Momentum in biotech IPOs continued this week as four more companies made their stock market debuts.
IPO activity is higher this year by several measures, according to IPO research firm Renaissance Capital. A total of 119 IPOs have been filed in 2020, up 250% from last year. Renaissance counts 95 IPO pricings that have raised $37.3 billion. Health care is the biggest sector represented among newly public companies, accounting for 47% of IPOs.
As usual, oncology figured prominently as two cancer drug developers went public to finance clinical development of their respective pipelines. But this week also saw IPOs from two biotechs taking novel approaches to treating rare diseases. Here’s a look at the newly public biotech companies.
Design Therapeutics draws $240M for drugs treating genetic diseases
Design Therapeutics had the biggest IPO this week, raising $240 million for its pipeline of drugs addressing rare, inherited diseases. The preclinical company priced its offering of 12 million shares at $20 apiece, which was the top end of its projected price range. Design’s shares will trade on the Nasdaq under the stock symbol “DSGN.”
Carlsbad, California-based Design is developing drugs for inherited diseases driven by nucleotide repeat expansion, in which stretches of certain nucleotide sequences repeat excessively. This type of DNA mutation has been found to be the root of dozens of degenerative diseases. There are currently no FDA-approved therapies that address repeat expansions.
A Deep-dive Into Specialty Pharma
A specialty drug is a class of prescription medications used to treat complex, chronic or rare medical conditions. Although this classification was originally intended to define the treatment of rare, also termed “orphan” diseases, affecting fewer than 200,000 people in the US, more recently, specialty drugs have emerged as the cornerstone of treatment for chronic and complex diseases such as cancer, autoimmune conditions, diabetes, hepatitis C, and HIV/AIDS.
Design is developing small molecule drugs called gene targeted chimeras (GeneTACS) that are intended to target the underlying cause of repeat expansions. The company says in its IPO prospectus that its GeneTACs are designed to selectively bind to genetic repeat sequences and modulate gene expression by restoring or blocking the gene’s ability to transcribe messenger RNA, which is the genetic template for making proteins.
So far, Design’s GeneTAC platform has produced drug candidates for two rare, inherited neuromuscular disorders: Friedreich ataxia (FA) and myotonic dystrophy type 1 (DM1). The company plans to spend $30 million to complete preclinical development of its FA program and advance it through Phase 1 testing. Another $35 million is budgeted for preclinical and Phase 1 work in DM1. Design also plans to spend about $35 million for the preclinical development of an undisclosed program.
Since the company’s start, it has raised more than $170 million from investors that include Logos Capital, SR One, Quan Capital, Cormorant Asset Management, and West River Capital, according to the IPO documents. Trusts for the family of Pratik Shah, Design’s co-founder and executive chair of its board of directors, hold a 14.25 percent post-IPO stake, the same percentage held by co-founder Aseem Ansari. Ansari’s expertise includes transcriptional regulation, and he is the chair of the department of chemical biology and therapeutics at St. Jude Children’s Research Hospital. SR One is Design’s largest institutional investor, owning 9.9% of the company.
Edgewise lands $176M to develop small molecules for muscular dystrophies
Edgewise Therapeutics priced its offering of 11 million shares at $16 each, which was the top end of its projected price range. At that price, the biotech raised $176 million to fund its research and development of small molecule drugs for muscular dystrophies. The company’s shares will trade on the Nasdaq under the stock symbol “EWTX.”
The newest FDA-approved drugs for Duchenne muscular dystrophy include genomic medicines designed to get a patient’s cells to produce a functional version of a dystrophin, a key muscle protein that they lack. Edgewise therapeutics is taking a different approach with small molecule drugs that improve muscle stability and reduce damage from muscle hypercontraction.
Lead Edgewise drug candidate EDG-5506 is designed to selectively block the fast-twitch skeletal muscle cells that hypercontract in patients who lack dystrophin. By moderating these fast skeletal muscles, the drug is intended to improve muscle stability and moderate injury that Duchenne patients can develop. In animal testing, Edgewise said in its IPO documents that its drug protected muscles from damage and improved muscle function. The company believes its treatment could work alongside currently available treatments as well as therapies now in development, including gene therapies.
Last October, Edgewise began a Phase 1 test of EDG-5506 in healthy volunteers. The company plans to spend about $80 million of the IPO proceeds on its lead drug candidate. The cash will enable the company to complete to complete that early-stage study and evaluate the drug in patients with Becker’s muscular dystrophy, another muscle disorder. If the drug proves tolerable, Edgewise plans to start a Phase 2a study in Becker’s patients. Data from both studies could support a pivotal study testing the drug in pediatric Duchenne patients and adults and teens with Becker’s. Edgewise estimates that the cash will be enough to get to a preliminary data readout for a planned Phase 2/3 study in Duchenne patients. Another $55 million of the IPO proceeds will be set aside for the research and development of three other preclinical programs, two for skeletal muscle and one for cardiac muscle.
Edgewise formed in 2017 with funding from OrbiMed. The company’s co-founders are Chief Scientific Officer Alan Russell; Peter Thompson, an OrbiMed partner; and Badreddin Edris, who is currently the chief operating officer at SpringWorks Therapeutics. Russell’s muscle drug experience includes work at GlaxoSmithKline and Cytokinetics. Edgewise has raised $160.7 million since its launch, according to the IPO filing. OrbiMed is the biotech’s largest shareholder with a 28.6% pre-IPO stake, followed by Novo Holdings with 10.6%.
Ikena Oncology nets $125M to advance its cancer drug pipeline
Ikena Oncology has been working in partnership with Bristol Myers Squibb, an alliance that could feed cancer drugs to the pharmaceutical giant’s pipeline. Now the company has $125 million in IPO cash to spend on the research and development of those drugs as well as its own cancer drug programs. Boston-based Ikena priced its offering of more than 7.8 million shares at $16 apiece, which was the midpoint of its targeted $15 to $17 per share price range. Those shares will trade on the Nasdaq under the stock symbol “IKNA.”
Formed in 2016, Ikena was first known as Kyn Therapeutics, a developer of targeted cancer drugs addressing signaling pathways driving the formation and spread of cancer cells. The company changed its name in 2019, the same year it struck up a research alliance with Celgene. BMS inherited the Ikena alliance with the close of its $74 billion acquisition of Celgene. The collaboration has produced two drug programs: IK-175 is in early clinical development for bladder cancer and IK-412 is being readied for clinical testing in solid tumors.
Some of the IPO cash will support the partnered programs, with between $55 million and $60 million earmarked for clinical development of both drugs through the completion of Phase 1b testing, according to the prospectus. At that point, BMS can exercise its right to license the compounds and continue their clinical development. If it does, Ikena would be eligible for a $50 million payment for IK-175, and $40 million for IK-412. Regulatory and commercial milestones could bring the company up to $450 million in milestone payments, plus royalties from sales.
Ikena plans to use the IPO cash to develop its wholly owned programs, including IK-930, a drug targeting genetic mutations of the Hippo signaling pathway. Up to $40 million is budgeted for advancing that program from preclinical research to the completion of Phase 1 testing. Another $25 million to $30 million is set aside for developing a drug that blocks ERK5, an enzyme in the RAS signaling pathway. The cash is expected to support the nomination of an ERK5 candidate and the start of human testing. Ikena plans to spend up to $15 million to complete a Phase 1b test of IK-007 in colorectal cancer, and up to $35 million to continue research on its preclinical programs.
Of the $239.2 million Ikena has raised since its start, $95 million is from an upfront payment received from BMS, according to the prospectus. That payment breaks down to $80.5 million cash and a $14.5 million equity investment.
Lava Therapeutics fetches $100M for targeted antibody cancer drugs
Lava Therapeutics began trading on the Nasdaq Thursday under the stock symbol “LVTX.” The Netherlands-based cancer drug developer priced its offering of 6.7 million American depositary shares at $15 each, which was the midpoint of its targeted price range, raising $100.5 million.
Lava is developing bispecific antibodies that are engineered to selectively activate a subset of gamma delta T cells that can distinguish tumor cells from healthy ones. The approach is intended to kill tumor cells directly and spark immune responses form innate and adaptive immune cells in the tumor microenvironment.
The most advanced Lava drug candidate, LAVA-051, is being developed as a treatment for chronic lymphocytic lymphoma, multiple myeloma, and acute myeloid leukemia. Lava has clearance from Netherlands authorities for a Phase 1/2a study, which is expected begin enrolling in the first half of this year. The company plans to seek FDA permission for a Phase 1 study in the first half of 2022.
According to Lava’s IPO paperwork, another $40 million is planned for the development of LAVA-206×207 for metastatic castration-resistant prostate cancer. The company expects to submit the regulatory paperwork for those studies second half of this year. Lava also plans to spend $10 million to advance its other drug candidates in its pipeline.
Lava’s U.S. stock market debut follows an $83 million Series C round of funding last September led by Versant Ventures and Sanofi Ventures. Those firms own 16.1% and 7% post-IPO stakes in Lava, according to the filing.
Photo: Spencer Platt, Getty Images
