The Cell & Gene Therapy Market Matures Wave of Collaborations Accelerate Vaccine Development Dealmaking in 2020 Faces New Ch
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June 2020 biopharmadealmakers.nature.com — Nurturing biopharma partnerships The cell & gene therapy market matures Wave of collaborations accelerate vaccine development Dealmaking in 2020 faces new challenges Originally published as an advertising feature in the June 2020 editions of DRUG DISCOVERY PLATFORM THE WORLD OF FULLY HUMAN ANTIBODIES et u Me s! BIO DIGITAL CONVENTION June 8–12 CONTACT US TO DISCUSS [email protected] YOUR NEEDS! www.yumab.com June 2020 FEATURE B3 Next-generation therapeutics sustain momentum The next-generation therapeutics space has continued to progress on several fronts in the past 12 months, but this could be set to change as the industry copes with the COVID-19 crisis. PROFILES B6 Elicio Therapeutics B14 Novadip Biosciences Cover image Gene therapy conceptual B8 NovaGo Therapeutics B15 Aruvant Sciences illustration; a double stranded DNA (deoxyribonucleic acid) molecule coming B9 Biovian B16 Antapodia out of a viral capsid. Science Photo Library / Alamy Stock Photo. B10 Cellatoz Therapeutics Nanotherapeutics BioPharma Dealmakers is a quarterly B11 inRegen B17 MiCAN Technologies publication that facilitates partnering by profiling the activities of organizations B12 Synplogen in the biopharma field, including biotech companies, global biopharma companies and academic institutions. Profiles of organizations are aligned with FEATURE relevant editorial features that cover different therapeutic and technological themes in each issue. B18 Partnering for a pandemic Note Companies that appear in this History has shown that collaborative efforts can have a crucial role in combating issue have paid for their advertisement viral outbreaks such as the current coronavirus pandemic. features and have final approval of their content. If you would like to appear in the next BioPharma Dealmakers PROFILES please contact Claire Thompson: [email protected] B20 GSK Vaccines B23 TAXIS Pharmaceuticals B21 Amicoat B24 Atriva Therapeutics Dealmakers team Editor Raveena Bhambra B22 Sanofi Pasteur Editorial Assistant Georgia Francis Profile Writers Emma Dorey, Suzanne Elvidge, Dan Jones, Jackie Kelly, Mary Lee Mackichan, Gaspar Taroncher- Oldenburg, Nick Taylor FEATURE Feature Writers Raveena Bhambra, Paul Verdin B25 Dealmaking in 2020: navigating a new landscape Production Simon Fenwick, Richard Thomas After a slow start to the year, dealmaking for the rest of 2020 is likely to be Head of Business Development profoundly affected by the COVID-19 pandemic. Claire Thompson Business Development Manager Verónica Zacatenco PROFILES Account Executives Rebecca Djaic, Carmen Ramirez B27 D&D PharmaTech B31 Panorama Medicine Marketing Kimberly Petit, Jack Holloway Publisher Richard Hughes B28 nference B32 Henlius Biotech B30 Noveome Biotherapeutics Human iPSC derived glutamatergic neurons for research and HTS When cells need precision and consistency www.bit.bio | [email protected] FEATURE Next-generation therapeutics sustain momentum The next-generation therapeutics space has continued to progress on several fronts in the past 12 months, but this could be set to change as the industry copes with the COVID-19 crisis. Vector Stock Gulenok / Alamy Vitalii Credit: Paul Verdin In April 2019, the combined 2024 sales forecast for the cell, that affect cell therapies in general. It is also too soon to assess the gene and nucleic acid therapy market was $41 billion, accord‑ impact of the COVID‑19 pandemic, which could dramatically ing to EvaluatePharma sell‑side consensus forecasts (Biopharma affect future forecasts. Dealmakers B15–17, June 2019). One year on, we analysed the area again, and here we highlight the latest trends in market fore‑ Key approvals casts, approvals, development pipeline focus, leading companies The past 12 months have kept up the recent approval momentum and dealmaking for next‑generation therapeutics. in the field. Among the new products with the greatest expecta‑ tions is AveXis/Novartis’s Zolgensma (onasemnogene abeparv‑ Fluctuating forecasts ovec), which became the first US Food and Drug Administration Although clinical progress has boosted the number of next‑ (FDA)‑approved gene therapy for spinal muscular atrophy (SMA) generation therapeutic products on the market since 2019, our in May 2019. This approval also set up an interesting dynamic analysis of the same landscape puts the overall forecast value in within the next‑generation therapeutics space, with Zolgensma 2024 at $38 billion—a 7% contraction of commercial expecta‑ launching into a market already occupied by Biogen’s Spinraza tions (Fig. 1). (nusinersen, an antisense oligonucleotide). Both therapies target Forecasts fluctuate of course, and volatility in forecast com‑ the underlying cause of SMA, but Zolgensma is theoretically a mercial performance is to be expected in such a pioneering area. ‘one‑and‑done’ treatment, while Spinraza requires repeated Looking beyond the headline value, however, reveals that forecasts administration. Zolgensma has checked Spinraza’s growth and for DNA and RNA therapeutics (such as antisense oligonucle‑ is forecast to be the leader in an increasingly crowded SMA market otides) and gene therapy have remained relatively stable—cell by 2024, with sales of $2.04 billion compared with $1.57 billion therapy is where the largest declines are apparent, as forecasts for Spinraza. have eroded by almost $3 billion (or approximately 20%) since Vyondys 53 (golodirsen), the second of Sarepta Therapeutics’ this time last year (Fig. 2). It is too soon to say whether this is the growing portfolio of exon‑skipping antisense oligonucleotides beginning of a cooling‑off of commercial expectations for some for Duchenne muscular dystrophy (DMD), was approved by cell therapy approaches. However, perhaps it could reflect grow‑ the FDA in December 2019. And in Europe, there was a first ing recognition of the strong competition in some areas such as approval for bluebird bio, with a European Medicines Agency blood cancers, as well as the access and affordability challenges (EMA) nod for Zynteglo (autologous CD34+ cells encoding the 40,000 18,000 2024 (April 2020) 35,000 Genome editing 17,000 Gene therapy 2024 (April 2019) 30,000 DNA and RNA therapeutics 16,000 Gene-modified cell therapy 25,000 Cell therapy 15,000 20,000 14,000 15,000 13,000 10,000 12,000 Sales forecast ($ million) Sales forecast Sales forecast ($ million) Sales forecast 5,000 11,000 0 10,000 2020 2021 2022 2023 2024 Gene therapy Cell therapy DNA and RNA therapeutics Fig. 1 | Sales growth forecasts of cell, gene and nucleic acid Fig. 2 | Changes in sales growth forecast trends of cell, gene therapy products from 2020 to 2024. Source: EvaluatePharma, and nucleic acid therapy products in 2024. April 2020. Source: EvaluatePharma, April 2020. biopharmadealmakers.nature.com | June 2020 | B3 FEATURE Duchenne muscular dystrophy Spinal muscular atrophy Non-Hodgkin lymphoma Amyloidosis Multiple myeloma Hemophilia A Acute myeloid leukemia Melanoma Sickle cell disease Hemophilia B Huntington disease Thalassemia Chronic heart failure Limb-girdle muscular dystrophy Hyperlipidemia Porphyria Parkinson disease Epidermolysis bullosa 2024 (April 2020) Urinary calculi/kidney stones 2024 (April 2019) Amyotrophic lateral sclerosis 0 500 1,000 1,500 2,000 2,500 3,000 3,500 4,000 4,500 Sales forecast ($ million) Fig. 3 | Top 20 indications in the field of cell, gene and nucleic acid therapies, based on 2024 sales forecasts. Source: EvaluatePharma, April 2020. βA‑T87Q‑globin gene) for patients with β‑thalassemia, as well as marketed (Zolgensma and Spinraza), and >99% of the 2024 forecast an EMA approval for Akcea/Ionis’s Waylivra (volanesorsen), an of $3.6 billion is contributed by these marketed products. antisense oligonucleotide therapy for familial chylomicronemia The risk inherent in these forecasts is therefore very different, syndrome. even without factoring in uncertainty around commercial per‑ The scale and diversity of progress being made in the next‑ formance once on the market. Across the top 20 indications by generation therapeutics space is a testament to the industry’s 2024 sales, only 6 are currently validated to the extent of regula‑ ability and effort in developing novel science into commercial tory approval and so clearly the space will continue to be highly products. Across the industry, EvaluatePharma lists >5,300 active dynamic. R&D programs in cell and nucleic acid medicine, an increase of 6% on the same analysis 12 months ago. Just over 2,000 of these Companies taking the lead next‑generation therapeutic programs are in clinical development This dynamism is also reflected in the company rankings (Fig. 4). (an increase of 8% over 12 months), with more than 200 programs This time last year Sarepta led the pack in terms of 2024 forecast in phase 3 trials or at the regulatory filing stage. sales, and few major biopharma players featured in the top 20 by forecast revenues. Again, only 5 of the top 20 companies are Leading indications large biopharma: Novartis, Bristol‑Myers Squibb (following the Current clinical development pipelines in the field have a large acquisition of Celgene), Biogen, Gilead and Roche. In this year’s focus on oncology, with 16 of the top 20 indications in cancer. The analysis, however, Novartis leapfrogs Sarepta—albeit margin‑ most studied non‑oncology indications are rare ophthalmology ally—to become the top‑ranked company in the field, with sales conditions, Parkinson disease, osteoarthritis and peripheral vas‑ forecasts of $4.25 billion versus Sarepta’s $4.14