In this week’s InnovationRX edition, we look at the latest drug pricing requirements, a biotechnology entrepreneur facing a rare genetic disease, the new technology aimed at ending animal drug testing and much more. To take it to your inbox, Sign up here.
THEThe weekendPresident Trump told reporters that his administration had Reduce drug prices “1500%.” On Sunday he returned this claim to say that it would happen in the future, despite the fact that it is a mathematical weakness, unless the administration was a bit of a drug to pay patients to use their products.
Still, Trump’s threats to drug prices have become stronger. On Friday, the White House sent letters to 17 pharmaceutical companies, such as AbbVie, Pfizer, Novo Nordisk and Eli Lilly, demanding to reduce prices by the end of September.
The administration wants these companies to reduce US prices according to other countries that pay as described in an executive mandate since May. This “most favored nation” pricing requires that drug companies reduce prices by 30% to 80% to match prices in other developed countries. Trump threatened to “develop every tool in our arsenal” to reduce prices if companies do not comply, but the administration does not have that many legal tools and Congress ignored Trump’s request to give him the power of enforcement earlier this year.
Another hiccups in Trump’s desire to reduce drug costs? His commercial policy. On Tuesday, the President told CNBC that he was planning to put into effect a 250% invoice For imported medicinal products, they must be gradually abolished over the next two years. Assuming that such an invoice has survived the judicial challenges (its power to implement invoices is the subject of multiple pipelines) or legislated the Congress correctly, will massively increase the price of drugs in the United States. Although drug companies have announced new production plants in the US, this is a very short time for many to get up and run. And the cost of US drug production makes it unlikely that generic manufacturers will move here at all.
How a 60 -year -old drug programmer created a 4 billion dollar craft to treat “butterfly skin disease”
Suma Krishnan of Krystal Biotech
Jamel Toppin for Forbes
largeOngtime Drug Developer Suma Krishnan It was in the late ’40s, when it had the idea of a local gene therapy to cure a rare and terrible skin disorder in which the skin becomes as fragile as butterfly wings. In 2016, at 51, after a few months of modeling the idea and the launch of the patent registration process, she and her husband Krishnan, with whom she worked in biotechnology for more than a decade, co -exists Krystal Biotech.
Focusing on a rare disease, with just thousands of patients in the US, was unusual. They were also avoided by business capital in favor of self -financing a biotechnology start, with about $ 5 million mainly made by previous biotechnology companies. But perhaps the biggest bet was science, adopting a completely new approach to a problem with a gene therapy that was delivered as a gel that could potentially deliver great if it succeeded, but also had a high chance of failure. “I had to work with the regulators because they had never seen it,” Krnnan said Forbes. “It was completely new.”
Just 18 months after the start of the company based in Pittsburgh, the Krishnans took it publicly on the Nasdaq Stock Exchange. Krystal now has a $ 4 billion (from the market market) with an FDA -approved treatment, Vyjuvek, for “Butterfly Disease”, called Bullosa’s dystrophic skin, market, and other genera treatments, including bullshit, including bullies, including bulls, including bulls Various stages of clinical development. Everyone is based on modified herpes simplex virus, but they have different delivery mechanisms. The company’s revenue amounted to $ 291 million for $ 2024, over five times more than $ 51 million in $ 2023. Net income increased eight times to $ 89 million from $ 11 million in the same period.
Krishnans have a combined 12% share in the business. SUMA, president of the company’s research and development, is worth about $ 275 million from Tower Estimates (from Tuesday), almost entirely from its share of business and stock sales over the years. Krystal’s stock was unstable: it is over 1,200% from the IPO, but has decreased almost 25% in the past year.
“You have to be brave and bold to do that,” says Krishnan. “I was never afraid of taking risks. I never felt like I needed a steady job.”
Read more here.
Biotechnology and pharmacy
Animal tests have been identified by law since 1937, when a new wording of a common antibiotic had a poisonous new ingredient – and killed more than 100 people. Nearly a century later, drugs still pull from the shelves because they have toxic effects, even though animal tests have shown that they were safe. Now politicians, scientists and entrepreneurs are pushing for new, more precise ways to test drugs before they reach human clinical trials – may save lives and billions of dollars in the process.
In 2022, a group of scientists ran an experiment with 27 known drug compounds that studies on animals had clusters of cells integrated into a lightweight electronic device that could simulate the behavior of an instrument. The researchers found that the liver-on-a-chip organs predicted accurately which compounds were dangerous. More accurate tests using instrument chips could save the industry more than $ 3 billion a year, the authors of the study were calculated.
In addition to safety, the cost is another reason to move away from animal tests. Today, drug companies can spend more than $ 2 billion to bring a single drug to the market, with industry spending nearly $ 300 billion a year on research and growth. Despite the huge R&D spending, over 90% of drug candidates fail. It is a wasteful process, contributing to the prices of drugs on the market.
Read the whole story.
Plus: Minister of Health and Human Services Robert F. Kennedy Jr. He announced that it canceled about $ 500 million grants and MRNA vaccines. This includes contracts in pharmaceutical companies working in MRNA vaccines. (Although one of the names of the companies controlled in the announcement, Moderna is quite interesting Tower that there is no current contracts.) The move threatens the United States’ ability to fight infectious diseases or a possible future pandemic and could undermine the American biotechnology industry.
Digital health and ai
The Hungarian start of the AI turbine signed a research cooperation With Europe Merck & Co. Develop AI models in the fight against Turbine Cancer will use the fundamental AI models to simulate hard to treat compact tumors in patients. There are many types of cancer that are difficult to deal with because cells cannot be cultivated in the laboratory, Turbine Sazboics chief executive Nagy said Tower. To train AI models, the turbine will use data from these cancers as shown in animal models, organoids and certain types of patient data. As soon as he trains, Nagy said, virtual cell models can test medicines in a way that is impossible in the laboratory, opening opportunities for “treatment of cancer in populations of patients with inadequate service”.
Plus: Ultromics based in Oxford, which has developed FDA algorithms for detecting heart disease, set $ 55 million led by investors L&G, Allegis Capital and Lightrock.
Mold
Setpoint Medical received FDA approval for tThe adjustment system, an implantable device for the treatment of patients with moderate to severe arthritis that cannot be treated with existing drugs. The device electrically stimulates the lung nerve to cause anti -inflammatory responses.
Public health and hospitals
Vaccination rates between children entering the kindergarten fell during the school year 2024-2025, According to new statistics from the disease control centers. The decline comes as the measles epidemic continues to grow, sick of more people than at any time since it is declared. THE New data shows vaccination coverage ranging from 92.1% for vaccine diphtheria, tetanus and abnormal granular (DTAP) up to 92.5% for measles, mumps and red (MMR).
Plus: Gates Foundation is bound by $ 2.5 billion for women’s health Initiatives.
What do we read
New pre -backed colon screening recommendations for patients under 50 have led to More people in the ’40s were diagnosed early When the disease is therapeutic.
Medicare and Medicaid will Experiment with weight loss drugs According to a proposed five -year plan by Trump’s administration, according to documents received from the Washington Post.
A new study finds that consuming highly processed food harder to lose weight– Even when the diet falls differently in the dietary guidelines.
The Kentucky Hospital Association warned These Medicaid cuts will force hospitals across the state to reduce services or even close their doors.
Google AI’s health care consisted of a part of the body in the brain, showing the possible severity of AI errors in clinical environments.
The chikungunya virus transmitted by mosquitoes has contaminated more than 7,000 people in at least 13 cities in China.
Cases of Legionnaires’ illness in New York arrived on Tuesday, as the developing outbreak in the city has led to three deaths.
More than Forbes
