From CRISPR patent wars, IPOs and acquisitions to major deals in China; what would be on your top ten list of 2018?
The opening plenary at Phacilitate Leaders World always involves a review of the top events, milestones and trends of the past year. Being in January, the meeting presents a timely opportunity to reflect, look ahead and set ourselves up for the coming year. Needless to say, the mood was more than optimistic, you might even say jubilant, after a great 2018. The plenary chair, Chris Mason of AVROBIO, declared that "Our field is growing because of very solid science but, more importantly, we’ve got life-changing therapies that are impacting patients and their caregivers."
Susan Nichols, CEO, Falcon Therapeutics delivered the top ten events presentation in place of Ed Field, Susan told us this is what happens when Ed calls and says 'Hey Susan, can you do me a favour..?" Perhaps she'll screen those calls in future!
The presentation began with a recap on the top spot from the last six years, which really illustrates how much progress has been made and the steep growth trajectory that our industry is on...
Now here's the countdown for the top ten industry events in 2018.
10. THE END OF THE CRISPR PATENT WAR
The patent dispute between MIT’s Broad Institute and UC Berkeley, whereby they both claim the original development and full license of CRISPR-Cas9 technology, came to an end this year. The Brock Institute won the US patent and subsequent appeal based on ‘non-obvious invention’ on September 10th 2018. Meanwhile, due to strict European laws, the Broad Institute’s patent was invalidated and the European Patent Office announced its intention to grant a broad patent to the University of California, the University of Vienna and Emmanuelle Charpentier.
CRISPR technology is the cornerstone of gene editing and this is why this has gained so much international attention. Susan Nichols commented “it’s interesting that the US patent office ruled on non-obvious invention and not on original development. As our industry grows, with the abundance of prior art, we’re going to encounter a lot of IP conversations.”
9. THE LARGEST, DEDICATED CELL AND GENE THERAPY MANUFACTURING FACILITY OPENS
In April 2018, Lonza opened the doors to the world’s largest dedicated cell and gene therapy manufacturing centre in Houston, Texas. In fact, this centre is part of a global restructuring of the organisation to create four centres of excellence for cell and gene therapy in strategic locations.
The centre in Texas may have topped the charts in terms of square footage but the trend was seen throughout the CDMO landscape with many expansions taking place.
There’s little sign of a slowdown in demand for manufacturing space, what will 2019 hold?
8. TIME MAGAZINE’S INCLUSION OF CARL JUNE IN TOP 100 MOST INFLUENTIAL PEOPLE
Dr Carl June was recognised for his pioneering work in CAR-T development by Time magazine. He was nominated by Emily Whitehead, the first paediatric patient to receive a CAR-T treatment as part of a clinical trial at the Children’s Hospital of Philadelphia. Emily went into a 14-day coma as a result of the treatment but awoke on her 7th birthday and has barely looked back.
Carl’s inclusion in Time magazine is truly an industry landmark; it means that advanced therapies are becoming ‘a household name’.
Susan Nichols stated “Thanks to people like Emily Whitehead and her foundation, the story is being told. Listening to Emily’s father, Tom, and Bruce Levine during the opening super plenary really brought home that the patients are the ones telling the story, which is no longer just of hope but of life-changing treatment."
7. FDA REPORT ‘BALANCING SAFETY AND INNOVATION FOR CELL-BASED REGENERATIVE MEDICINE’ IN NEJM
FDA commissioner Scott Gottlieb and CBER Director, Peter Marks, published a report titled ‘Balancing Safety and Innovation for Cell-Based Regenerative Medicine’ in the New England Journal of Medicine in March 2018. Both FDA leaders are constantly trying to help with the regulation of the 700+ stem cell clinics that are in operation without the oversight of the FDA.
One of the things the report seeks to do is to clarify and help bring the stem cell industry into compliance. There have been safety issues recorded, even patient lawsuits and multiple warning letters from the FDA have been sent.
At the heart of this is what Peter Marks said during the super plenary i.e. “quality, safety and efficacy are all intertwined.”
6. THE RISE OF DEALS IN CHINA
Despite US-China trade war concerns, the deals we’ve seen in China throughout 2018 have been phenomenal. Of course, China is the second largest pharma market, is very strong in regenerative medicine, has good government support, manufacturing capabilities and a complex regulatory landscape, which makes it an extremely attractive option for partnerships and collaborations.
- Kite/Gilead and Fosun Pharma headed into a joint venture to bring Yescsarta to China - more
- Novartis and Cellular BioMedicine Group have a license agreement for Kymriah - more
- Athersys expanded their collaboration with Healios to include other indications - more
- RepliCel closed an investment deal with Chinese partner YOFOTO - more
5. CRISPR BABIES
On November 28, at the Second International Summit on Human Genome Editing at the University of Hong Kong, Dr He Jianku from South University of Science and Technology in Shenzhen, China announced that he had genetically modified and implanted the first human embryos resistant to HIV and that the gene-edited twin girls were born earlier that month. Needless to say, the shock was widespread and palpable across the scientific community.
If hopeful scepticism is not an oxymoron, it could be used to describe the sentiment of the industry, however, Dr Jianku’s announcement has since been verified. On Monday 21st January, the Chinese government ruled that Dr Jiankui acted on his own, was self-funded and operated outside of national guidelines.
4. TAKEDA-SHIRE ACQUISITION
After just eight months, on December 5th 2018 Takeda acquired Shire for $62bn. Susan joked that “10 years ago we were begging for $1m!”. A testament to the rapid growth of the sector.
This move took Takeda into the global top ten pharma companies, creating a biopharmaceutical leader with its headquarters remaining in Japan. It’s also the largest foreign buy out ever executed by a Japanese firm and gives Takeda access to the lucrative rare diseases Shire pipeline.
3. MODERNA IPO SETS RECORDS AT $604M
2018 was an exciting year for IPOs. Rubius Therapeutics raised $240m in their IPO in Q3 and Allogene Therapeutics’ IPO was described as ‘eye-popping’ by Fortune at $324m.
Then on December 6th, Moderna set a staggering record with an IPO of $604m which was significantly beyond their initial aim of $500m and values the company at $7.5bn. Their pipeline also consists of an mRNA-based treatment platform, rather than the more popular CAR-T and gene therapies we’ve seen in pipelines of biotechs with headline-making IPOs.
Susan told the audience “What’s most interesting about this IPO is that it was in late Q4. Analysts were saying we burned out in Q1-3 and on a downward swing then up pops the largest IPO we’ve ever seen.”
2. TAKEDA/TIGENIX FIRST ALLOGENEIC APPROVAL
TiGenix’s Alofisel® received EU market approval in March 2018. This was a significant milestone for our industry, as it’s the first allogeneic stem cell therapy approval.
Alofisel has been approved for the treatment of complex perianal fistulas in adult patients with nonactive/mildly active luminal Crohn’s disease and has been licensed to Takeda for the exclusive development and commercialization outside of the US. Receipt of the market authorisation triggered a milestone payment from Takeda to TiGenix of €15 million and initiation of the process of transferring MA from TiGenix to Takeda.
1. REIMBURSEMENT CONVERSATIONS TAKE CENTRE STAGE
This is the conversation of 2019. The industry has proven advanced therapies can save lives and now the question is how are healthcare systems going to pay for them?
Susan remarked that "We have changed the treatment paradigm! We’re no longer a pay-as-you-go service, now we’re talking about potentially one-time curative therapies and our healthcare system has to adapt to ensure we get these life-saving therapies to patients."
The scale of this challenge is only going in one direction. Currently, there are over 800 active INDs in cell and gene therapy and it’s expected that there will be over 200 a year by 2020. The FDA has even hired 50 new receivers to handle this workload.
Michael Meyers, T.R Winston plc. said during the super plenary discussions that the conversation is now about how pharma, government and private sector can all work together to devise a plan, both long and short-term, to ensure we have reimbursement. He even suggested that the government could act as a backstop to help with funding.
So, how can we engage the capital market and how can they help us solve this problem?
We were definitely having this conversation last year during a three-part webinar series that is still relevant today and we’ll continue to bring the analysis and opinion of industry leaders to you through the Phacilitate:Exchange community, all year round.
Here’s more detail on that webinar, which you can access on demand.
Examining the approaches to gaining market access in Europe, as well as how to price your product and the lessons learnt from other pricing strategies.
Moderator: Graham Tatham, Director, Simon-Kucher & Partners
Speaker: Dr Thomas Lönngren, Strategic Advisor, NDA Group
Speaker: Panos Kefalas, Head of Health Economics and Market Access, Cell and Gene Therapy Catapult