Disappointed you missed the 2023 Lilly fantastic run this from around 365 to a high of year around 620 ( 255) due to their weight loss drug Tirzepatide positive trials and subsequent approval?

Gather round and listen to a tale of the next Blockbuster Pharma---Vertex. You know what is as big as weight loss? PAIN.

Pain, VX-548, and Breakthrough Designation: Pain is a multi-billion dollar industry and there have been no pain alternatives for decades. The race for a Non-Addictive Opioid Alternative is akin to the search for the Holy Grail. Vertex has developed a selective NaV1.8 inhibitor pain drug VX-548. It is being trialed for moderate to severe acute pain and diabetic peripheral neuropathy (different indications/different trials). The FDA granted Vertex Breakthrough Therapy Designation for their Pain drug VX-548 (a Non-addictive Opioid Alternative) based on clinical evidence from the Phase 2 studies demonstrating the potential to treat moderate to severe acute pain. Vertex is concluding Phase 3 VX-548 moderate to severe pain studies this month.

Opiod Problem: The opioid epidemic continues to ravage the U.S., with 50,000 people dying each year from overdose, and 10 million abusing the highly addictive medications annually. The U.S. Department of Health and Human Services (HHS) officially declared opioid abuse a public health emergency in 2017, but until there is a safe and effective alternative to opioids, acute and chronic pain present a silent scourge of their own. “There is an immense unmet need for new pain medications, even without the opioid epidemic,” Stephen Waxman, a Yale neuroscientist who penned another editorial accompanying the recent NEJM study, told BioSpace. (Reference: https://www.biospace.com/article/promising-vertex-pain-med-points-to-new-era-for-analgesics/)

Detail on VX-548 Phase 3 Pain Trials: The Phase 3 program has two double-blind, randomized, placebo-controlled trials to assess the safety and efficacy of VX-548 for moderate to severe acute pain treatment after bunionectomy or abdominoplasty surgery. These trials also have a hydrocodone bitartrate/acetaminophen treatment arm. A third, single-arm trial assesses the safety of VX-548 across various other moderate to severe acute pain types for up to 14 days.

Major Catalyst Coming: Phase 3 trials for VX-548 conclude this month with readout in Q1 FY24 (January to March timeframe). Successful P3 trials will be a huge forward catalyst leading to the company moving towards drug approval.

What has the stock been doing: In addition to moderate to severe pain trials, Vertex just concluded and provided a read-out from a positive P2 on VX-548 for diabetic peripheral neuropathy which caused the stock to increase from around 360 (day before announcement) to around 409 ( 49) as of today where it is holding steady. Notably-it has been holding steady in the lower 400s since the day of the P2 Announcement.

Various Articles about potential of VX-548:

Barrons: https://www.barrons.com/articles/vertex-pain-pill-non-opioid-573cae68

· A nonaddictive pain pill that works as well as an opioid, with none of the same addiction risk and side effects, would be an instant blockbuster. Drug companies have met with repeated failures in their efforts to develop one, but Vertex Pharmaceuticals (ticker: VRTX) could be close to a breakthrough.

· If the trials are successful, sales of VX-548 could hit $5.1 billion a year by 2030, according to Leerink Partners analyst David Risinger. That would be transformative for Vertex, which currently sells treatments for cystic fibrosis and records annual sales of under $10 billion.

· In a note out in early October, Goldman Sachs analyst Salveen Richter wrote that if VX-548 appears to work as well as an opioid in the Phase 3 trials focused on acute pain, Vertex shares, recently $362, would be worth an additional $58 each. If the drug works even better than opioids, she writes that it would be worth an additional $88 per share. In a best-case scenario—if the chronic-pain study is also positive—Richter estimates that the stock would be worth an additional $119 per share. Richter is less concerned about the downside risk if the trials fail. She says the market currently isn’t giving Vertex much credit for its pain program, and that the company’s cystic fibrosis franchise alone is worth $326 per share. Leerink’s Risinger is similarly undeterred by the risk. “It’s possible the stock could be down close to 20%” if all the trials fail, he says. “But the upside, if there’s success in those three trials, is quite significant.”

Other reasons to buy from Motley Fool: https://www.fool.com/investing/2023/12/27/3-reasons-to-buy-vertex-pharmaceuticals-stock-like/

· Rock Solid Revenue Base

· Groundbreaking Gene Therapy just got Approved. Note the Approval for Sickle Cell just happened with CRSP and approval for BT has a PDUFA date of March 30. This could be another large catalyst sending the stock forward.

· Plenty of Cash

Positions: This is an expensive stock, so you might have to borrow from your wife’s boyfriend. Shares are the easiest way to get into the stock but for leverage and significant returns the April 19 450Cs are tasty if you want safety of the full Q1 (Jan to March timeframe). If you think the readout could come quicker than you could try your luck with an earlier date such as Feb 16. Alternatively, if you think P3 is going to be a bust the April 19 350P will suit you well. Either way my personal theory is we are in for a move of 50 either way (positive or negative). I’m for the positive side and think P3 will be successful. Lastly, a few things to remember that can also pump the stock-the CEO is speaking at the JP Morgan Conference on Jan 8th and we have a Gene Therapy approval for BT could come in Q1 as well. Good luck to you all.