Freeline Bags Alnylam's Heggie As CEO
Hemophilia B Gene Therapy Advances
Executive Summary
Syncona-backed Freeline has nabbed an "accomplished commercial leader" in Theresa Heggie to lead the company as it prepares to take FLT180a, its gene therapy for hemophilia B, into Phase III.
Armed with a fresh cash injection from backer Syncona, Freeline Therapeutics Ltd. has poached Alnylam Pharmaceuticals Inc.'s Theresa Heggie as CEO as the UK gene therapy firm makes plans to put its hemophilia B treatment into pivotal trials.
Getting Heggie onboard is quite a coup for Freeline which described her as "an accomplished commercial leader in the biopharmaceutical industry with extensive global experience of rare diseases." Having led Alnylam’s Europe, Middle East & Africa and Canada (CEMEA) operations, where she oversaw the launch of the RNAi specialist’s Onpattro (patisiran) for hereditary transthyretin-medicated amyloidosis (hATTR), Heggie will take up her new position in June.
Chris Hollowood, chairman of Freeline and chief investment officer at Syncona, noted that Heggie grew Alnylam’s CEMEA unit from 20 to more than 200 employees. He added that she "had a deep understanding of medicines for genetic diseases and direct experience in some of the indications Freeline is pursuing." She was also previously CEO of German rare disease biotech Jerini AG, acquired by Shire PLC in 2008.
Heggie said she was impressed by Freeline’s ambition "to build an independent, global biotech company with end to end capability from research and manufacturing through to commercialization."
Hollowood said that "now is the right time to transition to a commercially experienced CEO,” as the company's adeno-associated virus (AAV) gene therapy for hemophilia B, FLT180a, progresses through the clinic. The company will be presenting further data from the Phase I/II B-AMAZE trial of FLT180a at the European Association for Haemophilia and Allied Disorder (EAHAD) meeting at The Hague, Netherlands later this week (7 February).
In September last year, Freeline disclosed that the first two patients on B-AMAZE who were treated with the lowest dose of FLT180a, had shown that Factor IX (FIX) activity remained stable and consistent at 40.5%, plus or minus 5.5%, through weeks 66 and 74, respectively, post administration. The normal range of FIX activity in the general population's blood is between 50% and 150%, the company noted, adding that both patients remained free of spontaneous bleeding episodes and did not require replacement FIX therapies.
Freeline believes its product compares favorably with the other players in the gene therapy hemophilia market. They include uniQure NV, which presented one-year data from three patients who received AMT-061 (etranacogene dezaparvovec) from the Phase II HOPE-B trial at the American Society of Hematology congress in December. The data showed that AMT-061 increased the activity of FIX to a mean of 41%, reaching up to 50% in the first patient dosed in the trial. (Also see "UniQure Aiming To Be First With Hemophilia B Gene Therapy" - Scrip, 6 Dec, 2019.) (Also see "UniQure Has High HOPE For Hemophilia B Gene Therapy " - Scrip, 11 Feb, 2019.)
Pfizer Inc. also has a gene therapy in Phase III for hemophilia B. Fidanacogene elaparvovec was licensed from Roche subsidiary Spark Therapeutics Inc. and at ASH, Pfizer presented data from a Phase II trial which showed that one year after infusion, 15 patients achieved an average FIX level of 23%.
On the financial side, Heggie will be taking over a company in rude health. At the end of 2019, Syncona invested another $80m in the company via a series C round with $40m in the first tranche. Freeline's founding investors, including Syncona, had previously backed the company with £34.8m and £88.4m series A and B rounds, respectively. (Also see "£88m Series B For Syncona-backed Freeline To Advance Liver Gene Therapy Focus " - Scrip, 19 Jun, 2018.)
The fresh cash injection will allow Freeline to advance FLT190, a gene therapy that has recently gone into a Phase I/II trial in patients with Fabry disease, which affects one in every 40,000 people. The company, which was founded in 2015 by Syncona along with Amit Nathwani of University College London, who is the firm's chief scientific officer, also has preclinical programs looking at gene therapies for Gaucher disease and hemophilia A.
The funds will also be used to continue the ongoing buildout of Freeline's manufacturing operations in Munich, Germany. Last April, it signed a multi-year agreement with Brammer Bio Inc., a leading US gene therapy contract development and manufacturing organization acquired in March 2019 by Thermo Fisher Scientific Inc. for $1.7bn, which the London-based group said would meet the commercial demand of the FLT180a program.