrine diseases, pain and addiction, which are developed in-house and in collaboration with international pharmaceutical companies. The company’s shares are listed on Nasdaq Stockholm under the ticker CAMX. For more information, visit www.camurus.com.

Forward-Looking Statements

This press release contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, including, but not limited to, statements regarding Ra Pharma’s collaboration agreement with Camurus and potential payments thereunder, statements regarding the potential, safety, efficacy, and regulatory and clinical progress of Ra Pharma's product candidates, including without limitation the zilucoplan FluidCrystal® extended release program, beliefs regarding preclinical study data, and statements regarding trial design, timeline, and enrollment of Ra Pharma's ongoing and planned clinical programs, including without limitation the clinical development of the zilucoplan FluidCrystal® extended release formulation. All such forward-looking statements are based on management’s current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include the risks that Ra Pharma's product candidates, including zilucoplan, will not successfully be developed or commercialized, in the timeframe we expect or at all; as well as the other factors discussed in the “Risk Factors” section in Ra Pharma’s most recently filed Annual Report on Form 10-K, as well as other risks detailed in Ra Pharma’s subsequent filings with the Securities and Exchange Commission. There can be no assurance that the actual results or developments anticipated by Ra Pharma will be realized or, even if substantially realized, that they will have the expected consequences to, or effects on, Ra Pharma. All information in this press release is as of the date of the release, and Ra Pharma undertakes no duty to update this information unless required by law.

Source: Ra Pharmaceuticals and Camurus AB

Ra Pharmaceuticals, Inc.
Investors:
Ra Pharmaceuticals, Inc.
Natalie Wildenradt, 617-674-9874
nwildenradt@rapharma.com

Media:
Argot Partners
David Rosen, 212-600-1902
david.rosen@argotpartners.com

Camurus AB
Fredrik Tiberg, President & CEO
Tel. +46 (0)46 286 46 92
ir@camurus.com

Fredrik Joabsson, Chief Business Development Officer
Tel. +46 (0)70 776 17 37
ir@camurus.com