Deal Watch: Syndax Gets Former Vitae Leukemia Program From Allergan

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.

Syndax Acquires Former Vitae Leukemia Candidates From Allergan

Syndax Pharmaceuticals Inc. announced a worldwide licensing agreement Oct. 17 with Allergan PLC, under which it will obtain a portfolio of preclinical, orally available small molecule inhibitors of the interaction of Menin with the Mixed Lineage Leukemia (MLL) protein.

Syndax will make a one-time upfront payment to Allergan and will be responsible for development, manufacturing and global commercialization of the portfolio. Allergan will receive development and commercial stage milestones and tiered royalties on net sales of commercialized products.

Part of Allergan’s acquisition of Vitae Pharmaceuticals Inc. in 2016, the compounds are thought to offer potential in treating a genetically defined subset of acute leukemias with chromosomal rearrangements in the MLL gene. [See Deal] Syndax expects to initiate clinical studies in 2019.

Syndax CEO Briggs Morrison, formerly an R&D chief at AstraZeneca PLC, said the candidates might significantly change the treatment paradigm for this subset of leukemia patients, and might qualify for orphan disease classification. (Also see "Syndax's Newly Arrived CEO Briggs Morrison Announces Clinical Collaboration With Genentech " - Scrip, 27 Aug, 2015.) These patients typically enter intensive chemotherapy, with a five-year survival rate of less than 50%.

"This agreement represents another strategic addition to our pipeline that we believe will enhance the long-term value of Syndax," Morrison added in a statement.

Syndax is developing entinostat and SNDX-6352 in multiple cancer indications. Entinostat has FDA breakthrough therapy designation and currently is in Phase III for advanced hormone receptor-positive, human epidermal growth factor receptor 2-negative breast cancer.

Merck Buys Stake In KalVista, Accesses Diabetic Eye Disease Drug

Merck & Co. Inc. signed a licensing agreement Oct. 10 worth around $760m with KalVista Pharmaceuticals Inc. to access the Cambridge, Mass.-based biotech's investigational diabetic macular edema (DME) treatments. [See Deal]

Merck is paying $37m up front for an option to buy KalVista’s DME portfolio of compounds based upon plasma kallikrein inhibition, which is led by the Phase II-ready drug KVD001. The US pharma also has agreed to pay $715m in milestone fees plus tiered royalties and invest $9.1m in return for a 9.9% stake in the firm. (Also see "KalVista’s Novel DME Approach Moves To Clinical Trials" - Pink Sheet, 22 Aug, 2014.)

As well as KVD001, an intravitreal injection, Merck also has an option to acquire investigational orally delivered molecules for DME developed by KalVista. However, the deal does not include the latter's portfolio of oral plasma kallikrein inhibitors for hereditary angioedema (HAE).

The deal represents something of a re-entry into the field of eye disease for Merck, which began the divesture of its ophthalmic business in 2014 with the sale of assets to Santen Pharmaceutical Co. Ltd. in a $600m deal. (Also see "Santen Buys Asia, Europe Rights To Merck's Eye Drugs For $600 Million" - Scrip, 13 May, 2014.)

Arcturus’ Second Collaboration With Janssen Addresses HBV

RNA therapy specialist Arcturus Therapeutics Inc. announced a research collaboration and worldwide license agreement on Oct. 19 with Janssen Pharmaceuticals Inc. Together, the two companies will develop and commercialize nucleic acid-based drug products for the treatment of hepatitis B, using Arcturus’ UNA Oligomer chemistry and LUNAR lipid-mediated delivery platform.

Arcturus will receive an upfront cash payment, R&D support and preclinical, development and sales milestone payments under the agreement facilitated by [Johnson & Johnson Innovation - JJDC Inc.] as well as royalty payments on any product sales. Janssen will undertake all development responsibilities and commercialization costs associated with the program. The pact also includes an option to expand into other infectious and respiratory diseases.

Arcturus CEO Joseph Payne noted that the deal signifies an expanded relationship between his company and Janssen. The two partnered in 2015 around Arcturus’ technology platforms in an undisclosed indication. [See Deal]

Protalix Licenses Ex-Us Rights For Fabry Compound To Chiesi

Protalix BioTherapeutics Inc. announced an ex-US licensing and collaboration agreement Oct. 18 with Chiesi Farmaceutici SPA for pegunigalsidase alfa (PRX-102), the Israeli firm’s chemically modified version of the recombinant protein alpha-galactosidase-A protein. Protalix focuses on the development and commercialization of recombinant therapeutic proteins expressed through its proprietary plant cell-based expression system ProCellEx.

Protalix will receive an upfront payment of $25m from Chiesi and additional payments of up to $25m in development costs, capped at $10m per year. The biotech also could earn up to $320m in regulatory and commercial milestone payments, as well as tiered royalties ranging from 15% to 35% on net sales. The agreement also specifies that Protalix will continue to manufacture PRX-102 for clinical development and commercial purposes.

PRX-102, currently in Phase III for Fabry disease, has demonstrated a significantly enhanced circulatory half-life and higher enzyme activity in target organs affected by this rare enzyme deficiency when compared to currently available versions of the drug. The candidate also has produced positive safety and efficacy data in a completed Phase I/II trial.

Moshe Manor, Protalix’s CEO, pointed to Chiesi’s 80-plus years of commercializing pharmaceuticals in “virtually all” markets outside the US and said its $50m upfront commitment will provide a significant non-dilutive cash infusion for the firm, as well as validation of its Fabry program. Protalix retains all rights PRX-102 in the US.

Roivant Licenses Mystery AstraZeneca Drug

Roivant Sciences GMBH disclosed on Oct. 19 that it licensed a drug from AstraZeneca PLC, but the biopharma start-up generating firm did not disclose the mystery compound's name, mechanism or disease area. Roivant said only that the drug has been deprioritized by AstraZeneca.

The company has global rights to develop and commercialize the asset and will pay AstraZeneca upfront and milestone fees plus royalties if the development program successfully brings a drug to market.

Roivant also did not disclose whether this compound will be developed by an existing Vant or if the company will launch another new subsidiary to take on the new asset. (Also see "What's Next For Roivant? It's Not More Publicly Traded "Vants"" - Scrip, 13 Jul, 2017.) Roivant's strategic decisions have come under additional scrutiny recently, however, because one of its biggest bets on a development program in-licensed from a pharma company was a bust when Axovant Sciences Ltd.'s lead Alzheimer's drug candidate intepirdine failed in Phase III last month. (Also see "Disappointed, Yes, But Roivant's Not Roiled By Axovant's Alzheimer's Failure" - Scrip, 26 Sep, 2017.)

AbbVie, Harpoon Collaborate In Immuno-Oncology

AbbVie Inc. and Harpoon Therapeutics said on Oct. 18 that they have agreed to combine resources in a research collaboration that will use Harpoon's tri-specific T cell recruiting biologic therapies (TriTAC) platform to discover novel drugs based on AbbVie's research-stage immuno-oncology targets.

AbbVie will have the right to pursue development and commercialization of any compounds provided by Harpoon, but the South San Francisco-based biotech firm did not disclose financial terms of its agreement with the big pharma.

This is the first pharma partnership for Harpoon, which closed a $45m Series B venture capital round in May. (Also see "Finance Watch: Bicycle Spins Toward Clinic With $52m Round; Stock Spikes Spur Offerings" - Scrip, 1 Jun, 2017.)

The company will use its platform to develop novel T cell engagers via the combination of T cell receptors with TriTACs. AbbVie Vice President of Oncology Early Discovery and Development Tom Hudson said in a statement that the deal "offers the potential for a unique approach to engage the immune system through AbbVie's investigational therapies in development."

ElsaLys Licenses Worldwide Leukotac Rights From Jazz

Jazz Pharmaceuticals PLC licensed global rights to ElsaLys Biotech SAS on Oct. 12 to develop and sell Leukotac (inolimomab) for the treatment of steroid-resistant acute graft-versus-host disease (GvHD). Jazz obtained Leukotac through its 2012 acquisition of EUSA Pharma, and has since brought the IL-12 monoclonal antibody into Phase III trials for GvHD following hematopoietic stem cell transplantation. [See Deal]

ElsaLys already has initiated discussions with regulatory officials in Europe and plans to commence US approval applications in 2018; Leukotac has orphan drug designations in both markets.

The company was formed in 2013 and has built a pipeline of therapeutic antibodies for cancer (solid and blood tumors) and vascular eye diseases. The company's other strategic partners include MabLife, Transgene SA and Institute Curie, among others.

Aimmune Signs Peanut Allergy Trial Collaboration With Regeneron, Sanofi

Aimmune Therapeutics Inc., Sanofi and Regeneron Pharmaceuticals Inc. teamed up in a new trial collaboration Oct. 16 to study the combination of Aimmune’s AR101 immunotherapy with Sanofi/Regeneron’s dupilumab in a Phase II trial for peanut allergies.

Sanofi and Regeneron co-developed dupilumab, a monoclonal antibody that inhibits the inflammatory cytokines IL-4 and IL-13, under a 2007 collaboration. (The drug is marketed as Dupixent for atopic dermatitis/eczema, and is in additional trials for asthma, sinusitis and eosinophilic esophagitis.) Aimmune is working on AR101 in Phase III trials as a peanut allergy desensitizer.

Since earlier AR101 studies revealed immunomodulatory effects on the IgE-IgG4 ratio, and because dupilumab regulates the IgE-IgG4 axis, the partners believe that a combination therapy could provide added benefit to patients with a life-threatening peanut allergy. A Phase II trial, sponsored by Regeneron, is expected to begin in 2018, overseen by a joint development committee seated by all three partners.

Codexis, Nestle Health Science team up in metabolic diseases

Codexis Inc. granted Nestle Health Science SA an option Oct. 12 to license exclusive worldwide development and commercialization rights to its CDX6114 for phenylketonuria (PKU). Nestle also receives access to Codexis' CodeEvolver protein engineering platform for use in discovering therapies for other metabolic disorders and for the development of enzyme products for Nestle's medical nutrition and consumer care businesses.

Preclinical CDX6114 is an oral therapeutic enzyme for PKU created with the CodeEvolver technology. CodeEvolver is covered by more than 175 issued and pending patents and enables the design and preparation of genetic libraries, automated screening methods, algorithms for the interpretation of screening data, and predictive modeling.

Codexis receives $14m up front, $3m if Nestle exercises the option, up to $90m in development and regulatory milestones, up to $250m in sales milestones if revenues exceed $1bn in a single year, plus royalties in the middle-single to low-double digits (Strategic Transactions assumes 4%-29%). [See Deal]

Codexis will pay for clinical development costs for CDX6114 up to and including Phase Ia trials, after which Nestle can opt to license CDX6114 and would take over and fund all development and commercialization activities. Nestle also gets the right of first negotiation to obtain an exclusive worldwide license to enzyme therapies – either currently in Codexis' pipeline or which the firm may discover in the next five years – for inborn errors of amino acid metabolism.

Aerie Gets Rights To Envisia IP For Ophthalmology

Envisia Therapeutics Inc. granted Aerie Pharmaceuticals Inc. the rights to use its PRINT (Particle Replication In Non-wetting Templates) technology in ophthalmology. Aerie also gets rights to intellectual property relating to the preclinical dexamethasone steroid ENV1105 for diabetic macular edema (DME). Aerie will pay $25m in cash and common stock, plus milestone fees tied to product approvals under the agreement announced Oct. 5.

The PRINT platform can engineer highly precise microparticles and nanoparticles of virtually any size, shape and chemistry – including small and large molecules – and polymeric drug delivery systems, such as extended-release formulations. Envisia's ENV1105 was created using the PRINT technology. Aerie will use the technology to develop candidates aimed at back-of-the-eye diseases such as age-related macular degeneration (AMD) and diabetic retinopathy. The firm will initially use PRINT to manufacture injectable implants containing its preclinical compound AR13154 for wet AMD and DME.

In July, Aerie obtained an option to license Royal DSM NV's bioerodible polymer implant technology. [See Deal] Aerie also is focusing on AR13154 under that agreement and will study the candidate with both the Envisia and DSM technologies.

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