Mad Hedge Biotech and Healthcare Letter
July 7, 2022
Fiat Lux
Featured Trade:
(A BIOTECH WITH A QUIVER FULL OF ARROWS)
(VRTX), (UNH), (SIGA), (CRSP)
Tag Archive for: (VRTX)
Even with the decline of the general market, several stocks have managed to buck the trend and thrive.
However, no stock is worthy of serious consideration if it isn’t at least delivering some positive returns.
In the biotechnology sector alone, there are roughly 750 biotech stocks on the major US exchanges.
Approximately 50 of these have been in the positive territory in the last 12 months. Among them, only 25 have shown a 20% or above jump.
In this very short list of promising biotechnology stocks in 2022, one name stands out as a huge winner amid a growing number of losers: Vertex Pharmaceuticals (VRTX).
Vertex shares have increased and soared near 50% over the past 12 months. In fact, the stock is up by over 30% thus far this year.
On top of stock performance, another factor to consider is the quality of the underlying business.
At the very least, the company needs to show potential to grow sales and deliver profits. Vertex once again delivers on these aspects.
The durability and dependability of revenue and earnings growth are critical.
This year, only three of the surviving biotechnology companies from the whittled-down list managed to generate positive top- and bottom-line growths over the past five years.
These are United Therapeutics (UNH), Siga Technologies (SIGA), and, of course, Vertex.
Vertex’s recent performance is a complete 180 from earlier times. The stock fell more than 30% from October 2020 until October 2021. This decline was primarily due to the investors’ anxiety over the company’s heavy reliance on its cystic fibrosis (CF) program.
Evidently, the tide has turned for Vertex. More importantly, this could only be the start.
While a biotech with an excellent track record is a good indicator, it’s not a guarantee that it can deliver the same results in the future.
However, Vertex appears to be doing an exceptional job of continuing its winning streak.
The company holds a rare advantage that only a handful of biotechs have: a rock-solid moat.
While investors may not like Vertex’s complete reliance on its CF business, it’s critical to remember that expansion is far from over for this particular therapeutic segment.
Moreover, Vertex is the market leader in this field worldwide—and it’s expected to keep this position until the late 2030s at the very least.
Four CF treatments have been approved in both the US and Europe, and Vertex makes all of them.
Sure, several companies are attempting to enter this market and compete against Vertex, but none of them have gotten past Phase 2. Actually, most of the potential rivals are still in the preclinical testing phase.
This monopoly enables Vertex to generate solid revenue and earnings growth continuously. In the first quarter of 2022, the company’s cash position reached $8.2 billion.
If that’s not enough to secure Vertex’s position in this market, then here’s another one. The biggest threat to Trikafta, one of Vertex’s CF blockbusters, is a candidate being studied and developed by none other than Vertex itself.
That’s right: Vertex’s biggest threat is another Vertex candidate.
In terms of patent exclusivity, Vertex has this concern covered as well because its best-selling CF treatment won’t expire until 2037.
Nonetheless, investors aren’t the only people hoping to expand Vertex’s portfolio. The company has been steadfastly working on that, too.
Aside from working on a potential groundbreaking mRNA-based CF treatment with Moderna (MRNA), Vertex has been developing candidates in several key segments, including diabetes, blood disorders, and pain.
Vertex and CRISPR Therapeutics (CRSP) are expected to seek regulatory approval for exa-cel (CTX001), a potential one-time cure for sickle cell disease and transfusion-dependent beta-thalassemia, within 2022.
It also moved its kidney disease candidate, VX-147, into late-stage trials last March. If this works out, the treatment can target a larger patient population than CF.
Another program expected to move into late-stage trials in the second half of 2022 is VX-548, an experimental non-opioid pain drug.
Meanwhile, its Type 1 diabetes pipeline is anticipated to grow soon. The company already has at least one cell therapy queued for early-stage testing, and the plan is to advance another program into clinical trials by the fourth quarter of this year.
Simply put, Vertex’s pipeline is akin to a quiver full of arrows. Considering the company’s track record, it would no longer be surprising if it hits all its targets.
Mad Hedge Biotech and Healthcare Letter
June 9, 2022
Fiat Lux
Featured Trade:
(THE FUTURE OF BIOTECH IS IN GOOD HANDS)
(CRSP), (VRTX), (EDIT)
No advancement in the biotechnology and healthcare sector is quite as mysterious, promising, and powerful as CRISPR.
It has long captivated the attention and imagination of serious scientists and researchers alongside the media and the public. It’s an understatement to say that it’ll be a significant area of development for decades to come, thanks to its capacity to remodel our genetic code accurately.
Among the frontrunners in the burgeoning CRISPR segment are CRISPR Therapeutics (CRSP) and Editas Medicine (EDIT).
Both biotechs have promising pipelines packed with innovative and potential cures for previously incurable genetic conditions.
The CRISPR programs appear to be the most advanced among their candidates despite still being in the early stages.
Nevertheless, any progress reported could likely push the entire CRISPR group to surge substantially.
Fortunately for this sector, there are at least two major catalysts in 2022.
CRISPR Therapeutics, which leverages the CRISPR technique, aims to submit its blood disorder treatment candidate, CTX001, for regulatory review by the end of 2022.
This program, which is developed in collaboration with Vertex Pharmaceuticals (VRTX), specifically plans to target sickle cell disease and beta-thalassemia.
So far, results have been positive, and the consensus is that it’s only a matter of time before both companies finally launch the much-awaited treatment commercially.
Another catalyst opportunity for CRISPR stocks this year is from Editas Medicine.
While this stock is not as popular as CRISPR Therapeutics, I think it holds the potential to become a massive star in the CRISPR gene-editing sector. What makes this biotech fascinating is the sheer number of candidates in its pipeline and the variety of options and applications they have for the technology.
At this point, Editas is focused on exploring the application of its in-vivo CRISPR genome editing therapy, called EDIT-101, to patients suffering from Leber congenital amaurosis 10 (LCA10).
LCA10 is an inherited and severe eye disorder that primarily affects the cornea and leads to a severe loss of vision or even blindness at an early age. This is caused by the mutation of the CEP290 gene.
This condition is an ideal target for Editas because CEP290 gene mutation can be found among 20% to 30% of LCA10 patients.
More than that, there is no existing approved treatment for it. This means that if Editas successfully completes the clinical trials for EDIT-101, it will possess a monopoly of this market.
Thus far, Editas has been delivering promising results on this treatment, and shareholders can expect definitive results from the study by the second half of 2022. Needless to say, positive data from the trials would tremendously boost Editas shares.
Aside from EDIT-101, Editas is also working on EDIT-301, which could be a competitor of CRISPR Therapeutics and Vertex’s CTX001.
However, the more exciting development for Editas is its advanced cell treatments in the oncology sector.
Recently, Editas announced its new gene-editing technology, called “SLEEK,” which means SeLection by Essential-gene Exon Knock-in.
This is groundbreaking because it improves cell therapies in a couple of factors. For example, it can deliver better knock-in of trans gene cargos. This means that SLEEK is more efficient in terms of artificially introducing a new gene into the genome of a patient.
It remains to be seen how Editas plans to take advantage of SLEEK in its future candidate. One thing’s for sure, though; this biotech has multiple shots on goal, making it hard for Editas to miss.
Taking all these into consideration, it’s safe to say that it’s not yet too late for investors to position themselves for lucrative long-term gains courtesy of these CRISPR stocks.
Despite the sharp decline in the gene-editing biotechnology sector over the past months, practically all the applications of CRISPR technology remain untapped to this day.
The most convincing tailwind for CRISPR-focused stocks is that the market for CRISPR-based treatments is estimated to multiply in value by approximately 13-fold between 2019 and 2030.
For that target to be reached, cutting-edge biotechnology companies should step up and develop the first generation of CRISPR-centered treatments. Considering where things stand right now, it looks like they are on the right track.
Mad Hedge Biotech and Healthcare Letter
May 24, 2022
Fiat Lux
Featured Trade:
(FROM A BORING BIOTECH TO A TRAILBLAZING PIONEER)
(VRTX), (ABBV), (MRNA), (CRSP), (EDIT), (BEAM), (NTLA)
When will the turmoil in the stock market come to an end? Unfortunately, nobody can offer a definitive answer.
At this point, there’s still no end in sight to high inflation, climbing interest rates, and the continuing war in Ukraine.
Needless to say, all these issues are affecting the stock market. However, not all stocks are getting negatively affected by the turmoil.
There are still relatively safe bets to buy, with some crushing the market these days.
One of them is Vertex (VRTX).
Vertex stock soared by over 30% year-to-date by mid-April.
While it has given up some of that gain in the past weeks, this biotechnology and healthcare stock is comfortably outperforming the rest of the market, with its shares still up by around 15%.
One reason for Vertex’s good performance is its undisputed monopoly in the cystic fibrosis (CF) market. In fact, its closest potential competitor is AbbVie (ABBV), which recently announced disappointing results for its Phase 2 trials for a CF combo.
This means Vertex’s dominance in the CF space is set to go on for quite some time.
Here’s a bit of background. Vertex has 4 CF treatments.
Among these, the latest treatment, Trikafta, generates the lion’s share of the profits. It raked in $1.7 billion in the first quarter of 2022 alone, with the total revenue for the entire CF pipeline recording $2 billion.
Considering its approved indications and potential approvals, Trikafta is anticipated to treat 90% of the entire CF patient population.
Looking at its current performance and how strong its hold is in the CF market, it appears that Vertex’s prediction that it can sustain its dominance in this segment until at least the late 2030s will be proven right.
Moreover, it’s evident that Trikafta has yet to reach its peak revenue. However, Vertex isn’t depending on this particular treatment alone.
Rather, the company is working on developing a worthy competitor to this top-selling treatment.
That is, Vertex is working on a CF candidate that may potentially be even more effective than Trikafta.
So far, this new drug candidate not only has the capacity to beat Trikafta in terms of efficacy but also offers a more convenient option.
Trikafta is a twice-a-day oral drug that comes in the form of 3 tablets. Meanwhile, this potential competitor is a once-a-day alternative.
If everything goes according to plan, the Phase 3 trial for this Trikafta challenger could start by the end of 2022 or early 2023.
This means that the closest potential rival for the company’s top-selling treatment is its own candidate.
Apart from this, Vertex is working with Moderna (MRNA) to come up with an mRNA therapy for CF patients.
The goal is to offer an alternative option to patients who are not eligible for the current CF therapies.
Although this continued dominance in the CF sector is already a good enough reason to buy Vertex shares, they may be an even better one.
To date, Vertex has at least 6 programs queued in mid to late-stage clinical studies, all of which are projected to become multi-billion dollar revenue streams.
Outside its CF segment, Vertex could have another big winner in the form of gene-editing treatment CTX001.
This is a treatment for sickle cell disease and transfusion-dependent beta-thalassemia that the company has been working on with CRISPR Therapeutics (CRSP).
While CTX001 is promising, it won’t be entering the gene therapy market without any competition. It has to battle the likes of Editas (EDIT), Beam Therapeutics (BEAM), and Intellia (NTLA).
Nonetheless, CTX001, if approved, is a game-changer because it is developed as a one-time cure for genetic blood disorders.
So far, trial results have been positive, and the collaborating duo is expected to file for regulatory approval by the end of 2022 and possibly launch the product by the first half of 2023.
This gene-editing therapy is a significant milestone for Vertex, with CTX001 expected to become another blockbuster, raking in roughly $1 billion in annual sales for the company even after giving CRISPR its share of the profits.
Recently, Vertex added another $900 million to its collaboration with CRISPR to boost its share from 50% to 60%, indicating that it values CTX001 at roughly $10 billion.
Other critical treatments outside the CF space are VX548, an opioid alternative targeting acute pain, and VX800, a stem cell-derived therapy developed to treat Type 1 diabetes.
Vertex has been accused as a company scared of getting out of its comfort zone for quite some time.
With these new ventures, Vertex has become something of a pioneer—a strategy that is projected to open long-term and lucrative revenue streams for the company.
Overall, all these efforts paint an obvious picture. That is, Vertex is a well-balanced company with a main business that capably and reliably generates billions and is complemented by an exciting pipeline that holds the potential to replicate the success of its already established portfolio.
Mad Hedge Biotech and Healthcare Letter
April 14, 2022
Fiat Lux
Featured Trade:
(A BIOPHARMA STOCK BENT ON REDEMPTION)
(MRK), (BMY), (ABBV), (ORGN), (PFE), (VTRS), (MRNA), (BNTX), (CRSP), (VRTX), (BLUE), (BIIB)
It looks like we’re about to bear witness to a redemption journey.
Once upon a time, Merck (MRK) was a major player in the cardiovascular sector. Over the years, it has gradually diminished to a minor league name.
However, Merck has plans to reverse this fortune and reclaim its dominance in the cardio market. To date, it has eight new drug approvals and a slew of expanded labels queued in the next couple of years.
This decision is evident in Merck’s move to outbid Bristol Myers Squibb (BMY) in the auction for Acceleron Pharma, shelling out a whopping $11.5 billion to boost its cardio pipeline considerably.
While the deal may seem like a massive risk, Merck is confident that this deal holds the potential to open up the path to single-product peak sales reaching $10 billion by the mid-2030s.
In fact, there’s no need to wait for long to see some solid proof of Merck’s multibillion-dollar bet, as Acceleron already has a candidate set to be put on display by the end of 2022 or early 2023.
This Acceleron acquisition forms part of the “New Merck” touted when the company welcomed a new CEO and came on the heels of the success of the leadership that brought the mega-blockbuster cancer drug Keytruda.
It also signifies Merck’s conscious efforts to ease their heavily criticized over-dependence on Keytruda.
While the drug will lose patent protection after 2028, Keytruda still holds a significant portion of Merck’s sales. The treatment accounted for roughly 35% of the company’s total revenues last year.
The patent loss of a significant moneymaker is a typical problem for virtually every Big Pharma company, with AbbVie (ABBV) and Bristol Myers Squibb coming to mind as the most recent examples.
The go-to solution to this is pursuing mega-money mergers: AbbVie acquired Allergan for $63 billion while Bristol splurged on Celgene at $74 billion.
This quickly bolsters the existing pipelines and portfolios of the companies and assuages the fear of investors over impending revenue losses.
Instead of following this pattern, Merck did the opposite in 2021.
The company decided to downsize and established a spinoff segment: Organon (ORGN). The idea is to offload its biosimilars and other legacy products to focus on its core strengths.
This is reminiscent of Pfizer’s (PFE) move to spin out its Upjohn unit and merge it with Mylan to form Viatris (VTRS).
This move looks to have worked well for Merck and Organon as it allowed the parent company to focus on its blockbuster brands.
For instance, Bridion recorded a 28% year-over-year rise in 2021 to reach $1.53 billion in sales, while ProQuad reported a 14% increase to hit $2.14 billion.
Meanwhile, Gardasil rose to an impressive 44% to contribute $5.7 billion.
Even Merck’s Animal Health sector grew by 18% to record $5.6 billion.
There’s also Keytruda, which is projected to become the highest-selling drug at $24.3 billion by 2026.
These are only some of the blockbuster products in Merck’s portfolio expected to continue increasing revenues this 2022.
In addition, the company expects at least $5 billion from its COVID-19 antiviral drug Molnupiravir.
Looking at the trajectory and growth of the pipeline and existing programs, Merck estimates an additional 17% increase in its year-on-year revenue in 2022 to reach $56.1 billion to $57.6 billion.
Despite the move to establish a spinoff unit, the Acceleron deal hints at the possibility that Merck might be shifting to an open checkbook strategy.
Considering how relentlessly it pursued the deal, there’s a chance that the company would be at the bargaining table for a while in search of ways to protect itself against the pending Keytruda patent loss.
Some contenders for a potentially splashy offer from Merck are Moderna (MRNA) and BioNTech (BNTX), which could bolster the bigger company’s mRNA pipeline.
It can also splurge on gene therapy experts by targeting CRISPR Therapeutics (CRSP) and even Vertex (VRTX).
However, given bluebird bio’s (BLUE) flailing performance as of late, this small biotech could very well be a contender for a bargain deal.
Speaking of discounted stocks, Biogen (BIIB) is also reportedly under consideration simply because of its deeply discounted price following its disastrous Alzheimer’s disease program.
Whatever move it makes, one thing is sure: Merck, with its $208 billion market capitalization, is in a healthy and stable place financially.
More importantly, it has an excellent product portfolio and an exciting pipeline.
It has shown remarkable growth in the past years and impressive efforts to secure a great future, making it a solid stock to buy and hold for a long time.
Mad Hedge Biotech and Healthcare Letter
April 5, 2022
Fiat Lux
Featured Trade:
(A BRIGHT SPOT IN A GLOOMY SECTOR)
(VRTX), (MRNA), (ABBV), (CRSP)
In an economy continuously plagued with a rising interest rate, it’s not unheard of for risk-averse investors to steer clear of businesses with high debt loads.
After all, those kinds of companies could be the most affected as climbing interest rates inevitably lead to lower profits.
The silver lining is that there’s no need to sacrifice putting money in growth stocks altogether.
You can simply load up on ultra-conservative businesses to ensure that you don’t come off the losing end in the battle of an ever-increasing interest rate.
In the biotechnology and healthcare sector, there are a handful of promising fast-growing businesses that are not saddled with tons of debt. One of them is Vertex Pharmaceuticals (VRTX).
A continuously growing business, Vertex recorded $7.5 billion in sales in 2021, showing off a 22% increase from 2020.
Its cystic fibrosis (CF) program is a major player in its growth, particularly Trikafta/Kaftrio. On its own, this blockbuster treatment contributed $5.7 billion to Vertex’s top line in 2021.
As it expands and goes after more growth opportunities, Vertex consistently ensures that it is backed by a solid balance sheet. In total, its short- and long-term liabilities amount to roughly $3.3 billion.
With a cash balance of $6.8 billion, the company has more than enough to clear that off.
In the past 12 months, Vertex has generated roughly $2.6 billion in cash from its daily operating activities.
This biotechnology company has been in such excellent shape that it managed to buy back shares with $1.4 billion last year. That’s practically three times the $539 million it allocated to repurchasing efforts in 2020.
Meanwhile, investors who feel they missed the boat on Moderna (MRNA) now have a second shot at investing in another high-growth biotechnology company.
Plus, it still has a Moderna connection and already has a strong track record of dominating a lucrative market.
Vertex and Moderna, which saw their stock price catapult to a record-breaking 800% in the past two years, are working on an mRNA-based therapy for CF patients.
Now, you might be wondering why Vertex is pursuing this program, considering its dominance in the CF market.
In fact, the closest rival would be AbbVie (ABBV). However, Phase 2 trial results for this candidate are due in two to three years. That means Vertex will likely remain the top name in the CF space for a while. Nevertheless, Vertex appears determined to keep its lead.
So, why bother with a new program instead of bolstering the existing Trikafta pipeline?
Well, right now, Vertex has virtually covered 90% of the CF market—and this is where Moderna comes in.
What the two are trying to do is to completely cover the market and target the remaining 10% not qualified to take the existing Vertex CF treatment.
As of the last update, the remaining demographic is at 25,000 patients. This would translate to another $4 billion in commercial sales.
If they succeed, the two would have created the biggest competitor to Trikafta. That means Vertex’s most formidable rival would be Vertex as well.
Needless to say, Vertex’s continuous dominance in the CF space guarantees blockbuster levels of profits in the years to come.
Vertex has been busy expanding into additional therapeutics segments despite its resounding success in the CF space.
Another potential blockbuster is CTX001, a one-time gene-editing treatment targeting blood disorders beta-thalassemia and sickle disease, developed in collaboration with CRISPR Therapeutics (CRSP). This is by far the most exciting venture of the company, with the partners expected to file for regulatory approval by the end of 2022.
Aside from these, Vertex’s pipeline is filled with promising candidates. One is VX-147, which is a groundbreaking therapy for severe genetic kidney diseases. There’s also autoimmune treatment VX-880.
VX-548 is another exciting candidate. While this drug is aimed to be an acute pain treatment, a key characteristic is the absence of drug addictiveness.
This is a breakthrough effort because it might just be the answer to the ongoing opioid crisis.
Given the unique mechanism of VX-548, this alternative aims to deliver treatment with low addictive effects.
There are roughly 75,000 deaths reported annually caused by overdose on opioid drugs in the United States alone. This could translate to $4 billion in the addressable market.
Although these candidates are not as advanced as Vertex’s CF program, they demonstrate that the company can go beyond its well-established niche and bolsters investor confidence.
With the rising inflation and economic turbulence, it’s advisable to prioritize companies with steady cash flow and promising growth prospects
Despite the rough couple of years for the broader market, Vertex easily meets these expectations and appears to be one of the positive stories in the healthcare and biotechnology sector.
