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Top Ten Events in Advanced Therapies 2020

 From a pandemic and record-breaking vaccine approvals to FDA rejections and a Nobel Prize; Phacilitate's annual top ten list is being delivered slightly differently this year...
What. A. Year.

The pace of scientific innovation and clinical development has been the mainstay of headlines around the world this year; it’s been simultaneously exhausting and invigorating! In addition, biologics manufacturing etc. has been a positive thing, overall. Our hope is that has inspired the future leaders of our cell and gene therapy industry and beyond.

Of course, 2020 has not just been about the race for a Covid-19 vaccine but it has, understandably, dominated the news. It’s also the reason this list of top ten events is not being delivered to you live from Advanced Therapies Week in Miami. The whole Phacilitate team are devastated not to be there with you (London in January is not so inviting!) but we’re looking onwards and upwards to June and welcoming you to the new home of ATW at Miami Beach Convention Center.

We did a quick mid-year review back in the summer and were blown away by some incredible financing deals. In the six months since, there have been even more noteworthy events; some good, some that have given us an opportunity to reflect, learn and improve. 
Yes, it’s time for Phacilitate’s Top Ten events of 2020.
Drum roll please…

10. ASH Readouts

Advanced therapy biotechs took the lion’s share of investment after ASH readouts in December 2020.

CRISPR Therapeutics and Vertex Pharmaceuticals rose on the stock market following the results from their first-in-human study of one-time CRISPR/Cas9 gene editing treatment, CTX001, in patients with beta thalassemia and sickle cell disease. The presentation at ASH showed that a total of 10 patients remained free from a once-routine need for blood transfusions and were rid of painful events related to their diseases.

Fate Therapeutics surged as much as 35% after positive updates for a pair of key therapies, FT516 and FT596. The update for an early trial of FT516 combined with rituximab in B-cell lymphoma showed some patients were responding to the treatment. The results from the early-stage study of FT596 also showed patients were improving after a second dose.

9.   Manufacturing capacity continues to expand

The manufacturing capacity bottleneck in our industry is well documented and there is no slowing of the pace to continue to increase this capacity. This was particularly pertinent in 2020 as competition for capacity and raw materials rocketed due to the development and mass manufacture of several coronavirus vaccines. Four major CMOs and CDMOs announced expansions, here’s a timeline:

JUN: FujiFilm announces further $928m expansion of ex-Biogen facility in Denmark with plans to double its capacity after spending $890 to acquire the site in 2019.

AUG: VGXI has started designing a gene therapy and DNA vaccine manufacturing facility in Conroe, Texas that it says will be operational in early 2022.

NOV: Cognate Bioservices expands manufacturing capacity and supply chain infrastructure in Memphis

DEC: KBI Biopharma expands its US operations with new $150 million commercial manufacturing facility based in RTP, North Carolina
We also saw significant consolidation across the CDMO landscape, providing further proof of the need for increased capacity as well as the maturation of the advanced therapies industry, as more therapies move towards commercial-scale manufacturing needs.

The Catalent acquisition of MasTherCell for $315m in February was big news in the mid-year review, extending Catalent’s global reach in the US and Europe. Since then, there have been two additional CDMO acquisitions.

In March, AGC Inc. launched a voluntary tender offer to obtain all issued shares in MolMed amounting to 240 million Euros. The acquisition completed on 31st July 2020 and in October 2020 MolMed became AGC Biologics S.p.A. It is now one of very few CDMOs offering both plasmid production and end-to-end cell and gene therapy services.

Charles River announced a definitive agreement to buy HemaCare for $380 million in cash on December 16th 2020. The acquisition had a strategic rationale, outlined in a statement from Charles River, to ‘expand Charles River’s scientific capabilities in the emerging, high-growth cell therapy sector; create a comprehensive cell therapy solution from discovery through commercialization, which will enhance client retention and accelerate biopharmaceutical clients’ speed-to-market; and provide a compelling value proposition for both clients and shareholders.’

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8.    Cell therapy IPOs are out of this world! 

Stellar IPOs were on the top spot of the 2020 mid-year review and we’ve continued to see amazing financial results for the rest of 2020. The largest IPO still goes to Chinese biotech, Legend Bio, with a truly legendary (sorry!) $424 million raised from 18.3 million shares after their ASCO myeloma data from the Phase Ib/II CARTITUDE-1 study.

In addition to Poseida (CAR-T), Nkarta (NK Cells), Generation Bio (gene therapy), Inventiva (pan PPR-agonist) and Philadelphia-based Passage Bio (gene therapy), the end of year frenzy has seen four more biotechs jump onto Nasdaq; Seer, Sigilon Therapeutics, Silverback Therapeutics, and Kinnate Biopharma raked in $175 million, $126 million, $241.5 million and $240 million respectively.

7.    FDA rejects Biomarin’s Haemophilia A gene therapy amid durability concerns

In August, BioMarin announced that the FDA issued a Complete Response Letter (CRL) in response to their Biologics License Application for valoctocogene roxaparvovec, a gene therapy for severe haemophilia A.

A statement released by BioMarin explained the FDA’s rejection was centred on durability concerns. After finding significant differences between phase I/II and phase III data meant the FDA could not rely on phase I/II data to establish durability, they introduced a new requirement for two years of phase III data ‘to provide substantial evidence of a durable effect using Annualized Bleeding Rate (ABR) as the primary endpoint.’

The phase III study began in November 2019 and the last patient will complete two years of follow up in November 2021. So, we may well be discussing this at next year’s top ten events presentation…watch this space…

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6.    Mesoblast’s highs and lows

Another Complete Response Letter made headlines in October, this time, Mesoblast were on the receiving end. Despite the FDA’s Oncology Drugs Advisory Committee voting nine to one that the available data supports the efficacy of Ryoncil and in favour of approval, the FDA want more data.

A statement released by Mesoblast stated that ‘the FDA recommended that Mesoblast conduct at least one additional randomized, controlled study in adults and/or children to provide further evidence of the effectiveness of remestemcel-L for SR-aGVHD.’. There were also some question marks around the
relationship between potency and the drug’s biological activity.

Mesoblast’s Ryonicil is for the treatment of paediatric steroid-refractory acute graft versus host disease (SR-aGVHD), with no other currently approved treatments for this life-threatening condition in children under 12.

Mesoblast hit by FDA rejection, request to run another trial

However, just weeks later in November, Novartis licensed the stem cell therapy with a deal that certainly brightened the spirits of Mesoblast investors. As part of the deal, Novartis will shell out $25 million in upfront cash and take a $25 million stake in the biotech, while offering up to $1.255 billion in potential milestone payments.

5.    Lily buys Prevail for $1.04 billion

Lilly announced its agreement to buy Prevail for $1bn on 15th December 2020. Prevail’s neuroscience heavy portfolio will bolster Lilly’s commitment and standing in the gene therapy space, continuing the trend in backing AAV-based gene therapies.

The deal consists of $22.50 per share plus a contingent value right of up to $4 a share, which is dependent on the first regulatory approval from Prevail’s pipeline.

4.    Emmanuelle Charpentier and Jennifer Doudna awarded the 2020 Nobel Prize in Chemistry for CRISPR

CRISPR technology has been blighted with patent wars in recent years, but this is cause for celebration. The ‘godmothers’ of CRISPR, Jennifer Doudna and Emmanuelle Charpentier, have been duly awarded the 2020 Nobel Prize for Chemistry. Another testament to the power of partnerships and a source of huge pride for our industry.

“There is enormous power in this genetic tool, which affects us all. It has not only revolutionised basic science but also resulted in innovative crops and will lead to ground-breaking new medical treatments,” says Claes Gustafsson, chair of the Nobel Committee for Chemistry.

3.    Bayer-ASKBio deal totals $4 billion

On October 26th 2020, Bayer acquired AskBio, based in North Carolina’s Triangle Research Park. The deal consisted of a $2 billion upfront payment and another $2billion in potential milestone payments, which follows Bayer’s 2019 acquisition of BlueRock Therapeutics in a bid to strengthen their gene therapy pipeline. AskBio are industry leaders in AAV-based gene therapies and have phase III candidates for the treatment of Pompe disease, Parkinson’s and congestive heart failure.

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2.    Bayer continues to strengthen its standing in cell and gene therapy with Atara $600m alliance

It’s not often we see the same company featuring in succession but Bayer’s alliance with Atara is significant, as it backs two areas of cell therapy that are yet to be realised; solid tumours and off the shelf, allogeneic therapies. The commercial potential of these two types of treatment are huge.

In December, Bayer acquired rights from Atara Biotherapeutics to two cell therapies for solid tumours, including mesothelioma and non-small cell lung cancer.

As part of the deal, Bayer gets a non-exclusive right to negotiate deals for additional Atara CAR-T programs and could shell out a further $610 million in conditional payments.

1.      mRNA has its day

This could be the biggest event of 2020 on any sector’s list! A truly exceptional moment in our history in so many ways.

BioNTech pivoted from its substantial oncology-centred pipeline to apply its mRNA technology to the creation of a coronavirus vaccine, in partnership with Pfizer. It was not only the first approved Covid-19 vaccine but also the first mRNA vaccine to ever be approved for human use. The UK regulator, MHRA, were the first to approve the vaccine on 2nd December 2020.

Moderna followed suit with its vaccine approved first by the FDA on 18th December.

Moderna and Catalent Announce Collaboration for Fill/Finish Manufacturing of Moderna's COVID-19 Candidate 

Let’s take a look at a summary of the top ten events in 2020:
10. ASH readouts
9. Manufacturing capacity continues to expand
8. Cell therapy IPOs are out of this world! 
7. FDA rejects Biomarin’s Haemophilia A gene therapy
6. Mesoblast’s highs and lows
5. Lily buys Prevail for $1.04 billion
4. The Nobel Prize for Chemistry goes to….CRISPR
3. Bayer-ASKBio deal totals $4 billion
2. Bayer-Atara $600m alliance
1. mRNA has its day