Cyklokapron fda approval

https://www.nbcnews.com/politics/joe-biden/biden-administration-plans-reclassify-marijuana-easing-restrictions-na-rcna149424
This is big for the psychedelics industry. It sets precedent and a framework for reclassifying LSD and MDMA in the future, if and when full FDA approval is granted.

This question was brought to mind by the FTC settling with Razer for allegedly making misleading claims about their Zephyr RGB face masks during the peak of the COVID-19 pandemic. (Yes, that Razer.)

The sellers of a supposed N95-grade face mask called the Zephyr will pay more than $1.1 million to provide full refunds to consumers nationwide, as well as a civil penalty, under a proposed settlement the Federal Trade Commission announced today. The order settling the complaint also bars Razer, Inc., from making COVID-related health misrepresentations or unsubstantiated health claims about protective health equipment and requires them to pay a civil penalty of $100,000. [1]

TLDR they weasel worded to make it sound like the mask was in some way approved by the FDA to meet N95 requirements, and heavily implied that it would protect against COVID-19 to the same extent as an N95 mask. ArsTechnica reporting says: "The FTC's complaint against Razer, which is best known for high-priced, RGB-riddled PC gaming peripherals, claimed that Razer continued promoting the Zephyr despite consultants highlighting the mask's lack of certification and protection." [2]
This is not a question regarding COVID-19 specifically, nor if N95 masks are adequate protection against COVID-19 variants. I know COVID-19 is a hot button issue still and I don't want to tackle that in this post. I'd also prefer not to address highly controversial agencies like the ATF (I've been assured there is no ATF agent on this forum btw) or the IRS, because those are spicy questions.
My question specifically revolve around the jurisprudence around more relatively boring agencies like the FTC, FDA, OSHA, EPA, SEC, NHTSA, NTSB, and my personal favorite, CSB (not biased) [3]. On one end of the scale, you have the position that no agencies should exist and that all the power of the administrative state should instead rest with congress. On the other end of the scale is the expansion of power of these agencies. I expect most people are somewhere near the middle.
So what I'd love to hear from you: (TL;DR 2: what does the administrative state look like to you?)

1. What is the difference between having an agency specializing in a subject (like the FDA for example) vs having legislative committees consulting with subject matter experts? Is it that the resulting laws don't pass through the legislature? I'm still reading here but that's my best guess.
2. How much power, if any, do you think should be delegated agencies like these in general, and why?.
3. Are there reasonable exceptions where they should or should not be allowed?
4. Does it make sense for an agency to be able to met out fines in a case like the Razer incident above?
5. Is there a way that industries can self-regulate and still care for public interests like public health? Example food for thought: technology (let's say ISPs) vs industrial safety.
6. Bonus question, no justification needed, just curious: what are your favorite agency, least favorite agency, and (in your opinion) most interesting niche agency?

My thoughts are that there are a few areas where agencies are useful:

1. Fast-moving and deep-knowledge subjects that the legislature would find difficult to tackle quickly. Examples would be the medical/drug research (FDA) or a technology agency (FTC?)
2. Public Health/Safety subjects for things like food safety (FDA), disease research and response (CDC...spicy take...), transportation safety (NTSB, NHTSA), and workplace safety (OSHA and CSB)

My logic for #1 is that some fields move way faster than legislature can ever hope to learn about. At the federal level we have 538 + 100 lawmakers, a number which has been fixed for a very long time, while the complexity of modern life increases exponentially. Some delegation feels necessary to me.
For #2, I feel like there are some specific areas where public safety needs to be prioritized. An example is the aviation industry. Ignoring Boeing for a moment, aviation has been getting safer and safer for decades. A large part of it is from incident analysis and regulation in response by the NTSB learning as much as it can from every aviation disaster. I don't know what would fill the gap without an agency like that. CSB is similar, although they are technically non-regulatory. Still, we fund them, because it's cheaper than a large-scale chemical disaster if you take their budget request at its word [4].
As for my favorites...you can probably tell that I like incident analysis. I think agencies like the CSB and NTSB are some of the coolest cats around, followed by NHTSA. For least favorite, my response is probably similar to a lot of people here: ATF. CSB is my favorite relatively niche agency!
Thanks for reading, I upvote all responses.
[1] https://www.ftc.gov/legal-library/browse/cases-proceedings/razer
[2] https://arstechnica.com/tech-policy/2024/04/ftc-fines-razer-for-every-cent-made-selling-bogus-n95-grade-rgb-masks/
[3] https://www.youtube.com/useUSCSB
[4] https://www.csb.gov/assets/1/6/fy24_congressional_budget_justification_-_final_3.8.pdf

When I tell you this medicine has changed my quality of life I really mean it. I am now a week into starting Voquezna for erosive gerd and my symptoms are quite literally GONE. I have tried about every reflux medicine there is with absolute no relief. I also have had chronic gastritis and intermittent gastroparesis flareups in the past year. Since starting this medicine my stomach has been extremely quiet and I can now eat much larger meals with no consequences. I know the drug is just FDA approved for erosive gerd at the moment, but will be approved for just gerd in a few months so please ask your doctor about this!

How do you make your own estrogel?
I get asked this question so many times in the comments or by private message. I have typed out the answer too many times. So I am making this post so I don't have to continue answering the same question.
I advise you to thoroughly educate yourself before you just follow a recipe. The transgender community has a lot more resources for education than the menopause community. That is where I began my search. Start with the reddit group transgenderdiy and estrogel . Those are both great resources. Read back through several years of posts. Ask questions. They are very helpful.
Look up the website hrtcafe. They list homebrew vendors and online pharmacies. They also tell you who not to buy from.
Also the website transfemscience list dose equivalents.
The recipe is simple. .3mg of raw estradiol powder dissolved in 3ml of 99% ethanol alcohol. (Use a scale that reads .001 grams. I bought the Gemini 20 on Amazon. ) Mix with 100ml of 62% Ethyl Alcohol hand sanitizer with Isopropyl Myristate. (Mix 10ml of hand sanitizer at a time. when mixed completely add another 10ml. This is easiest with a 10ml oral syringe) Amazon Basics Hand sanitizer has the perfect ingredients.
This recipe was created using the patent material for the original estrogen gel that was approved by the FDA. You can easily find the patent online. It is basically hand sanitizer mixed with raw powder. You can buy the hand sanitizer ingredients separately or just buy a hand sanitizer with the correct ingredients.
This recipe is 3 times the concentration of the patented gel. They mention in the patent that the gel is effective up to 5 times the concentration. 1ml of this gel is 3mg of estradiol. If you looked at the website transfemscience you know that 3mg of estradiol gel is equivalent to a .1mg patch. You can use more or less depending on your goals. I use a 1ml oral syringe to dose accurately. Again, 1ml of this gel is 3mg of estradiol.
Raw estradiol powder is not a controlled substance in the US. (I can't speak for other countries) So it is legal to buy. Reddit won't let me link the website I get my powder from but look up waterlily. It is $21 a gram. There are other vendors who are cheaper but waterlily has hers tested with a third party. You can cross reference the test from the website of the testing company to verify it is legitimate. She also sells raw progesterone if you want to make your own suppositories. Other vendors are based in China and charge about $6 a gram plus $50 shipping. If you are buying for all your meno friends this may be a good option. :)
Again. Please educate yourself before setting up your own lab. But once you understand it is very easy and super cheap to make.
estrogel has many other recipes. This is the most basic recipe. It has been extremely effective for me.
Best to all the ladies. <3

https://youtu.be/_ZRNeABDrsc?si=oRb41iRyX0hIlRAz
Key takeaways: They are making $$$, trying to ramp production but takes time, pill form phase 3 results in 2025 (usually first fda approved for diabetes, then weight loss 1-2 years thereafter)

MHLW Project Team Compiles Interim Proposal for Supporting Healthcare Startups
April 30, 2024
A project team under the Ministry of Health, Labor and Welfare (MHLW) recently put together an interim report proposing measures for promoting the incubation of healthcare startups, including a milestone-focused funding scheme.
The “milestone-based development support” model proposed in the report is similar to that of the US Defense Advanced Research Projects Agency (DARPA), in which subsidies are given to companies every time they achieve the prespecified milestones. The aim of this funding scheme is to accelerate the development of drugs and medical devices in areas that have been difficult to tackle.
The proposal also recommends
1) setting up a new consultation desk dedicated to receiving and deliberating on requests from healthcare startups for reimbursement fee revisions,
2) establishing a startup support strategy office under the MHLW to reinforce the functions and structure of the Medical Innovation Support Office (MEDISO), and
3) actively utilizing decentralized clinical trials (DCTs) and other digital strategies in clinical studies to significantly reduce the time and costs until product launch.
The project team is led by parliamentary vice health minister Akihisa Shiozaki and kicked off discussions this February with the aim of supporting the launch of startups and new businesses in the healthcare space. The team compiled the interim proposal on April 25 at its fourth meeting and plans to draw up a finalized version in June.
https://pj.jiho.jp/article/250882
Tweets exchange from Hardy's X account (machine-translated from Japanese):
Koby@VC, April 27
Interim report of the Ministry of Health, Labor and Welfare Healthcare PT.
I think it's a good idea for MEDISO and AMED to "discourage" PMDA and development in the domestic market in both medical devices and pharmaceuticals. (Although it's definitely not possible)
If there is a reason why a product should be developed in Japan even if it is dissed, it would be good to develop it in Japan, but just because it is a Japanese seed, the local market is prioritized, and I don't feel like overseas VCs are interested.
I feel that the number of companies/entrepreneurs/investors with this kind of mindset is gradually increasing, and we are about to see a time when saying things like "talking with the FDA to expanding overseas" will become a cliché, so the future is bright! (Even though the yen is weak)
Dr. Tadahisa "Hardy" Kagimoto, MD, April 27
No, it is understood that biotech can only be established if it is strongly determined and approved by the United States. In fact, even the development of pharmaceutical companies is only possible in the United States! In the end, it's the same story as the initial BBG [product developed by Hardy's first company - imz72] development strategy. This is the correct direction. Nurturing healthcare startups as an industry. This is a recommendation that is shared by the task force, including multiple ministries.
Koby@VC, April 27
Thank you, Committee Member Kagimoto!
Then, please include the statement that "MEDISO does not provide consultations related to PMDA/domestic market"! lol
Dr. Tadahisa "Hardy" Kagimoto, MD, April 27
That wouldn't be necessary. The fact that the investment recovery efficiency is highest in the United States is not the same as not developing it in Japan. Furthermore, in the case of Japan, by making physician-initiated clinical trials faster and cheaper, human POC can be accelerated and the overall development risk can be significantly lowered. Based on this, the probability of success in the global exam will increase, so PMDA consultation should be proactive in that regard as well.
Koby@VC, April 27
I often hear stories like this, but I think my lack of insight is a big part of it, but there are currently not many cases where development is successful globally after proceeding in Japan with mechanisms such as physician-led initiatives and conditional approval. That's what I think, and I think it's just a hypothesis.
Dr. Tadahisa "Hardy" Kagimoto, MD, April 27
Don't forget the BBG example we did together! The clinical research in Japan (in this case, it was not physician-led) and the POC led to the US phase 3 trial, and then to the partnership, and now the product is available in 92 countries. Without the initial POC, the technology would have just been published and that would have been the end of it.
Koby@VC, April 27
I don't remember much, but did BBG carry out the regulatory process in Japan?
Dr. Tadahisa "Hardy" Kagimoto, MD, April 27
Yes, probably approved next year, drug lag, about 15 years behind Europe!
Koby@VC, April 27
So much lag...
I think it was a good thing that there was no "venture support" such as MEDISO at that time, and Mr. Kagimoto was able to freely pursue development in the United States...
Dr. Tadahisa "Hardy" Kagimoto, MD, April 28
I wonder. The advisors around me at the time recommended development in Japan (or rather, that's the only experience I had), but based on numerical analysis, I think they just gave priority to America because it had the largest market. I feel like it's a question of whether a company is actually a company in the first place if management decisions are made based on the opinions of advisors...
Dr. Tadahisa "Hardy" Kagimoto, MD, April 30 [in English - imz72]
The Ministry of Health’s Task Force has been working on the new policy to embark the growth of Biotech industry in/from Japan.
Here is the English version of it and I am in charge of Biotechnology and Regenerative Medicine field.
Please let us know your thoughts on this.
[I removed the link to the document as it may cause the thread to be deleted by the reddit system - imz72]

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Product Info
Price: $39.99

• I have a Rx for Wegovy but insurance won’t cover it until I try three other drugs.
• My doctor is not enthusiastic about compounds (“not FDA approved). Which is annoying as obviously no compound drugs are, but they are used for all kinds of needs. The pharmacies are regulated, not the drugs. It seems she should know this….. but oh well.
• so I found a local board certified plastic surgeon offering a four month program. However, it is quite pricey. $699 a month. I am very aware that TeleMed programs are often much cheaper but personally, I do not want to go that route.

Question: have a fantastic local compounding pharmacy. Is anyone aware of how I could work with an online provider that would allow me to fill my prescription through my local compounding pharmacy rather than by mail?

OK here's the deal. Bluebird Bio (BLUE) topped out at $139/share in March of 2018 and has been on steady decline ever since. The company split into 2 entities in 2021 with ones goal to treat some forms of cancer, and BLUE continuing to focus on gene therapy using Crispr technology.
On Bluebird's recent investor conference call, they state that just secured a $175million loan from Hercules that will keep them afloat until 2026. They have very newly FDA approved drugs that cost millions of dollars per dose, and have secured reimbursement agreements with medicaid to cover the costs of administration. Their drugs - Lyfgenia (sickle cell), Skysona (cerebral adrenoleukodystrophy), Zynteglo (beta thalassemia) - rely on Crispr.
In sickle cell disease, patient's red blood cells are abnormally shaped causing repeated vast-occlusive events that are not only very very painful often requiring IV narcotics, but can cause kidney problems, lung infections, and even death. After one dose of Lyfgenia, severe events were resolved in 94% of patients, and completely gone in 88% of patients who received the drug. Life expectancy in Sickle Cell is only about 50 years and could be greatly extended if vast-occlusive events were reduced.
Patients with beta thalassemia are chronically anemic and are transfusion dependent their entire life-- after one does of Zynteglo, 9 out of 10 patients were TRANSFUSION FREE with normal blood counts.
Cerebral adrenoleukodystrophy is a devastating neurodegenerative disease of children with no treatment outside of an incredibly risky stem cell transplant from a matched donor, and over half of children diagnosed will die within 5yrs of diagnosis. With Skysona, it cuts in half the chances of having major functional disability at 2 years.
Yes, Lyfgenia is more expensive than it's FDA approved rival (Vertex’s Casgevy) which did NOT come with a black box warning, but, this was an earlier formulation of the drug, only TWO patients in the study developed blood cancer and patient's with Sickle Cell are already at higher risk of blood cancers than the general population, meaning this could just be coincidence. Additionally, Both Lyfgenia and Casgevy are similar in that you take the patients own stem cells, alter them with Crispr and give the altered cells back to the patient as a one time infusion. In order to tolerate the infusion, patients receiving either drug need high dose chemotherapy to calm down the immune system. The cancers mentioned on the black box warning for Lyfgenia are AML (acute myeloid leukemia), and MDS (myelodysplastic syndrome)-- BOTH of these types of cancers happen more frequently in ANY patient who received chemotherapy. It is very possible that the black box warning is just bad luck for Bluebird and it has nothing to do with the drug, it is a function of just the patient having sickle cell and getting chemo.
Cost wise, Bluebird's Lyfgenia is listed for $3.1 million for a dose and Casgevy for $2.2 million -- the most expensive medications of all time. Pricing is complicated, but Bluebird hopes to enroll about 100 patients this year and earnings will reflect that later in the year.
About 17% of the float is shorted, at about a 4.3:1 ratio of shorted shares to daily volume, so unlikely to short squeeze, but if shorted shares are under-reported (LOL), it could at least get interesting.
I'm bullish. THESE DRUGS WORK. The cancer risk is overblown. A stock that once traded for $139/share WITHOUT a viable/sellable product now trading in penny stock territory at the same time they are FINALLY rolling out a product into patients veins, securing $175 million of funding to keep afloat until 2026, and getting medicare to agree to pay for the drug? Count me in. I know a lot of people got burned riding this down from 2018, but don't let that scare you away right before we go parabolic. I predict a sharp rise to the upside after Fall earnings are reported, and continued growth from then on.
Who's with me? I bought low and I'm holding LONG. No pump and dump here. This could be good. 2024-2025 has lots of potential

Hey guys and gals. I'm a former analyst at one of the big biotech-specialist hedge funds in Boston. I just left to manage my own investments, and I started a blog because why not? Believe it or not there can be deep value in biotech. I think SPRB is a one of the most compelling risk/reward net-nets I've seen - market cap $28M (85% drop recently due to market overreaction to meaningless data [my opinion]); Ph2 readout in 3Q; validated MoA and good biomarker data; net cash after readout is ~60M (I estimate). I've spoken to management and it's not obvious they'll burn all the cash if Ph2 fails. Pasting the text here but if you like it you should check out my blog for similar content, and subscribe.
https://open.substack.com/pub/vulpescapital/p/spruce-biosciences-upside-from-a?r=3pksaj&utm_campaign=post&utm_medium=web
Standard disclosure stuff: I own a lot of this, not investment advice, do your own research, consult a financial advisor.
An overreaction to negative but largely irrelevant data has created an irrationally attractive opportunity for:

1. High probability of success (PoS) Ph2 readout (validated mechanism [CRF1 inhibition in CAH], comparable biomarker data to nearly approved CRF1 inhibitor crinecerfont)
2. Downside protection: Price to net-cash = 0.36. Price to net cash post-Ph2 readout = ~0.5

Summary
SPRB is developing tildacerfont, a second-generation CRF1 antagonist for the treatment of congenital adrenal hyperplasia (CAH). A discussion of CAH pathology and standard of care follow below. CRF1 is a validated mechanism (first-gen CRF1 antagonist, crinecerfont, will likely be approved in April).
SPRB is a single-asset company, and has everything riding on tildacerfont. In mid-March, tildacerfont failed in a Ph2 CAH trial (study 203). Typically, when single-asset biotechs fail in Ph2, the company is dead. The market has decisively taken this position, and sent shares down ~85% on the news, where they have stayed.
SPRB is not dead. The company will release results from a second, more relevant, Ph2 (study 204) in 3Q24. I would argue that 203’s failure has little impact on the future of the company and that the trial was designed such that it tells us nothing about the prospects of study 204, which are quite positive.
Study 204 is being run in a much more appropriate patient population, and has a good PoS (60%-70% in my opinion).
At present, the market is offering an irrational price at which one can make a bet on 204’s outcome with a reasonable degree of downside protection (cash).

• Market cap = $28M (not fully diluted – warrants priced way OTM)
• Net cash = $81M
• Burn = ~$12M per quarter
• Quarters to data = 2
• Projected net-cash post Ph2 readout = ~$57M

Though I’m typically not inclined to assume downside protection from cash in dev-stage biotechs (and SPRB management is not very invested), the cash here is considerable, and management has few other options to squander the cash.
Though I’m not relying on a short-term price prediction, it wouldn’t shock me if SPRB traded up into data as investors digest the fact the study 203 data weren’t interpretable positively or negatively, and I would consider selling before 204-readout if offered a fair price.
CAH
Patients with classic CAH are unable to make cortisol due to a genetic deficiency in the enzyme (21-hydroxylase) responsible for its production. Cortisol is a negative regulator of a signaling pathway called the hypothalamic-pituitary-axis (HPA). Below, I do my best to explain the HPA’s role in CAH, in plain English:
Androgens are hormones produced by the adrenal gland which have an effect on bone density, muscle mass, and reproductive development among other things. The best known example of an androgen is testosterone.
Androgens are produced in response to a signal from the pituitary gland to the adrenal gland. The signal molecule is called ACTH.
ACTH is produced in response to a signal from the hypothalamus to the pituitary. The signal molecule is called CRH. The pituitary receives the CRH signal through a receptor called CRF1.
The amount of CRH production by the hypothalamus (and thus ACTH and androgen production) is negatively impacted by the presence of cortisol, which is produced by the adrenal gland in response to ACTH. Thus, a hyper-active HPA will produce more cortisol, which puts on the HPA’s brakes and keeps it in check. Conversely, an under-active HPA will produce little cortisol, which can boost it. Lots of signaling pathways maintain homeostasis in a similar way.
Because patients with CAH cannot make cortisol, they have over-active HPA’s, and make way too many androgens. A whole lot of bad things happen when patients produce too many androgens—examples include deformed genitalia, early puberty, shortened stature, and inability to regulate salt concentrations.
Additionally, cortisol absence results in other problems unrelated to androgen production.
CAH treatment
The two main goals of treatment are 1) resupply the body with cortisol, because it needs it, and 2) resupply the body with cortisol to dampen the HPA and reduce excess androgen production.
As such, patients take a glucocorticoid (GC; a steroid; basically cortisol). Since they can’t make cortisol, they’ll have to take a GC for the rest of their lives.
CAH patients need to take very high GC doses to dampen androgen production, which leads to significant side effects such as stunted growth, obesity, increased risk of developing type 2 diabetes, cardiovascular disease, osteoporosis, skin toxicities, gastrointestinal disorders, and reduced lifespan. GC doses also need constant readjustment. Basically, they’re not fun to take.
CRF1 inhibition
If we refer back to the mechanism of CAH pathology, it would seem that if you could block the signal (CRH) from the hypothalamus to the pituitary, that might have the same effect of dampening the HPA as cortisol does. One could achieve this through the design of an antagonist (drug) that binds CRF1, blocking CRH’s access to the receptor.
Does this work? Yes, we already know that it does. Crinecerfont (Neurocrine) is a first-generation CRF1 inhibitor that generated positive Ph3 data in CAH. Patients on crinecerfont were able to reduce GC usage to a stat-sig greater extent than patients on placebo. Moreover, 68% of patients on crinecerfont reduced GC usage to a physiologic level, compared to 18% on placebo. The drug will likely gain FDA approval in the coming weeks (PDUFA date April 30). Estimates of peak sales are in the $1B range).
Tildacerfont
Tildacerfont is a second-generation CRF1 antagonist. Compared to crinecerfont, tildacerfont has better pharmacokinetics and is being dosed once-daily vs. twice-daily.
Does tildacerfont work? It appears to… we have proof of mechanism/target engagement. Here’s what we know:
In patients with poor disease control, tildacerfont (when they take it) significantly reduces elevated ACTH, 17-OHP, and A4 levels (the latter two are downstream markers). Biomarker levels rose again upon termination of treatment (data not shown - can't copy image here).
In patients with good disease-control (patients without severely elevated androgens), tildacerfont didn’t have much of an effect. That’s not such a bad thing— the goal is not to lower androgens (which are already successfully controlled in this population), but to allow these patients to reduce their GC usage.
These biomarker responses were approximately similar to those observed after 2-weeks of treatment with crinecerfont (data not shown - can't copy image here).
All this to say, tildacerfont has a good shot at reading out positive data in study 204, which has the same primary endpoint as crinecerfont’s phase 3, and a similar patient population.
Why this opportunity exists: Phase 2 failure
There are two types of CAH patient:

1. Patients with severe-CAH and poor-disease control. These patients struggle to control their HPA’s despite GC usage. The goal in treating these patients is to bring their androgen levels back towards the upper limit of normal.
2. Patients with good disease-control. These patients, through GC usage have androgen levels close to or within the normal range. The goal in treating these patients is to allow them to reduce their total GC usage.

Spruce decided to run two Ph2 trials–one in each patient group, with the goal of 1) improving disease-control in poorly controlled patients and 2) allowing patients with good control to lower their GC usage. The Ph2 in patients with severe-disease patients (study 203) read out last week. The Ph2 in patients with good disease control (study 204) will read out in 3Q24.
203 did not meet its primary endpoint – a reduction in A4. This looks like a very bad sign and the market reacted mercilessly. There are 40M shares outstanding, and 37M shares traded in the three days following data read-out.
Why did this trial fail and what does this mean for the next one?
The company is blaming their Ph2 failure on poor patient compliance, and I find this to be a reasonable explanation—only 50% of patients were >80% compliant.
The low compliance is cause for concern because a safety/tolerability issue could derail the drug. The company is making the argument that low compliance is an attribute of the poorly-controlled-CAH patient population. I don’t like to blindly trust management teams when they make statements like this, but take comfort in their disclosure that compliance is much higher in study 204. Moreover, discontinuations were quite low at 10% (typically you’d expect around 20%) and there were no safety issues.
The company found a KOL to attend their post-data investor call and say that “The CAHmelia-203 results underscore the complexities inherent in managing a patient group with challenges related to androgenic control and GC compliance. Based on my clinical experience, patients within this group may face difficulties adhering to any therapeutic interventions, potentially impacting treatment outcomes…”
When pressed on the call as to the reason for the low compliance in the 203 population, she insinuated that these patients are often poorly-controlled as a result of their low adherence to medication. This sounds reasonable, but again, what’s important is that we already know compliance is much higher in study 204.
The KOL added that the population in the ongoing Ph2 is more indicative of the patients that are seen in the clinic.
In sum, given the validated mechanism, observed biomarker data, and improved compliance, I give the ongoing Ph2 a 60%-70% PoS.
Valuation
The valuation range post-positive data is wide, but I’m not concerned with precision since the value is most assuredly multiples of $29M.
Sell-side analysts are projecting $500M peak sales (could be higher, as they project crinecerfont peak sales of $1B). At 2X peak sales, accounting for exercise of options/warrants plus 30% dilution on top, I arrive at $13/sh on approval. Discounted back two years and adjusted for PoS, I arrive at $7.5/sh of fair value today. It sounds a little crazy to say that the FV today is 10X the current price given study 204 data is on its way in two quarters, but I would consider that a month-and-a-half ago the stock was trading in the $5 range and, in my opinion, the prospects of the drug haven’t changed at all. Though I can’t predict short-term price action, I would not be surprised to see this trade up into data and would consider getting out before 204 read-out if given a fair price.
Disclaimer: The views and opinions expressed in this blog post are solely those of the author and should not be construed as investment advice. The author owns shares in the company discussed in this post. Please be aware that all investments involve varying degrees of risk, including the potential loss of principal, and there are no assurances that any investment will match or outperform any particular benchmarks or indices. As always, it is important to conduct your own research and consult with a qualified investment professional before making any investment decisions.

So I had a previous post that talked about some of the *weird* explanations of my benefits I got while on the phone with insurance and I've since then collected more info which I'll toss down here incase it helps anyone, because having the info helped me confirm I shouldn't be billed for the procedure.
My plan: IBX Personal Choice Bronze EPO Basic
Today's findings: I was told that my specific plan will cover 1 of each preventative procedure per year (1 colonoscopy per year, 1 birth control, etc) and after that the 2nd procedure would count towards the deductible (which is obvs not relevant for sterilization because there is only 1 procedure). BUT for my plan this ONLY works if it's billed as PREVENTATIVE care by the doctor. If it's a medical necessity or elective, it will NOT be covered fully on my plan (and this is apparently just a thing the doctor will say like '58661 because preventative care' when they send the codes to insurance). She also confirmed that related services (consultation for the procedure, anesthesia, etc) should be covered with no cost to me.
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Documents that helped me get my point across
(and bless the lady who put up with my questions because there was a bit of a language barrier and I'm sure she wanted to strangle me lol); But I legit had her pull up some of the docs and I referenced specific passages (I've bolded imo the most useful):

• Plan Benefit Book
  • page 7: discusses preventative code and links to another page that discusses preventative care
  • Page 20: says Adult preventative care is covered 100%
  • page 21: says women's preventative care is covered 100% with deductible not applying
  • page 92-93 (or search 'sterilization'): says peeps should have access to full range of female-controlled contraception and then lists 'sterilization surgery' under that list. It also mentions 'only certain contraceptive drug options in each category are covered at no cost' so perhaps heckle insurance about what would/wouldn't be covered.
• Generic page on preventative care
  • lists generic stuff covered and takes you to a more useful page which...
    • page 2 (bottom paragraph): "Most Independence Blue Cross health plans fully cover recommended preventive care services at an in-network provider, so you pay $0 out-of-pocket"
    • page 5: includes sterilization and contraception under women's preventative care
• The giant list of CPT codes I mentioned (direct link may or may not work)
  • just search '58661' or 'sterilization' and it'll take you to the miscellaneous section; as far as I can tell, this just lists the code as being covered, not necessarily that it's FULLY covered, but the other passages mention above should indicate it's fully covered since it's listed as preventative care.
  • There's a search bar in the upper-right where the lady searched '58661' and the first thing that came up was the gender affirming surgery.
  • if the direct link doesn't work... go to ibx.com/medpolicy (and accept the terms) > search 'sterilization' or the cpt code > it's doc 00.06.02aq also listed as 'preventative care'
  • miscellaneous policy says: "Sterilization procedures, patient education and counseling for contraception, and all Food and Drug Administration (FDA)--approved contraceptive methods are covered as a preventive service for all females with reproductive capacity."
  • related bulletin where they seem to have added the 58661 cpt code

External government resources:

• healthcare.gov
  • says must cover some preventative services at no cost if in network
  • link to preventative services for women
    • all market place plans must cover FDA approved contraceptive methods, sterilization procedures, etc
    • links to another page mentioning sterilization being covered

------------------------------------------------------------------------- Issues I ran into questioning people along the way: - They were searching a giant list of CPT codes, but they were searching the WRONG one. Using bisalp code 58661 they were searching in the 'gender affirming services' doc. I only found this out when I asked how she had gotten to the doc she was looking at. (see med policy cpt doc linked above. - She should have been looking at the 'preventative care' code. This might also be why the other people I talked to thought it counted towards the deductible, but idk. - people counting the cpt code as 'surgery' or an 'outpatient procedure' rather than 'preventative care' (which i have a feeling was tied into where they searched for the code - Apparently doctors may practice at different locations and some of the LOCATIONS aren't covered, but some are. Doctors may also not renew their being covered with insurance, so it's not a bad idea to call the doctor to verify they're in network and then call insurance to check and make sure that's not going to end randomly in a month if they're not planning on renewing. Super weird... Anyway...
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Also HELLA thanks to everyone who has helped me dig up policy content and find relevant info - you're all fantastic and I appreciate you <3
I'm legit so excited right now (even though I'm still paranoid I'm gonna have to fight with insurance later again) because the lady sounded very sure that it should be covered (compared to the other people I spoke to). Now off to heckle some more doctors on the list and see if any of them are properly available lol.

I can understand how reading about a disease that you have can cause you distress. However, I have seen multiple posts here and on other online tinnitus forums that go something like this.
Success story.
I accept it.
Moving on with my life.
I'm leaving the community.
Tired of reading about it.
rEaDiNg bAD!!!!!!!!!
READING ABOUT TINNITUS MAKE TINNITUS WORSE!!! ahhhhhhh!!!!!!!
They always follow this basic pattern and it is hilarious because the good for nothing quack shrinks promote the idea that people shouldn't read online tinnitus forums. The reason is because they want you stop thinking about it and "habituate" which just means ignore it. I have had 2 bad spikes in the last 2 months, one from prescribed drugs, another from noise. Mine is at a 1/10 right now and guess what? It's not bothering me. With the office air, and the typing on my keyboard I can't even hear it. So what even is "habituation"? Can you habituate, un-habituate, and habituate again in 3 weeks? Is it like Catholicism where you lose your salvation pretty much every week and get it back at the Mass or is it like Calvinism that teaches once saved always saved?
Habituation just means that it's so low it doesn't bother you or you're literally the kind of person like a hoarder living in a mountain of trash that can live in that chaos unbothered. Some of us are simply not in either of these situations. So, regarding these fake success stories that seem to be trying to convince us to stop reading here, I think they are just that, fake. There was recently a post made here that was just like the example I provided and it was literally the only post they had ever made on Reddit. Why would someone that had "success" feel the need to come here and try to convince the rest of us to leave too? Like, why do they care?
Habituation as a concept is BAD because it decreases the urgency to find a cure. Why would there be urgency? There is a "treatment" it's called CBT which teaches to habituate? There's hair cell regeneration technology and nerve regeneration technology that already exists and just needs a little further development and there are actually several other drugs to regenerate nerve damage being trialed right now. After the covid vaccine Operation Warp Speed I think we deserve something like that ESPECIALLY considering the government has sent so many people in the military to tinnitus doom by giving them bad earplugs. The government also doesn't regulate concert halls' noise levels like they do in factories. Let's also not talk about the government FDA approved procedure called MICROSUCTION that has landed many of us here. Also, drugs, also the covid vaccine also many many other things.
In before:
well the government is god FDA good and there's nothing we can do so all we have is habituation so it's a good thing shut up and stop noticing things and stop having ideas

​

Stock way up on quarterly earnings results. A few highlights from his comments:

• Company has added $11b in capital investments to support additional production but these projects have a substantial lead time.
  
• They are in late-stage trials on a GLP-1 pill and expect FDA approval next year. Pills are easier to produce than injections.
  
• New clinical trial is showing good results for sleep apnea, which is not surprising given its link with excess weight.
  

Everyone has seen the short interest on this stock and its insane as share price has doubled on shorts as well as cost to borrow having now doubled on shorts. Here are some reasons why this could actually blow.
Ok all of these are not catalysts but there are many reasons to like IBRX.
79.62% of all shares are held by INSIDERS
Cost to borrow has risen over the last several days from 44% to 447%
Earnings - May 9th to May 13th

1. 20,000 doses ready to be distributed which is the inventory for one year. Each does costs $35,000.

Assuming that 80% of that will be paying customers we will have a revenue of $560 Million for the first year.
2) Rollout will begin on May 6th.
3) Secured BCG supply for one year and have other options in place incase of supply issues.
4) 23 Global sites activated for international expansion.
5) All International Strategic Partners have shown great interest.
6) $240Million cash on hand. Sufficient until revenue generation. No additional funding needed.
7) Front ran most financial commitments, Moonshot, CMC and Inventory buildup behind us.
8) 54 new Sales and Marketing Access Team hired, trained and Certified for 5 sales regions in the US.
9) Lunch with major presence in San Antonio, TX (beginning of May) and Chicago, IL (beginning of June)
10) Improved results from the completed Phase II non-small lung cancer trail. This may fast track FDA registration and hopefully approval.

The generic injectables market is a crucial segment of the pharmaceutical industry, encompassing a wide range of injectable drugs that are bioequivalent to branded drugs but typically cost less. These drugs are essential for treating various medical conditions and are widely used in hospitals, clinics, and other healthcare settings.

Market Size and Growth

In 2023, the generic injectables market was valued at USD 114.47 billion. The market is expected to grow at a compound annual growth rate (CAGR) of 13% during the forecast period of 2024-2032, reaching a value of USD 343.87 billion by 2032. This growth is driven by several factors, including the increasing prevalence of chronic diseases such as cancer and diabetes, as well as the rising demand for cost-effective treatment options.

Market Drivers

One of the key drivers of the generic injectables market is the growing prevalence of cancer worldwide. Chemotherapy, which is a common treatment for cancer, often requires the use of injectable drugs to manage side effects and improve patient outcomes. Additionally, the rising demand for injectables in chemotherapy is further fueling market growth, as healthcare providers seek more affordable treatment options for their patients.

Competitive Landscape

The global generic injectables market is highly competitive, with several key players dominating the market. Some of the major players in the market include Fresenius Kabi, Hikma Pharmaceuticals PLC, Sagent Pharmaceuticals, Inc., Sanofi, and Sandoz (Novartis). These companies are constantly innovating and investing in research and development to maintain their competitive edge in the market.

Market Challenges

Despite the growth prospects, the generic injectables market faces several challenges. Regulatory challenges, such as the need for approval from regulatory authorities for the sale and distribution of injectable drugs, can hinder market growth. Pricing pressures and the presence of generic competition also pose challenges for companies operating in this market.

Future Trends

Several trends are expected to shape the future of the generic injectables market. Technological advancements in injectables, such as the development of novel drug delivery systems, are likely to drive market growth. Additionally, the increasing focus on biosimilars – which are generic versions of biologic drugs – is expected to create new opportunities for market expansion.

Regional Analysis

The market size and growth of the generic injectables market vary by region. North America and Europe are the largest markets for generic injectables, driven by factors such as the presence of a well-established healthcare infrastructure and favorable reimbursement policies. In contrast, the Asia Pacific region is expected to witness the fastest growth during the forecast period, fueled by factors such as the growing prevalence of chronic diseases and increasing healthcare expenditure in emerging economies.

Product Segmentation

The generic injectables market can be segmented based on the type of injectable drug. Common segments include oncology, anti-infectives, cardiovascular drugs, and others. Each segment has its own market dynamics and growth prospects, influenced by factors such as disease prevalence and treatment guidelines.

Distribution Channels

Generic injectables are typically distributed through various channels, including hospitals, clinics, retail pharmacies, and online pharmacies. The choice of distribution channel can impact market growth, as each channel has its own advantages and limitations in terms of reach and accessibility.

Regulatory Environment

The regulatory environment for generic injectables varies by country and region. Regulatory authorities such as the FDA in the United States and the EMA in Europe play a key role in ensuring the safety, efficacy, and quality of generic injectables. Compliance with regulatory requirements is essential for market entry and success.

Investment Analysis

Investing in the generic injectables market offers several opportunities for investors. The market is expected to grow significantly in the coming years, driven by factors such as the increasing demand for cost-effective treatment options and the growing prevalence of chronic diseases. However, investors should also be aware of potential risks and challenges, such as regulatory hurdles and pricing pressures.

Impact of COVID-19

The COVID-19 pandemic has had a significant impact on the generic injectables market. The pandemic led to disruptions in the global supply chain, affecting the production and distribution of generic injectables. However, the market has shown resilience, with companies adapting to the new normal and implementing measures to mitigate the impact of the pandemic.

Sustainability and Environmental Impact

There is a growing focus on sustainability and reducing the environmental impact of pharmaceutical manufacturing. Companies in the generic injectables market are increasingly adopting sustainable practices, such as reducing waste and energy consumption, to minimize their environmental footprint. These efforts not only benefit the environment but also contribute to long-term market growth and sustainability.
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We are students at need namin na bumuo ng isang business para sa project namin, we've decided to look upon scented candle products for our business proposal. So ang question ko lang po is need pa po ba namin kumuha ng approval from the FDA para masecure po ang business proposal namin at hindi mag cause ng harm sa iba. We have no plan on continuing this business po after naming ipasa ang subject na to