The biotech IPO queue has added two more companies. Freeline Therapeutics and Checkmate Pharmaceuticals have each filed the regulatory paperwork to offer shares to the public.
Freeline is raising cash to ensure that its lead program, a prospective hemophilia B gene therapy, keeps pace in the race to develop and commercialize a genetic treatment for the inherited blood clotting disorder.
In paperwork filed with securities regulators late last week, Stevenage, UK-based Freeline set a preliminary $100 million target for its IPO, through which it plans to offer American depositoInterry shares on the Nasdaq. The company says in its filing that it plans to apply for a listing on that exchange under the stock symbol “FRLN.”
Freeline’s gene therapies are based on adeno-associated viruses, a type of virus engineered to deliver a functional version of a gene into cells. The company says it can target delivery of its therapies to the liver, where they prompt the cells of the organ to produce therapeutic proteins that address disease. The company says it has engineered the capsid, or protein shell of a virus that encloses the genetic material, to more efficiently enter liver cells compared to strains of the virus found naturally. Freeline adds that this capability allows it to better address diseases that require high protein levels, such as enzyme deficiency disorders, as well as inflammatory and immune disorders. Furthermore, the company believes that the potency of its therapies allows it offer them in lower doses, making them more tolerable to patients.
“As a result, we believe our platform has the potential to expand the current reach of gene therapy and to develop one-time treatments for indications beyond the well-known monogenic diseases targeted by many current gene therapy clinical programs, including our current pipeline,” Freeline says in its filing.
The hemophilia B program, FLT180a, is currently in a Phase 1/2 study testing its ability to restore the production of Factor IX (FIX), a clotting protein produced in insufficient amounts in hemophilia patients due to a genetic defect. Last week, Freeline presented additional clinical data during the 2020 virtual meeting of the International Society on Thrombosis and Haemostasis. The results were for the first 10 patients treated with the gene therapy who were followed up to two years after receiving the treatment.
The data showed that the therapy was able to boost FIX levels in moderate-to-severe hemophilia B patients. Higher doses led to higher FIX levels. So far, none of the patients needed supplemental FIX infusions to address bleeding problems. But the higher FIX levels, which were at or near the range found in healthy people, were also accompanied by higher levels of liver enzymes, a side effect. Freeline is using steroids and immunosuppressants to try to manage these liver enzymes while also maintaining FIX levels.
Several other companies are also developing gene therapies for hemophilia, and some of them are further ahead. UniQure (NASDAQ: [[ticker:QURE]]) has reached Phase 3 testing for its hemophilia B gene therapy candidate. Last month, the Netherlands-based biotech licensed the therapy’s rights to CSL Behring for $450 million up front. Meanwhile, BioMarin Pharmaceutical (NASDAQ: [[ticker:BMRN]]) expects to receive an FDA decision next month for its hemophilia A gene therapy candidate.
Freeline says in its prospectus that it expects to report additional data from the FLT180a study in the second half of this year. The company plans to advance the gene therapy to a pivotal study in 2021. The other programs in its pipeline include FLT190, a gene therapy for the rare enzyme deficiency Fabry disease that is in Phase 1/2 testing. Two other programs, FLT201 for Gaucher disease, another rare disorder caused by an enzyme deficiency, and FLT210 for hemophilia A, are in preclinical development.
Freeline was formed in 2015 based on AAV capsid research from a University College London team led by company founder Amit Nathwani. The biotech has raised $282.8 million, according to the prospectus. Its largest shareholders include
