Business

FDA approves leucovorin for cerebral folate deficiency but not autism

Published

2 hours ago

10 March 2026

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FDA reversals on UniQure, Moderna approvals worry investors

The headquarters of the U.S. Food and Drug Administration in Silver Spring, Maryland, Nov. 4, 2009.

Jason Reed | Reuters

The Food and Drug Administration on Tuesday approved a decades-old prescription vitamin called leucovorin as the first treatment for a rare genetic disorder in certain adults and children.

The move comes months after the Trump administration touted leucovorin as a potential therapy for a broader group of patients with autism spectrum disorder symptoms. The claim sparked skepticism among some in the medical and research community, but fueled excitement among families, spiking prescriptions of the drug in the U.S.

One FDA official told reporters Monday that “we don’t have sufficient data to say that we could establish efficacy for autism more broadly” but said the agency is open to interest from companies in studying leucovorin in the autism population.

The medication, also referred to as folinic acid, is a synthetic form of vitamin B9 that has been used to treat the toxic side effects of chemotherapy. Just a handful of small trials have suggested that leucovorin could be effective as an off-label treatment for children with autism, and some families have reported that it helped their nonverbal kids develop more language and social skills.

FDA officials, who requested anonymity to discuss the decision, told reporters Monday that they started with a broad review of leucovorin as an autism treatment before narrowing its approval to a smaller population with cerebral folate deficiency, a rare genetic mutation that prevents folate – a key vitamin – from properly reaching the brain.

The condition shares overlapping features with autism, typically develops in young children under age 2 and can cause severe developmental delays, seizures, a lack of muscle control and other serious neurological complications.

The officials said the FDA found that using leucovorin in patients with that condition produced the “highest quality data” to support an expanded approval, which will apply to both generic versions of the drug and GSK’s old branded medication, Wellcovorin.

“That was the data where we saw the largest effect sizes,” one FDA official said on the call. “So we narrowed in on that population, just because we felt like that was the strongest both scientific rationale and also the largest treatment effects that could be used to then overcome some of the limitations in the data sources.”

The approval was based on a systematic review of published literature on the area, including patient case reports, but not a randomized controlled clinical trial. The same official acknowledged there can be biases with systematic reviews, but emphasized that the treatment effects were so large that they outweighed those concerns.

The FDA is encouraging existing manufacturers of leucovorin to increase production to match higher demand for the drug, the officials added. While GSK originally marketed the drug from 1983 until 1997, the company said in September that it has no plans to relaunch and manufacture the product itself.

In a release Tuesday, Dr. Tracy Beth Hoeg, acting director of the FDA’s Center for Drug Evaluation and Research, said the approval demonstrates the FDA’s commitment to “rapidly identifying effective treatments for ultra rare diseases while maintaining the same evidentiary standards for approval.”

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Business

Cardiff medtech firm IQ Endoscopes boosted with new equity investment round

Published

13 seconds ago

10 March 2026

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Cardiff medtech firm IQ Endoscopes boosted with new equity investment round

The latest round has been led by growth capital investor BGF

14:31, 10 Mar 2026Updated 14:32, 10 Mar 2026

The clinical team at IQ Endoscopes.

Cardiff-based medical devices firms IQ Endoscopes has been boosted with a multi-million-pound new equity round to support its commercialisation drive.

The company is developing a single‑use gastrointestinal endoscopy platform designed to improve patient access to endoscopy and help healthcare providers respond to growing demand.

Its latest round of funding led by BGF, which has also seen follow on investment by the Development Bank of Wales, will be used to support IQ Endoscopes’ early commercial rollout in selected UK centres, strengthen manufacturing and supply chain capability, and build a robust commercial pipeline, as the company prepares for future scale‑up in the UK and overseas. The investment will also support further regulatory work and market validation in the US.

Existing reusable endoscopes create significant time burden and carbon emissions due to complex decontamination and transport processes, contributing to hospital capacity constraints and workflow inefficiencies. IQ Endoscope provides sustainable alternative designed to do the opposite, freeing up beds and physician capacity.

Since being established it has secured key regulatory approvals and commenced production. The company has identified its first customers, received initial orders and is now preparing for early commercial use of its platform. The business currently employs 20.

The exact value of its latest funding round has not been disclosed.

Matt Ginn, IQ Endoscopes chief executive, said: “This investment is a significant step forward as we move into the next phase of our growth. It allows us to begin early commercial rollout in the UK, strengthen our operational foundations and build momentum for future expansion.

“Support from BGF and the Development Bank of Wales has been instrumental in helping us reach this point, and we’re excited to continue to work closely alongside them as we bring our technology into real‑world clinical use.”

Maggy Lau, investor at BGF said: “IQ Endoscopes is addressing a clear challenge facing healthcare systems, with a product that has the potential to make a lasting, positive impact. We’re pleased to continue to support Matt and the team as the business moves into this exciting next stage of growth.”

Tom Davies, investment executive with the Development Bank of Wales said: “We’re proud to continue supporting IQ Endoscopes as it enters its next growth phase. The team’s progress in manufacturing and early clinical use is impressive, and this investment will help accelerate adoption of a technology that could enhance patient access and strengthen endoscopy services across the NHS and beyond.”

Business

Shipley names new senior executives

Published

8 minutes ago

10 March 2026

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Company fills COO, CDO and CFO roles.

Business

Motilal Oswal Wealth launches bond trading platform to widen investors’ access to fixed income

Published

14 minutes ago

10 March 2026

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Motilal Oswal Wealth launches bond trading platform to widen investors' access to fixed income

Motilal Oswal Financial Services’ wealth management arm on Tuesday launched a digital bonds trading platform to enable investors to access fixed income securities.

The company said its newly launched Bond Provider Platform will allow investors to invest in government securities, PSU bonds and corporate bonds through a dedicated digital interface.

The move comes as the domestic bond market expands and investors increasingly seek predictable returns and capital preservation alongside equity investments.
India’s bond market has grown to nearly USD 3 trillion, making it the third-largest in Asia after Japan and China, and equivalent to roughly 100-110 per cent of India’s GDP, a release said.
“With Indian households increasingly allocating savings to financial assets and the inclusion of Indian government bonds in global indices expected to bring strong foreign inflows, the opportunity in fixed income is becoming more compelling,” said Ajay Menon, managing director and chief executive officer of wealth management at Motilal Oswal Financial Services.

The new bond platform comes at a time of heightened geopolitical tensions and global market uncertainty that have increased volatility in equity markets, prompting investors, particularly high-net-worth individuals, to increase allocations to fixed income, the company said.
Ashish Malaviya, head of distribution at Motilal Oswal Wealth Management, said that amid rising equity market volatility driven by global developments, investors, particularly HNIs, are increasingly seeking capital protection, predictable income, and portfolio stability.

Business

Stifel raises Cullinan Oncology stock price target on autoimmune progress

Published

22 minutes ago

10 March 2026

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Stifel raises Cullinan Oncology stock price target on autoimmune progress

Business

Crexendo: AI, Acquisitions And A Growing Software

Published

28 minutes ago

10 March 2026

NewsAdmin

Crexendo: AI, Acquisitions And A Growing Software

Business

Saba Capital’s Boaz Weinstein warns private credit problems are multiplying

Published

37 minutes ago

10 March 2026

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Saba Capital's Boaz Weinstein warns private credit problems are multiplying

Inside Alts: Saba Capital's Boaz Weinstein on private credit's liquidity problem

A version of this article appeared in CNBC’s Inside Alts newsletter, a guide to the fast-growing world of alternative investments, from private equity and private credit to hedge funds and venture capital. Sign up to receive future editions, straight to your inbox.

The problems in private credit are “multiplying by the quarter,” due in part to the “financial alchemy of promising liquidity that isn’t there,” Boaz Weinstein, founder of Saba Capital Management, told Inside Alts this week.

“What’s happening, big picture, right now is that, for a number of reasons, in the middle of a bull market, there are cracks, there are problems, there are frauds, there are companies that are going bad without being a fraud,” Weinstein said in an exclusive interview. “So for those reasons, investors are seeing their dividends being cut. They want their money back, and [on] Wall Street the No. 1 story right now is where the redemption is going to be for all these managers.”

Weinstein, of course, is a central figure in that story. His firm, Saba, alongside Cox Capital Management, just launched a tender offer to purchase 6.9% of shares in one of Blue Owl’s nontraded private credit funds at a 34.9% discount.

“We were hearing from investors in these funds that they wanted their money back,” he said. “They were trying to find someone to step into their shoes, so that happened in an organic way.”

That fund, known as Blue Owl Capital Corp. II, halted quarterly redemptions and sold $1.4 billion of direct lending investments to provide liquidity for its investors. It turned out to be among the first in a slew of nontraded, private credit funds that have been hit with redemption requests above the typical 5% quarterly cap.

Private wealth flows across products tracked by analysts at Jefferies were down 19% in the first quarter compared with Q4. Analysts said they expect redemption rates across retail credit products to increase.

Saba and Cox see an opportunity amid investors’ limited liquidity. They are launching similar tenders for stakes in several other funds at Blue Owl as well as Starwood Real Estate Income Trust. This has caused some to question whether Weinstein has been criticizing the private credit industry only to scare retail investors into selling their stakes to him at a discount.

While speaking with Inside Alts, Weinstein clarified that he doesn’t actually believe there will be a wave of private credit defaults or frauds, nor does he think people should redeem further. (“The redemptions have arrived,” he said.)

In fact, he’s actually bullish on several of the largest private credit managers. Weinstein said over the past few weeks, he bought shares in “the most amazing managers,” including Ares, Apollo and Blackstone. He said he is even long “a little bit” of Blue Owl equity.

“We’re long the stocks of these companies on the idea that, in case this is overdone, these are the guys that are going to be the winners at the end, when the smoke clears and their stocks may represent good value,” said Weinstein.

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Weinstein said he thinks private credit is trading at pessimistic levels and public credit is trading at “incredibly optimistic levels.” He’s shorted public credit through credit default swaps and credit derivatives. Weinstein said that the gating of private credit funds means that investors will have to sell more liquid assets to raise cash, which would weigh on the market.

“I think that public credit is incredibly mispriced and part of my short-term thinking about it is informed by the issues that the private credit markets are having,” he said.

Weinstein said it will be a “number of weeks” before they know what happens with the Blue Owl bids, and how much they’ll end up buying. Weinstein said the tender offers weren’t “personal” against the manager, but rather, he said, “if we go bid for something, it’s a sign we think the manager is good.”

However, Weinstein noted a firm called Cliffwater as one in the private credit space that they’re “watching the most closely.” He said Cliffwater operates similarly to a fund-of-funds model, where they don’t own the loans directly, but rather, they’re invested in other managers. As a result, they have limited control over fulfilling their own redemption requests – something Weinstein describes as a “turducken” (a chicken stuffed inside a duck, stuffed inside a turkey).

According to a Securities and Exchange Commission filing, Cliffwater disclosed that as of the end of last year, 69% of its Corporate Lending Fund was comprised of direct investments in underlying credit and the remaining 31% was exposed to funds.

Weinstein predicted that when Cliffwater announces its redemption rate — expected as early as Tuesday — it could be between 10% and 20%.

“I don’t know their exact cash position, but we think it’s very likely that they’re going to have to start redeeming and they’re going to get cut back when they redeem these funds that they’ve invested in,” he said.

Cliffwater was also the subject of a recent viral investor letter by the hedge fund Rubric Capital, which said the alternatives manager could be “a canary in a coal mine” and “the first domino in the bank run we foresee,” according to The New York Times, which cited two people who read the private note.

When asked about what happens to private credit if there’s a real credit cycle, Weinstein said, “it will fall harder than it should.”

He added that “one of the best opportunities” in his career would be investing in private credit at a massive discount “when the economy slows.”

“Maybe that’s not for a year, maybe it’s about to happen. Maybe it’s going to happen years from now,” Weinstein said. “It’s about to get super interesting.”

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Business

Cantor Fitzgerald raises Neurocrine Bio price target on Ingrezza outlook

Published

43 minutes ago

10 March 2026

NewsAdmin

Cantor Fitzgerald raises Neurocrine Bio price target on Ingrezza outlook

Business

DA Davidson reiterates Repay stock Buy rating on strong Q4 results

Published

52 minutes ago

10 March 2026

NewsAdmin

DA Davidson reiterates Repay stock Buy rating on strong Q4 results

Business

ABM Q1 2026 slides: revenue beats offset by margin pressure

Published

57 minutes ago

10 March 2026

NewsAdmin

ABM Q1 2026 slides: revenue beats offset by margin pressure

Business

Tekmar encouraged by momentum and record order book despite drop in revenues

Published

1 hour ago

10 March 2026

NewsAdmin

Tekmar encouraged by momentum and record order book despite drop in revenues

The County Durham offshore engineering group says it is seeing positive signs

Offshore technicians assembling Tekmar’s patented TEKLINK cable protection system during offshore installation on an offshore wind farm(Image: Unknown)

Offshore energy group Tekmar says it is encouraged by its latest results, despite seeing a drop in revenues and another year of losses.

The County Durham-based firm, which provides asset protection technology and offshore energy services, has released results for the year ending September 30 2025.

They show turnover falling slightly to £28.7m, while gross profit fell to £9.8m. After taking into account exceptional items, depreciation and other costs, Tekmar reported an overall loss for the year of £3.9m, though this was less than last year’s losses.

But Tekmar said that £43m of new orders since last July and currently had a record order book. It said its balance sheet had been strengthened, including by the sale of its former Innovation House building for £2.8m.

The company said its Project Aurora plan to scale the business through both organic growth and acquisitions, and to improve its financial strength, was progressing well.

CEO Richard Turner said: “FY25 has been a pivotal and highly productive year for Tekmar as we launched and started to execute on Project Aurora. The group delivered results in line with market expectations, alongside a material improvement in profitability in the second half.

Richard Turner, CEO Tekmar Group plc(Image: Tekmar Group plc)

“We are pleased to have been able to maintain our momentum post period end – in the first four months of FY26 we have delivered a record order book, with multi-year visibility and have unlocked further growth potential by significantly strengthening our balance sheet.

“We are encouraged by the strong start to the new financial year and healthy pipeline we see ahead of us and are focused on delivering sustained, profitable growth and enhanced value for shareholders.”