Thursday, May 15, 2025
Taysha's Rett Syndrome Gene Therapy Gets FDA Support: Key Trial Design OKd Without Phase Meeting
Taysha Gene Therapies (NASDAQ: TSHA) reported Q1 2025 financial results and provided updates on its TSHA-102 program for Rett syndrome. The company received FDA alignment on pivotal Part B trial design, allowing direct submission of the trial protocol as an IND amendment in Q2 2025, potentially expediting study initiation. Safety data shows both high dose (1x1015 vg) and low dose (5.7x1014 vg) of TSHA-102 are well-tolerated with no treatment-related SAEs or DLTs in 10 patients across REVEAL trials. Financially, Q1 2025 showed R&D expenses of $15.6M (down from $20.7M in Q1 2024), net loss of $21.5M ($0.08 per share), and cash position of $116.6M, expected to fund operations into Q4 2026. The company will present three oral presentations at the upcoming IRSF Rett Syndrome Scientific Meeting in June 2025.
George Floyd Riots 2.0
by Bill Glahn
The original was such a hit that Democrats are demanding a sequel. The Minneapolis Star Tribune breathlessly reports,
Walz, other Minnesota agencies weigh in on rumors Trump will pardon Derek Chauvin.
As the fifth anniversary of the death of George Floyd nears, the governor and other public officials are bracing for possibility the president will undo federal convictions.
You read that correctly. Chauvin was convicted in state court (based on questionable testimony). The Feds piled on with convictions for civil rights violations and tax evasion, to boot. He in currently being held in a Federal facility.
A “possibility.” Based on what? The Star Tribune quotes a Minneapolis police spokesman,
To be clear, we have no credible intelligence about any pardon or planned disruptions here in Minneapolis.
Four (4) reporters contributed to the Star Tribune piece, which contains zero (0) news content. The body of the article runs almost 1,100 words. That’s 1,100 words to say exactly nothing.
So, who is stirring the pot?
Responding to questions from reporters this week, Gov. Tim Walz said the White House has given him no indication that a federal pardon for Chauvin is imminent.
Gov. Walz channels his inner Inspector Kemp from Young Frankenstein (1974), “A riot is an ugly thing, and I think that it is just about time we had one.” Democrats are convinced, despite all available evidence to the contrary, that the George Floyd riots were crucial to defeating Trump in 2020. With 2025 being an election year in Minneapolis, and Trump back in office, they are desperate to trigger another round. Anything to distract the voting public from the colossal political failures in Minnesota at every level.
You will hear of riots and rumors of riots but see to it that you are not alarmed.
https://www.powerlineblog.com/archives/2025/05/george-floyd-riots-2-0.php
Is There a Method to Trump's Qatar Madness?
by Roger Simon
Qatar???
Yes, Qatar.
Qatar, the country that donated $6.3 billion with a b— more than any other nation, more even than communist China or the Saudis—to America’s terminally woke universities since such things were recorded (1986)?
Yes, Qatar.
Qatar, that oil-and-natural gas-rich potentate that has long treated their foreign employees like virtual slaves and human trafficking victims and, even now, after supposed reforms, works their domestic employees 14 hours a day, half of them for an incredible 18 hours, at ridiculously low salaries, their passports confiscated to prevent escaping, according to Amnesty International?
Yes, Qatar
You mean Qatar, the country Donald Trump once called (2017) a nation that has “historically been a funder of terrorism at a very high level” and whose former leader multi-billionaire Sheikh Hamad bin Jassim bin Jaber Al Thani (aka HBJ) just donated his used 787 to Trump as if it were his personal Kars for Kids? That same HBJ sheltered 9/11 mastermind Khalid Sheik Mohammed, is the bagman behind $30 million monthly payments to Hamas in Gaza and is said to be the major funder of the Oct. 7 massacre. Not inconsequentially, he apparently doesn’t care for Jews.
Yes, Qatar— the place about which Louisiana senator John Kennedy opined “I trust Qatar, like I trust a rest stop bathroom.”
So what’s going on?
I could say “beats me” or refer people to Eli Lake’s analysis at The Free Press—”Trump’s Serenity Prayer for the Middle East”—but there is a method to Trump’s seeming madnes . Not parenthetically Mr. Trump has also been making nice with the Syria’s new president and former al Qaeda terrorist with a $10m bounty on his head, Ahmed al-Sharaa, offering to lift sanctions on his country and urging him to recognize Israel and join the Abraham Accords.
What Trump is doing, clearly, is trying a whole new approach to the Middle East, emphasizing peaceful economic relations and positivity, almost like a latter-day Norman. Vincent Peale. Our president is abjuring the attempts at ideological rectification inherent in the Iraq War and in the policies of both Clintons, both Bushes, both Cheneys, Obama, Biden and practically our entire body politic—in other words those who are now tarred with the epithet neocons, perhaps unfairly since virtually everyone in both parties (except, intermittently, Trump himself) favored that war at that time and only learned subsequently how difficult it was to to turn Iraq into Denmark.
In Trump’s new—it might even be called revolutionary—approach, we may not agree with the Islamic world about the role of women, personal freedom and a host of other matters, but we can still be friends and trade together. We do not need to change you. You’re capable of making your own decisions and building your own societies. Look at the magnificence of Riyadh and Doha. You did that. Mohammed bin Salman (MBS) is a great leader and so forth
Trump even extended his hand, made a similar offer, in a more tentative way but we can be sure they got it, to Iran (whether will listen or want to is another matter).
Could this approach work? At first I gasped. Anybody who knows the region, has been there multiple times, has read Memri.org that translates Arab videos and news articles verbatim as assiduously as I have would have reason to be skeptical in the extreme. As recently as late this March, Memri had this item from Qatar’s Al Jazeera— “Trump Is A Terrorist Who Seeks To Destroy The World Order And Foment Chaos; He Presents Himself As A Hero But Isn't One.”
Oh, well. Whoever said it would be smooth sailing?
Neither of the Sinwar brothers, alive or dead, many of their ilk high and low, are likely to give up anti-Western, anti-Israel, anti Sunni or Shiite (deepening on which side they are on) frequently violent quasi-religious passions that have been dominating their culture pretty much unceasingly since the Seventh Century.
And yet, what other approach has worked? None, obviously. We all know that. We can assume Trump does too. So he has set out on another course as he has in so many areas. The man is truly remarkable and courageous for that. We have never had a president who has tried to do so much while being so roundly, and often irrationally, condemned for it, and then, almost miraculously, seeming to get positive results.
That does not mean he cannot be wrong. But the rest of us—politicians, pundits and citizens—have our own choice in this matter. We can support this initiative for Middle Eastern policy reform or not. The more of us who vocally support it, the more likely it is to succeed, to be heard across oceans. We are the American equivalent of the Arab Street.
At its best, Trump’s approach will bring about change more rapidly in the Islamic world than all the hectoring, lectures, sanctions or bombs could achieve. That’s how human behavior usually operates—the bird flies to the open hand and all that.
Unfortunately, it’s been tried a few times before in the Middle East and failed. This time, however, there is more behind it, the power of the United States and its president.
And if that fails, he, and we, can always change our minds and act differently. That happens pretty quickly nowadays.
Two final things: Not to be ignored in Trump’sMiddle East initiative, but for some reason missing from the many analyses I have seen, is that the president is about to enact the first trillion dollar defense budget, by far the largest in U. S. and world history and over three times the size of China’s.
This is peace through strength on steroids. Een though pundits may ignore it, the Emir of Kuwait, MBS, etc. certainly do not.
Two: As he landed in Qatar, President Trump said the following regarding concern for Israel that he skipped on this trip: “My visit to the Middle East does not sideline Israel; it benefits Israel,” and further noted, “This is good for Israel, having a relationship like I have with these countries.”
Probably so, but as always, trust but verify… and verify again.
https://americanrefugees.substack.com/p/is-there-a-method-to-trumps-qatar
Missouri Lawmakers Approve Referendum to Repeal Abortion-Rights Amendment
Six months after Missouri voters approved an abortion-rights amendment, Republican state lawmakers on Wednesday approved a new referendum that would seek the amendment's repeal and instead ban most abortions with exceptions for rape and incest.
The newly proposed constitutional amendment would go back to voters in November 2026, or sooner, if Republican Gov. Mike Kehoe calls a special election before then.
Republican senators used a series of rare procedural moves to cut off discussion by opposing Democrats before passing the proposed abortion-rights revision by a 21-11 vote. The measure passed the Republican-led House last month.
Immediately after the vote, protestors erupted with chants of "Stop the ban!" and were ushered out of the Senate chamber.
The Senate then blocked further Democratic debate and gave final approval to a separate measure repealing provisions of a voter-approved law guaranteeing paid sick leave for workers and cost-of-living increases to the minimum wage. That measure does not go back to the ballot. It will instead become law when signed by Kehoe, who has expressed his support for it.
After taking the sweeping votes, the Senate effectively ended its annual legislative session -- 2 days ahead of a constitutional deadline to wrap up work.
Democrats were outraged by the legislative actions and vowed to retaliate by slowing down any Senate work next year.
"Our rights are under attack," Democratic state Sen. Brian Williams said during debate. He accused Republicans of "trying to overturn the will of the voters."
Republicans contend they are simply giving voters a second chance on abortion -- and are confident they will change their minds because of the new rape and incest exceptions.
"Abortion is the greatest tragedy in the world right now," Republican state Sen. Mary Elizabeth Coleman said while explaining her efforts to repeal the abortion-rights amendment. If someone's fine with "taking the life of an innocent, then probably you can justify whatever you want."
Some GOP lawmakers said they needed to repeal the paid sick leave requirement, which kicked in May 1, because it's adding costs that threaten the financial viability of small businesses. Republicans had been negotiating with Democrats over an alternative to exempt only the smallest businesses before scrapping that and opting for the full repeal.
Missouri lawmakers have a history of altering voter-approved policies. They previously tried to block funding for a voter-approved Medicaid expansion and authored changes to voter-approved measures regulating dog breeders and legislative redistricting.
Missouri's abortion policies have swung dramatically in recent years.
When the U.S. Supreme Court ended a nationwide right to abortion by overturning Roe v. Wade in 2022, it triggered a Missouri law to take effect banning most abortions. But abortion-rights activists gathered initiative petition signatures to reverse that.
Last November, Missouri voters narrowly approved a constitutional amendment guaranteeing a right to abortion until fetal viability, generally considered sometime past 21 weeks of pregnancy. The amendment also allows later abortions to protect the life or health of pregnant women and creates a "fundamental right to reproductive freedom" that includes birth control, prenatal and postpartum care, and "respectful birthing conditions."
A limited number of surgical abortions have since occurred in Missouri, but medication abortions remain on hold while Planned Parenthood wrangles with the state over abortion regulations.
The new measure seeks to repeal the abortion-rights amendment and instead allow abortions only for a medical emergency or fetal anomaly, or in cases of rape or incest up to 12 weeks of pregnancy. It also would prohibit gender transition surgeries, hormone treatments, and puberty blockers for minors, which already are barred under state law.
Polling indicates "that most voters are opposed to most abortions in Missouri but do want to allow for abortions with limited exceptions," said Sam Lee, director of Campaign Life Missouri.
The ballot title that voters will see doesn't explicitly mention repealing Amendment 3. Instead, it says the new measure would "ensure women's safety during abortions, ensure parental consent for minors," and "allow abortions for medical emergencies, fetal anomalies, rape, and incest." It also states that it will "protect children from gender transition," among other provisions.
Democratic state Sen. Tracy McCreery called the measure "an attempt to mislead and lie to the voters," echoing similar accusations that Republicans had made against the original Amendment 3.
An abortion-rights coalition that includes Planned Parenthood affiliates, the American Civil Liberties Union, and others planned a rally Thursday at the Missouri Capitol and vowed a vigorous campaign against the measure.
"Abortion rights won in this state 6 months ago, and mark my words: Missourians will protect reproductive freedom again," said Emily Wales, president and CEO of Planned Parenthood Great Plains Votes.
FAA says Denver air traffic briefly lost communications on Monday
The Federal Aviation Administration said on Thursday that Denver air traffic control lost communications for about two minutes on Monday but was able to maintain contact through an emergency frequency.
Franklin McIntosh, the FAA's deputy head of air traffic control, said during a U.S. House of Representatives hearing that at the Denver Air Route Traffic Control Center both the main and backup frequencies failed for about two minutes but a controller was then able to transmit to the aircraft to move to a secondary frequency.
He said there was no loss of separation between airplanes during the outage.
"Anytime there are these outages, which are happening more regularly now, it is very concerning," said Representative Robert Garcia, a California Democrat, at an FAA oversight hearing.
The FAA said in a written statement it is investigating after part of the center experienced a loss of communications around 1:50 p.m. in Denver, when both transmitters that cover a segment of airspace went down.
"Controllers used another frequency to relay instructions to pilots. Aircraft remained safely separated and there were no impacts to operations," the FAA said.
FAA communications failures have gained urgent attention due to a series of telecom outages at the facility overseeing Newark air traffic after three incidents have shaken public confidence.
The FAA relocated control of the Newark airspace to Philadelphia last year to address staffing and congested New York City area traffic. But the FAA is about 3,500 air traffic controllers below targeted staffing levels.
The latest incidents highlight the air traffic control network's aging infrastructure and come after U.S. Transportation Secretary Sean Duffy last week proposed spending billions of dollars to fix it over the next three to four years.
FDA Delays Decision Date on Biohaven’s Spinocerebellar Ataxia Application
The FDA also changed its tune and is now planning to convene an advisory committee to discuss Biohaven’s application.
The FDA has pushed back by three months its decision date for Biohaven’s investigational glutamate modulator troriluzole for the treatment of spinocerebellar ataxia, the biotech announced Wednesday evening. Biohaven, whose stock slumped more than 12% in premarket trading Thursday, now expects to receive the verdict in the fourth quarter.
In a note to investors early on Thursday, analysts at William Blair called the delay “surprising,” especially given the “proximity to the completed mid-cycle review.” On Monday, Biohaven also said that it had not received communication from the FDA regarding its application for troriluzole and continued to expect the regulatory decision to come in the third quarter.
In its press announcement on Wednesday, Biohaven said that the regulatory delay was to give the FDA enough time to review additional data that the company submitted in response to a request from the agency. As per William Blair based on communication with Biohaven management, the FDA’s questions “were part of the routine review process,” which the company has responded to “in a timely manner.”
The company also revealed that the FDA now intends to convene an advisory committee to discuss the application for troriluzole, though that meeting has not been scheduled yet. William Blair analysts said they are “not surprised by the AdCom expectation given troriluzole would represent the first FDA-approved therapy for the treatment of SCA [spinocerebellar ataxia].”
Troriluzole works by modulating the neurotransmitter glutamate, a key excitatory signal. The drug candidate increases the uptake of glutamate from the synapse, in turn lowering the concentration of the neurotransmitter in the synaptic space, according to Biohaven’s website. The overall effect of troriluzole is to prevent the overexcitation of neurons.
In September 2024, Biohaven unveiled topline pivotal data for troriluzole, demonstrating that the drug slowed disease progression by 50% to 70% versus untreated comparators.
The troriluzole delay comes amid delays at the FDA. Last week, for instance, the regulator failed to meet its target decision date for GSK’s Nucala, which the pharma is pushing into chronic obstructive pulmonary disease. Last month, the FDA also ran into delays for Stealth BioTherapeutics’ Barth Syndrome application and Novavax’s COVID-19 vaccine.
William Blair addressed this possibility directly in its note. “There will be investor debate over whether this is a sign of FDA delays as a result of layoffs and restructuring at the agency,” the analysts wrote, however noting that the FDA has had a history of extending reviews in the neuroscience space. In January 2023, for example, argenx’s supplemental application for Vyvgart Hytrulo in myasthenia gravis also ran into a regulatory delay, William Blair said.
Despite the delay, “we remain optimistic that the FDA will show more regulatory flexibility” toward troriluzole “given the rare-disease nature of SCA, lack of treatment options for the disease, and troriluzole’s risk/benefit,” the analysts added.
Veri Medtech (VRHI) Reaches $100 Million Revenue Milestone
Veri Medtech (VRHI) announced reaching a significant milestone of $100 million in cumulative revenue since its 2017 inception, with Q1 2025 revenue of $3.3 million. The healthcare technology platform has achieved several key milestones, including reaching up to 400,000 patients and 400 physicians on its network. The company has recently relaunched two key platforms: Veriheal.com and DosePop.com, as part of its telehealth diversification strategy. VRHI has also retained a PCAOB auditor in preparation for a potential uplisting. The company positions itself to compete with LifeMD, Ro, and HIMS in the $114 billion telehealth market through its Infinite Wellness Loop platform.
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