.png)
In addition to earnings reports, we've had our Eyes On some of the latest Pharmaceutical news highlights including approvals, partnering, mergers, research programs, and vaccine supplies. What's caught your eye lately?
Ascendis Pharma’s hormone replacement therapy for hypoparathyroidism has been approved by the FDA, making it ready for its U.S. launch. The company had planned to release the drug to the market fifteen months prior, but it was delayed by the FDA’s initial rejection of the product, which was accompanied by a surprise complete response letter (CRL). In May 2024, the U.S. regulator stated that Ascendis needed an extra three months to make its judgment, even though the company was once more prepared to move forward.
TransCon PTH (the once-daily Yorvipath (palopegteriparatide) is the first product for hypoparathyroidism in adults to be FDA-approved. Ascendis anticipates the first supply to be accessible in the first quarter of 2025. Still, there is a chance the launch will be pushed up to the end of 2024 after requesting FDA approval to commercialize already produced doses. The UK approved Yorvipath earlier this year, and the European Union did so in November 2023. According to Ascendis, more than 250 people are utilizing Yorvipath in Germany and Austria, where the treatment was initially introduced.
Analysts predict that the annual price for Yorvipath could be higher than $160,000 and forecast 2025 sales of the drug to reach $200 million globally and $100 million in the U.S., which assumes 1,300 to 1,500 U.S. patients would be using the product.
For more details:
Exscientia has merged into Recursion, forming a single company with ten clinical readouts scheduled for the upcoming eighteen months. To enhance its scaled biology research and translational capabilities, Exscientia will integrate its small molecule automated synthesis technology and precision chemistry design into Recursion. In addition to a potential $20 billion in future royalties, if any medications from the pipeline are approved. The merged company will have $850 million in cash and approximately $200 million in planned milestones over the next two years.
Exscientia, a company that uses AI to advance drug discovery, partnered with several major pharmaceutical companies, including GSK, Bristol Myers Squibb, and Sanofi, during its early years. However, Bayer ended a $243 million (240 million euro) partnership in 2022, and even though Exscientia added a partnership with Merck KGaA in September 2023, one month later, the company started to reduce the size of its quickly growing pipeline.
According to Exscientia and Recursion, their merger could allow them to establish an asset portfolio with potential annual peak sales opportunities of over $1 billion. Key products include Exscientia’s CDK7, LSD1, and MALT1 oncology programs for PKC-Theta and ENPP1. Each company gives attention to particular areas: Exscientia is focused on oncology therapies, and Recursion on oncology, infectious disease, and rare disease medicines. The combined power of each biotech's technology platforms should support the new company’s efforts in drug discovery.
For more details:
Merck & Co. is taking on Amgen in the blood cancer market by paying an upfront sum of $700 million for worldwide rights to Curon Biopharmaceutical's CD3xCD19 bispecific, putting the Big Pharma in direct competition with AstraZeneca and Amgen in the oncology space and Cullinan Therapeutics in the autoimmune disease space. In return Curon Biopharma received rights to the phase 1/2 candidate CN201 in exchange
The mechanism that gave rise to the bispecific antibody business was the engagement of CD3 and CD19. Receiving FDA approval in 2014, Amgen’s trailblazing T-cell engager Blincyto meets the two targets to treat acute lymphoblastic leukemia. Despite Blincyto’s head start, companies have detected weaknesses that they make take advantage of, and new research indicates that there be unrealized autoimmune potential.
For more details:
In response to the 2022 epidemic of mpox, the disease formerly known as monkeypox, Bavarian Nordic is receiving a $156.8 million award from the Biomedical Advanced Research and Development Authority (BARDA) to partially restore vaccination supplies. Prior to this, BARDA contributed to the development of the vaccine in a freeze-dried form which was approved by the FDA in 2019. The vaccine bulk product is to be manufactured and invoiced in 2024, with Bavarian Nordic being expected to receive $139.7 million. According to Bavarian Nordic, the remaining $17 million part of the deal will support new services from 2025 to 2027, such as the storage of vaccination doses in the U.S. In 2024, Bavarian Nordic anticipates to make between $395 million (2.7 billion kroner) and $439 million (3 billion kroner) in public preparedness business.
Bavarian Nordic received their first Jynneos replenishment deal in 2023. A few months after the company launched Jynneos on the U.S. commercial market, the most recent replenishment arrangement was reached. The vaccine is currently available to interested parties in physician offices, local pharmacies, and a select few clinics. The U.S. CDC's Advisory Committee on Immunization Practices suggested Jynneos as a regular immunization for adults at risk of mpox infection in October 2023, which preceded its commercial introduction.
For more details:
Halda Therapeutics has raised $126 million to fund ‘hold and kill’ solid drugs as part of its RIPTAC (Regulated Induced Proximity Targeting Chimeras) program. This will involve the development of a heterobifunctional molecule aimed at a cancer-specific protein and essential function protein, with the ability to kill a cancer cell while protecting non-cancerous tissue that does not express the cancer-specific protein. One of the significant drawbacks of many existing cancer treatments is drug resistance, but this broadly applicable product is specifically designed to avoid this tendency. Funded by their $126 million series B extension, Halda is now prepared to accept candidates for a phase 1 trial in castration-resistant, metastatic prostate cancer in the first half of 2025. A portion of the funds will also be utilized to advance a different RIPTAC candidate into a trial for metastatic breast cancer.
In 2023, Halda revealed preclinical data that they stated demonstrated RIPTAC therapeutics could provide a more effective prostate cancer treatment than Pfizer’s Xtandi. Patients with earlier stages of cancer as well as advanced cancer patients with heterogeneous resistance adaptations may benefit from the medication.
For more details:
Daiichi Sankyo is investing in the advancement of MK-6070, a trispecific T-cell engager that Merck purchased earlier this year when it acquired Harpoon for $650 million. Originally developed as HPN328, MK-6070 is intended to bind DLL3 on tumor cells and CD3 on T cells. Albumin is bound by the third domain, extending the half-life. Over 70% of small cell lung tumors express DLL3. Ifinatamab deruxtecan, an ADC directed by B-7-H3 that has recently entered phase 3 in small cell lung tumours, was part of the original agreement between Merck and Daiichi. The companies want to investigate the ADC and trispecific in combination in certain patients with small cell lung tumors.
Merck & Co. has swiftly recovered a portion of the expenditures associated with its acquisition of Harpoon Therapeutics, earning $170 million up front by combining the major candidate into a co-development agreement with Daiichi. Phase 1/2 trials are in underway for MK-6070. Amgen’s tarlatamab, a competitor DLL3 candidate, is in phase 3, but it does not have the same opportunities for combination that the Daiichi-Merck agreement provides.
For more details:
PharmaTell Studio Hints
PharmaTell Studio includes many different types of research reports, from weekly/monthly trackers to Industry and Sector Overviews. Be sure to search each catalogue for topics of interest, as they may be filed under Therapeutic Areas or Industry Intel if several companies/areas are covered.
If you are a PharmaTell client and can’t access the PharmaTell-specific links, please contact us at helpdesk@pharmatell.com. Â
If you are not a client and would like to learn more about PharmaTell and a free trial, please email us at info@pharmatell.com, and we will be happy to assist you.Â
Comentarios