Mad Hedge Biotech and Healthcare Letter
December 14, 2023
Fiat Lux
Featured Trade:
(EDITING YOUR PORTFOLIO)
(CRSP), (VRTX), (BLUE), (BEAM), (CRBU), (EDIT), (NTLA), (PRME), (VERV), (LLY), (REGN)
Tag Archive for: (VRTX)
In the world of biotechnology, the buzz these days is all about gene editing – a frontier that’s moving at warp speed.
While the journey from sequencing the first human genome took a staggering 13 years, companies like CRISPR Therapeutics (CRSP) have sped up the process, bringing their revolutionary "molecular scissors" concept to market in a mere decade.
It's a thrilling time for investors, with the potential for staggering returns, but the path is littered with clinical and regulatory landmines. This turns choosing the best stocks to put your money into a tricky challenge.
Recently, the FDA gave the green light to two groundbreaking gene therapies for sickle cell disease, developed by Vertex Pharmaceuticals (VRTX) in collaboration with CRISPR Therapeutics and by Bluebird Bio (BLUE).
This disease, predominantly affecting African-American communities in the U.S., has been a target for medical advancement for years.
While the approval is a landmark, it's not without its tremors. Bluebird Bio's stock took a nosedive by 33.9%, triggered by the FDA’s warning label about potential cancer risks linked to their treatment.
In contrast, the treatment by Vertex and CRISPR dodged such warnings, possibly giving it an edge in the eyes of prescribing doctors.
And then there’s the money side of things. Bluebird Bio missed out on a priority review voucher from the FDA, which they were hoping to sell to Novartis for a cool $103 million. That's a tough break.
Meanwhile, the Vertex and CRISPR therapy, now known as Casgevy, boasts the honor of being the first FDA-approved drug using the trailblazing Crispr/Cas9 technology. It's a Nobel Prize-winning innovation that's finally reaching the patients it promises to help.
The approvals of Casgevy and Bluebird Bio’s Lyfgenia, which arrived earlier than expected, mark a significant moment for patients with sickle cell disease.
Although priced in the millions, these treatments offer a potential one-time cure, replacing the traditional, complex regimens. Unfortunately, they are not without their challenges, involving intensive procedures, lengthy hospital stays, and chemotherapy.
This brings us to the investment side of things.
The gene-editing arena is brimming with potential, but it's akin to navigating a labyrinth. With no specific exchange-traded funds (ETFs) focusing solely on gene editing stocks, investors might feel like they're trying to find their way in the dark.
However, a diversified approach could be the lantern in this darkness.
Companies like Beam Therapeutics (BEAM), Caribou Biosciences (CRBU), Editas Medicine (EDIT), Intellia Therapeutics (NTLA), Prime Medicine (PRME), and Verve Therapeutics (VERV) are some of the key players in this space, each with its unique technological platform.
But it's not just the pure-play gene editors that are worth your attention. Giants like Eli Lilly (LLY), Regeneron Pharmaceuticals (REGN), and Vertex Pharmaceuticals have thrown their hats into the ring, making substantial investments in gene editing.
So, how should you play this? If it were my money, I'd spread it around.
Put a chunk in leaders like CRISPR and Intellia. Then, combine these with established players like Eli Lilly, Regeneron, and Vertex to provide a safety net, balancing out the inherent risks of this high-stakes biotech game.
On the other hand, companies like Beam and Verve, representing the next wave of this technology, should not be overlooked, though perhaps with a more conservative stake.
And here's a little hedge for you: keep an eye on smaller players like Caribou Biosciences and Editas Medicine. In this high-stakes game, they could be your ace in the hole.
The gene-editing industry is a roller coaster of innovation, risk, and potential. It's a sector where fortunes can be made and lost in the blink of an eye.
For the savvy investor, a diversified, strategic approach, blending the bold with the stable, could be the key to unlocking the vast potential of this exciting field.
Remember, as with any investment, the key is not just in choosing the right horses but knowing how to spread your bets across the race.
Mad Hedge Biotech and Healthcare Letter
November 7, 2023
Fiat Lux
Featured Trade:
(OUTSMARTING OPIOIDS)
(VRTX), (LLY), (NVO), (BIIB)
Amid the stark realities of America's opioid crisis, with a staggering 80,000 annual fatalities due to overdose, the pharmaceutical industry is on the brink of a significant shift.
Vertex Pharmaceuticals (VRTX) stands out with its investigational drug VX-548, which promises a novel approach to pain management without the addiction risks of opioids. As the year winds down, this biotechnology company is poised to reveal findings from four clinical trials that could catapult VX-548 into the market spotlight.
Needless to say, the stakes couldn't be higher for Vertex.
The commercial success of VX-548, particularly in the chronic pain market, could mark a significant turning point. While generic opioids are cost-effective for short-term use, their potential for addiction and other risks make a non-addictive alternative like VX-548 an attractive proposition for insurers and patients alike.
Drawing parallels to the recent rise of GLP-1 obesity drugs by Eli Lilly (LLY) and Novo Nordisk (NVO), VX-548 could potentially mirror their impact.
Successful trials could see VX-548 generating annual revenues of $5.1 billion by 2030 — a substantial addition to Vertex’s current cystic fibrosis portfolio, which pulls in just shy of $10 billion.
Yet, it's essential to temper enthusiasm with a dose of reality. After all, the biotech sector is no stranger to the pitfalls of high expectations.
Past failures in the nonopioid pain sector underscore the importance of cautious optimism. Nerve growth factor inhibitors, once hailed as a breakthrough, faltered due to safety concerns, highlighting the unpredictable nature of drug development.
VX-548 aims to circumvent these issues with its unique mechanism of action that targets pain signaling at the peripheral nervous system—potentially a significant advantage over central nervous system-targeting opioids.
So, investors must weigh the risk-reward ratio of betting on Vertex ahead of these results.
This treatment’s success in acute pain management could result in a significant uptick in Vertex's stock value. Analyst projections suggest a potential increase of $58 per share if VX-548 matches opioid efficacy, with an $88 increase if it surpasses it. Should the chronic pain trials yield positive results, the stock could climb an additional $119 per share.
However, like I said, it's crucial to approach these numbers with caution. The market's response to trial outcomes can be unpredictable, and the memory of recent high-profile disappointments, such as Biogen's (BIIB) Aduhelm, still lingers.
In light of this, the downside should not be understated — a failed trial could see Vertex's stock take a substantial hit, potentially up to 20%.
Nevertheless, the financial health of Vertex remains strong even sans this pain management candidate. In fact, its top-selling TRIKAFTA/KAFTRIO patents are secured through 2037, accounting for a dominant 89.9% of sales.
This foundation provides a buffer against the inherent risks that come with drug development. With operating margins at a solid 45.6% and a GAAP EPS increase of 30.8% quarter over quarter, Vertex displays a financial resilience that may be reassuring to interested investors.
Taking everything into consideration, investors stand at a crossroads, with the potential of VX-548 offering both promise and uncertainty. The decision to invest now hinges on more than just the outcomes of the trials; it requires strategic consideration of the broader market, potential competitors, and the overarching trends in pain management.
As Wall Street watches with a trained eye, the early indications from Vertex’s trials suggest that VX-548 has a fighting chance to succeed where others have faltered.
If its subsequent tests affirm its potential, VX-548 could not only transform the company’s financial landscape but also mark a significant advancement in the fight against the opioid epidemic — a win for both public health and discerning investors. I suggest you buy the dip.
Mad Hedge Biotech and Healthcare Letter
November 2, 2023
Fiat Lux
Featured Trade:
(SLICING THROUGH DOUBT)
(CRSP), (VRTX), (BLUE), (BEAM), (AMGN), (REGN)
In the intricate world of medical breakthroughs, September 14, 1990, stands out like a sore thumb—or perhaps, a healing one.
On this day, the baseball world was left agog as Ken Griffey Jr. and Sr. knocked out back-to-back home runs, a feat as rare as hen’s teeth.
Meanwhile, in a quieter corner of the planet, a medical marvel was unfolding. Ashanti DeSilva, a 4-year-old with a genetic disorder ravaging her immune system, was about to become the poster child for gene therapy, receiving a groundbreaking treatment that involved a cocktail of modified white blood cells. The aim? To supercharge her immune system and give her a fighting chance at a normal life.
But let’s not sugarcoat it—the road from there to here was anything but a walk in the park. Gene therapy, the promising prodigy of the biotechnology and healthcare sector, had its fair share of teenage rebellion, grappling with safety concerns and delivery vehicle dilemmas. It wasn’t until the early 2010s when gene correction technologies got their act together and safer delivery systems stepped onto the scene, that gene therapy started living up to its potential.
Enter Sickle Cell Disease (SCD), the blood disorder that’s been playing hard to get, affecting around 70,000 Americans and causing everything from anemia to organ damage.
The cure seemed as elusive as a winning lottery ticket until exa-cel, the brainchild of CRISPR Therapeutics (CRSP) and Vertex Pharmaceuticals (VRTX), entered the scene.
This therapy, wielding the mighty CRISPR/Cas9 like a genetic scalpel, takes a patient's stem cells on a rollercoaster ride—harvesting, modifying, and infusing them back into the patient, with the end goal of producing healthy red blood cells.
Looking ahead, CRISPR Therapeutics and Vertex are gearing up for a potential launch of exa-cel in 2024, assuming all the stars align. This innovative gene therapy is poised to be a significant growth catalyst for both companies in the coming decade. Initially, the focus will be approximately 32,000 patients suffering from SCD and TDT.
However, investors need to brace themselves for the price tag, as gene editing therapies don't come cheap. The cost for exa-cel is anticipated to be well north of $1 million, reflecting the complexity and value of this cutting-edge treatment.
At this point, it's crucial to acknowledge that exa-cel is not the only player in this high-stakes game.
A variety of other gene therapies are also vying for the spotlight, with contenders like Bluebird Bio's (BLUE) lovo-cel, Beam Therapeutics' (BEAM) innovative base-edited candidates, and Editas Medicine's (EDIT) competitive CRISPR/Cas9 therapy all in the running.
Now, let’s talk turkey. The financial forecast for exa-cel is looking bright, with CRISPR Therapeutics poised to tap into a $48 billion market opportunity.
Although the treatment has yet to gain FDA approval, the company already has its ducks in a row. It set up 50 treatment centers in the US and 25 in Europe, as well as schmoozed with commercial payers to ensure exa-cel is as accessible as a cold beer on a hot day.
Still, let’s not put on our rose-colored glasses just yet. The biotech sector is as fickle as a cat on a hot tin roof, with CRISPR Therapeutics’ market cap doing the cha-cha in response to industry volatility. With a slew of gene therapies for SCD waiting in the wings, it’s a stark reminder that in biotech, it’s not enough to keep up—you’ve got to lead the pack.
Meanwhile, CRISPR Therapeutics is flexing its muscles with six other clinical trial programs targeting a spectrum of conditions from various cancers to type 1 diabetes, where it is ambitiously seeking a functional cure. With a robust $1.8 billion in cash, equivalents, and marketable securities as of the second quarter and a market capitalization of $3.2 billion, the company is in a strong financial position.
For the astute investors, the real gold is in playing the long game. Rather than getting caught up in the short-term ebbs and flows of the biotech market, the savvy should be pondering how to leverage the current market conditions to their advantage.
After all, CRISPR Therapeutics, with its pioneering gene-editing technology, has the potential to follow in the footsteps of biotech titans like Amgen (AMGN) and Regeneron Pharmaceuticals (REGN), both of which have turned early investments into veritable treasure troves.
Moreover, its financial stability, bolstered by its partnership with Vertex, ensures that funding woes common among smaller biotechs are less of a concern. While it may not be the largest or most prominent player in the biotech arena, the next decade could very well see CRISPR Therapeutics delivering returns that outpace the market. I suggest you buy the dip.
Mad Hedge Biotech and Healthcare Letter
October 3, 2023
Fiat Lux
Featured Trade:
(REDEFINING RESILIENCE)
(VRTX), (ABBV), (AMGN), (JNJ)
It’s not surprising that a number of investors might be hesitant to purchase stocks this month. A few might remember that a number of the most significant stock market downturns in history took place in October. Numerous stocks continue to be high-priced despite the stock market shedding a considerable portion of its earlier momentum from this year.
However, October has often proven to be a favorable month for the stock market — if you know how to choose. Moreover, while a multitude of stocks carry a high price tag, there are those that do not.
Now, imagine a pharmaceutical giant, a singular entity reigning supreme in a market valued in the billions, poised to unveil three revolutionary products by 2025, each potentially generating sales eclipsing $1 billion. Picture this company at the forefront, pioneering a cure for type 1 diabetes (T1D).
The fascinating part? This isn’t a fragment of imagination—it’s the reality of Vertex Pharmaceuticals (VRTX).
With its towering presence in the biotech sector, Vertex has a market cap surpassing $90 billion, establishing itself as the largest entity among U.S.-headquartered firms. Unlike its contemporaries—AbbVie (ABBV), Amgen (AMGN), and Johnson & Johnson (JNJ)—Vertex doesn’t distribute dividends.
Still, it remains one of the most consistent companies thanks to its remarkable trajectory starting from its inception in 1989. Since the advent of the SPDR S&P Biotech ETF (XBI) in 2006, Vertex has soared, achieving over 900% return, overshadowing the ETF’s 380% return.
The journey of Vertex is not just a tale of numbers and percentages; it’s a narrative of resilience and innovation. The company distinguishes itself with its innovative approach to addressing serious diseases, particularly focusing on cystic fibrosis (CF), and its continuous expansion in the CF treatment market.
As expected, the question of whether Vertex is a one-dimensional entity, solely reliant on CF therapies arises. Far from it.
CF isn't anticipated to be the sole catalyst for Vertex's expansion for much longer. The firm, alongside its partner CRISPR Therapeutics (CRSP), is aspiring to secure approval from U.S. regulatory bodies for exa-cel to treat uncommon hematological conditions such as sickle cell anemia and transfusion-reliant beta-thalassemia in the upcoming months. Additionally, the company envisions an imminent market introduction for VX-548, a potent, non-opioid pain medication.
Looking ahead, the future seems even more promising for this major biotech entity. Vertex is conducting a crucial clinical trial on inaxaplin, focusing on APOL1-mediated renal disease, affecting a broader patient demographic compared to CF.
Meanwhile, the financial prowess of Vertex is another facet of its diverse identity. The company has been a consistent beacon of positive free cash flow since 2016, and its financial robustness was highlighted by a 14% revenue growth in the second quarter, driven by robust international sales.
The company’s strategic investments in R&D and commercial capabilities are pivotal to leveraging the multibillion-dollar market opportunities looming on the horizon. These investments are not mere allocations of resources; they are the building blocks of Vertex’s future, the seeds sown today to reap innovations tomorrow.
An excellent example of this is Vertex’s ambitious stride into the type 1 diabetes market, marked by its acquisition of ViaCyte in a $315 million deal. Ultimately, the goal is to deliver innovative stem cell-derived cell replacement therapies as a functional cure for type 1 diabetes.
While the diabetes products are still navigating through phase 2, the anticipation is palpable regarding their role in fueling Vertex’s future growth. The company’s resilience against elevated rates and its propensity to bounce back make it a fascinating stock to consider during market corrections. It’s not just about the numbers on a balance sheet or the ticks on a stock chart; it’s about the relentless pursuit of innovation, and the unwavering commitment to making a difference in the lives of patients around the globe.
So, do these make Vertex the unstoppable stock poised to rule the next two decades? The signs are pointing to a resounding yes.
Mad Hedge Biotech and Healthcare Letter
July 25, 2023
Fiat Lux
Featured Trade:
(THE NEXT INVESTMENT DARLING)
(VRTX), (CRSP)
The pandemic stage lit a blazing spotlight on healthcare and biotech warriors like never before. Some biotech heroes, taking on the gauntlet of grave or lethal ailments, are still setting the market ablaze.
You see, the world's population isn't getting any younger, and diseases aren’t going away. These elements strap an ironclad defense around the healthcare sector - come hell or high water in the economy, the drumbeat of demand won't miss a beat.
One name in the biotechnology and healthcare sector that’s snubbing the bear market's growl with its groundbreaking panaceas is Vertex Pharmaceuticals (VRTX).
Widely considered an underdog in the sector, Vertex has been an overachiever for the past decade, running circles around the market. But guess what? The San Diego champ is also primed to repeat that glorious run over the next ten years.
Vertex has carved out its healthcare niche, nurturing a blossoming franchise in the world of cystic fibrosis treatments. The company's yearly sales are closing in on the dizzying heights of $10 billion.
While other Big Pharma names are staring down the barrel of a patent cliff later this decade, Vertex has shown that it’s playing the long game. Their cystic-fibrosis arsenal is locked and loaded till the mid-2030s at least, and they've got a robust pipeline of drugs chugging along nicely.
Specifically, its blockbuster drug Trikafta won't face the treacherous patent cliff until 2037. With its history of turning the complex task of developing CF therapies into child's play, Vertex has lorded over this territory for over a decade. Adding more feathers to its cap, Vertex is poised to bag fresh non-CF approvals, a move that will spice up its offerings and rev up its growth engine.
Not one to rest on its laurels, Vertex, along with Crispr Therapeutics (CRSP), is on the brink of clinching approval for a gene-editing treatment for red blood cell disorders such as sickle cell disease. The stage is set at this point, and the companies are patiently awaiting the green signal for exa-cel from Uncle Sam and our European friends across the pond.
Exa-cel, the brainchild of Vertex and CRISPR Therapeutics, is a game-changer in gene-editing therapy for rare blood diseases. But this ain't a one-trick pony. Vertex has more rabbits in the hat.
The company's brewing up a storm, geared towards hitting a home run by launching five new products within the next five years. Ambitious? You bet.
As Vertex's cystic fibrosis (CF) business continues to stride forward and the pipeline remains robust, a steady uptick in revenue and earnings is on the cards. The company stands tall, ready to deliver market-beating performances for some time to come.
Sure, the stock is inching closer to its 52-week zenith, but make no mistake - it's still a good catch, even at this high watermark.
Moreover, Vertex is far from being the wallflower at the party. The stock has leaped a whopping 25% this year, outpacing the respectable 18% gain of the S&P 500. The drug giant is the belle of the ball, sporting a market cap north of $90 billion.
Taking a cue from its CF monopoly, Vertex will kick things up a notch, potentially establishing a monopoly in the world of pain relief. The company should get its moment in the spotlight by year's end when clinical trial results for its ground-breaking non-narcotic drug for pain roll out.
Operating under the code-name VX-548, this trailblazer from Vertex intercepts pain signals in the peripheral nerves, outside the central nervous system. The modus operandi is somewhat akin to the topical drug lidocaine, but with a twist.
While lidocaine takes a toll on the heart when ingested, VX-548 sidesteps this issue, making it safe to pop as a pill. Plus, as it doesn't monkey around with the brain, it avoids the slippery slope of opioid addiction. Now, that's what I call a win-win.
Naysayers may voice doubts about the willingness of insurance companies to foot the bill for Vertex's high-priced, newfangled pain drug instead of sticking to the generic opioids or other neurological drugs usually prescribed.
But I don’t buy into the skepticism. There’s a huge possibility that the insurers will. The reason? The mounting political and public relations pressure will essentially force these organizations to back a non-opioid contender, given it can duke it out toe-to-toe in terms of effectiveness.
Given the market for this, VX-548 stands to amass a revenue jackpot of a whopping $5 billion by 2032.
Sure, the healthcare sector isn't going the way of the dodo anytime soon. But rest on your laurels, and you'll be yesterday's news. It's a dog-eat-dog world out there, and if healthcare companies don't stay ahead of the curve, they risk being left in the dust.
This is where Vertex shines as a leader and innovator in its corner of the healthcare world. Keep an eye on this stock. It has the potential to deliver some handsome returns over the next decade.
