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Thursday, November 6, 2025

Regenxbio Inc (RGNX) Q3 2025 Earnings Call Highlights

Cash Equivalents and Marketable Securities: $302 million as of September 30, 2025, compared to $245 million as of December 31, 2024.
Revenue: $30 million for the quarter ended September 30, 2025, compared to $24 million for the same period in 2024.
Upfront Payment: $110 million received from Nippon Shinyaku in the first quarter of 2025.
Net Proceeds from Royalty Monetization: $145 million received in the second quarter of 2025.
Cash Runway Guidance: Expected to fund operations into early 2027, excluding potential non-dilutive financing opportunities.

Positive Points

Regenxbio Inc (NASDAQ:RGNX) has completed enrollment in the Affinity Duchenne Pivotal trial ahead of schedule, indicating strong community enthusiasm for RGX 202.
The company has a robust manufacturing capability, producing up to 2,500 doses of RGX 202 per year, ensuring readiness for commercial supply.
Regenxbio Inc (NASDAQ:RGNX) has a strong financial position with $302 million in cash, equivalents, and marketable securities, expected to fund operations into early 2027.
The RGX 121 program for MPS-2 has shown positive 12-month data and has completed FDA inspections with no observations, boosting confidence in its approval.
The company has strategic partnerships, such as with Nippon Shinyaku for RGX 121 and AbbVie for retinal disease programs, enhancing its commercial prospects.

Negative Points

Regenxbio Inc (NASDAQ:RGNX) faces uncertainties with FDA interactions, particularly concerning the accelerated approval pathway for RGX 202.
The company is reliant on non-dilutive financing opportunities to extend its cash runway beyond 2027, which are contingent on market conditions.
There is potential regulatory risk in Europe, as EMA may require a placebo control arm for approval, which could complicate the RGX 121 program.
The competitive landscape in gene therapy, particularly for Duchenne muscular dystrophy, poses challenges with recent developments in competitor products.
Regenxbio Inc (NASDAQ:RGNX) has yet to finalize its pricing strategy for RGX 202, which could impact its market penetration and revenue potential.

Q & A Highlights

Q: Can you provide insights on the potential for the accelerated approval pathway for RGX 202 for Duchenne muscular dystrophy? A: Curran Simpson, President and CEO, explained that they expect several FDA interactions, including a pre-BLA meeting around the time of top-line data release in early Q2 2026. They are optimistic about accelerated approval due to significant functional improvements observed in older patients, which differ from natural history expectations. The safety profile also supports this pathway.

Q: What is the status of the confirmatory trial for Duchenne muscular dystrophy, and how is enrollment progressing? A: Curran Simpson noted that enrollment for the confirmatory study began at the end of October, following the completion of pivotal enrollment. They expect to be substantially through enrollment by mid-2026, with an additional 30 patients specified in the protocol. Stephen Pakola, Chief Medical Officer, added that the study design is similar to previous trials, focusing on ambulatory patients aged one and older.

Q: Could you discuss the manufacturing capacity at the Rockville site and its ability to meet demand for RGX 202 and RGX 121? A: Curran Simpson stated that the Rockville facility houses a 2000 L bioreactor, capable of producing up to 2,500 doses of RGX 202 annually. The Hunter program (RGX 121) uses less than 5% of overall capacity, allowing for efficient production without significant capacity constraints.

Q: How are you approaching regulatory interactions with the FDA for RGX 121 and RGX 202, especially in light of recent leadership changes at the FDA? A: Curran Simpson mentioned that for RGX 121, they are in the late stages of BLA review, with a Padua date set for February 8th. They expect a late-cycle meeting with the FDA and have had positive interactions, including no observations from facility inspections. For RGX 202, a pre-BLA meeting is anticipated between early Q2 2026 top-line data and BLA filing.

Q: What are your thoughts on the recent M&A and licensing activity in the gene therapy space for wet AMD, and how does your program compare? A: Stephen Pakola highlighted the strong interest in one-time gene therapy treatments among retina specialists, as evidenced by surveys. The partnership with AbbVie, which has significant ophthalmology experience, validates their program. The ongoing advancements and financial commitments from AbbVie underscore the program's potential.

For the complete transcript of the earnings call, please refer to the full earnings call transcript.

https://finance.yahoo.com/news/regenxbio-inc-rgnx-q3-2025-190833422.html

Paxton sues Roblox, claiming it puts 'pixel pedophiles and profits' over children's safety

Texas Attorney General Ken Paxton has filed a lawsuit against popular online gaming site Roblox, claiming the company behind it is putting "pixel pedophiles and profits" over children's safety.

Paxton announced the suit on Thursday, Nov. 6, saying that Roblox markets itself as a "safe digital space for children" but that "in reality, it has become a breeding ground for predators."

Roblox says that it is "deeply committed to child safety" and that its "policies are purposefully stricter than those found on many other platforms."

What they're saying:

"We cannot allow platforms like Roblox to continue operating as digital playgrounds for predators where the well-being of our kids is sacrificed on the altar of corporate greed," said Paxton in a release. "Roblox must do more to protect kids from sick and twisted freaks hiding behind a screen. Any corporation that enables child abuse will face the full and unrelenting force of the law."

Roblox released a statement to FOX 7 Austin in response to the lawsuit, saying:

"We share Attorney General Paxton’s commitment to keeping kids and teens safe online, which is why we have implemented industry-leading protocols in an effort to protect users and remove bad actors. We are disappointed that, rather than working collaboratively with Roblox on this industry-wide challenge and seeking real solutions, the AG has chosen to file a lawsuit based on misrepresentations and sensationalized claims.

"Roblox is deeply committed to child safety. Our policies are purposefully stricter than those found on many other platforms. We prohibit the sharing of images and videos in chat, use filters designed to block the exchange of personal information and our trained teams and automated tools continuously monitor communications to detect and remove harmful content. We have a strong record of working with law enforcement and continue to invest in advanced safety systems to set the gold standard in online safety for the industry.

"We have introduced over 145 safety measures on the platform this year alone, and as a first for the industry, we recently announced that we are working to implement age estimation for all users accessing chat features soon, which will help prevent adults from chatting with minors."

What is Roblox?

What we know:

Roblox is an online gaming platform where users can "create, play and connect with each other in experiences built by our global community of creators," according to its website.

Last quarter, Roblox says it had about 151.5 million active users, with almost 40 billion hours of engagement between them.

The platform allows people to play an infinite number of games designed by other users, and to even create their own games using Roblox's game-developing tool, Roblox Studio. Once a person signs up, they can play games, build them, and chat with others for free.

Roblox also offers in-game purchases, made through its own virtual currency called "Robux", which can be purchased as part of a Roblox Premium membership or through online gift cards.

Other state AGs filed suit

Big picture view:

Paxton is not the first state attorney general to file suit against the company.

Other state AGs that have filed suit include Louisiana AG Liz Murrill in August and Kentucky AG Russell Coleman in October.

Both also allege that the platform has allowed predators access to kids.

Safety controls on Roblox

Dig deeper:

Roblox does say it has a variety of controls and features to help keep users safe.

Parental controls

Roblox offers account controls that let parents restrict how kids can interact on the site and the types of games they can play.

The "Account Restrictions" feature gives parents and caregivers the option to limit chat to a curated list of contacts, or turn it off altogether. Parents can also choose to lock their kid’s "Contact Settings" so they cannot be changed, Roblox explains on its website.

To enable these settings, parents can add their email address to a kid's Roblox account and create a PIN that prevents kids from changing the settings back.

Kids of any age can create an account on Roblox with no parental restrictions. For accounts of kids under 13, Roblox automatically defaults to stricter settings — but a kid could change these if there's no parent PIN.

Parents can also now see their child's average screen time and friends list from their Parental Controls dashboard, as well as set daily screen-time limits.

Parents can learn more about those controls here in Roblox's Parent and Caregiver Guide.

Age verification/estimation tech

Roblox is currently testing out an age verification feature which would require users to provide a government-issued photo ID to prove they are at least 13 years old.

In July, the company announced it would be investing in "age estimation" technology to help confirm user ages and grant access to Trusted Connections. Users 13 and over who want to use this new feature will be prompted to provide a video selfie, which will then be analyzed to estimate their age.

Users must receive an estimation of at least 13 years old to access Trusted Connections and other chat functions that are restricted to users 13 and older, says Roblox.

If they are estimated to be under 13, the platform will remove access to age-inappropriate features. If the system cannot estimate the user's age, the user's age will remain "unconfirmed" and they will not be able to access the Trusted Connections feature. However, if they are over 13, they will be able to confirm their age by providing a government-issued photo ID.

Roblox says that unfiltered communication between users aged 18+ and those 13 to 17 is blocked unless a preexisting, real-life relationship exists, confirmed through a QR code or phone contact list.

Open-source AI detection system

In its November Safety Snapshot, Roblox says it is using an open-source "Roblox PII Classifier" to detect and block attempts to violate the company's policies about sharing personally identifiable information, or PII.

It says that the tool has a "98% recall of potential PII conversations in English text on Roblox."

Moderator team

In addition to the AI system, Roblox says it employs a large team of "thousands of trained professionals" to monitor uploaded images, audio and video.

The platform also says it filters text chats in multiple languages to "block detected inappropriate content, such as discriminatory speech, bullying, extremism, violence, sexual content, and more, as well as personal information and instructions on how to move off the platform."

Working with law enforcement

Roblox told FOX 7 Austin in a statement that it "works closely with law enforcement, government agencies, mental health organizations, and parental advocacy groups to create resources for parents and to keep users safe on the platform."

The company says it maintains direct communication with organizations like the FBI and the National Center for Missing and Exploited Children (NCMEC), for immediate escalation of serious identified threats.

https://www.fox7austin.com/news/ken-paxton-texas-ag-lawsuit-roblox-childrens-safety

Tandem stock rises after hours on Q3 beats, record sales

Tandem Diabetes Care (Nasdaq:TNDM) shares got a boost after hours today on third-quarter results that came in ahead of the consensus forecast.

Shares of TNDM rose nearly 20% to $15.89 apiece in post-market trading today.

The San Diego-based automated insulin delivery technology developer reported losses of $21.2 million. That equals 31¢ per share on sales of $249.3 million for the three months ended Sept. 30, 2025.

Tandem — one of the largest diabetes tech companies in the world — recorded a 9% bottom-line gain on a sales increase of 2.2%. The company said its third-quarter sales marked records for both the U.S. and internationally.

Losses per share landed 3¢ ahead of expectations on Wall Street. Sales also topped estimates as experts projected $235.8 million in revenue.

https://www.massdevice.com/tandem-stock-rises-after-hours-on-q3-beats-record-sales/

Intellia Third Quarter 2025 Financial Results and Recent Updates

Intellia Therapeutics (NASDAQ:NTLA) reported Q3 2025 results and clinical updates on November 6, 2025. The company said the FDA placed a clinical hold on the MAGNITUDE and MAGNITUDE-2 Phase 3 trials of nex-z after rare Grade 4 liver transaminase elevations; more than 650 patients are enrolled in MAGNITUDE and 47 patients in MAGNITUDE-2. Intellia completed enrollment in the Phase 3 HAELO trial of lonvo-z and expects topline HAELO data by mid-2026, with a potential U.S. launch in 1H 2027. Cash, cash equivalents and marketable securities were $669.9M at September 30, 2025, and are expected to fund operations into mid-2027. Third-quarter net loss was $101.3M.

https://www.stocktitan.net/news/NTLA/intellia-therapeutics-announces-third-quarter-2025-financial-results-kx0no312v8m7.html

Aveanna outlines 2025 revenue target above $2.375B

expanding preferred payer strategy and integration of Thrive

https://seekingalpha.com/news/4518095-aveanna-outlines-2025-revenue-target-above-2_375b-while-expanding-preferred-payer-strategy

Lantheus Announces CEO Retirement and Leadership Transition

On November 6, 2025, Lantheus announced a leadership transition with CEO Brian Markison retiring effective December 31, 2025, and Mary Anne Heino appointed as Executive Chair and Interim CEO. The company reported third-quarter 2025 financial results, including a revenue of $384 million and a decrease in net income, while also highlighting strategic initiatives such as the acquisition of Life Molecular Imaging and the advancement of its radiopharmaceutical pipeline.

https://www.tipranks.com/news/company-announcements/lantheus-announces-ceo-retirement-and-leadership-transition

Sen. Hagerty rolls out bill to bar foreigners from funding US elections

Sen. Bill Hagerty (R-Tenn.) and seven other Republican senators have rolled out legislation intended to prevent foreign donors from funneling cash into projects impacting US elections.

While it is illegal for foreign nationals to donate to federal, state or local campaigns, some have contributed to intermediaries backing specific policy goals and adjacent efforts like get-out-the-vote activity.

Hagerty’s Preventing Foreign Interference in American Elections Act targets those workarounds.

“After years of hysteria over Russiagate and alleged foreign influence in American elections, it turns out Democrats have recently benefited from hundreds of millions of dollars in election-related contributions from a shadowy foreign billionaire,” Hagerty told The Post in a statement.

“This type of influence undermines democracy and self-government here in America, and its staggering scope should be alarming,” he continued, adding that his “legislation that will put an end to covert foreign influence on our elections and protect Americans’ voice in electing their leaders.”

Hagerty appeared to be referencing Hansjorg Wyss, a lefty Swiss billionaire whose eponymous Wyss Foundation’s associated Berger Action Fund has pumped millions into the massive liberal dark money Sixteen Thirty Fund — also known as Families Over Billionaires.

Families Over Billionaires used “paid media, rapid response, surrogate operations, and grassroots mobilization” to whip up opposition to key aspects of President Trump’s agenda, such as his One Big Beautiful Bill Act.

The Berger Action Fund has dumped more than $280 million into the Sixteen Thirty Fund, records show.

On Wednesday, Nebraska Attorney General Mike Hilgers announced a lawsuit against Wyss and six groups he’s backed over cash sent to six committees working to gin up support for ballot initiatives.

The bill, which Hagerty introduced last year as well, bars foreign nationals from contributing to efforts related to get-out-the-vote activities, state ballot initiatives, ballot harvesting, and/or public communications that elevate a political party.

Under the Preventing Foreign Interference in American Elections Act, foreign nationals would be barred from donating to conduits that then use that money for activities related to American elections.

The measure also features safeguards aimed at stopping federal probes from being overly broad or demanding, including a mechanism that allows for petitions to squash subpoenas on the basis of bias.

Hagerty’s legislation has the backing of Sens. Marsha Blackburn (R-Tenn.), Ted Budd (R-NC), Ted Cruz (R-Texas), John Kennedy (R-La.), Mike Lee (R-Utah), Cynthia Lummis (R-Wyo.) and Roger Marshall (R-Kan.).

“Only Americans should get to participate in American elections — duh,” Kennedy said in a statement.

“The Preventing Foreign Interference in American Elections Act would safeguard our electoral system from Chinese Communist Party-linked actors and anti-American foreign billionaires trying to game our democratic process.”

House Committee on Administration Chairman Bryan Steil (R-Wis.) plans to reintroduce companion legislation in the lower chamber but the timing hasn’t yet been determined, The Post has learned.

“There is no place for foreign influence in American elections,” Steil said in a statement. “By eliminating the potential for foreign nationals to fund elections operations we are one step closer to securing our democratic process.”

https://nypost.com/2025/11/06/us-news/sen-hagerty-rolls-out-bill-to-plug-loopholes-bar-foreigners-from-funding-us-elections/

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