Deal Watch, Licensing Focus: Roche, Sanofi Exit Ongoing Anticalin Development Efforts With Pieris

Sarepta licenses gene therapy candidate from Lacerta, also makes equity investment. Neuren licenses North American rights to rare disease drug trofinetide to Acadia.

Scrip regularly covers business development and deal-making in the biopharmaceutical industry. Below is a roundup of some of the most noteworthy recent transactions. Deal Watch is supported by deal intelligence from Strategic Transactions.

Sanofi And Roche Bail On Pieris Deals

Roche is discontinuing its research collaboration and license agreement with Pieris Pharmaceuticals Inc. as of Aug. 21. The deal, signed in 2015, focused on the discovery of Anticalin proteins specific to an exploratory immuno-oncology target selected by Roche. [See Deal] Pieris will wholly own Anticalin proteins generated under the collaboration and says it will review the data, then consider its strategic options.

Sanofi also informed Pieris of its intention to return Anticalin rights, effective Aug. 23. [See Deal] Again, Pieris will gain full control over the program, which centered on tetraspecific Anticalins targeting Pseudomonas aeruginosa, subject to a collaboration signed in 2010. Pieris will review the data and then consider its options.

The company received $6.4m up front for the Roche deal and €3.5m up front from Sanofi, and earned fees totaling €1.2m related to the achievement of three different milestones under the two agreements. In March, a separate deal with Daiichi Sankyo Co. Ltd. related to CGRP antagonists was terminated. [See Deal]

Pieris continues to collaborate with AstraZeneca PLC on respiratory diseases [See Deal] and Servier SA [See Deal] and Seattle Genetics Inc.[See Deal] in immuno-oncology.

CEO Stephen Yoder said these ongoing agreements "represent a new partnering strategy for the company" involving "a collaborative approach to target selection, as opposed to our earlier model, which involved Pieris developing Anticalins to a target preselected by the partner." He noted that the newer model offers "higher upside for the company."

Sarepta Invests In, Licenses Gene Therapy Candidate From Lacerta

Sarepta Therapeutics Inc. will increase its gene therapy pipeline to 11 candidates, with the potential for more, under a financing, licensing and option agreement signed with Lacerta Therapeutics Inc. on Aug. 8. The Cambridge, Mass. biotech will make a $30m equity investment in Lacerta, license the rights to a Pompe disease candidate and take options on two other gene therapy candidates discovered by the Florida biotech.

Founded on adeno-associated virus (AAV) technologies developed at the University of Florida, Lacerta uses a range of proprietary AAV vector technologies to develop treatments for central nervous system (CNS) diseases and lysosomal storage disorders. In addition to the license and option rights, Sarepta expects to benefit under the deal from access to Lacerta's capsid screening and OneBac manufacturing platform. All three candidates subject to the agreement are CNS-focused, Sarepta said.

Acadia Gets North American Rights to Neuren's Trofinetide

Acadia Pharmaceuticals Inc. licensed exclusive North American development and commercialization rights Aug. 6 to Neuren Pharmaceuticals Ltd.'s trofinetide (NNZ2566). The drug candidate is a synthetic analog of the amino-terminal tripeptide of IGF-1 development for Rett syndrome – a neurological disorder occurring primarily in females, caused by X chromosome mutations on the MeCP2 gene – and other indications, including Fragile X syndrome, another rare X-linked disease of the central nervous system.

Acadia will pay Neuren $10m up front and potential milestone fees of up to $455m – $105m related to development achievements in Rett and Fragile X syndrome indications, and $350m upon the attainment of certain North American sales goals. Neuren also may receive tiered, escalating, double-digit royalties on net sales within the licensed territory. Acadia has a right of first negotiation to acquire a license outside North America.

In addition, Neuren is entitled to one-third of the market value of any rare pediatric disease priority review voucher for trofinetide if awarded by the FDA upon approval of a new drug application (NDA).

Acadia will take over funding and execution of remaining development for trofinetide in Rett syndrome in North America, except for the completion by Neuren of certain ongoing preparatory activities. The partners will form a joint steering committee to oversee development in all indications, including an upcoming Fragile X syndrome trial. Any data or regulatory filings generated may be used by either party within their respective territories.

Trofinetide demonstrated safety and tolerability in Phase II trials concluded last year as well as clinically meaningful improvement in core Rett syndrome symptoms. A placebo-controlled Phase III study is planned for the second half of 2019. A Phase II trial also was completed for trofinetide in Fragile X syndrome and a Phase III trial is expected to begin this year.

PTC Obtains Two Rare Disease Therapies From Akcea

Ionis Pharmaceuticals Inc. affiliate Akcea Therapeutics Inc. licensed PTC Therapeutics Inc. exclusive rights on Aug. 2 to develop, manufacture and commercialize its rare disease drugs Tegsedi (inotersen) and Waylivra (volanesorsen) in Latin America and certain Caribbean countries. Akcea also granted PTC a right of first negotiation to commercialize AKCEA-TTR-LRx, a follow-on candidate to inotersen, on an exclusive basis in the same territories.

Akcea receives $12m up front, $6m on the earlier of FDA or European Medicines Agency approval of Waylivra, and up to $8m in regulatory approvals in the licensed territories. PTC also will pay royalties in the mid-20% range beginning the earlier of 12 months after the first commercial sale of a product in Brazil or when PTC recognizes revenue of at least $10m in Latin America. The parties will create a joint steering committee to oversee the collaboration.

Akcea got exclusive rights to the drugs from Ionis earlier this year. [See Deal] That deal could be worth up to $1.9bn in the form of upfront and milestone payments to Ionis. Any milestone and royalty payments paid by PTC for Tegsedi will be split on a 60% Ionis/40% Akcea basis, and 50/50 for Waylivra.

Tegsedi is an antisense therapy in pre-registration in the US for hereditary transthyretin amyloidosis (ATTR), a disease characterized by a buildup of abnormal protein deposits in organs and tissues. It has a PDUFA date of Oct. 6. From the EU, the drug received marketing authorization approval for the treatment of Stage 1 or 2 polyneuropathy in adults with hereditary ATTR. (Also see " Aimovig And Tegsedi In Raft Of Products To Get EMA Thumbs up " - Pink Sheet, 1 Jun, 2018.)

Waylivra is under regulatory review in the US, Europe and Canada for familial chylomicronemia syndrome. Its PDUFA date is Aug. 30. (Also see "Akcea Waylivra's Surrogate Endpoint Gains Support At US FDA Advisory Committee " - Pink Sheet, 10 May, 2018.) The therapy also is in clinical trials for familial partial lipodystrophy.

Nevakar Gets Five Sterile Injectable Candidates From Endo

Endo International PLC expanded its sterile injectables portfolio and its pipeline of complex 505(b)(2) regulation pathway products under a licensing agreement with Nevakar LLC Aug. 8. The deal brings Endo US and Canadian rights to five product candidates for which Nevakar will seek US regulatory approval. Endo’s Par Pharmaceutical unit will launch and distribute the products after they obtain approval.

The specific products were not disclosed by the two firms, but Endo said if approved, they will provide new treatment options in the hospital and critical care settings. The deal continues an ongoing relationship between Par and Nevakar’s Founder and CEO Navneet Puri. Par currently markets multiple products originally developed by InnoPharma Inc., a company founded by Puri that was purchased by Pfizer Inc. in 2014. [See Deal]

Luoxin Gets Trulance Rights In China From Synergy

Synergy Pharmaceuticals Inc. granted Shandong Luoxin Pharmaceutical Group Co. Ltd. (Luoxin) exclusive rights Aug. 7 to develop and sell Trulance (plecanatide) for chronic idiopathic constipation and constipation-predominant irritable bowel syndrome in China, Hong Kong and Macau.

Luoxin is responsible for costs and activities surrounding the development, approval and commercialization of Trulance in its territories. Synergy gets $12m up front, up to $56m in regulatory and sales milestones and tiered royalties. The deal is the second out-licensing for Trulance this year; Cipher Pharmaceuticals Inc. paid $5mm up front for Canadian rights in February. [See Deal]

Luoxin’s portfolio includes treatments for oncology and allergy, as well as infectious, viral, respiratory, central nervous system, endocrine, cardiovascular and inflammatory conditions. It also has a wide offering of gastrointestinal therapies, including the ulcer/GERD products lansoprazole, pantoprazole, esomeprazole and others. In development, the company is working on LXI15028, a proton pump inhibitor in Phase III trials that it in-licensed from CJ Healthcare.

You can read more about deals that have been covered in depth by Scrip in recent days below:

(Also see "Allergan And Editas Sharpen Their CRISPR Focus On Ocular Drugs" - Scrip, 7 Aug, 2018.)	Allergan PLC and CRISPR specialist Editas Medicine Inc. have reinforced their gene editing R&D pact launched in March 2017.
(Also see "Regeneron And Bluebird Team Up In Cell Therapy "Joint Venture"-Style Deal " - Scrip, 6 Aug, 2018.)	Regeneron Pharmaceuticals Inc. will make a $100m equity investment in bluebird bio Inc. to collaborate on the discovery of antibodies and T cell receptors, with the hope of building out in immuno-oncology and overcoming some of the early cell therapy challenges. Regeneron's business development chief talks to Scrip about its strategy.
(Also see "Perrigo's OTC Core Could Expand To Include National Brands" - Pink Sheet, 9 Aug, 2018.)	Submitting an OTC switch of Nasonex would be a first for Perrigo Co. PLC, as would marketing a national brand OTC if the switch is approved. In tandem with announcing divesture of Perrigo's Rx business, CEO Uwe Rohrhoff says marketing national brands in some OTC categories fits in the company's "value-creation road map."
(Also see "Onward Ho! Glenmark Strikes First Onco Asset Deal With China’s Harbour BioMed" - Scrip, 6 Aug, 2018.)	Glenmark Pharmaceuticals Ltd. has sealed a licensing deal for its early stage immuno-oncology asset with China’s Harbour BioMed, opening up new possibilities around collaborations between the two Asian nations. The Indian firm appears upbeat about the prospects of building further on this new linkage.
(Also see "Boehringer Takes Out Option For Cystic Fibrosis Gene Therapy " - Scrip, 6 Aug, 2018.)	Boehringer Ingelheim GMBH is making its first foray into gene therapy and is looking to license a new viral vector-based product that could create a new therapeutic pathway for CF patients, regardless of their mutations.
(Also see "Yuhan Licenses Out Degenerative Disc Therapy To Spine Despite Earlier Trial Failure" - Scrip, 1 Aug, 2018.)	South Korean pharma firm licenses out to US venture Spine Biopharma novel peptide drug for degenerative disc disease YH14618, despite a Phase II failure and trial halt in 2016.