RFK Jr launches database project to explore autism causes

 Robert F Kennedy Jr has launched a project to trawl federal databases to try to identify why there has been an increase in diagnoses of autism, but there are deep concerns about the project's credibility.

The Health and Human Services (HHS) Secretary, who has claimed in the past that there is a link between vaccinations and autism, despite decades of evidence to the contrary, is putting $50 million behind a project that he has claimed will deliver answers within a few months.

A petition has been running for some weeks against the initiative, advocating strongly against the creation of a patient 'registry' that could be vulnerable to misuse and discrimination, with signatures overtaking 50,000.

According to HHS – which denies the creation of a registry – the project is a "landmark partnership" between the National Institutes of Health (NIH) and the Centers for Medicare & Medicaid Services (CMS) that will create "a real-world data platform enabling advanced research across claims data, electronic medical records, and consumer wearables."

It claims the data harvesting will be carried out "in a manner consistent with applicable privacy laws to protect Americans' sensitive health information."

Ryan Smith, a parent of two neurodiverse children who started the petition, said yesterday that despite assurances from the HHS that a registry would not be created, "the government is still building a data platform, and many questions remain about consent, oversight, and who will control that information."

Kennedy – who has repeatedly claimed that an investigation was justified by the increasing prevalence of autism diagnoses among American children over the past 25 years – said the objective is to uncover "the root causes of autism and other chronic diseases."

Still, it is clear that the primary focus is on autism spectrum disorder (ASD), which will be the pilot indication for the programme. It will be used to understand "healthcare utilisation, chronic disease aetiology and treatment, and the economic burden of chronic conditions," according to HHS.

Recent figures from the Centers for Disease Control and Prevention (CDC) suggest one out of every 31 children in the US was diagnosed with autism in 2020, compared with one in 150 in 2000, although that increase has been widely attributed to greater awareness, more testing, and broader diagnostic criteria.

Kennedy has argued repeatedly over the years that vaccinations were the cause, including in a Fox News interview in 2023, and there are deep concerns that the aim of seeking an "environmental cause" for ASD is an excuse to force the data to fit a link with childhood immunisations.

The Autism Society of America has acknowledged the withdrawal of the registry plan following public outcry related to ethical, scientific, and privacy concerns, but remains deeply worried by Kennedy's programme.

"The broader research initiative persists, led by individuals like David Geier, whose past misconduct has raised questions about the study's credibility," it said in a statement.

Geier was appointed to lead the project despite having a long track record of anti-vaccine rhetoric and no medical or advanced scientific qualifications. He has also been disciplined in the past for practising medicine without a license.

"Statements from the Administration perpetuating discredited theories linking vaccines and autism, contradict established scientific consensus," said the organisation.

"Additionally, definitive claims to find 'environmental toxins' in an accelerated timeline further conflicts with current science, along with the considerable cuts to autism research and supports that have already taken shape.

https://pharmaphorum.com/news/rfk-jr-launches-database-project-explore-autism-causes

Boehringer advances oral therapy for geographic atrophy

 Boehringer Ingelheim has started a proof-of-concept trial of a drug that could offer the first oral treatment for geographic atrophy (GA), a major cause of irreversible sight loss that affects millions of people around the world.

The phase 2 JADE trial is testing the efficacy and safety of BI 1584862, a phospholipid modulator that Boehringer has said could offer a first-in-class treatment for GA, an advanced and severe form of age-related macular degeneration (AMD) characterised by the loss of photoreceptors and retinal pigment epithelial (RPE) cells in the central portion of the retina known as the macula.

At the moment, there are two approved therapies for GA – Apellis' complement C3 inhibitor Syfovre (pegcetacoplan) and Astellas' complement C5 inhibitor Izervay (avacincaptad pegol) – which both have to be administered by injection directly into each of the affected eyes every few weeks.

Both drugs are approved in the US, but are not available in Europe, where marketing applications were submitted but faced resistance from the EU regulator.

"Vision loss associated with geographic atrophy profoundly affects the lives of those impacted, particularly older adults, making daily activities more difficult. The loss of independence has a significant burden for patients, caregivers and society overall," said lead JADE investigator Karl Csaky, head of the Retina Foundation of the Southwest.

"An oral treatment could alleviate the burden of existing treatment regimens and offer the opportunity to address both eyes simultaneously in patients with bilateral disease," he added.

BI 1584862 is thought to work by reducing inflammation associated with GA and preserving vascular function in the retina, said Boehringer. The JADE trial will enrol around 138 people aged 55 and older with at least one eye affected by GA, who will be treated with one of three doses of the drug.

The main outcome measure will be change in the area affected by GA – which tends to cause blind spots in the central vision – while secondary endpoints will include macular thickness and measurement of visual acuity. Results are due in 2026.

Boehringer has made GA one of its R&D priorities, and BI 1584862 is the second candidate in its pipeline to advance into mid-stage clinical testing after BI 771716, an antibody fragment-based therapy that would be delivered by intravitreal injection.

BI 771716 is being compared to Syfovre in the VERDANT trial, with results expected sometime in 2026 or 2027.

https://pharmaphorum.com/news/boehringer-advances-oral-therapy-geographic-atrophy

Viagra-maker Viatris beats quarterly estimates on demand for branded drugs in China

 Drugmaker Viatris beat Wall Street estimates for first-quarter profit and revenue on Thursday, helped by sales of its branded drugs in China.

The company’s shares, which rose over 4% in premarket trading earlier after its pain drug and birth control patch succeeded in late-stage studies, were up 1.4%, after Viatris reported a goodwill impairment charge of $2.9 billion.

Viatris attributed the charge to “a sharp and sustained decline in its share price and significantly increased uncertainty and volatility in the geopolitical and economic environments.”

When adjusted for divestitures, the company’s first-quarter revenue fell 2% to $3.25 billion, but still came ahead of analysts’ estimate of $3.24 billion, according to data compiled by LSEG.

Sales of its generic drugs were hurt after the U.S. Food and Drug Administration restricted imports of 11 products from its facility in Indore, India due to violations found during an inspection.

Revenue in its bigger branded drugs unit, under which the company sells anti-anxiety medication Xanax and Yupelri for lung disease, rose 3% to $2.12 billion, reflecting expansion in emerging markets, and strong growth in Greater China and developed markets.

The company maintained its annual revenue forecast at $13.5 billion to $14 billion, but raised its per share adjusted profit outlook by 5 cents at the mid point, including the impact of share repurchases.

The forecast, however, did not include “any potential impact of future tariffs and trade restrictions as they cannot be reasonably forecasted,” the company said.

As a result of the impairment charge, Viatris swung to a quarterly loss of $2.55 per share for the quarter ended March 31, from a profit of 9 cents a year ago.

On an adjusted basis, it reported a profit of 50 cents per share, compared with estimates of 49 cents.

Pittsburgh-based Viatris was formed by the merger of Mylan and Pfizer’s Upjohn business in 2020.

https://whtc.com/2025/05/08/viagra-maker-viatris-beats-quarterly-estimates-on-demand-for-branded-drugs-in-china/

FDA Vaccine Advisors to Meet for COVID-19 Vaccine Updates

 

The independent experts will meet on May 22 to discuss updates to the COVID-19 vaccine for the upcoming season.

Later this month, the FDA will convene its external vaccine advisors to decide on the formulation for COVID-19 vaccines to be used in the upcoming 2025-2026 season, according to a Federal Register notice posted Thursday. The meeting is scheduled for May 22.

Importantly, Federal Register notices should go live at least 15 days before the scheduled meeting. In this case, the announcement should have been posted on Wednesday, May 7. “FDA regrets that it was unable to publish this notice 15 days prior to the [meeting] due to technical issues,” the notice reads.

Still, the agency decided to push through with the panel, noting the “need for an immediate meeting of the Committee, including the time-sensitive need for input and public discussion” on the subject at hand. The FDA “concluded that there are exceptional circumstances that support holding this meeting without the customary 15-day public notice.”

This urgency is a notable change in tone for the current administration, which in recent weeks has expressed strong skepticism of the COVID-19 vaccines. In February, Health and Human Services Secretary Robert F. Kennedy Jr. postponed a meeting of the Centers for Disease Control and Prevention’s vaccine advisors. The panelists were supposed to have met on Feb. 27 to update immunization recommendations for a host of infectious diseases, including COVID-19.

Kennedy’s HHS has also frozen its support for drugmakers developing next-generation COVID-19 vaccines, including GeoVax and Vaxart (the latter of which has had its stop order lifted). And last month, the FDA missed its decision date for Novavax’s updated COVID-19 vaccine. Later, the company revealed that the FDA is asking for a post-marketing commitment to collect additional clinical data for the shot.

Late last month, Politico reported that Kennedy was considering pulling COVID-19 vaccination from the CDC’s immunization guidelines for children—a move that would put the U.S. in line with international standards, but would also show how comfortable the secretary is in “making unilateral decisions,” BMO Capital Markets said in an investor note at the time.

Most recently, HHS named Vinay Prasad as the new head of the FDA’s Center for Biologics Evaluation and Research—succeeding Peter Marks, who was a staunch supporter of vaccines during his term.

Prasad, an oncologist and hematologist at the University of California, San Francisco, has built a name for himself on YouTube as an outspoken critic of some public health policies. Most notably, Prasad came out strongly against university COVID-19 vaccine mandates and argued on his YouTube channel that the disease has become merely a “common cold respiratory virus.”

https://www.biospace.com/fda/fda-vaccine-advisors-to-meet-for-covid-19-vaccine-updates

Takeda Pledges $30B in U.S., Argues Against Trump’s ‘Most Favored Nations’ Proposal

 

Takeda’s Julie Kim argues that Trump’s idea to match drug prices to that of other countries could cost the industry up to $1 trillion over the next 10 years.

Takeda is pumping roughly $30 billion into its U.S. operations over the next five years, CEO Christophe Weber said Thursday during the pharma’s earnings call for its full fiscal year 2024 results.

“This reflects the fact that the U.S. is the world’s leading market for biopharmaceutical innovation,” Weber said, “and I hope that will continue.” This move puts Takeda in the same league as its major pharma fellows—including Eli Lilly, Johnson & Johnson, Bristol Myers Squibb and Novartis—all pledging billions of dollars in the U.S. in manufacturing and investments, partly in response to President Donald Trump’s tariffs threats.

Takeda on Thursday also spoke out against Trump’s proposed “most favored nation” health policy, which Trump claims could help bring drug prices down. According to a report from Politico earlier this week, the President could come out with an executive order as soon as early next week.

“Basically, it is a price control,” Julie Kim, president of Takeda’s U.S. unit, said during the call. The “Most Favored Nations”—or MFN—approach seeks to lower the cost of prescription medicines to no higher “than the lowest price charged in other similar countries,” according to November 2020 announcement from the Centers for Medicare and Medicaid Services.

President Trump tried to pass a similar initiative during his first term, though nothing came of it.

“From an industry perspective, if MFN were applied within the Medicaid setting . . . that would be an industry impact over 10 years of up to 1 trillion dollars,” Kim argued. “It would fundamentally be a significant challenge for the overall industry, Takeda included.”

“We think that there are better ways to encourage manufacturing investment in the U.S., R&D investment in the U.S. and to support continued innovation [in] the biopharmaceutical industry in America,” she added. “MFN would not be the appropriate way to do it.”

Tariff Impact ‘Limited’

On the call, Weber emphasized that Takeda’s exposure to these tariffs is likely to be “limited.” While about 50% of the company’s total revenue comes from the U.S., imports account for only about 8% to 10% of Takeda’s U.S. sales. Those imports primarily come from Europe, Japan and Singapore.

“Of note, our largest product by revenue in the U.S., Entyvio, is 100% U.S. country of origin,” Weber said. Rather than thinking of the $30 billion package as a means to soften the blow of tariffs, Weber insisted that such an investment is “not new.” Instead, he suggested, the money is in-line with its long-standing strategy in the U.S.

“This is to maintain [our] presence. This is to continue to develop the company. This is to make sure that our manufacturing sites are upgraded up to the best efficiency and productivity,” he said, noting that the sum will also include “our R&D spend in the U.S.”

Takeda recorded a 7.5% year-on-year revenue growth, bringing in ¥4,581.6 billion in 2024, or roughly $31.6 billion. Entyvio, the anti-inflammatory monoclonal antibody indicated for ulcerative colitis and Crohn’s disease, was the Japanese multinational’s strongest growth driver, climbing 8.5% year-on-year to make ¥914.1 billion ($6.2 billion).

https://www.biospace.com/business/takeda-pledges-30b-in-u-s-argues-against-trumps-most-favored-nations-proposal

Axogen Inc earnings missed by $0.04, revenue fell short of estimates

 Axogen Inc (NASDAQ: AXGN) reported first quarter EPS of $-0.02, $0.04 worse than the analyst estimate of $0.02. Revenue for the quarter came in at $48.56M versus the consensus estimate of $48.66M.

https://www.investing.com/news/earnings/axogen-inc-earnings-missed-by-004-revenue-fell-short-of-estimates-4032067

Wave Life Sciences (WVE) Misses Revenue Estimates, Advances Clinical Trials

 Wave Life Sciences (WVE, Financial) reported first-quarter revenue of $9.175 million, falling short of the expected $11.34 million. Despite this, the company is making significant progress in its clinical trials. In the field of obesity, dosing has been completed for the first cohorts in the INLIGHT trial, and advancements are being made in the RestorAATion-2 trial focused on Alpha-1 antitrypsin deficiency (AATD). Further data from these trials are expected later this year, showcasing Wave's expertise in RNA therapies.

The RestorAATion-2 trial is centered around WVE-006, an RNA editing treatment administered subcutaneously. This trial is expected to yield data from various dose cohorts within the year, highlighting the potential of WVE-006 alongside Wave's other GalNAc-conjugated RNA editing programs. Additionally, the company is conducting the INLIGHT trial for WVE-007, a GalNAc-siRNA therapy targeting obesity, with initial clinical data anticipated in the year's latter half. This treatment aims to offer a revolutionary approach to managing obesity, promoting healthy weight loss and the maintenance of muscle mass, with the convenience of infrequent dosing.

https://www.gurufocus.com/news/2843164/wave-life-sciences-wve-misses-revenue-estimates-advances-clinical-trials-wve-stock-news