Fail to Launch: Affordable Gene Therapy from China - A Realty Within Reach?

Recent developments in the gene therapy industry have cast doubt on the commercial viability of these treatments, particularly in the context of hemophilia.

Notably, bluebird bio, once a frontrunner in gene therapy innovation, has agreed to be acquired by private equity firms Carlyle Group and SK Capital Partners for approximately $29 million.

?This valuation is a stark contrast to the company's peak market capitalization of around $9 billion. Under the terms of the agreement, bluebird shareholders will receive $3.00 per share in cash, with an additional contingent value right of $6.84 per share, payable if the company's products achieve $600 million in net sales within 12 months before December 31, 2027.

Pfizer's Setback

In a parallel move, Pfizer has announced the discontinuation of its hemophilia B gene therapy, Beqvez, less than a year after its FDA approval in April 2024. The company cited limited interest from both patients and healthcare providers as the primary reason for halting global development and commercialization of the therapy.

Beqvez, a one-time treatment, was initially priced at $3.5 million per patient, mirroring the cost of CSL Behring's Hemgenix, another hemophilia B gene therapy. Despite its potential, patient uptake has been minimal, leading to Pfizer's strategic withdrawal from this segment.

These events raise critical questions about the sustainability and affordability of gene therapies in their current form. The high costs associated with these treatments, coupled with modest demand, suggest that alternative approaches may be necessary to make gene therapy a viable option for a broader patient population.

Emerging developments from Chinese gene therapy developers offer a glimmer of hope in this challenging landscape.

One-Tenth of the Price

An executive from a Chinese gene therapy company, which has entered into a commercialization agreement with a multinational pharmaceutical firm, indicated that their production costs are approximately one-tenth of the current U.S. market prices for similar therapies.

This significant reduction could potentially make gene therapies more accessible and affordable for patients worldwide.

Moreover, when compared to alternative treatments such as Pfizer's once-weekly antibody drug, Hympavzi (marstacimab-hncq), the cost of the Chinese-developed gene therapy is estimated to be equivalent to just six months' worth of the antibody treatment.

Hympavzi, approved by the FDA for hemophilia A and B, offers a more convenient administration route but still entails ongoing costs that may be burdensome for patients without adequate insurance coverage.

Hemophilia, a bleeding disorder that can lead to severe joint damage and disability due to recurrent bleeding episodes, affects a significant number of individuals worldwide. In China, over two-thirds of patients experience bleeding-associated functional disabilities.

Once-Weekly vs. One-Time Treatment

While Pfizer has announced plans to introduce Hympavzi in Hainan Boao Special Zone prior to obtaining full regulatory approval in China, the lack of insurance coverage means that patients would bear the full cost of this once-weekly treatment, posing a substantial financial challenge.

The Chinese gene therapy company acknowledges that antibody injection needs one in a week, compared to just a one-time treatment for the gene therapy.

Similar hurdles also exist in securing public health insurance coverage for gene therapies within China.

As a result, their initial strategy focuses on targeting the self-pay market, aiming to provide a more cost-effective solution for patients willing and able to bear the out-of-pocket expenses.

In conclusion, while recent setbacks in the gene therapy sector highlight the financial and logistical challenges inherent in bringing these advanced treatments to market, innovative approaches from emerging markets like China may offer a pathway to more affordable and accessible solutions.

Collaborations between multinational pharmaceutical companies and cost-efficient developers could play a pivotal role in realizing the full potential of gene therapy for patients globally.

Other Significant News:

U.S. President Trump is considering levying 25% tariffs on pharmaceutical products from overseas, if enacted, it will be the first tariffs on drug products in three decades -- The Hill

Riding its obesity drug wave, Eli Lilly to invest $ 27 billion in U.S. drug manufacturing to build 4 production sites. - USA Today

Amid Trump's America First Investment order, Hong Kong is pushing forward its listing reforms and launch a “Tech Express" initiative to make public offerings by technology and biotech companies -- Caixing Global

Will Russia Help Washington to Challenge China's Rare Earth Dominance? -- South China Morning Post

South Korea records first growth in newborns in 2024 in nine years, due largely to the weddings during the COVID pandemic -- Yonhap News

Celltrion, South Korea's major biosimilar maker, saw growth accelerating due to strong demand for its bio-generic drugs -- Yonhap News

Merck, also known as MSD outside the U.S. and Canada, plans to significantly expand its workforce in India, aiming for around 2,700 employees by the year's end. The company focuses on therapy areas such as oncology, diabetes, and vaccines and has recently inaugurated a new technology center in Hyderabad -- Economic Times

(Comments, email to brianhxyang @yahoo.com)