< PreviousM&A ROUND-UP 10 Pharma Business International www.pbiforum.net Novo Nordisk - Forma Therapeutics Novo Nordisk is set to acquire Forma Therapeutics, a clinical-stage biopharmaceutical company focused on sickle cell disease (SCD) and rare blood disorders, for $20 per share in cash, representing a total equity value of $1.1 billion. The acquisition of Forma Therapeutics, including its lead development candidate, etavopivat, aligns with Novo Nordisk’s strategy to accelerate its scientific presence and pipeline in haemoglobinopathies - disorders in which there is abnormal production or structure of the haemoglobin protein in the red blood cells. Ludovic Helfgott, executive vice president and head of Rare Disease at Novo Nordisk, said: “Novo Nordisk has worked for more than 40 years to develop and deliver transformative medicines to patients around the world with rare and devastating diseases. By adding Forma’s differentiated approach to address unmet needs for patients, we are taking a step forward in enhancing our sickle cell disease pipeline. We have an ambition to build a leading portfolio with standalone and combination treatments to tackle the complications and underlying causes of sickle cell disease.” Etavopivat, an investigational oral, once-daily, selective pyruvate kinase-R (PKR) activator, is being developed to improve anaemia and red blood cell health in people with SCD. Etavopivat is currently being evaluated in a global phase 2/3 clinical trial (Hibiscus) in patients with SCD, and in a phase 2 trial (Gladiolus) in patients with transfusion- dependent SCD and another inherited haemoglobinopathy called thalassemia. Roche - Good Therapeutics Meanwhile, Good Therapeutics has entered into a definitive merger agreement to be acquired by Roche, giving the latter rights to the former’s innovative, conditionally active, PD-1- regulated IL-2 program and an exclusive right to the platform technology for the development of PD-1-regulated IL-2 receptor agonist therapeutics. Roche will make an upfront cash payment of $250 million and additional payments based on the achievement of predetermined development, regulatory, and commercial milestones. The transaction is expected to close in the third quarter of 2022. Setting sights on smaller deals Though sightings of mammoth deals have been lacking since our last issue, smaller acquisitions have been fruitful in the pharma industry, with inorganic growth on the cards as firms look to strengthen their portfolios. 10-13.qxp_Layout 1 10/10/2022 13:21 Page 1Pharma Business International 11 www.pbiforum.net M&A ROUND-UP © stock.adobe.com/ Kenishirotie Following the close of the Roche acquisition, the Good Therapeutics team plans to apply the technology for the design of conditionally active therapeutics to other targets in immuno- oncology and beyond in a new company, Bonum Therapeutics. John Mulligan, Ph.D., founder and CEO of Good Therapeutics, said: “Good Therapeutics was founded to create a new class of conditionally active therapeutics that will be more effective and avoid the problem of systemic immune activation seen with previous versions of such drugs. We have focused on PD-1-IL-2 as a biology that has great potential for benefiting patients. Roche is a leader in immuno-oncology and has pioneered the field of engineered PD-1- targeted IL-2 therapeutics. Given their expertise in this field and broad capabilities in oncology, we believe they are a perfect choice for taking this important program forward.” James Sabry, M.D., Ph.D., global head of Pharma Partnering at Roche, added: “We are excited to bring Good’s innovative PD-1-regulated IL-2 program into our existing oncology pipeline, which nicely complements our efforts on next- generation PD-1-targeted IL-2 therapeutics and our broader oncology strategy of providing cancer patients with innovative solutions and improving health outcomes. With our proven track record in cancer immunotherapy, we are well- positioned to leverage our deep discovery, development, clinical, and manufacturing capabilities and worldwide reach to potentially bring innovative products from this program to patients as fast as possible.” Incyte - Villaris Therapeutics Incyte is swooping for Villaris Therapeutics, an asset-centric biopharmaceutical company seeded by Medicxi and focused on the development of novel antibody therapeutics for vitiligo. Its lead asset, auremolimab (VM6), an anti-IL-15R? monoclonal antibody (mAb), is expected to enter clinical development in 2023. Incyte will acquire Villaris and the exclusive global rights to develop and commercialize auremolimab for all uses, including in vitiligo and other autoimmune and inflammatory diseases, 12 Á 10-13.qxp_Layout 1 10/10/2022 13:22 Page 2M&A ROUND-UP 12 Pharma Business International www.pbiforum.net making an upfront payment of $70 million. And Villaris shareholders will be eligible for up to $310 million upon achievement of certain development and regulatory milestones, as well as up to an additional $1.05 billion in commercial milestones on net sales of the product. “This acquisition complements our current portfolio, providing us the opportunity to further enhance treatment options for people with vitiligo, leveraging the expertise we established in the Dermatology space through our successful launch of Opzelura (ruxolitinib) Cream,” said Hervé Hoppenot, Chief Executive Officer, Incyte. “People living with immune-mediated dermatologic diseases like vitiligo face significant challenges and we are committed to developing novel therapies like auremolimab, which may address these unmet needs.” Rocket Pharmaceuticals - Renovacor Rocket Pharmaceuticals is looking to snap up biotechnology company Renovacor in a $53 million all-stock equity deal, with the transaction expected to close by the first quarter of 2023. Renovacor’s most advanced program, REN-001, is an AAV-based gene therapy targeting BAG3- associated dilated cardiomyopathy (DCM), a severe form of heart failure. BAG3-DCM represents a significant unmet medical need in a patient population with rapidly progressive cardiac dysfunction in whom no treatments targeting the underlying mechanism of disease exist. “The acquisition of Renovacor aligns with our strategy to expand our leadership position in AAV-based gene therapy for cardiac disease and gives us a perfect opportunity to continue on our mission to transform the lives of heart failure patients through the power of gene therapy,” said Gaurav Shah, M.D., Chief Executive Officer of Rocket. “Building on our success in Danon Disease to date, I am particularly excited to expand our cardiology focus and capabilities and address a clear unmet medical need in BAG3-associated dilated cardiomyopathy. By combining Renovacor’s compelling preclinical work with our joint clinical, regulatory and CMC expertise, we believe we will be well-positioned to bring the highest impact gene therapy with the best chance for success to these patients in the most productive and efficient manner possible.” GENFIT - Versantis GENFIT, a late-stage biopharmaceutical company “dedicated to improving the lives of patients with severe chronic liver diseases,” has entered into an exclusivity agreement to acquire all the share capital and voting rights of Versantis, a private Swiss-based clinical stage biotechnology company focused on addressing the growing unmet medical needs in liver diseases. The $109 million deal fits within GENFIT’s strategic vision of becoming a global leader in ACLF (acute-on- chronic liver failure), and involves the integration of a clinically advanced asset presenting a solid scientific rationale supported by encouraging Phase 1b and preclinical data. GENFIT will also further expand its pipeline in other liver diseases characterized by high unmet medical needs with additional product candidates developed by Versantis. Versantis’ main asset, VS-01, is a first-in-class innovative liposomal- based therapeutic product candidate currently in clinical development as a potential first-line therapy for the timely recovery of ACLF and urea cycle disorder (UCD). A planned 60- patient, randomized and controlled Phase 2 Proof-of-concept trial of VS- 01 in ACLF is expected to launch in the fourth quarter 2022. 10-13.qxp_Layout 1 10/10/2022 13:22 Page 3Pharma Business International 13 www.pbiforum.net M&A ROUND-UP © stock.adobe.com/nazif 10-13.qxp_Layout 1 10/10/2022 13:22 Page 4TRACEABILITY 14 Pharma Business International www.pbiforum.net Tracked and traced © stock.adobe.com/Melinda Fawver The traceability market is experiencing high levels of growth, driven by a need to combat ever more pervasive counterfeit drugs, and the ever more cunning criminals that are pushing them to market. 14-17.qxp_Layout 1 10/10/2022 13:22 Page 1Pharma Business International 15 www.pbiforum.net TRACEABILITY © stock.adobe.com/pikselstock T raceability is taking the world by storm – in food, clothing and in pharmaceutical product. In the pharma industry perhaps more than any other (barring food) traceability is most reasonable, both as a means of ensuring the safety and hygiene of product, but also in combating counterfeit drugs. It’s little surprise then that the market continues to grow, with studies suggesting it should grow by over $9.7 billion between 2021 and 2026 alone. By far the most common type of traceability employed in pharmaceuticals is the barcode; it is hardly unique in this sector, but reliable and easily included on packaging to enable accurate and affordable traceability. Less expensive than RFID, barcodes on pharma products are being increasingly adopted by Governments around the world and 16 Á 14-17.qxp_Layout 1 10/10/2022 13:22 Page 2TRACEABILITY 16 Pharma Business International www.pbiforum.net put into legislation. This is, again, to combat one of the biggest concerns in the world of counterfeit drugs. The counterfeit market remains a pervasive and troubling one, and it isn’t limited to less developed nations as some figures would have us believe. Counterfeit cancer and rare disease drugs target vulnerable people who may well be new to their situation, and who are inexperienced in telling genuine from counterfeit. It hardly helps that counterfeiters have become increasingly adept at mimicking packaging in recent years. During the COVID-19 pandemic, the counterfeit industry saw yet another opportunity to profit, and released a wave of counterfeit vaccines and drugs to the market, targeting people’s fear and paranoia, and further exacerbating the problems the industry faces in securing the trust of the populace. With counterfeiters profiting more than ever, the supply of counterfeit drugs has increased, driving the push toward heightened traceability and – perhaps most importantly – awareness of this. It is no use after all to have anti-counterfeit measures if consumers are not aware of them, so informing is just as important as having in this case. Though many are also looking to blockchain technology and its potential in the traceability market, this remains a niche application. It is growing in popularity, however, due to the comparable safety of serial codes stored within, and the ability for brands to trust in the absolute security of the content within. It is expected that the 14-17.qxp_Layout 1 10/10/2022 13:22 Page 3Pharma Business International 17 www.pbiforum.net TRACEABILITY usage of blockchain will grow in the coming years to combat malicious hackers especially. As pharmaceutical companies scramble to increase traceability on packaging, online and even in some cases on the medicine itself, those in the industry are also warning them not to forget case labelling. Wholesalers and distribution centres may well be left in the dark, and unable to read serial numbers or barcodes in a busy warehouse environment, especially if fragile goods are pallet-wrapped or shrink-wrapped, obscuring them. If the labels are unable to be read then there is a heightened risk of a wholesaler waving through product that might be counterfeit, and at that point the end- user may well believe they don’t need to double check as the wholesaler will have done it already. In law, traceability is the responsibility of the manufacturer, and thus any problems or errors will be traced back to the very source. It’s not just a matter of self-interest when legal matters are involved, and as governments across the world scramble to combat counterfeit drugs putting their vulnerable at risk, it’s expected the legislation will become even more stringent. This will drive traceability further, but it will be a forced change for many. It may be time to look more into blockchain, given it is at the highest end of technology, but even that needs to be backed up by physical labels, both on the packaging and on the transit cases. © stock.adobe.com/BabelFX 14-17.qxp_Layout 1 10/10/2022 13:22 Page 4MACHINERY AND EQUIPMENT 18 Pharma Business International www.pbiforum.net © stock.adobe.com/Mike Mareen Dealing with demand Dealing with demand 18-21.qxp_Layout 1 10/10/2022 13:23 Page 1Pharma Business International 19 www.pbiforum.net MACHINERY AND EQUIPMENT T he manufacturing of medications themselves, with multiple stages and continuous processing to meet demand, requires especially close focus on detail. One of the first and most important of these is choosing the most effective machines for each task. Careful consideration in this area not only upholds efficiency on the factory floor, but provides peace of mind, and the confidence that each machine will carry out tasks with minimal need for monitoring or maintenance. Tablet Processing Pharmaceuticals can be processed in many forms, including liquid suspensions, powders, and aerosols. However the reliable tablet or capsule is the most common means of dosing and administering medications, as these are convenient for controlling dosage levels, as well as being easy to self-administer. Before pressing a tablet into its neat shape, or filling a capsule, it is often important to prepare the pharmaceutical powders for granulation. In some cases, well-mixed powders won’t require granulation and can skip straight to the compression stage. But otherwise, there’s a choice to be made between wet and dry granulation, and many machines are available to achieve both. Granulation Fluidised bed granulators are some of the most used in the pharma industry, combining mixing, granulation and drying into one step along the production line. The gas plenum is the inlet for fluidisation gas or air. The gas is then distributed through the product container, agitating the bed of solid particles until they behave the same similarly to a fluid substance. This ensures the thorough separation of solid particles, resulting in an even distribution of pharmaceuticals through the final product. Though the fluid bed technique is commonly used, it’s far from the only option. High shear granulation machines use an agitator blade for the high- powered mixing of pharmaceutical ingredients, combining this effect with a chopper for the prevention of granule overgrowth. The chopper has the dual effect of distributing granulation fluid, for the same even results as the fluid bed. Single-Pot Granulation is an equally effective agitator blade system, which enables mixing alongside high-shear wet granulation and drying. The pharmaceutical industry is a high-demand one, not only at the consumer end but at every manufacturing and processing stage along the way. Attention to quality and cleanliness is crucial, from mixing and blending to preparing dosages, and all the way through to packing and transport. 20 Á 18-21.qxp_Layout 1 10/10/2022 13:23 Page 2Next >