Medication – Page 104 – Relatix Health

April 6, 2026

Rates of Treatment-Related Blood Cancers Are Increasing in Japan

Research led by Osaka International Cancer Institute in Japan shows that rates of therapy-related acute myeloid leukemia, linked to treatment for an earlier cancer, are going up as the overall number of cancer survivors increases.

As reported in the journal Cancer, the researchers showed the rates of treatment-related acute myeloid leukemia (AML) almost tripled between 1990 and 2020. The share of overall AML cases taken up by these patients has also increased from 4.4% to 8.2% over a similar time period.

With increasing numbers of cancer survivors in the U.S. and elsewhere, understanding the adverse effects of cancer treatment is becoming increasingly more important. Therapy‑related AML is a rare but serious complication of cytotoxic chemotherapy, and different chemotherapy drugs carry different levels of leukemia risk. Radiotherapy can also lead to this condition, but the risk is considered lower than that of chemotherapy.

“Several population‐based studies have analyzed the long‐term trend of therapy-related AML incidence, and the results are controversial,” write lead author Kenji Kishimoto, MD, PhD, a researcher at the Osaka International Cancer Institute, and colleagues.

“A national population‐based study did not demonstrate an increase in the incidence of therapy-related AML in Denmark between 2000 and 2013. In contrast, a significant increase in therapy-related AML incidence between 1997 and 2015 was identified in a Swedish nationwide study.”

As few such studies have been carried out in Asia, Kishimoto and colleagues analyzed how such rates have changed over time and whether the initial cancers leading to therapy-related AML have changed in Japan.

The researchers identified 9841 patients with AML in the Osaka Cancer Registry, 636 (6.5%) of whom had therapy-related AML. These patients were older when diagnosed than other AML patients at a median of 69 versus 66 years. There were also slightly more women with therapy-related AML than standard AML at 45% versus 40%. The time lag between first cancer and resultant AML was between two and 11 years (median five years).

In 1990, the incidence of therapy related AML was 0.13 per 100,000 people, but this had increased to 0.36 per 100,000 people by 2020. The share of all AML cases made up by therapy-related cases almost doubled from 4.4% in 1990 to 8.2% after 2010.

The most common earlier cancers before therapy-related AML were blood cancers (23%), breast cancer (15%), colorectal cancer (12%), and gastric cancer (9%). The mix shifted over time and gastric cancer became less common as the first cancer, while breast, head‑and‑neck, and lung cancers became more prominent.

“The study provides an important step towards better understanding how the nature of therapy-related AML is changing with the increasing number of cancer survivors,” said Kishimoto in a press release.

“Findings from this study lay the foundation for further studies to elucidate the mechanism of the change in therapy-related AML epidemiology,” add the authors.

The post Rates of Treatment-Related Blood Cancers Are Increasing in Japan appeared first on Inside Precision Medicine.

April 6, 2026

Top 5 Firms Engineering Healthcare in the CNS Space

Central nervous system (CNS) treatments are having a major comeback. These five precision medicine players plan to ride the resurgence.

After a decade of stagnation, the CNS space is seeing a revival in sales and R&D spending as the market was last year projected to surpass $80 billion for the first time since 2013 and hit around $127 billion.

Recent landmark approvals have brought attention back to the CNS, including the U.S. Food and Drug Administration (FDA)’s greenlight of Eisai/Biogen’s lecanemab (Leqembi) for the treatment of Alzheimer’s disease in 2023, and the FDA approval of Bristol-Myers Squibb’s schizophrenia treatment xanomeline/trospium chloride (Cobenfy) in 2024.

At the same time, Johnson & Johnson’s depression treatment, esketamine (Spravato), is on its way to blockbuster status, showcasing the growth potential of the CNS market.

These successes accompany an emerging shift in psychiatry clinical trials from subjective rating scales to more objective endpoints, including digital and physiological measures, with the potential to better tailor treatments to a patient’s biological makeup.

Startups and scaleups are attracting increasing investor attention for their potential to change the way we treat CNS conditions. Check out our list of the most exciting companies that have netted the biggest investor dollars.

1. Aerska

Founded: 2025 | Headquarters: Dublin, Ireland

Aerska’s name is derived from an Irish proverb stating that people survive in each other’s shelter, emphasising the strength of its team.

This team includes co-founder Jack O’Meara, previously co-founder of the liver-focused RNA interference (RNAi) biotech Ochre Bio, who is driven by the experience of loved ones suffering from Alzheimer’s disease.

Aerska is developing RNAi therapies for neurodegenerative conditions, including Parkinson’s and Alzheimer’s disease.

While there are already FDA-approved RNAi therapies on the market, such as Alnylam’s patisiran (Onpattro), these are typically focused on liver and cardiometabolic conditions rather than the CNS.

Aerska’s technology consists of antibody “brain shuttles” that bind to proteins on the blood-brain barrier (BBB). They then carry a payload RNA into the brain.

The payload, which is designed based on data-driven patient stratification and disease biomarkers, then silences specific genes driving the disease.

Aerska has already raised $60 million since its launch, including a $21 million seed round in October 2025 and a $39 million Series A round in February 2026, co-led by EQT Life Sciences and age1.

The company, which has research operations in the U.K., is using the latest funding to drive its pipeline programs toward clinical testing.

2. Beacon Biosignals

Founded: 2019 | Headquarters: Boston, Massachusetts, U.S.

Beacon Biosignals was co-founded by a team including its CEO—MIT neuroscientist Jacob Donoghue, MD, PhD—and its CTO, the machine learning researcher Jarrett Revels.

Boasting more than 100 employees, the company’s goal is to provide objective biomarkers in drug development that neurology and psychiatry have traditionally lacked compared with other areas of precision medicine.

Its FDA-cleared Waveband device measures the brain’s activity, known as electroencephalography (EEG), while patients sleep at home. The EEG data is then stored, quality-controlled, and fed into AI models that can guide the design of clinical trials.

For example, Beacon’s EEG data can identify patients with Alzheimer’s disease who have worse outcomes and might need a more targeted treatment or a different clinical trial than other patients.

Beacon raised $27 million in a Series A round in 2021 and an oversubscribed Series B round worth $86 million in November 2025.

The B round, which included investors such as Innoviva, Google Ventures, and Nexus NeuroTech, will help the startup to accelerate the discovery of neurobiomarkers and broaden clinical adoption of the technology.

Beacon acquired the French sleep monitoring company Dreem in 2023 to access its monitoring data and headband technology. Beacon then acquired the Ohio-based CleveMed in April 2025 to harness technology measuring breathing, oxygen, and other signals.

3. Brainomix

Founded: 2010 | Headquarters: Oxford, U.K.

Brainomix was founded by a team including CEO Michalis Papadakis, PhD, who was scientific director of the preclinical stroke lab at the University of Oxford.

Brainomix is dedicated to speeding up patient care in cases of stroke, where speedy treatment is key.

Brainomix’s flagship product, Brainomix 360 Stroke, is designed to harness AI to interpret brain scans and detect blood clots in patients with stroke, speeding up clinical decision-making.

The product involves a group of tools that automatically analyze images, including results from computed tomography (CT), CT angiography, magnetic resonance imaging (MRI), and CT perfusion.

Brainomix’s technology doubled the rate of thrombectomy treatment in patients with stroke and reduced hospital triage and transfer delays, according to a 2025 study.

The University of Oxford spinout is at a commercial stage, with operations in more than 20 countries, and is expanding into the U.S.

Brainomix raised a $21.2 million Series B round in 2021 and extended its Series C round from $6.5 million in March 2025 to $25.4 million in February 2026, with leading investors including Parkwalk Advisors and Hostplus. The proceeds will fuel the company’s expansion into the U.S. market.

Brainomix has also partnered with heavyweights, including Nvidia, Boehringer Ingelheim, Medtronic, and GE Healthcare.

Brainomix also has a product dedicated to disease monitoring in pulmonary fibrosis.

4. Circular Genomics

Founded: 2021 | Headquarters: San Diego, California, U.S.

Circular Genomics was spun out of the University of New Mexico, with its founders including CSO Nikolaos Mellios, PhD, and Alexander Hafez, PhD.

The company later moved its headquarters from Albuquerque to San Diego in March 2025 to access scientific and operational know-how from Eli Lilly at Lilly Gateway Labs.

Circular Genomics aims to equip medical professionals with a blood test to detect CNS conditions early, in addition to stratifying and guiding the treatment of patients.

Its technology involves using a polymerase chain reaction (PCR) test of a patient’s blood sample to screen for specific circular RNA molecules produced in the brain that can cross into the blood and be measured as a biomarker of disease in the CNS.

Commercially launched in 2024, Circular Genomics’ MindLight SSRI Antidepressant Response Test predicts whether a patient will benefit from common antidepressants called SSRIs with around 77% accuracy. This is designed to predict a patient’s most suitable antidepressants without needing months of trial-and-error approaches.

The company is applying its technology in Alzheimer’s disease, where the approvals of disease-modifying therapies such as Leqembi have led to demand for tests that can detect the disease at earlier stages than traditional tests.

Circular Genomics raised $15 million in a Mountain Group Partners-led Series A round in December 2025 to finance the development of its technology and expansion of its technology in Alzheimer’s disease.

The company also has its sights on other CNS conditions, including multiple sclerosis and Parkinson’s disease.

5. Omniscient Neurotechnology

Founded: 2019 | Headquarters: Sydney, Australia

Omniscient (o8t)’s founders include CMO Michael Sughrue, MD, a neurosurgeon aiming to improve anatomy maps for other surgeons, and machine learning expert Stephane Doyen, PhD.

o8t’s FDA-approved product Quicktome involves using a patient’s MRI brain scans and AI models to map out a patient’s brain circuitry. These maps, accessible from an electronic tablet, can guide surgery to minimize the risk of brain damage compared to using a generalized anatomical diagram.

Quicktome is already in use at major hospitals around the world, including major centers in the U.S. Its partners include U.S. surgical support firm META Dynamic and the U.S. medical device innovation center, The Jacobs Institute.

o8t has raised more than $60 million, and bagged $14 million (AUD 20 million) in January 2026 as part of a Series D round targeted to reach $25 million (AUD 36 million). The round was led by Australia’s National Reconstruction Fund (NRFC) and OIF Ventures, with the aim of keeping the company based in Australia.

The funding is earmarked to fuel the development and commercialization of Quicktome, and grow o8t’s Australian workforce by more than 40. The company also has operations in Atlanta, Georgia, U.S.

o8t also plans to expand the technology into high-growth markets, including brain computer interface targeting, stroke and traumatic brain injury.

Jonathan Smith, PhD, is a freelance science journalist based in the U.K. and Spain. He previously worked in Berlin as a reporter and news editor at Labiotech, a website covering the biotech industry. Prior to this, he completed a PhD in behavioral neurobiology at the University of Leicester and freelanced for the U.K. organizations Research Media and Society of Experimental Biology. He has also written for medwireNews, Biopharma Reporter, and Outsourcing Pharma.

The post Top 5 Firms Engineering Healthcare in the CNS Space appeared first on Inside Precision Medicine.

April 6, 2026

Isatuximab, carfilzomib, lenalidomide and dexamethasone in newly diagnosed multiple myeloma: a randomized phase 3 trial

Nature Medicine, Published online: 06 April 2026; doi:10.1038/s41591-026-04282-0

In the phase 3 EMN24 IsKia trial, transplant-eligible patients with newly diagnosed multiple myeloma who received isatuximab with carfilzomib, lenalidomide and dexamethasone pretransplant induction and post-transplant consolidation showed higher rates of measurable residual disease negativity after consolidation than patients who received carfilzomib, lenalidomide and dexamethasone.

April 6, 2026

STAT+: Neurocrine Biosciences to buy Soleno Therapeutics in $2.9B deal

Neurocrine Biosciences said Monday that it would buy Soleno Therapeutics and its treatment for Prader-Willi syndrome for $2.9 billion.

Neurocrine is paying $53 a share for Soleno, a 34% premium to its closing price on Thursday.

Soleno’s drug, Vykat, was approved in March 2025 to treat hyperphagia in patients with the rare genetic disease. Hyperphagia is one of the defining features of Prader-Willi syndrome, causing relentless hunger and leading patients to overeat. Vykat is the only approved treatment for hyperphagia in Prader-Willi patients.

Continue to STAT+ to read the full story…

April 6, 2026

Diabetes Drug Mimics Benefits of Exercise in Prostate Cancer Patients

Scientists have discovered that metformin, a widely prescribed diabetes drug, can have benefits similar to those of regular exercise in prostate cancer patients, whose movement may be limited by their condition or treatment. Published today in EMBO Molecular Medicine, their findings show that metformin increased levels of N-lactoyl-phenylalanine (Lac-Phe), a molecule naturally produced by the body after intense exercise.

Exercise can significantly benefit prostate cancer patients both during and after treatment. These patients often receive hormone therapy, which can disrupt metabolism, contribute to weight gain, increase insulin resistance, and affect their overall cardiovascular health. While physical activity is key to supporting their recovery and addressing these side effects, fatigue, pain, and other common symptoms can limit the ability of prostate cancer patients to regularly exercise.

“Cancer therapy often affects the body in ways that go beyond the tumor,” said Priyamvada Rai, PhD, professor of radiation oncology and co-leader of the Tumor Biology Program at Sylvester Comprehensive Cancer Center in the University of Miami Miller School of Medicine. “Supporting metabolic health can influence how patients tolerate treatment and how they feel over time, even if it doesn’t directly change tumor growth. This study was an opportunity to investigate molecular pathways that can be therapeutically activated for better outcomes to treatments that induce metabolic stress.”

Rai and colleagues found that metformin raises levels of Lac-Phe in prostate cancer patients, even in the absence of physical activity. Known for its role in regulating energy levels and weight gain, Lac-Phe is formed by combining lactate, a molecule produced during muscle contraction, and the amino acid phenylalanine. Previous preclinical and clinical studies have reported levels of Lac-Phe spiking after intense exercise and linked it to a reduction in appetite and improved weight control.

“Metabolism is involved in everything cells do,” said David B. Lombard, MD, PhD, professor of pathology and laboratory medicine and co-leader of the Cancer Epigenetics Program at the Miller School. “These findings suggest Lac-Phe may be a very informative signal for understanding how metformin affects metabolism in prostate cancer patients.”

Prostate cancer patients treated with metformin were found to produce similar levels of Lac-Phe compared to levels reported after intense exercise in previous studies of healthy volunteers. The benefits of Lac-Phe persisted even after hormone therapy began.

“From a clinical standpoint, seeing a metabolic signal that mirrors what we associate with intense exercise was striking,” said Marijo Bilusic, MD, PhD, genitourinary medical oncologist and professor of medicine and medical oncology at the Miller School. “The result isn’t a new cancer biomarker, but a clearer understanding of how a widely used drug may support metabolic health during prostate cancer treatment—an outcome that matters to patients and clinicians alike.”

Although a drug like metformin can never fully replace physical activity, these findings offer an alternative approach to accessing some of the benefits of exercise in patients with limited ability to engage in it.

“What’s encouraging about this work is that it reminds us cancer care isn’t only about targeting tumors—it’s also about supporting the whole patient,” said Rai. “By better understanding how treatments affect metabolism, we can begin to identify ways to help patients maintain strength, resilience, and quality of life throughout their care.”

The post Diabetes Drug Mimics Benefits of Exercise in Prostate Cancer Patients appeared first on Inside Precision Medicine.

April 6, 2026

Opinion: When my child is in psychosis, the pediatric health care system can’t help us

I am sitting in a firm recliner with a wipeable surface during a two-day hospital admission for testing at our local children’s hospital. The chair is designed for durability, not sleep. The pillow beneath my head is flat and smells faintly of disinfectant. A thin hospital blanket scratches against my arms as I shift, unsuccessfully, trying to rest. The room is dim but never quiet. Monitors beep. Machines hum. Footsteps pass the door. Hospital noise does not fade. It embeds itself in the nervous system.

My 13-year-old is finally asleep. His thin body is curled beneath a blanket identical to mine. One shoulder peeks out, bruised from repeated injections of calming medication. A neon orange bandage marks the most recent one, given about an hour ago. I watch his chest rise and fall and allow myself a brief moment of relief.

Read the rest…

April 6, 2026

StockWatch: Price War Dampens Lilly Surge After Oral GLP-1 Wins FDA Nod

Eli Lilly (NYSE: LLY) won the approval it sought when the FDA authorized the company’s oral obesity drug Foundayo (orforglipron), but the pharma giant’s post-approval stock bounce was short-lived.

Lilly shares rose 4% from $919.77 to $954.52 on the day of the announcement. But the momentum reversed into a 2% loss to $935.58 on Thursday, and for one important reason beyond simply the overall market decline triggered by investors losing confidence in a speedy end to the Iran war.

Investors (as reflected in analysts’ mixed observations) appeared divided on how quickly Lilly can generate sales this year for Foundayo, a small molecule glucagon-like peptide-1 (GLP-1) receptor agonist. That division results from the competition shaping up on the drug’s price with obesity arch-rival Novo Nordisk (shares traded on Nasdaq Copenhagen as NOVO-B; ADRs traded on NYSE as NVO).

Novo Nordisk got a jump on Lilly in the oral obesity drug front in December when the Danish biotech giant won FDA approval for oral Wegovy® (semaglutide), a once-daily 25 mg tablet indicated for chronic weight management. Analysts consider oral Wegovy sales to have started strong, with total prescriptions reaching 577,000 and 52,000 filled during the week ending March 20.

Lilly is expected to make Foundayo available directly to patients through its LillyDirect direct-to-consumer services and support platform starting Monday. There, a starting dose of 0.8 mg is being priced at $149/month, rising to $199/month for 2.5 mg, $299/month for the 5.5 mg and 9 mg doses, and up to $349/month for the highest doses of 14.5 mg and 17.2 mg. However, high-dose patients will automatically receive the $299/month price on their first purchases and keep it if they refill their prescription within 45 days of their previous prescription.

Patients paying through commercial insurance plans will be eligible for discounts that reduce Foundayo’s out-of-pocket cost to patients for 1-, 2-, or 3-month prescription fills to $5 a month.

“A positive surprise is that the approval was for a tablet formulation, which is less expensive to manufacture” than the capsule versions studied by Lilly during clinical trials, David Risinger, a senior managing director and senior research analyst covering diversified biopharmaceuticals at Leerink Partners, shared in a research note.

Lilly told Risinger that it conducted a bioequivalence study comparing capsules to tablets, which, according to the company, can be manufactured more efficiently than capsules and use less active pharmaceutical ingredient. The high dose studied in Lilly’s Phase III trials of orforglipron, 36 mg, corresponds to the highest capsule dose of 17.2 mg.

Price competition

Lilly has sought to price its oral obesity drug competitively with Novo Nordisk’s oral Wegovy, which starts at $149/month for the lowest dose of 1.5 mg, then rises to $199/month for the 4 mg dose, with new patients paying $149/month through August 31. Prices rise to $299/month for 9 mg and 25 mg doses.

However, patients who sign up for a 12-month subscription to oral Wegovy through Novo Nordisk’s telemedicine partner-providers enjoy a $50 discount that brings their monthly cost down to $249. And commercial insurance patients who agree to local pharmacy pickup with a savings offer can pay as little as $25/month, subject to a maximum savings of $100/month.

The price competition explains why buyers of securities for themselves or clients—the “buy side” in Wall Street jargon—have lowered their 2026 forecasts for Foundayo sales by more than half, from about $4 billion to less than $2 billion, Trung Huynh, an analyst with RBC Capital Markets, wrote in a research note. Huynh cited a consensus of analysts which is projecting approximately $1.6 billion in 2026 sales, though a Reuters spot check of investment brokerages found a range for this year’s projected sales of $1.5 billion to $2.8 billion.

“Although there have been headwinds on pricing erosion in the GLP-1 space, we believe there is substantial upside with the expected Medicare Part D expansion later this year,” Huynh added. The expansion of sales to the Medicare Part D program would cap patient copays at $50 per month.

Huynh and RBC Capital have projected Foundayo will reach peak-year sales of $36 billion—14% above the $31.68 billion racked up by last year’s best-selling prescription drug, the multi-indication cancer immunotherapy blockbuster Keytruda® (pembrolizumab) marketed by Merck & Co. (NYSE: MRK).

The highest peak sales forecast comes from Citi Research, where Geoff Meacham, PhD, the firm’s head of healthcare research and a managing director specializing in U.S. pharma and biotech research, has projected more than $40 billion. At the low end, a consensus of analysts surveyed by Bloomberg News expects Foundayo peak sales to reach $18 billion by 2030.

$50M upfront

Both ends are a far, far cry from the $50 million upfront that Lilly paid in 2018 to license orforglipron, then called OWL833, from Chugai Pharmaceutical, which discovered the drug and is owned by Roche Holding (SIX Swiss Exchange: ROP and RO; OTCQX: RHHBY). Lilly also agreed to pay Chugai up to $390 million in potential payments tied to achieving milestones.

At the time, OWL833 was deemed Phase I ready for clinical studies in type 2 diabetes—the indication for which Lilly markets its GLP-1/GIP (glucose-dependent insulinotropic polypeptide) receptor agonist tirzepatide as Mounjaro®, and Novo Nordisk markets its GLP-1 receptor agonist semaglutide as Ozempic®.

“Overall, we continue to believe that injectable anti-obesity medications will retain the majority of market share (roughly 80%, based on our estimates) in the United States due to the high potency of injectables coupled with a more elevated U.S. BMI population,” Andy T. Hsieh, PhD, a partner and biotechnology analyst with William Blair, wrote in a research note.

Beyond competitive pricing and lower manufacturing costs, Lilly has emphasized a convenience advantage over oral Wegovy—starting with its public announcement of the approval, whose headline referred to Foundayo as “the only GLP-1 pill for weight loss that can be taken any time of day without food or water restrictions.”

While Foundayo can be taken morning, afternoon, or evening, oral Wegovy, by contrast, requires patients to take the pill with up to 4 ounces of water on an empty stomach as soon as they wake up, then fast for 30 minutes before they can eat or drink.

“Accordingly, we expect Foundayo to blunt the uptake of oral Wegovy upon its availability (starting on April 6), though oral Wegovy retains a pricing advantage,” Hsieh concluded.

Huynh of RBC Capital agreed, citing a survey by his firm of about 200 patients, payers, and prescribers: “Our recent survey indicated that Foundayo would be a preferred oral option amongst patients since it has no dosing restrictions.”

Not so, Novo Nordisk CEO Maziar (Mike) Doustdar told CNBC last month: “People are really interested because it’s the most efficacious pill right now in the market.”

Novo Nordisk cites efficacy

Novo Nordisk sought to reinforce that message via an announcement trumpeting that its 25 mg dose of oral Wegovy showed “significantly” greater mean weight loss than the 36 mg dose of Foundayo, according to a population-adjusted indirect treatment comparison using data from two clinical trials:

OASIS 4 (NCT05564117), a 307-patient randomized study which evaluated once-daily oral Wegovy 25 mg in overweight or obese adults with at least one self-reported unsuccessful dietary effort to lose body weight, to measure their percentage change in body weight and whether it was ≥5% at the end of treatment at week 64.
ATTAIN-1 (NCT05869903), a 72-week, randomized, double-blind, placebo-controlled trial comparing the efficacy and safety of orforglipron (6 mg, 12 mg, and 36 mg) as monotherapy vs. placebo in 3,127 adults with obesity, or overweight with at least one of the following comorbidities: hypertension, dyslipidemia, OSA, or cardiovascular disease, who did not have diabetes.

Novo Nordisk plans to present details of its study, called ORION, at the Obesity Medicine Association’s annual Obesity Medicine 2026 conference, set for April 10-12 in San Diego.

News of the FDA approval for Foundayo caused Novo Nordisk shares to dip 0.1% Wednesday from DKK 231.15 ($35.65) to DKK 230.90 ($35.61). But the shares finished the trading week climbing nearly 3% Thursday to DKK 236.90 ($36.53).

U.S. and major European markets were closed on Friday in observance of Good Friday.

Foundayo is the fifth prescription drug to be authorized under the FDA’s Commissioner’s National Priority Voucher program, through which the agency awards vouchers to drug developers whose work will address a health crisis in the United States, deliver more innovative cures, address unmet public health needs, and increase domestic drug manufacturing as a national security issue.

The approval came nearly 10 months ahead of Foundayo’s target decision date of January 20, 2027, under the PDUFA (Prescription Drug User Fee Act) program, and just 50 days after Lilly filed a new drug application (NDA) for the oral obesity drug with the FDA.

Leaders and laggards

Inovio Pharmaceuticals (NASDAQ: INO) shares nosedived 35% from $1.74 to $1.13 Thursday after the developer of DNA therapies to treat and prevent HPV-related diseases, cancer, and infectious diseases announced that it priced at $1.40 a share an underwritten public offering of 12.5 million shares of common stock, plus accompanying Series A and Series B warrants, each series enabling potential purchase of up to 12.5 million shares (or pre-funded warrants in lieu thereof). The dilutive offering is projected to raise $17.5 million in gross proceeds—$16 million in net proceeds, rising to $18.4 million if underwriter Piper Sandler exercises in full its 30-day option to purchase up to 1.875 million additional shares and all Series A and Series B warrants (1.875 million shares each series). Net proceeds are intended to fund a potential commercial launch of INO-3107, a recurrent respiratory papillomatosis candidate under FDA review with a target decision date of October 30, as well as a confirmatory trial for INO-3107 if required, pipeline development, and general corporate purposes.
Oric Pharmaceuticals (NASDAQ: ORIC) shares tumbled 19% from $12.67 to $7.47 Wednesday after the cancer drug developer said it will advance rinzimetostat (ORIC-944) into a 600-patient Phase III trial (Himalaya-1 ) after generating positive data from its Phase Ib trial (NCT05413421) assessing rinzimetostat plus the Bayer (XETRA: BAYN)-marketed Nubeqa® (darolutamide) as a once-daily treatment for metastatic castration-resistant prostate cancer (mCRPC) in patients previously treated with abiraterone acetate (abiraterone). At a median follow-up of 4.9 months, the combination showed radiographic progression-free survival (rPFS) rates of 93% at 3 months, 84% at 4 months, and 84% at 5 months—rates consistent, Oric said, with the competitor PRC2 inhibitor currently in Phase III in post-abiraterone mCRPC patients and superior to available standard-of-care therapies showing rPFS rates of approximately 60% to 75%. Also, 47% of patients (7/15) achieved a PSA50 response, with 33% (5/15) confirmed, while 71% of patients (10/14) achieving >50% circulating tumor (ctDNA) reduction. Jefferies analyst Maury Raycroft, PhD, reported that some investors viewed rinzimetstat as “a better fit strategically” with Johnson & Johnson (NYSE: JNJ)-marketed Erleada® (apalutamide).
Sangamo Therapeutics (NASDAQ: SGMO) shares climbed 20% from 25 cents to 30 cents between Tuesday and Thursday after the genetic medicine developer said it expects to complete this summer its rolling Biologics License Application (BLA) submission seeking FDA approval for isaralgagene civaparvovec (ST-920), a gene therapy candidate being developed to treat Fabry disease, subject to securing adequate additional funding, while continuing business development discussions for a potential Fabry commercialization agreement. Sangamo said it is advancing the chemistry, manufacturing, and controls (CMC) module ahead of completion of the rolling BLA submission. Since launching its rolling BLA submission in December under an Accelerated Approval pathway, Sangamo has submitted preclinical and clinical modules, as well as submitted its antibody assay companion diagnostic, designed to screen patients for eligibility with isaralgagene civaparvovec, to the FDA’s Center for Devices and Radiological Health (CDRH), seeking Premarket Approval (PMA).

The post StockWatch: Price War Dampens Lilly Surge After Oral GLP-1 Wins FDA Nod appeared first on GEN – Genetic Engineering and Biotechnology News.

April 3, 2026

Low Birthweight Increases Risk of Early Stroke

Research led by the University of Gothenburg in Sweden suggests that low birthweight is a risk factor for having a stroke in younger adulthood.

In a study including just under 800,000 people, the investigators found that risk for early stroke events was 18-23% higher in men and women who had a birth weight under the median level than those with a higher birth weight.

Around 795,000 people in the U.S. have a stroke each year. Although it can affect people of any age, it is much more common in older individuals with estimates of prevalence suggesting that 0.9% of 18–44 year olds have strokes versus 3.8% of those in the 45–64 year age group and 7.7% of people aged 65 and over.

Low birth weight has been previously linked to an increased risk for stroke in several studies. Researchers think that low birth weight is an indicator of exposure to an adverse environment in the womb that may adversely affect the cardiovascular system of the fetus in a way that increases stroke risk—for example, by increasing the risk of high blood pressure.

Over the last 10-15 years, stroke prevalence has stayed the same in older adults but has gone up by 14-16% in 18-64 year-olds. Lina Lilja, a doctoral student at the University of Gothenburg, and colleagues aimed to investigate whether low birth weight increased the risk of stroke in younger adults.

They included 420,173 men and 348,758 women from Sweden who were born between 1973 and 1982 and followed up from birth until 2022. The researchers collected data on birth weight, gestational age, and body mass index in young adulthood, as well as information on first stroke and the type of stroke.

Overall, 2252 first stroke events were recorded at an average age of 36 years. Of these, 1624 were ischemic stroke (average age 37 years) and 588 were intracerebral hemorrhage (average age 33 years).

The results, which will be presented at the European Congress on Obesity in Istanbul later this year, showed that birth weight below the median (3.5kg) increased the risk for all stroke by 21%. The rates of stroke were slightly higher in men with a low birthweight at 23% versus women with a low birthweight at 18%.

Notably, gestational age at birth and young adult body mass index were not linked to stroke risk in this study.

The post Low Birthweight Increases Risk of Early Stroke appeared first on Inside Precision Medicine.

April 3, 2026

Pancreatic Cancer Development Driven by NADPH Disruption

Researchers at the University of Michigan have uncovered metabolic pathways that explain how pancreatic cells transition from acinar-to-ductal metaplasia to pancreatic ductal adenocarcinoma (PDAC). The study, published in Nature Metabolism, detailed on how reduced production of a molecule central to biosynthesis and oxidative stress control called NADPH alters cellular conditions to favor cancer progression. By examining precancerous pancreatic lesions, the team found that disruptions in enzymes responsible for NADPH production increase oxidative stress, accelerating the formation of lesions and, in some cases, the progression to PDAC.

“We know a lot about how pancreatic tumors behave and look, but we don’t know how they become cancerous,” said lead author Megan Radyk, PhD, a former postdoc in the lab of Costas Lyssiotis, PhD, at the University of Michigan and now an assistant professor at Roswell Park Comprehensive Cancer Center. “We wanted to learn about what metabolic changes happen before you get an established tumor.”

PDAC is the most common form of pancreatic cancer and has a low five-year survival rate. The disease develops through a stepwise process that begins with acinar-to-ductal metaplasia (ADM), a reversible state in which pancreatic cells respond to injury or inflammation by adopting a duct-like phenotype. Under normal conditions, these cells can revert to their original state. However, in the presence of oncogenic KRAS mutations, this process is disrupted, leading to persistent ADM and progression to pancreatic intraepithelial neoplasia (PanIN), which can ultimately become PDAC.

NADPH’s normal role is in maintaining cellular homeostasis. It supports the synthesis of lipids, cholesterol, and nucleotides, and it aids antioxidant systems that regulate reactive oxygen species (ROS). Under normal conditions, NADPH helps neutralize ROS, preventing cellular damage. The current study, however, demonstrated that lower levels of NADPH impair antioxidant defenses, leading to increased ROS and lipid peroxidation, which in turn promote the formation of precancerous lesions.

The researchers identified two NADPH-producing enzymes for their work: glucose-6-phosphate dehydrogenase (G6PD) and malic enzyme 1 (ME1). Both enzymes support the production of the appropriate levels of NADPH needed for biosynthesis and ROS regulation.

Using a multimodal approach involving RNA sequencing, metabolomics, and mouse models with oncogenic KRAS mutations, the Michigan team studied how the loss of these two enzymes affects pancreatic tissue. They observed that deficiency in either G6PD or ME1 increased ROS levels and accelerated the formation of ADM and pancreatic intraepithelial neoplasia (PanIN) lesions. Antioxidant treatments, including glutathione and N-acetyl cysteine, reduced lesion formation, further bolstering the current understanding of the role of oxidative stress in early tumorigenesis. The team achieved similar results when these methods were applied to human pancreatic tissue samples.

But results from later in the study showed that the two enzymes played distinct roles in the later stages of PDAC. While they both contributed to early lesion formation, only the loss of ME1 promoted progression to PDAC. This suggests that although both enzymes regulate NADPH and oxidative stress, they have distinct roles in later metabolic demands of cancer cells.

The study builds on prior research showing that KRAS mutations drive metabolic reprogramming and ROS production in pancreatic cells. Previous work has also indicated that antioxidant pathways, including those regulated by NRF2, are activated during tumor initiation.

Clinically, these findings suggest that targeting metabolic pathways involved in NADPH production could provide a strategy to intercept pancreatic cancer before it fully develops. Measuring levels of G6PD, ME1, or related metabolites could also serve as biomarkers to identify patients at higher risk of lesions progressing to cancer.

“Our study can help the search for new biomarkers that can intercept pancreatic cancer before it progresses,” the researchers wrote.

Future research will focus on identifying additional enzymes that regulate NADPH levels and determining how these pathways can be targeted safely. The researchers also plan to study whether patients with mutations in G6PD, ME1, or related pathways have an increased risk of pancreatic disease.

The post Pancreatic Cancer Development Driven by NADPH Disruption appeared first on Inside Precision Medicine.

April 3, 2026

How an Alzheimer’s Risk Gene Rewires the Brain Decades Before Symptoms

For millions of people worldwide, carrying the APOE4 gene variant means a significantly higher risk of developing Alzheimer’s disease. Yet one of the biggest unanswered questions has been when, and how, that risk begins to take hold in the brain.

New research from the Gladstone Institutes, published in Nature Aging, suggests that the effects of APOE4 emerge far earlier than previously understood. The study shows that subtle but important changes in brain activity occur long before memory loss begins, offering a potential window for early intervention.

Early changes in a seemingly healthy brain

Alzheimer’s disease is typically diagnosed after cognitive symptoms appear, but growing evidence suggests that the disease process begins decades earlier. The new study adds to this picture by demonstrating that brain circuits in young individuals carrying APOE4 are already functioning differently.

We found fundamental changes in brain circuits occurring in young mice that still had normal learning and memory, and importantly, that those changes predicted the development of cognitive deficits at older ages, ” said Misha Zilberter, PhD, principal staff research scientist at Gladstone and senior author of the study.

The researchers observed increased neuronal activity in the hippocampus, a brain region essential for learning and memory. Similar patterns of hyperactivity have been reported in human APOE4 carriers, even before clinical symptoms arise.

According to the scientists, this suggests that Alzheimer’s risk is not simply a matter of late-stage degeneration, but may instead involve long-term changes in how brain circuits are wired and function.

Smaller neurons, stronger signals

To understand what drives this early hyperactivity, the team examined individual brain cells. They found that neurons in key regions of the hippocampus were physically smaller in APOE4 carriers compared to those with the more common, lower-risk APOE3 variant.

While this might seem like a minor structural difference, it has functional consequences. Smaller neurons are more easily activated, meaning they fire more readily in response to stimuli. This heightened sensitivity can lead to persistent hyperactivity within neural circuits.

Over time, this imbalance may place stress on the brain and contribute to the gradual decline seen in Alzheimer’s disease.

A surprising source of dysfunction

For years, researchers believed that APOE4’s effects were primarily driven by astrocytes, support cells in the brain that produce most of the APOE protein. However, the new findings challenge this assumption.

The team discovered that the disruptive effects on brain activity were instead linked to APOE4 produced directly by neurons themselves. When APOE4 was removed from neurons, their size and activity returned to normal. Removing it from astrocytes, by contrast, had little effect.

This shift in understanding refocuses attention on neurons as key drivers of early disease processes, rather than passive victims of surrounding dysfunction.

A reversible pathway—and a new target

Perhaps the most striking finding of the study is that these early changes may not be permanent.

The researchers identified a protein called Nell2 as a central player in the process. Levels of Nell2 were elevated in APOE4 neurons and appeared to drive both the reduction in cell size and the increase in neuronal activity.

By reducing Nell2 levels in adult mice, the team was able to restore normal neuron structure and function—even after the changes had already occurred.

“What’s exciting about Nell2 is that we were able to reverse the disease manifestations in adult mice by lowering its level,” said Yadong Huang, co-senior author of the study. “That tells us the damage is not irreversible […].”

This raises the possibility of developing therapies that target Nell2, potentially slowing or preventing disease progression in individuals at high genetic risk.

Implications for early intervention

APOE4 is present in roughly one in four people and in the majority of Alzheimer’s patients. Despite this, current treatments largely focus on late-stage symptoms rather than early prevention.

The new findings suggest that intervening earlier, before cognitive decline begins, could be key. If brain circuit changes can be detected and corrected at an early stage, it may be possible to delay or even prevent the onset of Alzheimer’s disease.

The study also highlights the importance of understanding how genetic risk translates into functional changes in the brain. Rather than acting as a simple risk marker, APOE4 appears to actively reshape neural activity over time.

A shift in perspective

More broadly, the work reflects a growing shift in Alzheimer’s research, from focusing solely on hallmark features such as amyloid plaques and tau tangles to examining earlier, subtler changes in brain function.

By identifying a concrete pathway linking genetic risk to altered brain activity, the study provides a clearer framework for understanding how the disease develops.

“This study is a big breakthrough for the field of Alzheimer’s research,” Huang said. “It opens the door to a better understanding of how APOE4 alters the function of neurons at a young age to increase risk of cognitive decline, and to the development of therapies that could block the detrimental effects of APOE4 early on.”

While the findings are based on mouse models, they align closely with observations in humans and offer a strong foundation for future research. The next steps will involve determining whether targeting Nell2 or similar pathways can produce similar benefits in human patients.

If successful, such approaches could transform how Alzheimer’s disease is treated, not as an inevitable consequence of aging, but as a process that can be detected early and potentially reversed.

The post How an Alzheimer’s Risk Gene Rewires the Brain Decades Before Symptoms appeared first on Inside Precision Medicine.