Iveric Gets FDA Approval in Geographic Atrophy After Being Acquired by Astellas

 The FDA on Friday approved Iveric Bio’s intravitreal avacincaptad pegol, now to be marketed as Izervay, for the treatment of geographic atrophy secondary to age-related macular degeneration.

The regulatory win comes three months after the New Jersey biotech was bought by Astellas Pharma for $5.9 billion. The companies completed the acquisition last month.

Friday’s approval makes Izervay the first authorized geographic atrophy (GA) treatment that has significantly slowed down GA progression at 12 months across two Phase III studies, according to Astellas’ announcement. The eye injection also offers a new treatment option to physicians and patients across the U.S., Iveric Bio President Pravin Dugel said in a statement.

Discovered and developed by Iveric, Izervay is an intravitreally administered inhibitor of the complement C5 protein. By blocking the cleavage of C5, the eye injection also disrupts more downstream processes, including the formation of the membrane attack complex and inflammasome, which in turn gives rise to the hallmark symptoms of GA.

Iveric tested the therapeutic potential of Izervay’s mechanism of action in the Phase III GATHER1 and GATHER2 trials. GATHER1, a prospective, randomized and sham-controlled pivotal Phase II/III trial, showed that at the 2-mg dose level Izervay slowed GA growth by 27.4% over 12 months, while the 4-mg dose was slightly more effective at 27.8%. Both dose levels were statistically superior than sham.

The company followed this data up in September 2022, announcing that topline data from the Phase III GATHER2 study had met its prespecified primary endpoint. At 12 months, patients treated with Izervay also saw significantly slower growth in GA area, as compared with sham controls.

In both studies, Izervay also had a tolerable safety profile. Across the GATHER program, bleeding beneath the clear lining of the eye was the most common side effect observed in patients treated with Izervay, followed by increased intraocular pressure and blurred vision.

Izervay’s label reflects this safety profile and carries precautions against retinal detachment, an increase in intraocular pressure, neovascular age-related macular degeneration (AMD) and eye infections. Izervay does not have come with a boxed warning.

Astellas and Iveric expect Izervay to be available in two to four weeks, hitting U.S. shelves approximately six months after its main competitor, Apellis’ Syfovre (pegcetacoplan), was approved by the FDA.

Both treatments are intravitreally administered and work by disrupting the complement cascade to slow lesion growth. Neither, however, have yet been shown to elicit significant benefits on visual function.

https://www.biospace.com/article/iveric-gets-fda-approval-in-geographic-atrophy-months-after-being-acquired-by-astellas/

Why Quantum-Si Stock Crashed

 

Cathie Wood fave Quantum-Si stock could be as much as six years away from profitability.

Shares of protein sequencing company Quantum-Si (QSI -31.77%) collapsed on Monday after the company reported its financial results for Q2 2023, falling 35.2% through 12:10 p.m. ET.

A recent favorite of tech maven Cathie Wood, who has snapped up more than 3.4 million shares over the last month and a half, Wall Street analysts had pegged Quantum-Si stock for a $400,000 revenue quarter, and a $0.20 per share loss. As it turned out, Quantum-Si's GAAP loss for the quarter was only $0.18 per share, an apparent beat. Actual revenue for the quarter, however, was only half what was expected -- $205,000 total.   

That's apparently not the number that growth investors wanted to hear.

Management claimed to have "made great strides in the build out of our commercial team and have received valuable feedback from customers as we continue our thoughtful and controlled commercialization of Platinum." (Platinum is Quantum-Si's single-molecule protein sequencing platform, comprising sequencing hardware, consumable sequencing kits, and software.) And management promised "additional product improvements before the end of 2023."  

But investors wanted to see a more rapid uptake of Quantum-Si's product. Instead, what they got was a sequential slowdown in sales, as Q2's revenue actually declined 19% from the $254,000 in sales booked in Q1 -- and losses inched 6% higher.  

Quantum-Si didn't give much solace about the future, either, giving no guidance on full-year revenue or profits, and stating only that it doesn't expect non-GAAP operating expenses to increase in comparison to last year.

On the plus side, that means that if revenue increases, and gross margin holds steady at its present 38% level, then Quantum-Si should move closer to profitability as the year progresses -- but not much closer.

Consider: Quantum-Si's operating costs last year were $114 million. At a 38% gross profit margin on revenue, this means Quantum-Si stock won't be able to break even until it reaches at least $300 million in annual revenue -- a number that analysts don't see happening before 2029 at the earliest.

For Quantum-Si investors, there's still a very long road ahead before anything resembling profitability can be expected.

https://www.fool.com/investing/2023/08/07/why-quantum-si-stock-crashed-35-today/

Why Sage Therapeutics Stock Is Crashing

 

Sage's regulatory win wasn't enough to offset an even bigger regulatory loss.

Shares of Sage Therapeutics (SAGE -53.45%) were crashing 51.2% lower as of 11:27 a.m. ET on Monday. The huge sell-off came after the company and its partner, Biogen, announced following the market close on Friday that the U.S. Food and Drug Administration (FDA) didn't approve Zurzuvae (zuranolone) for major depressive disorder (MDD).

It wasn't all bad news for Sage, though. The FDA did approve Zurzuvae for treating postpartum depression (PPD). However, the company was betting on winning approval in the MDD indication as well.

The big problem for Sage -- and the main reason the biotech stock is plunging -- is that Zurzuvae will have a much smaller market size to target now. Millions of Americans suffer from MDD compared to hundreds of thousands of women who experience PPD.

The FDA stated in its Complete Response Letter (CRL) to Sage and Biogen that it didn't see enough evidence of effectiveness in the MDD indication. As a result, the agency wants another clinical study to be conducted.

Sage and Biogen are in the process of reviewing the FDA's feedback in the CRL to determine their next steps. The two partners expect to launch Zurzuvae in the PPD indication in the fourth quarter of 2023. The drug must first be scheduled as a controlled substance by the U.S. Drug Enforcement Administration, though.

With the more lucrative MDD market not in the cards for now, Sage plans to take steps to stretch out its cash. The company reported $1 billion of cash, cash equivalents, and marketable securities as of June 30. However, it posted a loss of $160.3 million in the second quarter. Sage Therapeutics CEO Barry Greene said that the company is "evaluating resource allocation, including pipeline prioritization and a workforce reorganization with a goal of extending our cash runway."

https://www.fool.com/investing/2023/08/07/why-sage-therapeutics-stock-is-crashing-today/

Vistagen Surges on Social Anxiety Spray Trial Success

A beaten down micro-cap biotech company, Vistagen Therapeutics Inc., is having its best day ever, surging north of four digits after its experimental nasal spray for social anxiety disorder hit targets in a late-stage trial.

Shares of the drug developer surged by a record 1,370% on Monday, sending the stock to its highest level in more than a year. The unexpected success pushed the stock’s market value to above $130 million, a level it had been trading below for the better part of a year amid a broad market selloff in risky assets and a prior trial setback.

Vistagen said the study of fasedienol nasal spray met its main goal, showing improvements on patients’ reported distress in a public speaking challenge while a secondary goal was also met with no severe or serious adverse events reported. The stock had been setting record lows this year after a previous late-stage trial of the drug failed to reach its main goal using the same scale measuring patient distress, dubbed SUDS, last year.

“Fasedienol demonstrated a rapid and very clinically meaningful reduction in SUDS score, indicating a single administration has the potential to reduce anxiety symptoms during an anxiety-provoking situation,” Michael Liebowitz, a founder of the Anxiety Disorders Clinic at the New York State Psychiatric Institute and a member of Vistagen’s advisory board, said in a statement.

An additional late-stage study is planned. That will include multiple administrations of the spray over several weeks on what the company said will be “a patient-tailored, as-needed basis.”

The stock had four analysts covering it with a buy rating as of last July, before the stock plunged. Now, the lone analyst tracked by Bloomberg that covers the stock rates it a hold

https://finance.yahoo.com/news/biotech-firm-vistagen-surges-1-175147101.html

Why Shares of Tabula Rasa HealthCare Are Soaring

 

The company announced it was entering into a merger agreement to be acquired by Nautic Partners and will combine with ExactCare Pharmacy, a portfolio company of Nautic.

What happened

Shares of Tabula Rasa HealthCare (TRHC 30.65%) were up more than 31% at 10:55 a.m. on Monday after the company announced that it had entered into a definitive merger agreement to be acquired by the private equity firm Nautic Partners for $10.50 in cash per share and will combine with ExactCare Pharmacy, a portfolio company of Nautic. The stock reached a 52-week high on Monday and is up more than 108% so far this year.

Tabula Rasa is a healthcare company that focuses on managed care for value-based care organizations. Basically, the company uses its platform to help healthcare organizations improve medication regimens for better patient outcomes, lower healthcare costs and lower risk. The combination with ExactCare is viewed as complementary and should provide some synergy as the company looks to increase its client base.

The deal will pay shareholders a 34% premium on the stock's closing price from Aug. 4. The all-cash deal is worth roughly $570 million, including debt of around $262 million. The transaction is expected to close by the fourth quarter.

It's an easy win for current investors in Tabula Rasa. The company has been growing revenue, but has not been profitable.

Tabula Rasa also reported second-quarter earnings before the markets opened on Monday. The company said it had revenue of $90 million, up 24%, year over year, and 2% sequentially. It also reported a net loss of $9.7 million, compared to a $12.7 million loss in the same period a year ago. Due to the impending merger, Tabula Rasa said it wouldn't provide guidance for the remainder of the year.

https://www.fool.com/investing/2023/08/07/why-shares-of-tabula-rasa-healthcare-are-soaring-m

Nektar sues Eli Lilly over autoimmune disease treatment

 Nektar Therapeutics on Monday sued Eli Lilly, accusing the drugmaker of undermining the prospects for Rezpeg, which Nektar was developing as a treatment for various autoimmune diseases.

The complaint filed in San Francisco federal court accuses Lilly of breach of contract, negligent misrepresentation, unfair competition and other wrongdoing. It seeks compensatory and punitive damages, among other remedies.

https://finance.yahoo.com/news/nektar-therapeutics-sues-eli-lilly-142336423.html

Controversial Eyeball-Scanning Worldcoin To Allow Governments To Use Its Digital ID System

  by Christina Maas via ReclaimTheNet.org,

Allowing state actors to use the dystopian technology...

OpenAI CEO Sam Altman’s Worldcoin is a good example of private companies doing their bit to push and introduce digital ID schemes to as many people as possible – although this effort is usually done by governments, and supported by various lobbies.

And now, Worldcoin has announced that it will be even more helpful to governments, by allowing them to use the system of biometric scanning it employs to sign users up. Other companies will be given the same privilege.

The intention is clearly to get as many people as possible on board, hence the “generosity” with sharing the iris scanning tech, as well as that designed to verify people’s identity.

And it’s no secret: “We are on this mission of building the biggest financial and identity community that we can,” is how Tools for Humanity (a company behind Worldcoin) executive Ricardo Macieira put it.

The mission marches on despite concerns not only from privacy focused non-profits and advocates, but also institutions in various countries that are tasked with protecting data privacy.

People – and the number mentioned in reports these days is 2.2 million so far – sign up to Worldcoin by giving up biometric data contained in their eyes, i.e., irises.

What they get in return is a digital ID, and citizens of some countries are incentivized to do this by being given some free crypto, too.

And, this last point seems to play a major role in why anyone would sign up for this – Reuters said that a majority of those the agency spoke to in the UK, India and Japan said they were doing it to get the free tokens.

But if you listen to what Macieira has to say, Worldcoin is clearly eager to build an image for itself of much loftier goals – not to mention ones that can turn controversial, and fast.

These are some ways Worldcoin can be used, as mentioned on its website: providing a way to tell human from artificial intelligence, possibly paving the way for universal basic income – but also, “enabling global democratic processes.”

Worldcoin currently targets countries in Africa, Latin America and Europe for growing adoption, and doesn’t seem to have much problem in raising capital, either, with a $115 million round in May alone.

https://www.zerohedge.com/technology/controversial-eyeball-scanning-worldcoin-allow-governments-use-its-digital-id-system