Deal Watch: Bristol Accesses Tech Platforms From SyntheX, Autolus

Pharma will partner with SyntheX on targeted protein degradation, with Autolus on cell therapy safety. Novo Nordisk gets rights to Ventus’s lead compound in NASH and chronic kidney disease.

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Scrip regularly covers business development and deal making in the biopharmaceutical industry. Deal Watch is supported by deal intelligence from Biomedtracker.

BMS Calls On SyntheX, Autolus In Protein Degradation, Cell Therapy R&D

Bristol Myers Squibb Company inked a pair of technology collaborations on 4 October, with San Francisco protein degradation specialist SyntheX and with UK-based Autolus Therapeutics plc for access to safety switch technology in cell therapy applications.

The tie-up with privately held SyntheX involves using the biotech’s ToRNeDO platform technology to discover small molecule molecular glue degraders against targets selected by BMS. SyntheX said its platform uses functional intracellular drug selection, rather than in vitro screening, and discovers candidates using a prespecified E3 ligase and a neosubstrate of interest.

BMS is paying undisclosed upfront cash and making an investment in SyntheX under the agreement. SyntheX also could realize up to $550m in performance-based milestones and potential sales royalties, the companies said.

Autolus, which develops next-generation programmed T-cell therapies, is granting BMS rights to incorporate its proprietary RQR8 safety switch into an initial set of cell therapy programs on a target-by-target basis. BMS also gets an option to incorporate the technology into additional cell therapy candidates.

The London biotech said the RQR8 safety switch works by binding rituximab to engineered CD20 epitopes and triggering cancer cell apoptosis. The technology enables precise targeting and controlled, enhanced and sustained CAR-T activity within the tumor microenvironment, Autolus added.

Autolus receives an undisclosed upfront fee under the agreement and can realize option fees, development milestones and potential sales royalties as well.

Novo Nordisk Moves Into NLRP3 Space With Ventus Agreement

Novo Nordisk A/S announced 29 September that it will pay Ventus Therapeutics Inc. $70m up front in exchange for worldwide rights to its lead peripherally restricted NLRP3 inhibitors in indications such as non-alcoholic steatohepatitis and chronic kidney disease. Ventus will also get R&D funding under the deal and could realize up to $633m in clinical, regulatory and commercial milestones plus sales royalties.

The deal will add to Novo’s R&D pipeline in NASH, which also includes a Phase III program for its GLP-1 agonist semaglutide (branded as Ozempic for diabetes and Wegovy for weight loss) and Phase II candidate NN9500, an FGF21 analog. (Also see "Novo Nordisk And Gilead Expand NASH Alliance" - Scrip, 18 Mar, 2021.) Ventus retains rights to develop NLRP3 inhibitors in other indications, including inflammatory and respiratory diseases. The Waltham, MA-based firm also retains all rights to its brain-penetrant NLRP3 program.

The pact continues a recent spate of deal-making for the Danish pharma, which signed a multi-year research partnership on 20 September with Octagon Therapeutics Inc. and acquired rare blood disorder-focused Forma Therapeutics Inc. for $1.1bn on 1 September. (Also see "Deal Watch: GSK Gets Rights To Spero’s Phase III Antibiotic" - Scrip, 22 Sep, 2022.) More generally, the biopharma industry has seen multiple deals involving NLRP3 inflammasome receptors, including Roche Holding AG’s acquisition of Jecure Therapeutics in 2018 and Inflazome Ltd. in 2020. (Also see "Roche Bulks Up In NLRP3 With €380m Inflazome Buy" - Scrip, 21 Sep, 2020.)

Pfizer Opts In On Voyager AAV Capsid

Gene therapy company Voyager Therapeutics, Inc. announced 4 October that Pfizer Inc. has exercised its option to license a novel capsid generated from Voyager’s TRACER next-generation adeno-associated virus (AAV) capsid discovery platform. Pfizer will use the novel capsid for a potential gene therapy program against an undisclosed rare neurological disease target.

The option triggers a $10m payment to Voyager, which is eligible for up to $290m in development, regulatory and commercial milestones, plus tiered royalties. The original license option agreement was announced in October 2021 and Voyager received a $30m upfront payment. (Also see "Pfizer Gets AAV Capsid Rights From Voyager For Gene Therapy R&D" - Scrip, 6 Oct, 2021.)

Voyager – which is both working with partners on their gene therapy programs and advancing its own internal pipeline of candidates for GBA1 Parkinson’s disease, SOD1 ALS and other neurologic diseases – hailed the option as “further validating the potential of the TRACER capsid program.”

Pfizer has elected not to exercise its option to license a capsid for a cardiac target under the original agreement, and those rights are returned to Voyager.

Sanofi, MiRecule Collaborate On Antibody-RNA Conjugate

Next-gen RNA therapeutics specialist miRecule, Inc. announced on 4 October the first partnership for its proprietary DREAmiR platform: a strategic collaboration and licensing agreement with Sanofi to develop and commercialize an antibody-RNA conjugate (ARC) for facioscapulohumeral muscular dystrophy (FSHD), the second most common form of muscular dystrophy.

The partners’ goal is “a best-in-class … disease-modifying treatment that selectively targets and suppresses the underlying cause of FSHD in muscle tissue.” MiRecule’s anti-DUX4 RNA therapy will be combined with Sanofi’s proprietary muscle-targeted NANOBODY technology to create an ARC using miRecule’s NAVIgGator conjugation and formulation chemistry. Sanofi will hold an exclusive worldwide license to the FSHD therapy.

Under the agreement, miRecule and Sanofi will collaborate on research through lead candidate selection, at which time Sanofi will take over development and commercialization activities. MiRecule is receiving an upfront payment and near-term milestone payments that “combined could exceed $30m.” Additional development, regulatory and commercial milestones could increase the total payments to miRecule to nearly $400m, plus tiered royalties on sales.

Genmab Licenses IO Antibody From Oxford BioTherapeutics

Oxford BioTherapeutics (OBT) licensed Genmab A/S a novel antibody related to one of OBT's immuno-oncology programs on 3 October. The target was discovered using Oxford’s proprietary OGAP drug discovery platform, which incorporates a proteomic database, which integrates clinical, experimental and expression data.

Genmab obtains exclusive rights to develop and commercialize novel antibody therapeutics utilizing OBT's IO antibody. Under the agreement, Genmab will be responsible for the future development and commercialization of any products incorporating this antibody. In addition to the upfront payment, OBT can earn development and regulatory milestone payments as well as royalties on any future product sales.

Aspire Acquires Morningside, Eyes International Growth

Aspire Pharma, a portfolio company of H.I.G. Capital, announced the acquisition of Morningside Healthcare and Morningside Pharmaceuticals on 3 October, at undisclosed terms. Both UK-based firms focus on generics, value-added medicines and specialty products – including some branded products – with “highly complementary” portfolios.

Morningside brings over 160 AKUs across more than 80 product families in therapeutic areas such as endocrine, psychiatry, central nervous system, infectious disease and gastrointestinal diseases. The firms noted the combined company will be “one of the largest and fastest growing UK pharma companies” and will better position it for M&A and international growth.

Solid Merges With AavantiBio Following DMD Setbacks

After multiple FDA clinical holds, Solid Biosciences Inc. is moving on from its Duchenne muscular dystrophy candidate SGT-001 and merging with gene therapy firm AavantiBio Inc. in a transaction announced on 30 September. After the deal closes, Solid shareholders are expected to own 85% of the new company, with AavantiBio shareholders owning 15%. Nonetheless, AavantiBio CEO Bo Cumbo will lead the combined company.

In tandem with the merger expected to close in Q4, venture capital firms Perceptive Advisors, RA Capital Management and Bain Capital Life Sciences are leading a $75m financing for the company – which will retain the name Solid Biosciences – enabling it to hit the ground running with about $215m in cash and expected runway into 2025.

Solid’s lead DMD candidate SGT-001 was the subject of a pair of FDA clinical holds in 2018, with the company relaunching a Phase I/II study in 2021 with some promising interim data. (Also see "Solid Restarts DMD Gene Therapy Trial, With Encouraging Interim Data To Back It Up" - Scrip, 16 Mar, 2021.) Now, however, the company plans to move on from ‘001 and advance the next-generation gene therapy candidate SGT-003 in DMD instead.

The combined company also will focus on AavantiBio’s pipeline, including preclinical candidates AVB-202 for Friedrich’s ataxia and AVB-401 for BAG3 Mediated Dilated Cardiomyopathy (BAG3) and other cardiac indications.

Idera Acquires Aceragen In Stock Deal

Idera Pharmaceuticals, Inc. said it completed its acquisition of privately held Aceragen, Inc. on 28 September at undisclosed financial terms. The transaction was an all-stock deal bringing in Aceragen’s pipeline focused on rare, orphan pulmonary and rheumatic diseases.

The two companies’ combined cash is expected to provide runway into Q3 2023, funding the advancement of Aceragen’s pipeline, including ACG-701 and ACG-801, through 2023 clinical milestones. The company estimates annual peak sales potential of $650m from those lead programs.

ACG-701 is a proprietary formulation of sodium fusidate being developed to treat acute pulmonary exacerbations (PEx) associated with cystic fibrosis (CF) and for melioidosis. The Phase II REPRIEVE trial of ACG-701 in CF PEx is expected to begin in Q4, funded in part by the Cystic Fibrosis Foundation. ACG-801 (recombinant human acid ceramidase) is an investigational biologic enzyme replacement therapy being developed for the treatment of Farber disease. The company expects to initiate the potentially registrational ADVANCE study ACG-801 in Q1 2023 with data expected in Q1 2024.

Visus Enhances Ophthalmology Pipeline By Acquiring ViewPoint

Visus Therapeutics, Inc. expanded its ophthalmology pipeline on 28 September via the acquisition of all patents and other assets of ViewPoint Therapeutics, a developer of alpha-crystallin aggregation inhibitors. No financial terms were disclosed for the merger of the two US eyecare-focused biotechs.

Alpha-crystallin aggregation inhibitors are intended to restore elasticity and lens clarity, potentially reversing presbyopia and cataracts without the need for surgery, the companies said. With the acquisition, Visus obtains an extensive library of drug candidates with desirable pharmaceutical properties for ocular delivery. Lead candidate selection is anticipated in the first quarter of 2023.

Visus is currently conducting two Phase III pivotal studies of its preservative-free eyedrops, Brimochol PF and Carbachol PF, for the treatment of presbyopia. The Seattle biotech said the ViewPoint assets are a natural complement to that program and will add to its pipeline targeting the leading causes of age-related loss of vision. AbbVie Inc.’s Vuity (pilocarpine) was the first eyedrop approved for presbyopia, in 2021, but there are several candidates in development. (Also see "With Vuity Approval, AbbVie Continues Stepping Into Eye Care" - Scrip, 3 Nov, 2021.)

In August 2021, Visus enhanced its pipeline by licensing glaucoma and age-related macular degeneration candidates from Cella Therapeutics LLC and partnering on long-acting delivery mechanisms with DelSiTech Ltd. (Also see "Deal Watch: Visus Begins Charting Its Direction In Ophthalmology With Two Deals" - Scrip, 20 Aug, 2021.)

Aquestive Licenses Ex-US Libervant Rights To Pharmanovia

New Jersey-headquartered Aquestive Therapeutics, Inc. licensed rights to a buccal film version of the anti-seizure medication Libervant (diazepam) on 28 September to Pharmanovia in the European Union, UK, Switzerland and Norway, as well as countries in the Middle East and North Africa (MENA). Aquestive gets $3.5m up front under the deal and can earn milestone payments and double-digit sales royalties in Pharmanovia’s territories if the product obtains full approval.

Libervant obtained tentative approval from the US FDA on 30 August after the agency determined that the orphan drug exclusivity granted to Neurelis’s Valtoco (diazepam nasal spray) blocked full approval. (Also see "Keeping Track: US FDA Novel Approvals Return With Xenpozyme, Spevigo Nods; Stimufend Survives COVID-19 Delays" - Pink Sheet, 5 Sep, 2022.) Valtoco’s exclusivity ends 10 January 2027; the product was approved 10 January 2020 for the same indication Aquestive is seeking: acute treatment of intermittent episodes of seizure clusters or acute repetitive seizures in patients on stable antiepileptic drug regimens.

Aquestive will serve as the exclusive manufacturer and supplier of the product, while Pharmanovia will be responsible for all regulatory and commercialization activities. Libervant is a buccally administered film formulation of diazepam, a benzodiazepine intended for the acute treatment of seizure clusters that are distinct from a patient’s usual seizure pattern. Aquestive developed Libervant as an alternative to the device-based products currently available for patients with refractory epilepsy, including a rectal gel and nasal spray products.

Cybin Licenses Psychedelic Class Of Molecules From Mindset

In a deal between Canadian psychedelic therapy-focused firms, Cybin Inc. acquired an exclusive license on 27 September to a class of tryptamine-based molecules from Mindset Pharma, Inc. Pursuant to the agreement, Cybin paid Mindset a license fee of $500,000, with additional milestone payments that could total up to $9.5m along with potential for commercial sales royalties of 2%.

Cybin said the agreement will:

• give it access to a catalog of molecules that complement its current preclinical library, with both complementary chemistry and properties;

• expand its discovery and development platform to provide a potential pipeline of future drug candidates;

• broaden its protected access to patent-eligible molecules and formulations;

• and strengthen its intellectual property reach and scope of protection to support continued investment in psychedelic therapeutics for mental health conditions.

Vernalis, Contera Combine Capabilities In Neurological Disease

Vernalis PLC and Contera Pharma entered into a drug discovery collaboration centered on an undisclosed neurology target on 27 September. The agreement will combine Vernalis's expertise in fragment and structure-based drug design with Contera’s knowledge of RNA-based drug development within neuroscience to design small molecules against a target for a rare neurologic disorder.

All costs will be shared equally until a predefined milestone event, the companies said. If the first stage is successful, the partners can opt to extend the collaboration, with Vernalis entitled to funding, downstream milestones and royalties on candidates resulting from the partnership. Denmark-headquartered Contera will be responsible for the development and commercialization of the candidates. The agreement also includes an option to extend the collaboration beyond the initial target.

Albireo Gets $115m For Royalty Rights To Bylvay

Albireo Ltd. agreed to a royalty monetization deal on 22 September with Sagard Healthcare Partners, obtaining $115m up front in return for a mid-single digit average royalty rate on global annual net revenues of Bylvay (odevixibat). Bylvay obtained FDA approval in July 2021 for the treatment of pruritus in progressive familial intrahepatic cholestasis (PFIC) patients. (Also see "Albireo Dashes Into PFIC Market As FDA, EMA Approve Bylvay" - Scrip, 21 Jul, 2021.)

Previously, Albireo realized $105m in September 2021 from the sale of the rare pediatric disease priority review voucher it was granted upon the FDA’s approval of Bylvay. The AstraZeneca PLC spinout also is earning royalties on Japanese commercial rights to Bylvay under a October 2021 agreement with Jadeite Medicines. (Also see "Asia Deal Watch: Hansoh Teams Up With OliX On SIRNA Therapies" - Scrip, 14 Oct, 2021.)

Under the current agreement, Sagard will receive a 12.5% royalty rate on Bylvay annual net revenues up to $250m with the royalty rate decreasing to 5% for annual net revenues above $250m. The rate then will decrease again to 1% on annual net revenues exceeding $350m if the US FDA approves Bylvay in a second indication, biliary atresia. Total royalties are capped at 1.6x invested capital if repaid by the end of 2028 and 2.0x thereafter.

Stay tuned for the next edition of Deal Watch. You can read more about other deals that have been covered in depth by Scrip and Generics Bulletin in recent days below:

(Also see "Nanopharm And Fluidda Join Forces To Speed Up Inhaled Drug Approvals" - Generics Bulletin, 5 Oct, 2022.)	Nanopharm and Fluidda are collaborating on a computational approach for assessing bioequivalence in inhaled generics. Their project aims to ease the USFDA approvals process for a drug class whose pharmacokinetic properties are notoriously difficult to observe.
(Also see "AstraZeneca’s LogicBio Buyout Will Give Other Troubled Gene Therapy Companies Hope" - Scrip, 4 Oct, 2022.)	Many gene therapies companies have faced R&D and financial problems this year but the longer term potential of the field and low prices might tempt big pharma into deals.
(Also see "Myovant Spurns Sumitovant Offer, Remains Open To Higher-Value Deal" - Scrip, 3 Oct, 2022.)	Sumitovant offered $22.75 per share in cash, valuing Myovant at $2.4bn total, to acquire what it does not hold as a result of its 2019 agreement to buy majority shares of five Roivant companies.
(Also see "Incyte Signals Increasing Focus In Dermatology With Villaris Buyout" - Scrip, 3 Oct, 2022.)	With Opzelura approved for vitiligo in July, Incyte acquires Villaris and its preclinical IL-15-targeted antibody to potentially add to its vitiligo portfolio.
(Also see "Canada’s Apotex Is Snapped Up By SK Capital" - Generics Bulletin, 28 Sep, 2022.)	Apotex is to be acquired by SK Capital, the private investment firm has announced. The Canadian generics giant’s new owner has promised to invest in the firm to “support Apotex’s next phase of growth and continued innovation for patient affordability.”
(Also see "Sanofi ThiNKs Scribe’s CRISPR Approach Can Advance Its NK Cell Therapy Goals" - Scrip, 27 Sep, 2022.)	CRISPR-Cas9 co-inventor Jennifer Doudna-founded gene-editing firm inks its second large collaboration, teaming with Sanofi on off-the-shelf natural killer cell therapies for cancer.
(Also see "ArsenalBio Adds Genentech As A Partner While Embarking On Internal CAR-T Program" - Scrip, 27 Sep, 2022.)	The start-up will secure $70m from Genentech, after closing a series B financing, while it moves its first CAR-T program into the clinic for ovarian cancer later this year.
(Also see "Seagen Digs Deep For Lava’s Bispecific Antibody" - Scrip, 26 Sep, 2022.)	Lava Therapeutics planned to move its preclinical candidate, the EGFR-targeting LAVA-1223, into the clinic later this year. Seagen has shown interest in business development as Merck merger talks have quieted down.