With the aspiring vision of delivering transformative innovation in the areas of gene therapy and neuroscience, Novartis has enteredinto an agreement to acquire AveXis, a clinical stage gene therapycompany, with focus on rare and critical diseases with a lead candidate for Spinal Muscular Atrophy (SMA). With a deal value of USD 8.7 Billion, Novartis will get access toAveXis’s prime candidate AVXS-101, potentially the first one time gene replacement therapy for SMA, a debilitating disease anda leading genetic cause of death in infants.While AVXS-101 is granted breakthrough therapy designation by FDA for SMA type 1, scheduling it under orphan drug category, the BLA will be filled in H2 2018. For EU and Japan, the product has received PRIME and Sakigakedesignations and submission is planned for 2019 and late 2018 respectively.
This comes in line of several strategic moves made by Novartis to solidify its leading position in the emerging field of cell and gene therapy. While its journey began with bagging the first FDA approval for cell therapy Kymriah, prior to the Avexis deal, earlier this year, Novartis also closed an exclusive ex-US worldwide licensing agreement with Spark Therapeutics for Luxturna, the only approved product that delivers a gene RPE65 into a patient’s retinal cells, in turn to produce a protein to restore vision loss.

Light adaptive contact lens became a reality this month, as Johnson& Johnson Vision Care’s Acuvue Oasys received FDA clearance for its Contact Lenses with Transitions Light Intelligent technology.The working principle behind this new technology is a photochromic additive that reconstructs the amount of visible light filtered to the eye based on the amount of UV light to which they are exposed, resulting in slightly dark colored appearance of lenses in bright sunlight which returns to stable stage upon exposed to normal or dark light. This is a significant technology development given the difficulty of applying photochromatic dye coatings uniformly to the delicate, soft surface of a contact lens. The Acuvue Oasys soft Contact Lenses is 510(k) cleared which is a pre market submission made by device manufacturers to the FDA to demonstrate that the new device is substantially equivalent to a legally marketed predicate device.Indications for use of these contact lenses with transitions light intelligent technology are for people with non-diseased eyes who are nearsighted (myopia) or farsighted (hyperopia) for daily usage.Market response to this new product will be only evident in early 2019 when Acuvue with transitions will be commercially available. The contact lens doesn’t cover the entire eye and hence doesn’t offer the same level of protection as photochromic sunglasses. The visual impact of darkening of the contact lens and appeal to the user will again only be known when the product is commercially available. While J&J refers to the innovation as ‘being born out of deep research into consumer lifestyle’, a year forward will reveal if this is extreme consumerism pervading B2C medical devices or a clinically relevant technology breakthrough.

Alexion Pharmaceuticals struck an acquisition deal with Wilson therapeutics AB, aSwedish pharmaceutical company developing therapies to treat rare copper mediated disease. The deal provides Alexion access to lead candidate in pipeline WTX101,a Phase 3 orphan drug for the treatment of Wilson disease which is a rare inherited disorder wherein copper accumulates in vital organs. FDA has given fast-track tag to the study after analyzing phase 2 data generated in 24-weeks. Current practice follows metal chelators to remove serum copper and requires maintenance with zinc to prevent re-accumulation. WTX101 on the other hand provides an alternate copper-protein transport mechanism with instant formation of copper-protein complexes, with very high specificity for copper. It de-toxifies free copper in the liver and blood, thus reducing copper overload by stimulating biliary excretion. This USD 855 Million cash deal transaction reinstates ever-growing interests in high value rare disease therapy. Contrastingly, GlaxoSmithKline has announced divestiture of its rare disease gene therapy portfolio to Orchard Therapeutics in order to focus R & D efforts on prime assets such as treatments for respiratory conditions, HIV and infectious diseases.In exchange GSK will get a 19.9 per cent stake in Orchard and a seat on its board, along with royalties and milestone payments from the treatments. Named Strimvelis, the treatment involves usage of patient’s own stem cells used for treating inherited genetic disorders that are extremely vulnerable to infections. While companies continue to covet high value rare disease therapies, greater selectivity is emerging and companies are seen to be exercising more caution and taking calculated risks in nature of assets pursued.

At the outset of world immunization week 24 to 30 April French Pharma giant Sanofi Pasteur announced their investment worth USD 431.5 Million in a vaccine manufacturing plant in Canada.Despite of setbacks caused from regulatory issues surrounding Dengvaxia, the company has always enjoyed good leadership in the global vaccines arena and is positioned well for growth in the coming years. Sanofi’s first Hexavalent vaccine is a well-received product across the globe and has seen substantial commercial success. Envisioning increasing demand in future as well as Institutional adoption by WHO, Sanofi has decided to invest in 1,50,000 square ft bulk biologics facility to manufacture 5 component acellular pertussis antigen as well as antigens for diphtheria, tetanus and whooping cough. The facility is set to open by 2021. This further expands Sanofi’s vaccine development and manufacturing footprint in Canada, a country where it has enjoyed strong technology development backbone in combination vaccines.
Similar offshore investment in vaccine expansion was announced by Merck earlier this month. Merck has development plans for vaccine manufacturing plant in Ghana making it the first country in sub –Saharan African region to be equipped with a human vaccine facility. This initiative is an important stepping stone in this part of the world which despite high volume-requirements, remain entirely import dependent for vaccines. In the context that vaccine manufacturing capacity today is largely concentrated in a handful of countries, this is a positive move, as high incumbent capacity is beneficial to the country. India is a classic example, where high indigenous vaccine success has made the country almost self-sufficient in its UIP needs and played a significant role in advancing the country to the cusp of GAVI graduation in the near future. Long term impact of Merck’s investment on the Sub-Saharan African region will depend on sustainable capacity development in the region in biologic manufacturing and catalytic impact of this announcement in attracting manufacturing investments by other vaccine companies as well.

Sanofi has entered into an exclusive negotiation contract with PE firm Advent international to acquire the former’s European generic business Zenvita for USD 2.35 Billion, which could result in Advent creating a new independent entity in the European generics market. The opportunity was much coveted and was being evaluated by a number of industry majors and funds for the past several months.This comes in the backdrop of big pharma companies strategically offloading their non-core assets and businesses to focus and strengthen on their core business. Several such efforts have been executed in the recent past including majors such as Merck and Novartis. Merck sold its consumer-health business to Procter and Gamble and products included in the USD 4.3 Billion deal are vitamins, Femibion supplements for women, Seven Seas cod liver oil and Nasivin nasal decongestant. At the other end, just before its Avexis buyout, Novartis was seen to execute the sale of its 36.5% stake in its consumer-health joint venture with GlaxoSmithKline for USD 13 billion, clearing re-directing its focus in frontier science of cell and gene therapy. We anticipate continued momentum in this trend of larger pharma companies trimming to the core focus areas and deepening investments only in prioritized product categories and geographies.