Connor Haskins didn't want to make the drive to save his own life anymore...
In 2022, Haskins started feeling faint and had trouble breathing while playing basketball with his friend. He blacked out. And the next thing he knew, he was in a hospital bed.
Doctors diagnosed the then-11-year-old Maine boy with late-onset Pompe disease ("LOPD"). About 1 in 40,000 people suffer from this terminal genetic condition.
People with LOPD lack an enzyme called alpha-glucosidase ("GAA")...
GAA normally breaks down glycogen inside tiny compartments within cells called lysosomes. But without enough GAA, glycogen builds up.
This buildup happens throughout the body. And essentially, it causes muscle weakness and pain. It can also harm the carrier's organs and tissues.
Although no cure exists yet, LOPD is treatable.
But to get the treatment, Haskins and his family had to drive roughly six hours each way from Maine to Boston.
In Boston, Haskins would receive enzyme replacement therapy ("ERT").
These IV infusions gave Haskins the GAA that his body couldn't make. But each ERT session took 13 hours.
Fortunately, Haskins' treatment could soon get easier...
He got a medical port – basically a small, implantable IV – put into his chest. So next year, he hopes to start doing his ERT sessions at home.
Haskins can now do a lot of things that seemed impossible just a couple of years ago. He loves hunting with his family and playing basketball with his friends.
Despite his LOPD, the ERT sessions allow Haskins to live a mostly normal life.
Stories like this show why rare-disease research matters. But there's a big problem...
When a disease only affects 1 in 40,000 people, it's hard to find enough patients to test new treatments. So it's difficult to figure out what works best to fight these disorders.
Biotech companies can't take shortcuts or make exceptions in clinical trials, either...
The technicians conducting the trials need to have the right qualifications. The testing sites need to have the right equipment. And the patients in the study need to be both diverse and medically qualified.
Trials often run at the same time across multiple countries, too. That means navigating multiple regulatory regimes – each with its own requirements for approval.
Because of all that, the cost of each trial is high. And failures or delays are expensive as well.
A Phase III trial currently costs somewhere between $20 million and more than $100 million. The total costs depend heavily on the type of drug, the locations of the trial, and more.
The average daily cost of a trial can be up to $55,000. But industry research shows that if faulty data delays a drug, it could cost an average of $500,000 per day in missed sales.
That's where clinical contract research organizations ("CROs") come in...
In short, CROs serve as the "bridge" between a breakthrough and regulatory approval.
Biotech companies turn to CROs to help solve their problem. The CROs have the networks, the expertise, and the reach to find the patients needed to test new treatments.
They connect with patient registries. They work with specialized medical centers. They coordinate trials across dozens of countries and languages.
Without CROs, most rare-disease treatments would never make it out of the lab.
That's why this type of business should grow significantly in the years ahead...
Last year, estimates put the size of the CRO market at about $85 billion. It's expected to grow to about $175 billion by 2032. That's a compound annual growth rate of nearly 10%.
And when it comes to investing, it ties into a bigger story with biotech right now...
The Power Gauge Still Sees Major Strength for Biotech Stocks
Regular readers will recall that last month, I discussed a changing landscape for biotech...
In the Power Gauge, we can track this corner of the market with the SPDR S&P Biotech Fund (XBI). As I noted last month, the fund is down sharply from its 2021 highs. However, I also pointed out that it had surged since its lows in early April.
The Power Gauge has taken note. And it still sees a lot of strength in this corner of the market right now...
Today, XBI earns a "very bullish" rating in the Power Gauge. In fact, out of 21 market subsectors, it's one of just four with that top-notch grade.
And out of XBI's 130 individual holdings with ratings, 43 are "bullish" or better. So our system sees plenty of strong stocks to choose from in this industry.
Put simply, the Power Gauge still says biotech is a great place to look for opportunities today.
Good investing,
Joe Austin
