There is no most expensive, only more expensive. The list of the world's most expensive drugs is updated with the launch of Bluebird Biologics' new drug Zynteglo. There is a group of companies that are committed to developing "noble medicines" for a small number of people, and a needle of ten million yuan has become a reasonable existence, what supports this ecology? How are they doing? Is sinicization possible?
On August 17, BlueBird Bio-in vitro genetically modified cell therapy Zynteglo was approved for marketing by the FDA, a one-time treatment for patients who need regular blood transfusions due to β-thalassemia, priced at $2.8 million (about 19 million yuan), more than novartis' gene therapy Zolgensma (priced at $2.1 million).
For this pricing, Bluebird's chief commercial officer revealed in an interview with the media that the drug pricing regulator has said that the therapy is cost-effective as up to $3 million per dose. Considering that a patient's lifetime blood transfusion can cost $6.4 million, the pricing of the therapy is actually reasonable.
Bluebird Bio said it would reimburse up to 80 percent of the cost of treatment to private and commercial health insurance companies if the therapy didn't work within two years of the patient receiving treatment. According to Bluebird Biologics, there are about 1,500 people in the United States who rely on blood transfusions β-thalassemia, of which an estimated 850 are physically eligible for Zynteglo therapy.
But Zynteglo's fate is as tortuous as the bluebird creature's. Before the U.S. listing, Zynteglo was actually approved for listing in the European Union in June 2019, and the United Kingdom, Iceland, Liechtenstein and Norway also approved the listing of the drug, the price of the drug was as high as 1.77 million US dollars, but due to the lack of price consensus with German regulators and the unsatisfactory commercialization results, Bluebird Bioan orderly closed its European business for serious genetic diseases and focused on the US market.
Not only that, Bluebird biology has contributed to the world's first approved BCMA targeted CAR-T therapy, ABECMA.
In addition, its Skysona was approved for marketing in the European Union in July 2021 for the treatment of early cerebral adrenoleukodystrophy (CALD) patients under the age of 18 with ABCD1 gene mutations, and the patients could not obtain matching hematopoietic stem cell (HSC) transplant donors. The product is currently under review by the FDA, with a PDUFA date of September 16, and if the drug is successfully launched, Bluebird Bio will also receive a priority review ticket. At present, the FDA has not approved the marketing of any CALD treatment drugs.
But even if it is excellent, the development of bluebird creatures has not been smooth.
The data shows that Bluebird Biologics is now worth $484 million. The company's revenue fell 90.8% in the first half of the year to $20.26 million. Its semi-annual report shows that Bluebird Bio has about $218 million in restricted cash, cash and marketable securities. Cash consumption for the full year 2022 is expected to be below $340 million. Bluebird Bio warned investors of "serious doubts" about its ability to remain solvent for the next 12 months.
Bluebird Biology, which is still in an existential crisis, has also undergone a major change of internal management, and its former CEO Nick Leschly, Chief Scientific Officer Philip Gregory and Chief Financial Officer Chip Baird, Co-Operating Officer and Head of the Oncology Business Unit Joanne Smith-Farrell, chief medical officer David Davidson, MD, have left Bluebird Biology. Bluebird Biology is also struggling to survive, splitting into two companies, focusing on serious genetic diseases and tumors.
Not only Bluebird, according to the 10 most expensive drugs on the market in 2021 and their companies released by the US drug price tracking website GoodRx, the only companies that are still developing independently are Horizon, Eiger, Y-mAbs, and BioMarin.
Among these 4 independent development companies, BioMarin and Horizon have a market value of nearly 20 billion US dollars, and have come out of the situation of relying on a drug alone for development, with more than 5 commercial products listed, and there are nearly 2 billion US dollars in cash and cash equivalents in hand.
Y-mAbs and Eiger, two companies with a market capitalization of less than $1 billion, remain on the brink of danger. Both companies currently have less than $150 million in cash and cash equivalents, and the two companies expect to support the company's growth through 2024.
BioMarin
Market capitalization of $17.536 billion
BioMarin received a $1.5 million investment from Glyko Biomedical in March 1997 and began operations focused on developing transformative therapies for patients with serious, life-threatening rare diseases.
To date, BioMarin has 7 commercial products (mostly enzyme replacement therapies). Its semi-annual report shows that in the first half of this year, the company's total revenue was $1.053 billion, an increase of 7% year-on-year, and net revenue from product sales was $960 million, an increase of 10% year-on-year. The product with the highest sales volume was Vimizim, which achieved sales of $356 million in the first half of the year, an increase of 8% year-on-year. BioMarin said the breakthrough growth in the first half of the year was mainly due to the commercialization of the newly launched voxzogo, which achieved sales of $54 million in the first half of the year, and was driven by Aldurazyme, which is responsible for sales by Sanofi, which achieved sales revenue of $78 million, up 21% year-on-year. Its listed product, Briineura, had net revenue of $74 million, up 28% year-over-year.
In terms of new product development, its Roctavian is the first approved hemophilia A gene therapy in Europe, and BioMarin plans to submit a BLA to the FDA in September.
As of June 30, the company's cash, cash equivalents and investments totaled $1.522 billion. The company expects full-year 2022 total revenue of $2.06-$2.16 billion.
Horizon
Market capitalization of $14.2 billion
According to Horizon's 2022 semi-annual report, the company's first-half net sales were $1.762 billion, up 50% year-on-year. Horizon expects full-year 2022 net sales of $3.53 billion to $36 billion. As of June 30, Horizon had $1.89 billion in cash and cash equivalents.
At present, Horizon's commercialized products mainly include 8 orphan drug products and 4 inflammatory products. The data shows that the net sales of Horizon orphan drugs in the first half of the year increased by 13% to $840 million. Ravicti, the most expensive drug on its list, costs $690,000 a year at a list price.
However, the product is not a pillar of Horizon's performance, but a monoclonal antibody teprotumumab (trade name: Tepezza) that targets IGF-1R, and the data shows that sales of the product reached $981 million in the first half of the year, an increase of 116% year-on-year.
Compared with other companies, Horizon's development model is completely different from Bluebird's own research and development, Horizon has been acquiring products through mergers and acquisitions since its listing.
Founded in 2008, Horizon focuses on the discovery, development and commercialization of drugs to meet the needs of patients with rare diseases, autoimmunity, and severe inflammation. Listed on the NASDAQ (formerly known as Horizon Pharma in 2019, it will be changed to Horizon Therapeutics in 2019) and has since grown through mergers and acquisitions.
After 2014, the company acquired Vidara Therapeutics International for US$660 million and Nuvo Research for the distribution rights of Pennsaid (diclofenac sodium) for US osteoarthritis drugs in the United States; Acquired Crialta Holdings in 2015 for $510 million; Acquired Raptor Pharmaceutical, a rare and severe drug research and development company, for $800 million in 2016; Acquired River Vision Development Corp for $145 million in 2017 for Teprotumumab, the company's research ophthalmic product; In 2021, it acquired Viela Bio for $3.05 billion...
In addition, Horizon is also collecting good ideas from entrepreneurs around the world, setting up a horizon prize to encourage entrepreneurs and innovators around the world to think creatively on a problem every year, and selecting up to two winners among the thousands of innovators, scholars and inventors who submit solutions, sharing $150,000 in prize money.
Y-mAbs
Market capitalization $756 million
Y-mAbs' net profit attributable to common shareholders of the parent company for the first half of 2022 was -69.199 million US dollars, down 760.55% year-on-year; Operating income was $21.283 million, up 30.3% year-on-year.
Its main product, DANYELZA (naxiltimab), was approved in November 2020 for an annual treatment cost of nearly $980,000. In the first half of this year, the product generated $20.3 million in revenue. The company forecasts that DANYELZA's full-year 2022 revenue will be $45 million to $50 million.
Y-mAbs disclosed that as of June 30, the company had a cash position of $133.7 million, which could support the company's development through mid-2024. Total cash consumption in 2022 was $78 million to $83 million, and operating expenses were $162 million to $167 million.
Y-mAbs is currently expanding its indications for DANYELZA (narcetimab) and is also advancing the development of another product, OMBLASTYS (omburtamab).
It is worth noting that in December 2020, the domestic pharmaceutical company Saison Pharmaceutical has reached an exclusive indefinite licensing agreement with Y-mAbs for two products, DANYELZA and OMBLASTYS, and obtained the exclusive right to cooperate in the development, registration and commercialization of these two products in the Greater China region (Chinese mainland, Hong Kong, Macau and Taiwan). In July 2021, Sci Johnson Pharmaceutical submitted its listing application to NMPA for DANYELZA and was qualified for priority review in September 2021.
OMBLASTYS' Biological Product Licensing Application (BLA) was accepted by the U.S. Food and Drug Administration ("FDA") in June this year primarily for the treatment of neuroblastomas with central nervous system/pia mating in children and was granted priority review. Under the U.S. Prescription Drug Declarant Fees Act (PDUFA), the fda's target date for making a decision is set for November 30, 2022. The product is expected to be the world's first targeted therapy for patients with neuroblastoma with central nervous system/pia mating.
Eiger
Market capitalization of $400 million
According to Eger's semi-annual report, its net profit attributable to common shareholders of the parent company in the first half of 2022 was -44.527 million US dollars, down 542% year-on-year; Operating income was $6.764 million, up 17.78% year-on-year. The company had $140 million in cash and cash equivalents as of June 30, and the company said it would fund its operations by 2024, the data showed.
For Eiger, its product, Zokinvy (lonafarnib), is instrumental in the company's growth. Approved for the treatment of Progeria and related diseases in 2020, Zokinvy is the first FDA-approved drug to treat Progeria at an annual cost of up to $1.03 million.
The product has been approved in Europe and has reached an exclusive partnership with AnGes japan to seek approval and commercialization of lonafarnib in Japan. However, Zokinvy's main clinical program targets infection with hepatitis D (HDV), the most severe form of viral hepatitis in humans. In addition, Zokinvy's clinical research for hepatitis D (HDV) has entered the clinical phase III phase. It is understood that 5 products in the Eiger product pipeline have been recognized by the FDA as breakthrough therapies.
Its founder and CEO, David Cory, has many years of successful entrepreneurial experience, having previously served as president and chief operating officer of Prestwik before being acquired by Biovail for $160 million. Cory was also a co-founder of Pulmonary Hypertension Orphan Drug Company CoTherix, which was later acquired by Actelion for $425 million. In addition, he worked at InterMune, a company specializing in the development of idiopathic fibrotic orphan drug products, and was a senior vice president and key figure in charge of the company's first IPO, which was acquired by Roche in 2013 for $8 billion.