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Monday, November 15, 2021

Catalyst Biosciences Down 40% After Announcing MarzAA Development Halt

 Catalyst Biosciences Inc (NASDAQ:CBIO) shares tumbled more than 40% after it told investors it has made a strategic decision to discontinue the development of MarzAA, an investigational therapy designed to prevent acute bleeds in hemophilia patients.

In addition, the company said it will seek a buyer for its hemophilia assets.

In the last couple hours of trading before the weekend, the company's shares are trading around $1.74.

"Based on several factors including a recently updated feasibility assessment, we determined that we cannot continue to develop MarzAA through completion of the ongoing trials," said Nassim Usman, CEO of Catalyst Biosciences.

"Enrollment in our MarzAA clinical trials has been adversely impacted by several factors, including pandemic-related logistical challenges, competition for subjects, and increasing availability of prophylaxis therapy globally. Given these factors, it is no longer feasible for us to deliver topline data in 2022," added Usman.

The decision by the company to halt the development of the therapy will allow it to reduce the current burn rate by 40%.

While it will report on the data obtained in the Crimson-1 trial for subcutaneous MarzAA, CBIO will now focus its attention on its complement therapeutics and protease platform.

Alongside the change in strategy, the company also reported its third quarter earnings on Friday, recording a loss of 80 cents a share on revenue of $2.3 million. Analysts predicted a loss of 66 cents a share on revenue of $675,000.

Cash, cash equivalents and, investments at the end of September were $64.5 million.

Friday's announcement prompted analysts to downgrade the stock and lower price targets. Raymond James downgraded CBIO to market perform from outperform, while JonesTrading downgraded it to Hold from Buy.

Piper Sandler lowered their price target on CBIO shares to $4 from $16.

https://finance.yahoo.com/news/catalyst-biosciences-down-40-announcing-145451974.html

Decibel Therapeutics started at Buy by Wainwright

 Target $23

https://finviz.com/quote.ashx?t=DBTX

Biden's spending plan could cost nearly $5T if made permanent

 President Biden has unveiled a framework for a modified social spending plan that imagines spending close to $1.75 trillion over the next decade, but budget experts say the true cost of the legislation would be closer to $5 trillion because of the way the programs are structured.

The newest "Build Back Better" proposal – pared down from the original $3.5 trillion request – would expand Medicaid, establish universal preschool, provide new funding for child care and offer green energy tax credits, though it notably omits progressive priorities like free community college and Medicare coverage of dental and vision. It relies on $1.95 trillion in new taxes, including a 15% corporate minimum and a surcharge aimed at ultra-millionaires.

But findings from the Committee for a Responsible Federal Budget, a nonpartisan group, released on Monday suggest Biden's plan could actually cost more than $4.9 trillion in a scenario where the spending and revenue provisions are made permanent, nearly doubling the size of the legislation.

The bulk of the programs and spending initiatives in the bill are poised to sunset in just a few years. But lawmakers often pass multiyear extensions down the road, even though the Congressional Budget Office assumes those measures will expire when it scores the legislation – a tactic that critics deride as "budget gimmicks."

"To be sure, lawmakers may choose not to extend some or all of these provisions. However, if they do, they would need to more than double current offsets in order for the bill and the extensions to be paid for," the group said. "The alternative would be a substantial increase in the debt."

Democrats have made it clear that by taking an across-the-board cuts approach to the spending package, they intend to force a future Congress to extend popular programs in a few years (whether or not they will be able to do so ultimately depends on the 2022 midterms and 2024 presidential election). 

For instance, in the plan rolled out last month, Biden proposed a temporary, one-year extension of the expanded child tax credit, which provides up to $3,600 per child to families. The White House estimated the temporary expansion would cost about $100 billion annually. But Democrats and the president have both pushed for a permanently expanded child tax credit, which would actually cost about $1.13 trillion.

It's not uncommon for lawmakers to take this approach: For instance, in 2017, Republicans passed the Tax Cuts and Jobs Act that included several income tax provisions slated to sunset within a few years – but lawmakers indicated at the time they had no intention of letting that happen. 

If Democrats made the legislation permanent without including additional offsets, the measure would increase the U.S. deficit by a stunning $1.5 trillion over the course of five years and by $3 trillion through 2031. As the proposal is written and currently stands, it would increase the deficit level by $800 billion over the first five years and a total of $200 billion through 2031 (in the second five years, it would actually reduce the deficit by $600 billion).

Sen. Joe Manchin, a moderate West Virginia Democrat who has become one of the most powerful votes in the upper chamber, has accused the Biden team of using "budget gimmicks" to conceal the true, higher cost of the president's signature economic spending plan. 

"As more of the real details outlined in the basic framework are released, what I see are shell games and budget gimmicks that make the real cost of this so-called ‘$1.75 trillion dollar’ bill estimated to be twice as high if the programs are extended or made permanent," Manchin said during a news conference. "That is a recipe for economic crisis."

If Biden succeeds in passing this proposal along with a $550 billion bipartisan infrastructure plan, Congress will have approved a staggering $5 trillion in spending in the less than one year since he took office, an unprecedented level. The nation's debt level is already at a historic high of $28 trillion and is on track to surpass $30 trillion.

https://www.foxbusiness.com/politics/biden-spending-plan-true-cost

Cassava probed by unnamed 'government agencies'

 Cassava Sciences likes to fight fire with fire, with CEO Remi Barbier calling a citizen petition to halt development on its lead drug a “short attack” in September. But the noise surrounding the saga has apparently drawn the attention of the US government.


The biotech reported in an SEC filing Monday that “certain government agencies” have asked Cassava to provide them with “corporate information and documents.” It’s not clear which agencies are involved in probing Cassava, or if multiple agencies are doing so, but the company says it’s cooperating.


Though Cassava reported that the government said it had not informed the biotech that any wrongdoing had occurred, the company left the door open to potential penalties.


“We cannot predict the outcome or impact of any [of] these ongoing matters, including whether a government agency may pursue an enforcement action against us or others,” Cassava wrote in the filing.


Cassava was originally accused by the law firm Labaton Sucharow in August of data manipulation, with the shareholder rights activists petitioning the FDA to stop development of the Alzheimer’s drug simufilam. The biotech fought back with Barbier’s comments as well as an unusual Q-and-A style press release put out around that time.

https://endpts.com/cassava-being-investigated-by-unnamed-government-agencies-rocket-pharma-gives-updated-data-on-danon-disease/

Rocket Pharma reports updated PhI data for Danon disease

 New Jersey biotech Rocket Pharmaceuticals has been working on an investigational gene therapy for gene disorder Danon disease — and the pharma announced updated data from the Phase I trial today.


The trial was looking at a single IV infusion of gene therapy RP-A501 at two different dosages in five patients — “low-dose” with three patients (6.7e13 vg/kg) and “high-dose” with the other two (1.1e14 vg/kg). While the gene therapy was well-tolerated at the low dose and conferred sustained clinical benefit, the same can’t be said for the high dose.


As previously made public, dose-dependent toxicity was seen in one of the two patients on the high dose regimen — and developed thrombotic microangiopathy that fully resolved with treatment, which included transient hemodialysis.


As such, the biotech will be cutting off the higher dosage and sticking with the lower amount moving forward.


“We are excited to announce positive data from our RP-A501 trial for Danon Disease showing clinical, functional and biomarker improvements at one year or beyond and potential early separation from the natural course of disease in adult and adolescent patients,” said Rocket CEO Gaurav Shah. “We have initiated treatment in the pediatric cohort at the low-dose and anticipate moving as rapidly as possible toward Phase II.”

https://endpts.com/cassava-being-investigated-by-unnamed-government-agencies-rocket-pharma-gives-updated-data-on-danon-disease/

Vaxxinity, hoping to turn bodies into their own drug factories, makes Wall Street debut

 Immunotherapeutic vaccine developer Vaxxinity, with an eye on COVID and some serious CNS diseases, has pulled off a fairly small IPO.

The Dallas-based company (with bases around the world), created back in the spring when COVAXX and United Neuroscience came together under the new biotech, had been gunning for a $101 million offering, looking to sell nearly 7 million shares at a price range between $14 and $16.

But today it priced at just $13, below its original range, selling 6 million shares and earning $78 million. The company will trade on the Nasdaq under the "VAXX" symbol.

The company, tapping tech from its two parents, is working on a synthetic peptide vaccine platform—aka the Vaxxine Platform—which it said in a recent Securities and Exchange Commission filing has the potential to enable a new class of medicines.  

Its platform is set up to harness the immune system to convert the body into its own "drug factory," stimulating the production of antibodies with a therapeutic or protective effect.

It has a of host early- to midstage shots on goal, including for Alzheimer's disease, Parkinson's disease, migraine and hypercholesterolemia.

Its leading candidate, UB-311, targets amyloid to try to beat Alzheimer's, with a phase 2b early efficacy trial slated for next year.

It’s also working on a COVID-19 prevention program, UB-612, designed to activate both B- and T-cell arms of the immune system to fight against SARS-CoV-2; the program is in several phases of development around the world.

Both of these are high-risk, high-reward plays, and Vaxxinity still has a long road ahead.

https://www.fiercebiotech.com/biotech/vaxxinity-hoping-to-turn-bodies-into-their-own-drug-factories-makes-a-subdued-wall-street

Protagonist posts phase 2 hormone replacement data, eying lead in genetic iron overload disease

 Protagonist Therapeutics wants to take the lead in a genetic iron disease, delivering midphase data on a hormone replacement therapy it envisages offering an alternative to blood draws.

Rusfertide is Protagonist’s attempt to improve on the druglike properties of the hormone hepcidin to alter the regulation of iron homeostasis. In people with hereditary hemochromatosis, who absorb too much iron from food, the mechanism of action could enable physicians to stop excessive iron accumulation—and the negative outcomes it causes—without relying solely on blood draws.

The 16 subjects in the phase 2 clinical trial self-administered a subcutaneous dose of rusfertide once or twice a week, with physicians adjusting the dose based on disease biomarkers over the course of the six-month study.

Average serum iron and the ratio of serum iron to total iron-binding capacity, the primary endpoints of the study, fell from baseline, with the p-values coming in at 0.0106 and 0.0051, respectively. Drops in serum iron and the transferrin saturation ratio were seen 24 hours after the first rusfertide dose.

The improvements correlated to less need for the blood draws, known medically as phlebotomies, that are the main treatment for the disease today. Participants were averaging 0.28 phlebotomies per month pre-study. That fell to 0.009 phlebotomies per month during the study. 

“Rusfertide has the potential to offer a treatment option for patients in whom phlebotomy may be difficult or contraindicated,” Kris Kowdley, M.D., the Washington State University physician who served as the study’s principal investigator, said in a statement. 

Most participants experienced a treatment-emergent adverse event, but the only report higher than Grade 2 was a preexisting case of pancreatic cancer deemed unrelated to the drug. Around half of the subjects suffered mild or moderate treatment-emergent injection site reactions.

Protagonist now plans to talk to clinical investigators and regulatory agencies to determine the next steps.

https://www.fiercebiotech.com/biotech/protagonist-posts-phase-2-hormone-replacement-data-pitching-for-lead-role-genetic-iron