The financial pressure is now causing concern among some biotech companies that have already launched their products.

Recently, X4 Pharmaceuticals announced that it will lay off 43 employees, accounting for approximately 30% of its global workforce. This wave of layoffs includes the closure of the "Center of Excellence" located in Vienna, Austria, and the suspension of the development of all preclinical drug candidates. Layoffs and business downsizing will save the company $30 million to $35 million.

This company just welcomed the launch of its core pipeline XOLREMDI (mavorixafor) less than a year ago (April last year), however, the product launch does not mean that X4 has reached the safe line of cash flow.

According to last year's Q3 financial report, excluding XOLREMDI's revenue, X4 company's cash is only sufficient to operate until the end of 2025. However, after layoffs and integration, X4 is expected to extend its cash flow to the first half of 2026.

Mechanism of Core Pipeline

X4 Pharmaceuticals itself is more like a new venture of employees who left the old Jianzan after the Sanofi/Jianzan acquisition, relying on old and undeveloped CXCR4 inhibitor projects. For example, co-founder Dr. Alan Walts, CEO Dr. Paula Ragan, and COO Alison Lawton are all veteran employees of Jianzan with over ten years of work experience.

The key is that the older generation CXCR4 inhibitor Plerixafor is an old drug developed by Jianzan.

Initially, X4 planned to focus its development on the field of cancer, particularly renal cell carcinoma. Around 2019-2020, it was found that the therapeutic effect of WHIM syndrome was more prominent, so the focus shifted to the development of WHIM syndrome.

WHIM syndrome is a rare primary immunodeficiency disease, named after the English acronyms of four main clinical manifestations: Warts, Hypogammaglobulinemia, Infections, and Myelokatexis.

WHIM syndrome is caused by acquired mutations in the CXCR4 gene. CXCR4 itself is a chemokine receptor that normally binds to its ligand CXCL12 to regulate the migration and homing of immune cells. In patients with WHIM syndrome, excessive activation of CXCR4 leads to the retention of immune cells (especially neutrophils) in the bone marrow, which cannot be released into the peripheral blood normally, resulting in immune deficiency and infection.

By blocking the CXCR4 signal with the CXCR4 inhibitor mavorixafor, the retention of neutrophils in the bone marrow can be reduced, allowing them to be released normally into the peripheral blood, thereby improving neutropenia. At the same time, it also helps regulate immune cell function and enhance overall immune response.

Can it hold up a sky for the company?

However, in the end, WHIM syndrome is a rare disease, and in many cases, patients may not even realize that they have this disease and lack understanding of its treatment. This is also one of the reasons why X4, after being approved for market in April, will launch a doctor peer lecture program in Q3 to raise awareness and understanding of the disease.

As the first rare disease drug targeting WHIM syndrome, the price of mavorixafor has also skyrocketed.

For patients taking a daily dose of 400 mg, the annual cost of mavorixafor is $496400. For patients taking a dose of 300 milligrams, the annual cost is $372300.

In 2019, X4 Company conducted research on the US market and found that there were over 1300 cases of WHIM syndrome in the United States, and if undiagnosed potential cases were included, the highest number could reach 3700. In theory, if full patient coverage can be achieved, future revenue would indeed be very considerable, and could even become a "heavyweight bomb" worth over $1 billion.

However, theory is theory, whether patients are willing to purchase and whether they can detect the disease in a timely manner is another matter. Currently, the net income of mavorixafor Q3 is $600000, and there is no clear conclusion on whether the future of mavorixafor is promising.

However, in markets outside the United States, the company is working hard to advance the process of going public.

Last month, X4 signed a licensing agreement with European pharmaceutical company Norgine to commercialize Mavorixafor in Europe, Australia, and New Zealand. The agreement brought X4 a prepayment of 28.5 million euros (approximately 29.55 million US dollars) and regulatory and commercial milestone payments of up to 226 million euros (approximately 234.29 million US dollars). The listing application submitted in the EU is expected to have a result by early 2025.

Among other indications, chronic neutropenia is another one that the company is vigorously developing, and transitioning from phase II to phase III in clinical practice requires greater costs. And this may be a larger market indication for the pipeline to break through the upper limit of rare disease treatment.

By comparison, the importance of the pre clinical pipelines X4P-002 and X4P-003 that were cut off this time does not seem to be as high.

summary

Overall, mavorixafor does have some potential, but it may take several quarters to observe sales performance. The clinical success of chronic neutropenia will play a decisive role in the future prospects of mavorixafor.