Late-stage clinical biopharma company Acelyrin Inc said on Monday that it was seeking a valuation of up to $1.5 billion in its initial public offering in the United States that comes at a time when new listings are testing investors' appetite.
The Los Angeles-based company is aiming to raise up to roughly $370 million, by selling 20.6 million shares at nearly the top-end of its proposed price range of $16 to $18 per share, the company said in a filing.
There are signs that the IPO market is beginning to thaw. Johnson & Johnson Inc is preparing to list its consumer health business Kenvue Inc in New York this Friday, hoping to raise about $3.5 billion.
Last week, SoftBank's ARM Ltd filed with regulators confidentially for a U.S. stock market listing, setting stage for largest IPO offering of the year.
U.S. IPOs, excluding listings for special purpose acquisition companies, is down about 22% to a total of just $2.35 billion year-to-date, according to Dealogic, as the volatility in stock market and economic uncertainty had put many IPO hopefuls off.
Following the completion of offering, Acelyrin will be listed on the Nasdaq with its shares trading under the ticker symbol "SLRN".
Morgan Stanley, Jefferies, TD Cowen and Piper Sandler are acting as joint book-running managers for the offering.
RadNet, Inc., (NASDAQ: RDNT) today announced that it has received FDA clearance for the third-generation release of its Quantib Prostate™ 3.0 MRI artificial intelligence (AI) software, further enhancing the offering with new and updated tools. Quantib Prostate™ software for MRI reading support was first introduced in the US, followed by a class IIb CE marking in Europe in 2021.
FDA cited concerns related to the manufacturing control strategy for variability of delivered dose in the TransCon PTH drug/device combination product
No new preclinical studies, or Phase 3 clinical trials to evaluate safety or efficacy, were requested in the letter
Ascendis will work collaboratively with the FDA to make TransCon PTH available to patients in the U.S. as quickly as possible
Conference call today at 8:30 am ET
Ascendis Pharma will host a conference call and webcast today at 8:30 am Eastern Time (ET).
Those who would like to participate may access the live webcast here, or register in advance for the teleconference here. The link to the live webcast will also be available on the Investors & News section of the Ascendis Pharma website at https://investors.ascendispharma.com. A replay of the webcast will be available on this section of our website shortly after conclusion of the event for 30 days.
Sanofi hasgambled against gene therapy before, and now it has done so again. The group today licensed Maze Therapeutics’ MZE001, an oral contender for Pompe disease.
Treatments for Pompe already exist in the form of enzyme replacement therapies, and here Sanofi dominates the market with Myozyme (also known as Lumizyme) and its newer drug Nexviazyme. But competition could be coming from the likes of Amicus’s AT-GAA and eventually gene therapies, a look at the pipeline shows.
The move for MZE001 therefore looks like a shoring up of Sanofi’s existing franchise. Maze reckons that its project, a glycogen synthase 1 (GYS1) inhibitor, could be used both as monotherapy and in combination with enzyme replacement.
Enzyme replacement replacement
Pompe disease is caused by mutations in the GAA gene, which encodes the enzyme acid alpha glucosidase. This enzyme usually breaks down glycogen, which builds up in Pompe patients, leading to muscle damage.
According to Maze, enzyme replacement is only partially effective in clearing glycogen. MZE001 inhibits GYS1 – the enzyme that controls glycogen production – and could address the “therapeutic gap”, the company says.
Sanofi apparently agrees. The deal looks to have been triggered by phase 1 data released this year, showing reductions in glycogen in healthy volunteers. Maze had planned to start a phase 2 trial this year; this will now be Sanofi's responsibility.
The French group is not making a big bet here, however. The collaboration is worth $150m in up-front cash and future equity, the companies said, without breaking this down. $600m in milestones are up for grabs under the agreement, which also covers Maze’s back-up GYS1 assets.
Gene therapies, offering the promise of a once-and-done treatment, could pose a big threat to both enzyme replacement and MZE001. But regulators have been cautious about such projects, particularly in diseases with existing treatments, given questions about durability and toxicity.
A more advanced candidate, Astellas’s AT845, was placed on clinical hold last June after a serious adverse event of peripheral sensory neuropathy. This was lifted in January, and in February Astellas reported initial data from four patients in the phase 1/2 Fortis trial. Three of the four have come off enzyme replacement, but three participants also developed transient transaminitis deemed possibly related to AT845. Astellas said these cases were resolved with changes to immune suppression.
A drug shortage in theUnited Statesaffecting Adderall, Ozempic and more than 300 total meds has no clear resolution,.government officials and academics say
Unexpected manufacturing problems, demand spikes, and tightened ingredient supply lines have all contributed to the shortages, which have subsequently caused treatment delays, medication switches and other hassles for doctors and patients.
Through the first three months of the quarter, there were 301 active national drug shortages, according to the University of Utah Drug Information Service. The figure is 49% higher than the 202 shortages recorded in the first three months of 2018.
While many of these prescription drug shortages have existed for years, their availabilities have been squeezed further by the pandemic as tightening supply lines were met with increased demand.
The U.S. Food and Drug Administration announced last year that Adderall, a medication used to treat Attention-Deficit/Hyperactivity Disorder (ADHD), was facing a shortage due to a manufacturing problem. Other stimulants that treat ADHD were similarly affected.
Shortages have also affected the availability of injectable drugs used in hospitals, such as IV saline and some cancer treatments as the pandemic spurred demand.
These drugs are more than twice as likely as tablets or topical treatments to experience a shortage, according to a recent report written by Sen. Gary Peters, D-Mich., chairman of the Senate Committee on Homeland Security and Governmental Affairs.
At the same time, the pandemic and nationally implemented health restrictions forced the closure of health offices and clinics. So, regulators allowed doctors to write prescriptions without first seeing a patient in person — further increasing demand.
For example, prescriptions for Adderall and its generic equivalents jumped at least 20% between February 2020 and the end of last year, according to IQVIA, while supplies were stretched thin.
Ozempic, a treatment for diabetes that is being prescribed for weight loss, is also facing a shortage as prescriptions have doubled since the summer of 2021, according to the health data firm IQVIA. It is widely praised by celebrities and social media personalities.
Pricing was also a contributing factor with some drugs as alternatives were too pricey to be considered by patients.
Ozempic is a diabetes drug. The same medication, semaglutide, which is sold under the brand name, Wegovy can be more than twice as much as Ozempic.
"They’ve been having a run on Ozempic because people don’t want to spend that much on Wegovy," said Stephen Schondelmeyer, a University of Minnesota College of Pharmacy professor.
Another contributing factor is the generally thin profitability of medications like Adderall and amoxicillin. Companies are not incentivized to make and store large amounts in case a shortage develops, according to University of Utah Health researcher Erin Fox.
"Once a shortage starts with something you make just in time anyway, it’s really hard to resolve it unless all the suppliers are back," she said.
Finally, as factories in China and India supply most of the raw materials used in American medicines, international supply lines being squeezed restricted their availabilities.
India, for example, restricted exports of 13 active pharmaceutical ingredients to protect its domestic drug supply early in the COVID-19 pandemic.
Companies have said they aren't getting enough raw materials to make the drugs, which could help ease the shortages. The federal government argues companies are not using the raw materials they do have.
"There's been a lot of finger-pointing back and forth," Fox said.
Joe Biden is a nostalgia president. He’s a link to the Obama era, of course.
But he’s also a living reminder of the days when Democrats were automatically the party of white ethnics, especially Irish Catholics.
For senior white voters in the Democratic coalition, the professed Catholic and Irish-ish Biden is an older, lesser Kennedy — but an heir to JFK nonetheless.
Only now Biden has to contend with a real Kennedy for next year’s Democratic presidential nomination.
Overall, the survey found some 70% of Americans think Biden shouldn’t run.
An octogenarian Joe Biden knows history bears a warning for him. The last Democratic president to be challenged for re-nomination was Jimmy Carter.
The challenger then, too, was a Kennedy.
Carter beat Ted Kennedy in the primaries only to lose the 1980 general election to Ronald Reagan.
Republican precedents are equally alarming.
A recent NBC News poll found that a majority of Democrats don’t wan’t President Biden to run in 2024.AP Photo/Carolyn Kaster
Pat Buchanan’s success marshaling GOP discontent against President George H.W. Bush in 1992 wasn’t sufficient to deny Bush the nomination. But it did portend his defeat by Bill Clinton that November.
Above all there’s the example of RFK Sr. His insurgent challenge to Lyndon Johnson in the 1968 Democratic primaries forced the president to withdraw from the race.
Only an assassin’s bullets stopped Bobby Kennedy from becoming the Democratic nominee that year.
He also holds the CIA culpable for the murder of his uncle in Dealey Plaza 60 years ago.
Robert Kennedy Jr.’s penchant for “conspiracy theories” leads Biden-friendly commentators and political strategists to dismiss him.
He threatens to spoil their myth that Republicans are the crazy party, whether or not he poses any risk to Biden.
But in fact conspiracy theories have as much of a home in the Democratic Party as in the GOP, if not more of one.
The difference is that Democratic conspiracy theories, such as those alleging Russian responsibility for the election of Donald Trump in 2016, often come with the imprimatur of prestigious media outlets.
RFK Jr., on the other hand, is a Democrat whose conspiratorial beliefs don’t dependably align with the elite media’s prejudices.
Kennedy supporters holding signs at his campaign launch event in Boston on April 19, 2023.Matthew McDermott
He’s long believed that vaccines contribute to autism. And he’s a fiery critic of Anthony Fauci and the response by government and the medical establishment to COVID-19.
Views like those are supposed to be the province of QAnon, not Democratic primary voters, according to the commentators who routinely burnish the party’s image — and tarnish the GOP’s.
But the guardians of the Democratic Party’s respectability are in for a rude awakening.
The Kennedy name, Biden’s weakness and the profound distrust millions of Americans feel toward institutions such as the CIA, the media and the pharmaceutical companies will make RFK Jr. formidable.
The fact thatMarianne Williamson, a longshot who shares many of Kennedy’s views but not his family fame, is polling at 8% to 9% in the Democratic race is further evidence that a reckoning is at hand.
Whatever the sordid realities of Camelot, for millions of baby boomers the Kennedys still represent a dream deferred.
JFK personally showed how an Irish Catholic could assimilate all the way to the top of America’s institutions, and if he could, then so could a kid of any background.
Irish Americans like the Kennedys could be proud of their heritage while also being proud Americans.
Raising up a John F. Kennedy didn’t mean tearing down a Thomas Jefferson.
JFK’s assassination only prolonged the dream.
The nightmare of the Lyndon Johnson years, of Vietnam and the triumph of the civil-rights movement turning into the tragedy of Martin Luther King’s murder and the burning of America’s cities, couldn’t taint the memory of the martyred hero.
RFK Sr.’s murder spared him, too, of responsibility for his party’s capture by the culturally radical left and the Democrats’ defeats by Richard Nixon.
The Kennedys are the enduring symbol of a liberalism, and a party, that died in the 1960s. It lives only in the nostalgia of an idealistic generation that’s now far from youthful.
When the baby boomers die, the Kennedy dream, of a Democratic Party that is inclusive yet proud of America’s past rather than ashamed of it, will also be over.
Biden, leader of a party whose liberalism is very different, hides behind the Kennedy dream. RFK Jr. takes that away.
And the bottomless sense of betrayal that animates Robert F. Kennedy Jr. is an epitaph for a generation.
Boomer Democrats loved the American dream. But they failed the real America.
Daniel McCarthy is the editor of Modern Age: A Conservative Review.
