The pandemic will pass, and the biomedical industry will continue to march forward. While 2020 was hit hard by the COVID-19 pandemic, it also saw a surge in research and commercial activity in the biomedical industry. The pandemic and other hot areas are driving venture capital, along with an increase in m&a activity and ipos. Clinical and commercial activity has increased in the fields of traditional drug discovery, cell and gene therapy, despite the death of four participants in two clinical trials of gene therapy. Investors have long been enthusiastic about emerging technologies, including gene editing and synthetic biology.
According to a recent paper published in GEN, the following are some of the trends to watch for in the biomedical industry in 2021.
1.COVID-19: See the end
When Pfizer and BioNTech announced positive phase III data in November 2020, they immediately sought emergency authorization (EUA) for their BNT162b2 vaccine candidate. Such hopes for a COVID-19 vaccine were further boosted when Moderna reported progress on its mrN-1273 candidate. In addition, AZD1222(Astrazeneca/Oxford), JNJ-78436735(Johnson & Johnson), NVX-COV2373 (Novavax), MRT5500(Translate Bio/ Sanofi), V591(Merck) and an unnamed vaccine being developed by Sanofi and GlaxoSmithKline.
According to Michael’s research report, vaccination capacity is expected to increase nearly 150-fold, from 35 million in the fourth quarter of 2020 to 5 billion in the fourth quarter of 2021, as multiple new vaccines are approved. In addition to the COVID-19 vaccine, Veklury, the first COVID-19 treatment approved by the FDA in 2020, eli Lilly’s Bamlanivimab mab and regenerated antibody cocktail Regn-Cov2 have also received emergency use authorization (EUA) for the treatment of COVID-19.
2.Gene therapy: Big pharma, big deal
Now big pharma is starting to get really excited about gene therapy, and through acquisitions to expand their operations in 2021 and beyond.
In October 2020, Bayer acquired AskBio for $4 billion; Novartis acquired Vedere Bio, an eye gene therapy company, for $280 million; Roche has signed a $1.8 billion partnership with Dyno Therapeutics to use its CapsidMap™ platform to develop next-generation adenoassociated virus (AAV) vectors for gene therapy for central nervous system diseases and targeted therapies for the liver.
Pfizer has invested $60 million in Homology Medicines after it submitted positive data for pheNIX in a phase I/II clinical trial (NCT03952156). PheNIX is a gene therapy for adults with phenylketonuria (PKU). In addition, Biogen and Sangamo Therapeutics are collaborating to develop and commercialize their gene regulation therapy candidates for Sangamo, which could bring Sangamo more than $2.7 billion in revenue.
However, preventing the tragedy caused by clinical trials of gene therapy remains a challenge in 2021. In October 2020, a five-year-old girl with mucosaccharidosis type IIIA was reported to have died in a phase II/III trial (NCT03612869) designed to evaluate Lys-SAF302.
In January 2020, Astellas Pharma acquired Audentes Therapeutics for $3 billion, The company announced three patient deaths between May and July in a Phase I/II trial (NCT03199469) evaluating its X-linked myomyopathy (XLMTM) candidate AT132. All 3 patients received a high dose of 3 × 1014vg/kg. All three of the patients who died showed significant characteristics, including older age, heavier weight, and evidence of preexisting hepatobiliary disease, Audentes said.
Despite these deaths, gene therapy has not seen the kind of industry devastation that followed the death of Jesse, 17, in 1999. According to statistics from the Regenerative Medicine Alliance, gene therapy financing has nearly tripled year-over-year, rising 178 percent in the third quarter of 2020 to reach $3.5 billion. Between January and September 2020, the amount raised rose 114 percent to $12 billion.
In addition to the increase in funding, clinical trials based on gene therapy are also on the rise, with 373 gene therapy trials in q3 2020, up 4% from 359 in q2. Of these, 115 were in phase I, 223 were in phase II and 32 were in phase III.
3. Gene editing: CRISPR
In November 2020, Lilly entered into a potential $2.7 billion partnership with gene-editing company Precision BioSciences, which opens the way for gene therapy for Duchenne muscular dystrophy (DMD). Leaps by Bayer, Bayer’s investment arm, co-led A $65 million Series A funding round for Metagenomi, A company developing crisPR-based gene-editing systems, to develop cell and gene therapies.
CRISPR Therapeutics and Vertex Pharmaceuticals are continuing to study CTX001, a gene-editing therapy based on CRISPR-Cas9, The treatment demonstrated proof-of-concept in two transfusion-dependent β -thalassemia patients and efficacy in another patient with sickle cell disease in two Phase I/II trials in June 2020.
Business Research Company’s Market report, released in November 2020, forecasts that the global CRISPR technology market will grow from $1.65 billion this year to $2.57 billion in 2023, then jump to $6.7 billion by 2030.
4.Fundraising: Impressive venture capital, M&A and ipos
The record amount of risky investments shows that a lot of capital has flowed into the biomedical industry. This trend is expected to continue in 2021, with strong growth in MERGERS and acquisitions and ipos.
According to the quarterly MoneyTree report from PwC and CB Insights, investment in biopharmaceutical related industries reached a record $5.9 billion in the third quarter of 2020, with $3.9 billion invested in 104 biotech deals alone, more than double the $1.9 billion invested in 74 deals in the third quarter of 2019.
Cancer is also driving a series of large mergers and acquisitions in 2020. Gilead acquired antibody-coupled (ADC) developer Immunomedics in A $21 billion acquisition in October, acquiring the pioneering TROp-2 targeted ADC drug Trodelvy for the treatment of adult patients with metastatic triple-negative breast cancer (mTNBC) who have received at least two previous therapies for metastatic disease. Illumina acquired Grail, a developer of cancer blood tests, for $8 billion ina deal that should close in 2021.
Ipos were also strong in 2020. In the first three quarters of 2020, biomedical companies raised a total of $9.32 billion in 51 initial public offerings, more than double the $3.6 billion raised in 41 ipos in the first three quarters of 2019, according to EvaluatePharma.
Overall, biotech stocks have performed well during 2020. As of November 2020, the NASDAQ Biotech index was $4,364.15, up 22% from $3,581.05 a year earlier.
5.Drug development: Whether a new Alzheimer’s drug is approved
One of the most talked-about drug development stories of 2021 is whether or not the FDA will approve Aducanumab, an Alzheimer’s disease candidate being developed by Biogen and Eisai.
Biogen, which has been working on aducanumab since 2015, stopped the EMERGE and ENGAGE phase III trials in March 2019 after analysis showed the drug was unlikely to meet its primary endpoint. In October 2019, however, Biogen reported that a larger data set from the EMPER trial was available, and analysis of that data set showed that aducanumab alleviated cognitive decline by targeting and eliminating the toxic protein amyloid beta. Biogen also noted that data from a subset of patients in the ENGAGE trial supports the new findings about the EMPER trial. So the company went back to the FDA for approval.
On November 6, 2020, the FDA convened an independent advisory panel to review the experimental data for the candidate antibody drug called Aducanumab, which concluded that even the strongest clinical trial data were sufficient to support its effectiveness. The FDA is expected to decide by March 2021 whether to approve Aducanumab, which if approved would be the first Alzheimer’s drug to slow cognitive decline.
“We believe aducanumab will receive a complete response letter in early 2021 requesting another large trial prior to approval. We assume a 40 percent chance of approval in 2024, “Andersen said. “If Biogen receives a full response letter, they will have to decide whether to continue to invest in Aducanumab or focus on a similar antibody, BAN2401, which has been studied in several Phase 3 studies with Eisai.”
Andersen added that aducanumab could either lead to more attention and higher valuations for Alzheimer’s projects or dampen enthusiasm for treatments based on clearing amyloid plaques: “Either way, we hope to continue to invest in other mechanisms of action, particularly Tau, and other models that can better cross the blood-brain barrier.”
6.Cell therapy: Buyers large and small
In November 2020, PerkinElmer acquired Horizon Discovery Group for approximately $383 million. The transaction was designed to expand PerkinElmer’s portfolio of life science research and applied genomics solutions, Better able to collaborate with scientists in academia and pharmaceutical/biopharmaceutical fields. The next generation of cell engineering will also be explored. The deal will close in 2021; Sanofi bought Kiadis Pharma, a developer of natural killer cell therapies, in November for about $364 million. Most of these therapies are focused on cancer, but one of the preclinical therapies is for COVID-19.
In September 2020, Takeda Opened a new R&D cell therapy manufacturing facility at its Research headquarters in Boston. The facility provides end-to-end research and development capabilities and will accelerate Takeda’s efforts to develop next-generation cell therapies, initially focused on oncology with the potential to expand into other therapeutic areas.
In addition, financing for cell therapies is growing at an alarming rate. According to the Regenerative Medicine Alliance, cell therapy financing reached $3 billion in the third quarter to November 2020, an increase of 97% and 242%, respectively, compared with the first and second quarters. The number of phase I trials of cell therapy increased from 41 in 2019 to 50 in 2020.
7.Synthetic biology: The appeal is getting stronger
SynBioBeta recently shared some numbers that show the growing appeal of synthetic biology to investors. In the first half of 2020, 56 companies in the sector raised a total of $3.041 billion. In the first half of 2019, 65 companies raised just $1.9 billion. The largest round of funding in 2020 was $700 million from Sana Biotechnology, a developer of engineered cell therapies.
In September 2020, Zymergen raised $300 million to promote its expansion into the chemical and materials industries and accelerate production of bio-prefabricated transparent membranes for electronic applications; Other products are expected to be commercialized for applications in electronics, agriculture, consumer health and healthcare.
“COVID-19 has not hindered the $4 trillion bioeconomy driven by synthetic biology,” John Cumbers, founder of SynBioBeta, recently commented in Forbes.
If anything, COVID-19 is helping to advance synthetic biology. Among them, Ginkgo Bioworks raised $70 million in May 2020 to develop a large-scale testing infrastructure. In July, the company announced an award of approximately $40 million from the NIH Advanced Technology Platform for Rapid Diagnostics Acceleration (RADX-ATP) program to expand its research into automation technology for next-generation sequencing sarS-COV-2 tests.