Osteoporosis

President Joe Biden has called on agencies to integrate women’s health across the federal research portfolio to ensure the government is best using all of the funds to advance research. Ten days after asking Congress to fund women’s health research with $12 billion, President Joe Biden is doing what he can to boost investment via an executive order to expand and improve research on women’s health. The orders come with 20 new actions across federal agencies such as the Department of Health and Human Services, under which the FDA sits. Within the actions is a $200 million National Institutes of Health initiative for fiscal year 2025 to fund new women’s health research. This would be the first step in the call to action issued by Biden at the State of the Union on March 8. Through the orders, Biden has called on the agencies to integrate women’s health across the federal research portfolio to ensure the government is best using all of the funds to advance research. The executive order also calls on the Office of Management and Budget and the Gender Policy Council to determine gaps in federal funding for women’s health research. “Agencies will take action to ensure women’s health is being considered at every step in the research process—from the applications that prospective grantees submit to the way that they report on grant implementation,” the White House said in a fact sheet issued Monday. The president also directed federal agencies to prioritize investing in women’s health research, specifically the Advanced Research Projects Agency for Health (ARPA-H), which received $100 million in February for the “Sprint for Women’s Health.” One key focus of the new orders is on women’s midlife health, including health issues that are more likely to occur after menopause, like rheumatoid arthritis, heart attack and osteoporosis. The FDA has specifically been asked to help address gaps in research and the availability of products that address conditions that primarily impact women, or for conditions that impact women differently. The agency will also provide industry guidance on the inclusion of women in clinical trials and reach out to stakeholders to discuss new opportunities to advance women’s health. The NIH has pledged to up funding of the Small Business Innovation Research Program and the Small Business Technology Transfer Program by 50% to support smaller companies engaged in R&D on women’s health. Another part of the order addresses the standardization of data collection that could help researchers share and combine datasets, promote interoperability and improve accuracy of data related to women’s health.

AstraZeneca is bolstering its rare disease pipeline with a deal to buy Amolyt Pharma, a biotech company in late-stage development with a therapy designed to treat a rare hormone deficiency whose only FDA-approved treatment will soon cease production. According to deal terms announced Thursday, AstraZeneca is paying Amolyt shareholders $800 million up front. Another $250 million is tied to the achievement of a regulatory milestone that was not disclosed. Considering that the drug is in Phase 3 testing, that milestone could be a regulatory approval. Lyon, France-based Amolyt is developing a therapy for hypoparathyroidism, a disease in which the parathyroid glands do not produce enough parathyroid hormone. This hormone is important for regulating calcium and phosphate levels in the blood. Disease symptoms include tingling or burning sensations in the fingertips and toes, muscle aches or cramps, muscle twitching or spasms, and fatigue. Hypoparathyroidism disproportionately affects women. Calcium and vitamin D supplements can help, but at high doses, calcium supplements can lead to gastrointestinal problems such as constipation. When supplements alone aren’t enough to manage hypoparathyroidism, the only FDA-approved therapy is Natpara, an engineered version of parathyroid hormone sold by Takeda Pharmaceutical. Amolyt could bring patients a different way to manage hypoparathyroidism. The biotech’s drug candidate, eneboparatide, is a peptide designed to bind to PTH receptor 1, the receptor that native parathyroid hormone binds to. In Phase 2 results reported in 2022, Amolyt said its drug led to normal calcium levels in the blood as well the potential to eliminate the need for daily calcium and vitamin D supplements. Furthermore, the drug preserved bone mineral density, which is important for patients at higher risk of developing bone problems such as osteopenia or osteoporosis. The market need for new hypoparathyroidism treatments is becoming urgent. Takeda’s Natpara (Natpara outside of the U.S.) has had a rocky commercialization history since its FDA approval in 2015. In 2019, the FDA issued a recall due to potential contamination from tiny rubber particles from the cartridge housing the injectable product. Takeda’s efforts to resolve the problem to the FDA’s satisfaction have been unsuccessful. In late 2022, Takeda said it planned to discontinue manufacturing of Natpara/Natpar by the end of 2024. After that, the company said it would supply the remaining doses of the drug until inventory is depleted or the product is expired. The Amolyt acquisition brings AstraZeneca into a group of companies vying to fill the hypoparathyroidism treatment need that will be left when Takeda’s drug is no longer available. Extend Biosciences is in Phase 1 testing with a drug derived from parathyroid hormone. MBX Biosciences has reached Phase 2 testing with a peptide drug. Both drug candidates are injectable. Septerna is developing an oral drug for hypoparathyroidism. Last year, the South San Francisco-based startup raised $150 million in financing to advance the small molecule to Phase 1 testing. Amolyt last raised money in early 2023, a €130 million (about $138 million) Series C financing to support its pipeline. Besides the hypoparathyroidism program, that pipeline includes AZP-3813, a potential treatment for acromegaly, a disease in which the body produces too much growth hormone. Phase 1 testing began last summer. Amolyt has said it expects preliminary data in the first half of 2024. AstraZeneca expects to complete the Amolyt acquisition by the end of the third quarter of this year. When the deal closes, Amolyt will become part of Alexion, AstraZeneca Rare Disease, the pharmaceutical giant’s Boston-based rare disease division. AstraZeneca said the acquisition will enable it to expand its rare disease research into endocrinology. “Chronic hypoparathyroid patients face a significant need for an alternative to current supportive therapies, which do not address the underlying hormone deficiency,” Marc Dunoyer, CEO of Alexion, AstraZeneca Rare Disease, said in a prepared statement. “As leaders in rare disease, Alexion is uniquely positioned to drive the late-stage development and global commercialization of eneboparatide, which has the potential to lessen the often debilitating impact of low parathyroid hormone and avoid the risks of high-dose calcium supplementation.”

AstraZeneca’s rare disease portfolio generated $7.76bn in global sales in 2023, as per the company’s Q4 financials. Image Credit: EllaSt / Shutterstock. AstraZeneca has signed a definitive agreement to acquire Amolyt Pharma, which specialises in therapies for rare endocrine diseases, in a deal worth $1.05bn. As per the contract, AstraZeneca will pay $800m upfront along with potential regulatory milestone payments of $250m. The deal is expected to close in Q3 2024. Amolyt’s lead candidate, eneboparatide (AZP-3601), was a central part of the acquisition. Eneboparatide is a subcutaneous parathyroid hormone receptor 1 (PTHR1) agonist for the treatment of hypoparathyroidism. This condition is caused by a deficiency in the parathyroid hormone that results in decreased calcium and elevated phosphorus levels in the blood. Dysregulation of parathyroid hormone can lead to osteoporosis and chronic kidney disease. Amolyt launched a Phase III CALYPSO trial (NCT05778071) for eneboparatide in May 2023. Topline data from the trial is expected by the end of this year. Another candidate in Amolyt’s pipeline is AZP-3813. The therapy is a peptide growth hormone receptor antagonist, which is being developed as a potential add-on to somatostatin analogues for the treatment of acromegaly, a condition associated with abnormally high secretion of growth hormone. It can lead to a variety of complications including heart failure and impaired glucose tolerance. The company initiated a Phase I trial for AZP-3813 in June 2023. See Also: Almirall to license Eloxx’s ZKN-013 for rare skin conditions Risk adjusted net present value: What is the current valuation of AstraZeneca’s AZD-3427? AstraZeneca’s rare disease portfolio generated $7.76bn in global sales in 2023, as per the company’s Q4 financials. The UK pharma giant has been investing heavily in rare disease therapies starting with the $39bn acquisition of Alexion in 2021. Last year, the company paid $1bn to acquire an early-stage gene therapy portfolio for rare diseases from Pfizer. According to a GlobalData report, innovation in rare diseases is seen as a risky investment, with rare oncology indications dominating most of the rare disease pipeline. GlobalData is the parent company of Pharmaceutical Technology . Despite this, AstraZeneca has seen some success amongst rare diseases that are not cancer-related. In September 2023, the Phase III trial for a rare vasculitis therapy, Fasenra (benralizumab), met its primary endpoint. The study found that Fasenra was non-inferior to the current standard of care, Pfizer’s Nucala (mepolizumab). Earlier this year, AstraZeneca reported positive outcomes from its Japan Phase III clinical trial of acoramidis, an investigational treatment for a rare cardiomyopathy, transthyretin-mediated amyloid cardiomyopathy (ATTR-CM).