Shares of Revance Therapeutics Inc. (RVNC) gained 8% premarket on Monday after the U.S. Food and Drug Administration approved the biotech company's Daxxify injection for treatment of cervical dystonia in adults. Cervical dystonia is a chronic disease that causes painful, involuntary neck muscle contractions and affects about 60,000 people in the U.S. Daxxify, a Botox competitor, previously received FDA approval late last year for the temporary improvement of frown lines. The approval announced Monday is the injection's first therapeutic indication. Revance shares have gained 2.4% in the year to date, while the S&P 500 is up 16%.
Monday, August 14, 2023
China Abandons Clean Energy Goals Making U.S. Efforts Painful and Pointless
Bidenomics and the EPA have America on a path of inflationary and environmental madness that’s all pain and no gain.
Painful and Pointless
Please consider the Heritage Foundation article China Abandons Paris Agreement, Making U.S. Efforts Painful and Pointless
Three Key Takeaways
- China has repeatedly stated that it has no intention of going along with the Western push to net-zero.
- EVs are not emissions-free, because they need electricity to charge them, and electricity generation creates emissions.
- All these costs will result in no reduction in global emissions. The EPA has America on a path to all pain and no gain.
It was a bad week for anyone who thought China would cooperate on emissions reduction. President Xi Jinping reiterated that his country would set its own path on the issue and not be influenced by outside factors, according to the Washington Post and Bloomberg. This contradicts Xi’s 2015 Paris Agreement pledges to reduce its carbon emissions at the latest after 2030.
This should not be news, because Xi gave the same message last fall. In October 2022, he said that China would not abandon coal-fired power plants before renewables could substitute for the lost fossil fuel.
In April, the EPA released a proposed tailpipe rule that would require 60 percent of new vehicle sales to be battery-powered electric by 2030, and two-thirds by 2032. And in May, the EPA proposed a power-plant rule that would require most power plants to sequester, or bury, 90 percent of their carbon emissions, or go out of business by 2040.
These rules would result in tens of billions of dollars in annual costs to the U.S. economy—and with no reduction to global emissions, if China replaces U.S. emissions with its own emissions.
EVs are not emissions-free, because they need electricity to charge them, and electricity generation creates emissions. Even the EPA states in the proposed rule that “we expect that in some areas, increased electricity generation would increase ambient SO2, PM 2.5, ozone, or some air toxics.”
The power-plant rule would raise the cost of electricity just as the EPA plans to have millions of new EVs access the grid. Sequestering 90 percent of carbon emissions on such a large scale has never been done before, and it is not an “adequately demonstrated” technology. The only proven option for a power plant to comply with the proposed regulation is to close down.
The rule would remove power from the grid at a time when America needs more power for planned electrification, and it would likely cause more blackouts. Blackouts can have serious consequences, including death, especially if they occur during periods of unusually high or low temperatures when power is most needed.
In addition, higher costs of electricity will have adverse economic effects. Prices will rise, manufacturing will go offshore, and layoffs and unemployment will increase. All this will lower GDP growth and reduce Americans’ standard of living.
The EPA has America on a path to all pain and no gain.
Second Thoughts in the EU
Because of rising costs to achieve climate goals, the EU is having second thoughts .
Also, support for the Green party in Germany is crumbling and support for Marine Le Pen is rising in France.
Behold, the Rise of the Anti-Greens
Please consider mu July 24, 2023 post Behold, the Rise of the Anti-Greens
A major revolt is underway in the EU. Citizens have finally had enough of Green nonsense. The latest polls provide all the evidence you need.
The German AfD party is now polling 22 percent ahead of every party other than Union (CDU/CSU).
None of this should be surprising. The costs of the EU’s climate change mandate are soaring and people have had enough of it.
Electric Vehicles for Everyone?
On July 19, I asked Electric Vehicles for Everyone? If the Dream Was Met, Would it Help the Environment?
My follow-up post was What Do MishTalk Readers Think About “Electric Vehicles for Everyone?”
Math Does Not Add Up
The EV math does not add up in the EU or here. But the Economic and Monetary Union (EMU), better known as the Eurozone, has economic debt brakes and budget rules that make matters more painful for the 20 EMU countries.
In the US, deficits pile up as do the economic impacts of a massive wave of Bidenomic regulations and mandates.
We pretend that deficits don’t matter and mainstream media not only looks the other way, but is in on the act with countless fearmongering stories.
As a direct consequence, the US is riding a huge wave of inflationary and environmental madness. The only way to stop it is for Republicans to oust Biden in the next presidential election.
A reader ignorantly commented “My Tesla S can easily over a hundred miles per gallon equivalent. As utilities get cleaner so does my car. ”
I replied: Well la de da.
Where did the minerals come from for your battery? At what cost? At what cost if everyone stupidly did the same?
At what environmental cost to extract the minerals.
At what cost to build the infrastructure so everyone can plug in?
No one has ever scaled EVs to estimate the mining costs and infrastructure costs if everyone did the same thing.
It’s like all these free money experiments of giving people money to see if it makes their lives better.
No one has ever scaled EVs to estimate the mining costs and infrastructure costs if everyone did the same thing.
It’s like all these free money experiments of giving people money to see if it makes their lives better.
Zuckerberg Supports Universal Basic Income
For discussion, please see Zuckerberg Supports Universal Basic Income
Your Flaw Is Your Strength
While writing my next book, I came across a thought-provoking essay from a well-known Jewish Rabbi, Zalman Schachter-Shalomi. His topic was the opal stone that we see in jewelry.
The opal gem consists of small silica spheres and gaps between the spheres, which in one context could be considered flaws. However, the very gaps that occur on the opal's surface are what create the opal's beauty when light is shined. The light rays are diffracted by the spheres and gaps, which break the light into a fiery array of component colors. The Rabbi's point is that we are like opals: what are our flaws when viewed one way become our greatest sources of beauty in a different light.
Our flaws, in the right light, are our strengths.
For example, in one light, our ambition and achievement orientation are flaws, leading us to become so wrapped up in our work that we neglect our health and relationships. Those flaws lead us to trade from the ego, overreact to gains and losses, and chop ourselves up when big moves aren't realized.
In a different light, our ambition and achievement orientation lead us to define and seek goals and ideals and realize our vision for being the best possible version of ourselves.
There are so many ways in which our flaws and strengths mirror one another. Think about how our sensitivity in relationships can lead to caring, but also hurt and disagreements. Think about how our desire for self-development can result in personal growth--and in an insensitivity to others. Think about how our commitment to not losing money can stand in the way of making significant money.
We are like opals, and our challenge is to find the light that enables us to shine.
Here's a simple exercise to help with that challenge: Each week, identify the one most fulfilling, meaningful event that occurred to you during the past seven days. Then identify the one most frustrating, negative event over that same period. Then reflect on how the two are related and what made the positive experience so special and what made the negative experience so disappointing. What is the light in which you are simply a stone with spheres and gaps, and what is the light in which you shine? Over time, keeping this simple journal, you'll discover the contexts in which you display your fire.
The goal is not to eliminate your flaws, but to turn those into sources of beauty.
https://traderfeed.blogspot.com/2023/08/your-flaw-is-your-strength.html
BioXcel pivots focus to agitation drug
BioXcel Therapeutics Inc said on Monday it will reduce its workforce by more than 50% to 80 employees from about 190, as the drugmaker shifts its focus towards developing its agitation treatment.
Shares of BioXcel fell about 36% to $4.75 in premarket trading.
The company said it will prioritize the development of its drug BXCL501, which is being evaluated for use in at-home settings for treatment of agitation related to schizophrenia, bipolar disorders and in patients with mild-to-moderate dementia due to probable Alzheimer's disease.
In June, the company had flagged issues related to the recording of safety data at a site where a late-stage trial of the drug for treatment of agitation in Alzheimer's patients was being conducted.
BioXcel, in a conference call on Monday, said it has initiated an external audit for the issue and expects an update by the end of the year.
The drug, sold under the brand name Igalmi in the U.S., is already approved for use in hospital settings to treat agitation in adult patients with schizophrenia or bipolar disorder.
The company plans to reduce in-hospital commercialization expenses, and suspend trials that it no longer deems core to its business. With the job cuts, BioXcel aims to reduce more than 50% of its cash burn to about $80 million on a go-forward annualized basis. Its cash and cash equivalents totaled to $127.5 million as of June 30, compared with $165.5 million as of March 31.
In the absence of additional funding from its existing agreements with Oaktree Fund Administration and Qatar Investment Authority, the company said it expects the current cash to last through mid-2024, compared with its previous forecast of 2025.
https://finance.yahoo.com/news/1-bioxcel-announces-job-cuts-112139034.html
Taysha: Initial Clinical Data from First Adult Rett Syndrome Phase 1/2 Trial
Data from first adult patient dosed in REVEAL Phase 1/2 trial showed TSHA-102 was well-tolerated with no treatment-emergent serious adverse events (SAEs) as of six-week assessment and improvement in key efficacy measures, including Clinical Global Impression – Improvement (CGI-I), Clinical Global Impression – Severity (CGI-S) and Rett Syndrome Behavior Questionnaire (RSBQ), four weeks post-treatment
Principal Investigator (PI) observed clinical improvement in multiple domains, including autonomic function (sleep and breathing), vocalization, as well as gross motor skills (gained ability to sit unassisted for three minutes) and fine motor skills (gained ability to hold objects), supported by initial clinical data and video evidence
United States (U.S.) Food and Drug Administration (FDA) cleared the Investigational New Drug (IND) application for TSHA-102 in pediatric patients with Rett syndrome
Clinical Trial Application (CTA) submitted to the United Kingdom (U.K.) Medicines and Healthcare products Regulatory Agency (MHRA) for TSHA-102 in pediatric patients with Rett syndrome
Private placement financing (“PIPE”) is expected to result in gross proceeds of approximately $150 million from new and existing investors and, net proceeds from PIPE, along with existing cash and cash equivalents, extends cash runway into the third quarter of 2025
Conference call and live webcast today at 8:30 AM Eastern Time
Taysha management will hold a conference call and webcast today at 8:30 a.m. ET to review its financial and operating results and to provide a corporate update. The dial-in number for the conference call is 877-407-0792 (U.S./Canada) or 201-689-8263 (international). The conference ID for all callers is 13740092. The live webcast can be accessed here: https://viavid.webcasts.com/starthere.jsp?ei=1624983&tp_key=25b742b70a. An archived version of the webcast will be available for 30 days and can be accessed by visiting Taysha’s website at https://ir.tayshagtx.com/news-events/events-presentations.
https://finance.yahoo.com/news/taysha-gene-therapies-reports-initial-110200128.html
HARMONY BIOSCIENCES TO ACQUIRE ZYNERBA
Acquisition expands pipeline and diversifies portfolio to drive long-term growth
Innovative potential new therapeutic option for rare/orphan neuropsychiatric disorders with high unmet medical needs
Lead asset in pivotal Phase 3 trial for Fragile X syndrome and has completed Phase 2 proof-of-concept study in 22q11.2 deletion syndrome
Conference call and webcast to be held today at 8:30 AM ET
At 8:30 AM ET Harmony will host a live webcast to review this proposed acquisition. The live and replay webcast of the call will be available on the investor relations page of our website at https://ir.harmonybiosciences.com/. To participate in the live call by phone, dial (800) 245-3047 (domestic) or +1 (203) 518-9765 (international), and reference passcode HRMY0814.
https://finance.yahoo.com/news/harmony-biosciences-acquire-zynerba-pharmaceuticals-113000390.html
Sunday, August 13, 2023
FDA Action Alert: Ipsen, Revance, Neurocrine and Regeneron
This week, the FDA is due to decide on four therapeutic applications, including one for Huntington’s disease and two for ultra-rare diseases.
Is the Third Time the Charm for Ipsen’s Palovarotene?
After stumbling in the past, Ipsen Biopharmaceuticals is once again awaiting an FDA verdict for palovarotene, which it is proposing for the treatment of the ultra-rare disease fibrodysplasia ossificans progressiva (FOP). The regulator’s deadline is Aug. 16.
FOP is a genetic disorder that arises when muscle and connective tissues are gradually replaced by bone. This extra-skeletal formation of bone hinders patients’ movements and, when the joints become afflicted, may ultimately lead to the loss of mobility. FOP patients also suffer from difficulties in speaking and eating, along with breathing problems.
Palovarotene is an oral agonist of the retinoic acid receptor gamma, which plays a crucial role in the regulation of skeletal development. By mediating the interactions between the receptor and its ligands, as well as other growth factors and proteins involved in the retinoid signaling pathway, palovarotene can decrease the formation of abnormal bone.
Ipsen has put its candidate in front of the FDA twice before. The first time was in 2021, but the company voluntarily withdrew its application in August of the same year after the regulator indicated the need for more data during a meeting. Ipsen filed a resubmission in 2022, which the FDA rejected in December.
In its third filing, Ipsen provided data from a Phase II study with several open-label extension phases and a single-arm, open-label Phase III trial. Palovarotene failed this late-stage study, though the company maintains this was due to statistical discrepancies, as well as the biases that come with using historical controls. An advisory committee in June 2023 took Ipsen’s side and voted 11–3 that palovarotene’s benefits outweighed its risks.
Revance Seeks Label Expansion for Daxxify in Cervical Dystonia
On Aug. 19, the FDA is set to decide on Revance Therapeutics’ supplemental BLA proposing Daxxify (daxibotulinumtoxinA-lanm) for the treatment of cervical dystonia in adults.
Daxxify, a neuromuscular blocking agent and acetylcholine release inhibitor, was approved in September 2022 for the temporary improvement of moderate to severe frown lines in adults. Commonly positioned as an alternative to Allergan’s (AbbVie) Botox, Daxxify is a peptide-formulated injection that has durable treatment effects.
In its sBLA, which the FDA accepted in January, Revance included data from the Phase III ASPEN clinical development program, which includes the ASPEN-1 and ASPEN-OLS trials. In ASPEN-1, a pivotal late-stage study, both 125U and 250U doses of Daxxify were effective and generally well-tolerated in cervical dystonia patients and even reduced the frequency of treatments by up to 50% annually.
Meanwhile, ASPEN-OLS validated the long-term safety and efficacy of Daxxify in this indication for up to four consecutive treatments.
In February, Revance presented new data for Daxxify at the 2023 Association of Academic Physiatrists annual meeting, further supporting the therapeutic value of the injection. Across 88 weeks of follow-up, corresponding to up to five injection cycles and a total of 1,240 treatments, Daxxify remained safe and effective in cervical dystonia patients.
Neurocrine Proposes Ingrezza for Chorea in Huntington’s
Neurocrine Biosciences is proposing to expand Ingrezza’s (valbenazine) label to include chorea in Huntington’s disease. The FDA accepted the company’s supplemental NDA in December 2022 and set a target action date of August 20.
Approved in 2017 to treat tardive dyskinesia, Ingrezza is a selective vesicular monoamine transporter 2 inhibitor. In its sNDA, Neurocrine included data from the Phase III KINECT-HD trial and the ongoing KINECT-HD2 open-label study to establish the safety and efficacy of Ingrezza in this proposed indication.
In April 2022, the company presented results from KINECT-HD at the American Academy of Neurology 2022 annual meeting and followed this up with a Lancet Neurology publication in May. Data from the late-stage trial showed that Ingrezza treatment significantly reduced scores in the Unified Huntington's Disease Rating Scale Total Maximal Chorea score by 3.2 units versus placebo, an effect that was statistically significant.
Ingrezza likewise outperformed placebo on secondary endpoints, including the Clinical Global Impression of Change and Patient Global Impression of Change response status.
Huntington’s disease is a hereditary, progressive neurodegenerative disease characterized by the gradual destruction of neurons, leading to motor and cognitive problems, as well as psychiatric symptoms.
A common symptom of the condition is chorea, which refers to abnormal, unpredictable and involuntary movements affecting various parts of the body. Chorea can interfere with movement and posture, as speech and swallowing.
Regeneron Awaits Verdict for Pozelimab in CHAPLE Disease
The FDA’s last scheduled decision date this week is for Regeneron’s investigational antibody pozelimab, which the company is developing to treat the ultra-rare CHAPLE disease. The regulator is scheduled to release its verdict by Aug. 20.
CHAPLE disease, also known as CD55 deficiency with hyperactivation of complement, angiopathic thrombosis and protein losing enteropathy, is a life-threatening immune system disorder characterized by the over-activation of the complement system. In turn, the hyperactive immune system also targets healthy cells, ultimately damaging blood and lymph vessels, as well as the digestive tract.
Most patients with CHAPLE disease suffer from a wide variety of life-threatening symptoms, such as bloody diarrhea, malnutrition and recurrent infections. There are no known cures for CHAPLE.
Pozelimab, a fully human monoclonal antibody, works by binding to the C5 complement factor, thereby disrupting the complement cascade and preventing associated diseases.
Regeneron backed pozelimab’s regulatory bid with data from a Phase II/III open-label study, which showed that at 24 weeks, all 10 treated patients achieved “rapid and sustained normalization” of albumin, a key marker of CHAPLE disease activity. Pozelimab treatment also eased clinical manifestations, including abdominal pain and number of daily bowel movements.
The FDA accepted pozelimab’s application in February and granted it Priority Review. If approved, Regeneron’s antibody would become the first treatment authorized for use in CHAPLE disease.
https://www.biospace.com/article/fda-action-alert-ipsen-revance-neurocrine-and-regeneron/
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