Deal Watch, Licensing & Alliances: Amgen Places $66m Bet On Genetic Sequencing

Already partnered with Oxford Nanopore through its acquisition of deCODE Genetics, Amgen invests in the UK biotech to enhance its genomic capabilities. Aclaris licenses dermatology product Rhofade from Allergan. Other recent licensing and alliance deals... 

Scrip regularly covers business development and deal-making in the biopharmaceutical industry. Below is a roundup of some of the most noteworthy recent licensing and alliance transactions – mergers and acquisitions are covered in a separate column (see sidebar). Deal Watch is supported by deal intelligence from Strategic Transactions.

Amgen Invests $66m In Oxford Nanopore

In a move to deepen its genomics capabilities, Amgen Inc. made a direct investment in the genetic sequencing firm Oxford Nanopore Technologies Ltd. Oct. 18 to the tune of $66m (£50m). The big biotech said the investment aligns with its goal of using human genetics to deliver novel medicines to patients.

Amgen subsidiary deCode genetics EHF, acquired in 2012 for $415m, already employs Oxford Nanopore's sequencing platforms in its genome research, including efforts to identify and validate new targets. [See Deal]

Kari Stefansson, deCODE’s founder, said in a statement that Oxford Nanopore’s technology has enabled the company to sequence several hundred genomes and offers long-read sequencing capabilities that provide access to parts of the genome once believed out of reach. The platform also helps deCODE assess genetic variants that can create risk for a variety of diseases, the exec added.

Oxford Nanopore noted the scalability of its technology, with its MinION pocket-sized sequencer that can be used in any location; other applications offer high-throughput benchtop sequencing.

No specific terms of the investment were provided, although the Oxford, UK-based biotech said the pricing would the same as in its £100m (roughly $140m) funding round announced March 20. [See Deal] Intended to finance commercial expansion, the round included participation by Asia-Pacific investors GIC (Singapore), China Construction Bank International, Hostplus (Australia) and returning investors. Medtech Insight estimated that the financing brought its total fundraising to £451m at that point, with a capitalization of roughly £1.5bn. (Also see "Oxford Nanopore Hits £1.5bn Valuation With Latest £100m Round " - Medtech Insight, 22 Mar, 2018.)

Aclaris Obtains Allergan's Rhofade For Rosacea

Aclaris Therapeutics Inc. acquired Allergan PLC's Rhofade (oxymetazoline hydrochloride 1% topical cream) on Oct. 15 including an exclusive license to related intellectual property. Aclaris pays $64.5m up front ($58m in cash and $6.5m to be placed in escrow), a $5m milestone related to the potential development of an additional dermatology product, plus royalties ranging from a mid-single-digit to the mid-teens. To finance the deal, Aclaris entered a concurrent $65m loan and security agreement with Oxford Finance.

Rhofade cleared the US FDA last year for facial erythema (redness) associated with rosacea. Allergan initially gained the drug (then in Phase II) through its 2011 acquisition of Vicept Therapeutics Inc., a company established in 2009 by many Aclaris senior management team members. [See Deal]

This deal supports Allergan's recent efforts to offload its non-strategic medical dermatology assets; Allergan divested most of its US medical dermatology portfolio to Almirall SA in August. (Also see "Almirall Inks 'Transformational Deal' Buying Allergan's US Derma Portfolio" - Scrip, 3 Aug, 2018.) The addition of Rhofade complements Aclaris' topical dermatology portfolio, including Eskata (hydrogen peroxide) for seborrheic keratosis, which was launched earlier this year.

Roche, SQZ Enter New Pact On Antigen-Presenting Cells

Roche and SQZ Biotechnologies Co. agreed Oct. 15 to jointly develop and share commercialization rights for certain oncology therapies based on antigen-presenting cells (APCs) engineered by SQZ's platform and derived from peripheral blood mononuclear cells.

Watertown, Mass.-based SQZ is entitled to up to $125m in upfront and near-term milestone payments; $250m in clinical, regulatory and sales milestones per product; additional development milestones up to $1bn; and royalties. Through native immunity, APCs, which display antigens on their surface that are complexed with major histocompatibility complexes, can induce CD8 T-cell activity against tumors expressing a target antigen.

This new deal replaces Roche and SQZ’s 2015 alliance, which could have been worth up to $500m and was focused on B-cell modification using the biotech’s CellSqueeze membrane-disruption platform. [See Deal] Since signing the first agreement with Roche, SQZ has gone on to raise a significant amount of venture funding. It completed a $15.7m Series B round in 2016, co-led by NanoDimension and Polaris Partners, and earlier in 2018 closed on an oversubscribed $72m Series C, which included several new investors. [See Deal] SQZ previously raised $6m in seed and Series A rounds.

Sweden's Sinntaxis Targets Post-Stroke Brain Function In Acturum Pact

Sinntaxis announced Oct. 9 that it has inked an option agreement with fellow Swedish group Acturum for an exclusive license to a pain drug candidate initially developed by AstraZeneca PLC that will be tested as a potential treatment helping stroke survivors recover brain function.

AZD2066 is a metabotropic glutamate receptor 5 (mGluR5) antagonist that AstraZeneca advanced into Phase II studies in neuropathic pain. Sinntaxis said that its preclinical research has shown that substances that modulate the mGluR5 receptor can help recover lost brain functions by stimulating the healthy parts of a damaged brain, even when treatment starts as late as a week after a stroke.

The agreement provides Sinntaxis with rights to receive an exclusive license to AZD2066 for a three-year period in return for shares to be granted to Acturum. The latter got rights to a number of drug candidates developed by AstraZeneca when it purchased the pharma's facility in Södertälje in 2013.

Privately owned Sinntaxis plans to initiate a Phase II study in 2019/2020 and is confident that the development timeline can be significantly reduced given that the molecule that has already demonstrated satisfactory clinical pharmacokinetic properties and safety profile in the previous Phase I and II trials carried out by AstraZeneca. Michael Oredsson, CEO of the Lund-headquartered biotech, said the option to license a candidate that modulates the receptor that has proven capable of recovering key brain functions in preclinical trials "gives us a unique opportunity to develop a potentially pioneering treatment to help overcome stroke related brain damage in a time and cost-effective way."

There is no drug treatment available to help stroke patients recover brain function. Treatment currently involves rehabilitation, particularly physiotherapy and speech therapy.

Jnana, Neurocrine Enter CNS Discovery Alliance

Jnana Therapeutics Inc. and Neurocrine Biosciences Inc. are teaming up to discover multiple targets for small-molecule therapeutics for central nervous system disorders. Under the deal announced Oct. 9, Jnana gets an upfront payment, committed research funding to support discovery efforts, and milestones and royalties on resulting therapeutics.

A 2017 start-up, Jnana contributes its drug-discovery platform that targets the solute carrier (SLC) family of transporters. SLC transporters facilitate the passage of a wide range of substances across biological membranes and play a significant role in metabolic processes, including cellular uptake of nutrients and absorption of drugs.

The parties will work together to identify CNS compounds, after which Neurocrine will take over further lead optimization as well as development and commercialization activities for any potential therapies. Jnana also gets one-time access to a subset of Neurocrine's own compound library for Jnana to screen for a select number of non-CNS targets.

Neurocrine’s Ingrezza (valbenazine), an inhibitor of the vesicular monoamine transporter 2 (VMAT2) from the SLC family, was approved by the FDA last year for tardive dyskinesia.

Sarepta Gains Ex-EU License To Lysogene's LYS-SAF302

Sarepta Therapeutics Inc. licensed exclusive worldwide commercialization (excluding Europe) and manufacturing rights to Lysogene 's LYS-SAF302 gene therapy candidate Oct. 15 for the lysosomal storage disorder mucopolysaccharidosis type IIIA (MPS IIIA, also known as Sanfilippo A syndrome).

Sarepta is making an upfront payment of $17.5m ($15m in cash and $2.5m through the purchase of the purchase of 951,000 Lysogene ordinary shares at $2.63, a 30% premium). Lysogene stands to get $125m more in total compensation (including up to $11m more in committed cash this year and a $19m payment in 2019), plus royalties.

Caused by mutations in the N-sulfoglucosamine sulfohydrolase (SGSH) gene – which codes for the enzyme required for heparan sulfate recycling in cells – MPS IIIA is a rare autosomal recessive disorder leading to rapid neurodegeneration, severe behavioral problems and early death. LYS-SAF302 is a Phase II adeno-associated virus (AAV)-mediated gene therapy candidate, which uses the AAVrh10 virus to replace the faulty SGSH gene with a healthy copy of the gene. In MPS IIIA preclinical models, proof-of-concept was established with the compound demonstrating strong expression, broad distribution and the ability to correct lysosomal storage defects. Additional data showed that LYS-SAF302 was not associated with negative safety findings in the brain.

Lysogene is responsible for completion of the Phase II/III pivotal trial (for which the company just received IND clearance with the FDA last month) expected to start in the fourth quarter to assess the efficacy of intracerebrally administered LYS-SAF302 in improving or stabilizing the neurodevelopmental status. In addition to commercialization outside Europe, Sarepta is also tasked with global manufacturing of LYS-SAF302 and will supply Lysogene for its territory.

Under the deal, Sarepta also has an option to another undisclosed CNS-targeted gene therapy candidate. Sarepta, which has an existing gene therapy pipeline focused mostly on Duchenne muscular dystrophy (DMD), is looking to expand into other therapeutic areas. So far this year, the company has signed multiple collaborations, gaining programs and options on CNS candidates from partners including Lacerta Therapeutics Inc. (Pompe disease) [See Deal] and Myonexus Therapeutics Inc. (limb-girdle muscular dystrophies). [See Deal]201820169 The current deal gives it yet another disease focus beyond DMD.

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

(Also see "AbbVie Buys In On Morphic’s Oral Integrin Inhibitors, To Tune Of $100m" - Scrip, 18 Oct, 2018.)	After years of industry unsuccessfully trying to find an oral version of the successful I.V. integrin inhibitors, Morphic Therapeutic Inc. has a prolific platform – and AbbVie Inc. is paying $100m up front for preclinical oral integrin inhibitors for fibrosis indications, potentially including NASH.
(Also see "Tie-Up Moves Kangstem's Novel Stem Cell Therapy Towards Globalization" - Scrip, 17 Oct, 2018.)	Helped by a new partnership with Belgium-based MaSTherCell SA, the South Korean biotech  is aiming to initially challenge the European market with a stem cell therapy for atopic dermatitis, derived from human umbilical cord blood.
(Also see "BMS, Compugen Pursue Multiple Checkpoint Inhibitions In Antibody Collaboration" - Scrip, 16 Oct, 2018.)	Bristol-Myers Squibb Co. is teaming up with Compugen Ltd. to test the latter’s investigational antibody that targets a new checkpoint, PVRIG, in combination with its own antibody Opdivo. Combined inhibition of checkpoint mechanisms, such as PVRIG and TIGIT, is also on the cards for the future.
(Also see "Vertex’s Danish Reimbursement Deal Brings Budget Predictability" - Scrip, 16 Oct, 2018.)	Vertex Pharmaceuticals Inc.’s portfolio-based access deal in Denmark will give patients quicker access to a range of cystic fibrosis drugs and give budget predictability to the company and government.
(Also see "Sandoz And AbbVie Biosimilar Humira Settlement: What Does It Mean?" - Scrip, 12 Oct, 2018.)	Sandoz International GMBH has become the latest firm to sign a settlement agreement over biosimilar Humira with AbbVie, heralding imminent launch in Europe. In the US, it will hold off launching until September 2023.
(Also see "Carrick Targets Ovarian Cancer With BTG Deal, Entices Celgene’s Former Top Deal Maker" - Scrip, 11 Oct, 2018.)	In a busy week, Carrick Therapeutics Ltd. has added to its oncology pipeline, as well as bringing in Celgene Corp. deal maker George Golumbeski.
(Also see "Ionis Catches The Eye With New Roche Alliance" - Scrip, 11 Oct, 2018.)	October has been a very good month for Ionis Pharmaceuticals Inc.. Following US approval of Tegredi, it has now signed a potentially lucrative deal with existing partner Roche for another antisense drug, this time to tackle advanced dry AMD.