Which new therapies/technologies are most sought after in the biopharmaceutical industry today? Which startups are most favored by investment institutions? Which disease areas are the “heavy” areas for new drug development? An overview of global new therapeutic/new technology-related investment and financing events may help you to find out.
According to PharmaInvest database, in the first quarter of 2021 (2021Q1), there were more than 224 funding events (including IPOs) focused on new therapies/new technologies worldwide, involving more than 200 companies and a cumulative funding amount of more than $16.9 billion. By month, the number of financing events continued to increase from January to March, with 58 events in January, 68 in February, and 98 in March. In terms of the affiliation of financing companies, the cumulative number of announced financing events related to new therapies/new technologies is relatively high, with a total of 158 events.
In terms of funding rounds, Series A (including Pre-A, Pre-A+, A, A1, A+ and A++) had the most funding events, with 74, followed by Series B (including Pre-B, B and B+) and Series C (including C and C+), with a distribution of 60 and 36. It is worth mentioning that in 2021Q1, a total of 24 companies focusing on new technologies/new therapies formally IPOed, raising over $4.576 billion in cumulative capital. Among them, Sana Biotechnology set a record for the highest IPO in the quarter, raising $675.6 million, followed by Instil Bio and Immunocore, raising $368 million and $297.1 million, respectively.
Founded in 2018, Sana is a company dedicated to the development of engineered cell therapies that brings together the original Juno lineup at the top, “made a name for itself” last June with the announcement of an initial round of funding of up to $700 million, and is currently developing “fusogens “Fusogens can bind to the cell surface proteins of target cells and then deliver the gene payload directly to the cell’s cytoplasm. Based on this technology, Sana is developing innovative in vivo cell therapies, including in vivo modification of T cells to fight cancer, in vivo modification of hepatocytes to treat liver-related genetic diseases, and in vivo modification of hematopoietic stem cells to treat sickle cell disease and beta-thalassemia. The company is also laying out in vitro cell therapies (similar to CAR-T, which is modified in vitro and then transfused back into patients), including in vitro modification of T cells to fight cancer, beta cells derived from stem cells to treat type 1 diabetes, and glial progenitor cells derived from stem cells to treat central nervous system disorders such as Huntington’s chorea.
It is worth noting that all Sana products are currently in preclinical development and Sana reported at the ASGCT 2021 meeting in April that the latest preclinical data: 1) For T cells, CD8-targeted Fusogens showed cell-specific selectivity and efficacy in in vitro and in vivo gene transfer and were able to achieve in vivo tumor eradication (a single intravenous injection of fusosomes specifically deliver CD19 CAR transgenes to CD8+ T cells to generate CAR-T cells in vivo, and such CAR-T cells show dose-dependent antitumor responses); and 2) for hepatocytes, effective transduction of fusogens was achieved in both engineered cell lines and human hepatocytes. sana concluded that these results suggest that fusogen-targeted vectors have the potential to deliver genes effectively as well as specifically in vivo, laying the groundwork for the development of gene and cellular therapies for a variety of diseases.
Founded in 2018, Instil Bio is also a cell therapy company dedicated to the development of autologous tumor-infiltrating lymphocyte (TIL) therapies. The company is using its own platform to optimize the production of TIL and improve the scalability, logistics and accessibility of TIL therapies on the one hand; on the other hand, they are also developing novel genetically engineered TIL therapies using the Co-Stimulatory Antigen Receptor (CoStAR) platform. From its announced pipeline, the company is currently developing two main products, a conventional TIL therapy (ITIL-168) and CoStAR-TIL (ITIL-306). ITIL-168 has entered Phase I clinical studies for melanoma; in April this year, Instil Bio announced that the FDA granted ITIL-168 orphan drug eligibility designation for the treatment of patients with stage IIB to IV melanoma. The company plans to initiate a global Phase II trial of ITIL-168 in patients with advanced melanoma this year, and clinical studies of ITIL-168 in other types of solid tumors are expected to begin in 2022.
Immunocore, founded in 2008, is a company focused on the development of ImmTAX (Immune mobilizing monoclonal TCRs Against X disease), a novel TCR bispecific immunotherapy designed to treat a wide range of diseases including cancer, infectious diseases and autoimmune diseases. Based on the ImmTAX platform, Immunocore has established a rich pipeline, with the most advanced candidate being tebentafusp, which is currently in Phase III clinical phase with a BLA filing scheduled for the third quarter of 2021. In February, Immunocore announced that the FDA had granted breakthrough therapy designation to tebentafusp for the treatment of unresectable or metastatic uveal melanoma.
In addition to tebentafusp, the company has several other products in clinical development, including IMC-C103C (Phase I/II, solid tumors), IMC-F106C (Phase I/II, solid tumors), and IMC-I109V (Phase I, HBV). Phase I data for MC-C103C are expected in the fourth quarter of this year, and Phase I data for IMC-F106C are expected in the fourth quarter of this year. Phase I data for F106C are expected in mid-2022, and MC-I109V just completed its first patient dosing this month.
Founded in 2017, Design Therapeutics closed a $125 million Series B round of funding in January and launched an IPO in March, ultimately raising $276 million. The company is focused on developing novel small molecule drugs – gene targeted chimeras (GeneTAC). The company is using its proprietary GeneTAC platform to design and develop nucleotide repeat amplification-driven therapeutic candidates for genetic diseases. geneTAC is a heterobifunctional molecule consisting of two major components: 1) a DNA-binding component and 2) a ligand component. the DNA-binding component is linked to the ligand component by a linker, and the ligand participates in and regulates the transcriptional machinery. design GeneTAC is expected to target mutant alleles and regulate the transcriptional machinery to specifically act on disease-causing nucleotide repeat amplification sites, enabling cells to restore normal protein expression.
The company’s programs are currently in preclinical development and are focused on Friedreich’s ataxia (FA) and ankylosing dystrophy (DM1). In the case of the Friedreich ataxia program, the DNA-binding portion of Design Therapeutics’ GeneTAC molecule was designed to bind the first intron extension GAA sequence of the FXN gene in FA patients, and the ligand portion was designed to recruit an endogenous transcriptional elongation complex to unblock the transcriptional machinery and to production of functional natural FXN proteins back to therapeutic levels.
Sensei Biotherapeutics, a company focused on developing immunotherapeutics for cancer as well as other diseases, announced the closing of a $30 million Series B financing in January and launched an IPO in February to raise $152.6 million. The company has two main platforms: 1) the ImmunoPhage™️ platform (ImmunoPhage™️), with the most advanced ImmunoPhage™️ program, SNS-301, in Phase I/II clinical studies targeting the treatment of squamous cell carcinoma of the head and neck (SCCHN); and another ImmunoPhage™️ program, SNS-401, being developed to treat Merkel cell carcinoma, with an IND filing scheduled for the first half of 2022; 2) monoclonal antibody and nanobody platform, the fastest progressing antibody program targeting VISTA, with an IND-enabling study expected to be completed by the end of 2021.
Founded in 2017 and headquartered in California and Shanghai, Terns Pharmaceuticals is focused on developing small molecule monotherapies and combination therapies for NASH and other chronic liver diseases, announcing the completion of an $87 million Series C financing in January and launching an IPO in February to raise $146.6 million. Other projects in clinical development include TERN-501, a THR-β agonist, which began patient dosing in a Phase I study in March, and TERN-201, a VAP-1 inhibitor, which is expected to start patient dosing in H1 this year. patient dosing (Phase Ib).
Founded in 2016, Ikena Oncology, a company dedicated to developing targeted cancer therapies, announced the closing of a $120 million Series B financing in January and launched an IPO in March, raising $143.8 million. The company’s main projects include TEAD inhibitors (IK-930, IND-enabling study underway), ERK5 inhibitors, AHR antagonists (Phase Ib), etc.
In terms of indications, more than 50% of the 217 companies announcing funding events are conducting drug development for cancer, involving a very diverse range of therapeutic types, including small molecules, lysing viruses, cellular therapies, gene therapy/gene editing therapies, RNA therapies, cytokines, antibodies (mono- and dual-antibodies), vaccines, radiotherapy, ADCs, antibody-coupled immunostimulants (ISACs), peptide-coupled drugs (PDCs), virus-like drug couples (VDCs), and others.
Other popular disease areas include autoimmune/immune-mediated diseases, neurological/neurodegenerative diseases, genetic diseases, inflammatory/inflammatory diseases, infectious diseases, etc.
In the past few years, we have witnessed major breakthroughs in cancer treatment brought by immunotherapy represented by PD-1/PD-L1 antibodies and CAR-T therapy; witnessed fruitful R&D of ADC drugs; witnessed the development of RNAi therapy from 0 to 1 and from 1 to N. …… In the next 5-10 years, we will witness which pharmaceutical R&D breakthroughs, primary market investment and financing events are very referenced. Where the money goes, the probability is that the breakthrough will be born. Exactly where the money in the primary market in the field of medicine will mainly go in the future, we will continue to pay attention.