The president and CEO of Global Blood Therapeutics (GBT) is defending the list price of his company’s recently-approved sickle-cell disease (SCD) drug Oxbryta™ (voxelotor) from criticism that the company set it too high.

The Institute for Clinical and Economic Review (ICER), a Boston-based nonprofit that analyzes the clinical and cost effectiveness of new treatments, concluded last month that Oxbryta and two other SCD therapeutics approved since 2017—Novartis’ Adakveo® (crizanlizumab) and Emmaus Life Sciences’ Endari™ (L-glutamine oral powder)—were overpriced based on a threshold of $150,000 per quality-adjusted life year. ICER recommended that GBT set an annual cost for Oxbryta of $9,218.

But speaking to an audience of investors in New York, GBT CEO Ted W. Love, MD, insisted that the drug’s efficacy against SCD justified its $125,000/year list price; GBT has promised not to raise that price for three years. He also cited the savings to SCD patients of over $700,000 in otherwise lost lifetime income due to the disease.

The mean cost per patient per year of the top 100 orphan treatment products in the U.S. surpassed $150,000 in 2018, compared with $33,654 for non-orphan drugs, according to the most recent edition of EvaluatePharma’s Orphan Drug Report, issued in April 2019.

GBT’s workforce is more than half female—including its head of manufacturing—and more than half people of color, CEO Ted W. Love, MD, said during the recent Biotechnology Innovation Organization (BIO) CEO & Investor Conference in New York. [BIO]

Price point

Addressing investors at the Biotechnology Innovation Organization (BIO) CEO & Investor Conference, held in Times Square, Love said GBT priced Oxbryta within the lower half of the average list price for drugs indicated for orphan diseases. Half of Oxbryta’s users will be on Medicaid, so the company will give the Centers for Medicare and Medicaid Services the 23.1% mandatory rebate off the list price. And he cited one unidentified payer that told him some SCD patients it covers cost more than $1 million a year in coverage.

Love also noted that Oxbryta was the first FDA-approved drug designed to treat the root cause of SCD by directly inhibiting sickle hemoglobin polymerization, through which red blood cells become sickled, causing anemia and blockages in capillaries and small blood vessels that can lead to frequent pain crises, stroke, and irreversible organ damage. He said 20% of children with SCD had cognitive brain injury before their 10th birthday.

“If you could convert those individuals into people who don’t have cognitive brain injury, who have a fair shot at life, whose organs don’t prematurely deteriorate before they’re 10 years old, that is a big intervention. And the idea that a typical orphan price is not justified makes no sense to me,” Love asserted.

“If you go into a disease area where there’s nothing available, looking at the alternative price value of the alternative is a silly discussion. The alternative is to continue to let these patients suffer and die.”

“Despite ICER, most people realize that if you can fundamentally stop [SCD], it’s cheaper than dealing with the cost of it,” Love added. “I think we priced it very responsibly.”

During an hour-long fireside chat, Love also discussed the development of Oxbryta; detailed the company’s approach to commercial expansion; highlighted additional patient populations the company plans to treat; and offered a broader defense of the biopharma industry against criticism that its development and pricing of new drugs have not generated a proportionate benefit to patients and the general public.

Oxbryta won accelerated FDA approval in November 2019 as a treatment for SCD in patients 12 years old and older. The FDA based its approval on positive clinical data Oxbryta generated in the Phase III HOPE (Hemoglobin Oxygen Affinity Modulation to Inhibit HbS PolymErization) trial (NCT03036813). Of 274 patients evaluated, more than 50% treated with Oxbryta achieved greater than 1 g/dL increase in hemoglobin, compared with 6.5% receiving placebo.

The approval came three months in advance of the FDA’s Prescription Drug User Fee Act (PDUFA) target priority review decision date of February 26: “I started to cry,” Love told moderator Alethia Young, managing director, head of Healthcare & Senior Biotech Research Analyst at Cantor Fitzgerald, upon hearing news of the agency’s speedy decision.

Love said the drug’s commercial launch has gone “very well.” Details should emerge when the company releases its fourth-quarter and full-year 2019 results on February 25. However, Love did say the company was able to start packaging and shipping Oxbryta the day of its FDA approval because it spent months preparing to bring the drug to market.

GBT’s commercialization effort, Love said, included the hiring of about 200 staff over the past year, bringing the company’s workforce to about 350. The company sorted through more than 500 applicants to find ten U.S. regional sales managers, and considered more than 2,400 applicants before selecting the 65 field sales representatives those managers will oversee.

“Patients are dying”

Oxbryta™ (voxelotor) won accelerated FDA approval in November 2019, three months ahead of its PDUFA target priority review decision date of February 26. [GBT]

“Patients are dying, and our company exists to try to save their lives. And part of saving their lives is to not only make a good therapy, but it’s to make it available. And part of making that available is having an organization, MSLs [medical science liaisons], that can interact with physicians, interact with patients, building a patient support hub, which we built,” Love said, referring to GBT Source™, designed to enroll patients within two weeks.

GBT’s workforce is more than half female—including its head of manufacturing—and more than half are people of color, Love said, reflecting what he termed the company’s commitment to building a diverse workforce by creating a supportive culture.

The goal of workforce diversity across the industry won reinforcement from BIO on January 30, when it issued its first annual report on workforce diversity and inclusion, “Measuring Diversity in the Biotech Industry: Building an Inclusive Workforce.”

“If you have an organization where you don’t look like the community from which you‘re recruiting, you’re probably experiencing some bias in how you’re recruiting and how you’re building the organization,” Love said.

“People at other companies who are really good look at GBT and say, ‘I think this is an environment where, as a black man, I’ll be treated really equally, without hesitation.’ ‘As a woman, I’ll be treated equally without hesitation.’ ‘As a person who is gay, I’ll be treated equally.’ We live that every day. We embrace that, and the best people from every background want to work for our company. It’s an enormous advantage when you’re trying to double the size of your company every year.”

Love and passion

No matter their color or gender, Love said, GBT seeks to hire people with passion for the company and its mission in those it hires: “We want people that are committed to changing the world for the patients that we’re focused on.”

GBT is working to broaden its patient focus to include two additional populations: People with SCD who are experiencing severe vaso-occlusive crises (VOCs), and most children younger than 12.

Oxbryta’s mode of action distinguishes the drug from earlier SCD treatments that focused on the painful VOCs. GBT’s pipeline includes inclacumab, a novel fully human monoclonal antibody designed to reduce VOCs through P-selectin inhibition. In 2021, GBT anticipates launching a pivotal study of inclacumab, which is being developed through an exclusive worldwide licensing agreement with Roche.

Already underway is HOPE-KIDS 2 (NCT04218084), a confirmatory Phase III study designed to measure transcranial doppler (TCD) flow velocity in order to demonstrate a decrease in stroke risk in children ages 2-15 years following treatment with Oxbryta. Half the study’s 224 participants will be randomized to 1500 mg of Oxbryta. The study includes a 24-week primary analysis treatment period, and a 96-week total treatment period.

Love joined GBT in June 2014 from Onxy Pharmaceuticals, where he was EVP, research and development and technical operations. Earlier he served as president, CEO, and chairman of Nuvelo, and as SVP development at Theravance. He also held senior management positions at Genentech, where he oversaw the development strategy and execution leading to approvals of Rituxan®, Herceptin®, Xolair®, TNKase®, Raptiva and Avastin®.

Love ended the fireside chat by saying the biotech industry was “wonderfully positioned to make more innovations and to help more patients, in particular patients for which we had not had good solutions.”

The biggest threat he sees is “all the political rhetoric that really tries to characterize our industry, I think, unfairly. Most of the people in our industry are more committed to the health and well being of this country than are estimated by many senior elected officials. We need to say that. I think we need to demonstrate that, and the public needs to understand that, and get away from the rhetoric that the industry is the evil empire.”