Ionis Pharmaceuticals (IONS) stock plummeted when GlaxoSmithKline (GSK) stopped pursuing a late stage trial of Ionis-TTR the TTR amyloidosis called familial amyloid polyneuropathy (FAP). This product represents one wagon in Ionis train, which has several wagons representing the firm’s pipeline products. GSK is still occupying one of these wagons known as the familial amyloid cardiomyopathy (FAC).
All the other BIG names riding Ionis’ train are still there, no one abandoned wagon and all of them are contributing knowledge and know how towards perfecting the indispensable technology behind the RNA-based therapeutics. Among those, Roche and Biogen are there, in addition to four other firms and the number of interested firms and institutions are increasing not decreasing.
We kept repeating in the Prohost Letters that antisense therapeutics are extremely hard to achieve, but all experts in the area agree that their successes are indispensable for certain diseases that cannot be treated otherwise. That’s what explains the long time taken to develop antisense products and the exaggerated fear and skepticism that jump up faster than Spiderman when the slightest negative news is announced, causing exaggerated selloffs in the antisense developing firm’s stocks prices.
What happened does not need all the guessing we are reading in investors’ blogs. A few months ago, when the FDA had asked GSK to answer questions about patient-monitoring protocols after reporting thrombocytopenia in the FAP drug, experts in the antisense field, including the FDA knew well that the drug causes thrombocytopenia and they also knew that this adverse effect can be dealt with successfully if the patients are successfully monitored. All the FDA wanted is confirm that GSK representatives at the trials are doing the monitoring homework meticulously and persistently on the candidates taking Ionis-TTR.
Regarding whether there are any important drugs in Ionis’ pipeline that have exciting news, the answer is yes. All the updates coming from the antisense drug nusinersen, which the firm has discovered in collaboration with Dr. Adrian R. Krainer at Cold Spring Harbor Laboratory are positive and exciting. Nusinersen is to treat babies with spinal muscular atrophy, or SMA — a severe motor neuron disease — the leading genetic cause of infant mortality. SMA occurs from a deletion or mutation of a gene responsible for producing a protein critical for normal cellular function.
Nusinersen is designed to increase the production of this protein by modulating the splicing of a closely related gene, thereby compensating for the underlying genetic defect.
All the updates coming from Nusinersen trials are positive and exciting and second to no other treatment on the market or in clinical trials. When the firm began the study, its only hope was to delay the progression of SMA to keep these babies from losing more muscle function and succumbing to respiratory failure.
What is being observed in the trial results now is the study has exceeded by far the firm’s expectations. The infants treated with nusinersen are not only living longer, but also improving. All the infants are continuing the treatment and are becoming toddlers. They are all over the age of 2 and several over 3 years old. The children are also doing things that were never expect from an infant with Type 1 SMA. They are not requiring permanent respiratory assistance.
Of the unexpected results:
– Increase in children muscle function.
CHOP INTEND scores that correlates with the achievements of new and unexpected development milestones.
– Infants are sitting unaided.
Usually, babies diagnosed with Type 1 SMA, who are never expected to sit. As of the latest data analysis in January, even more babies are sitting unaided and guess what? Babies are crawling, standing and even walking.
Bottom line, the RNA-derived drugs are created to treat diseases that no other existing treatments, or approaches are capable to treat. The technology is very hard and the therapeutics are difficult to create as they differ based on the targeted tissue and organ and disease. There are still problems on the road towards perfection, but the in many diseases, the drugs are safe, and in others, the benefits seem to outweigh the risk, which is in many cases early age death.
Negative news, regardless of how minimal they are causes selloff in a market where many investors thrive on stocks’ selloffs. We can do nothing about that. Investors should know this reality and make their own decision, based on their personal circumstances.
This is the picture. We personally believe that the technology will be improved and drugs under development now will be granted approval sooner or later.
This is our personal opinion.
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Advancing the Diagnosis of Underdiagnosed Diseases
The FDA Approved a Safer, Accurate, Cost Effective and Easy to Use Instrument
For the Diagnosis of:
Bone Index Ltd., a research-based company and one of Europe’s top new manufacturers of medical devices was founded in 2011 by researchers from the University of Eastern Finland and based in Kuopio.
In the NEWS, the FDA sent 510(k) clearance to Bone Index Ltd for the osteoporosis instrument Bindex® point-of-care, which safely, effectively and rapidly measures the cortical bone thickness of the tibia. The algorithm calculates the Density Index, a parameter which estimates bone mineral density at the hip as measured with Dual-Energy X-Ray Absorptiometry (DXA), which is the current standard of care for measuring bone density.
The Bindex is pocket-sized instrument that has no radiation and does not require physicians’ referrals, which enables new and cost-effective diagnostic pathways for osteoporosis.
Bindex detects osteoporosis with 90% sensitivity and specificity and will significantly help physicians with a safe, quick, and early diagnosis of osteoporosis, which is expected to improve the management of osteoporosis.
Dr. Janne Karjalainen, Bone Index’ chief technology officer (CTO) said that the FDA clearance process was straightforward despite the fact that Bindex brings to the clinic a new approach. The technical validated was based on extensive clinical evidence and the clinical validation for Bindex has been conducted with a total of over 2,000 patients.
“A recent article published in “Osteoporosis International” presented an approach to how the international fracture risk calculator FRAX and the Bindex test can be used together to identify patients in need of medication. This is extremely important because an estimated 75% of osteoporosis sufferers are undiagnosed and are not taking the the treatment they need,” says Professor Heikki Kröger, MD.
Osteoporosis is underdiagnosed and when diagnosed it is not always correctly managed. As a matter of fact, this disease is yet to be taken seriously in spite of being a debilitating disease with tremendous bad consequences when left untreated.
A major problem is that osteoporosis is still viewed by patients and some physicians as a natural phenomenon that affects most post menopausal women and both old age males and females.
The time has come, though, for osteoporosis to be considered among the diseases that if not taken care of early, they might cause tremendous complications. New guidelines should be put in place for the diagnosis, assessment and treatment of osteoporosis with all the information about its causes, which are numerous and not only restricted to the lack of estrogen in menopausal women.
The diagnostic methods and approaches, including the hormonal testing for the possibility of hyperparathyroidism, for example, continue to be controversial being still disputed by the specialists and so is the use of the sestamibi scans, the standard of care screening for parathyroid nodules that are require to be surgically removed. The same can be said about the ultrasound that accurately diagnose less than half of the patients with hyperparathyroidism that, if not taken care of early enough through a simple surgical procedure, it could devastate not only the skeletal system, but other body systems and organs.
Also, drugs taken in abundance for arthritis pain (steroids) or for atrial fibrillation (warfarin) and other diseases, add to the problem while complicate the cases. In addition to all of the above, eating the wrong food, drinking too much alcohol, smoking and many other bad habits such as not regularly exercising – all add to the problem of bone fragility, which leads to bone fracture.
Indeed, Osteoporosis, is an underdiagnosed and when diagnosed it is still mismanaged, not to say, badly managed. That why osteoporosis is currently responsible for two million broken bones every year in the USA, while costing around $19 billion a year. Experts forecast that by 2025, the costs due to fractures will rise to 25.3 billion dollars.
Currently, one of the biggest challenges in osteoporosis management is the low availability of reliable accurate diagnostics. Bone density scans are mostly performed in hospitals with large DXA X-ray machines that entail high costs. The fact that they are X-ray makes them harmful, in addition to being costly.
We believe the FDA has done a great job approving Bone Index Ltd instrument Bindex® point-of-care. Although this small safe instrument does not solve all the problems surrounding osteoporosis, it is surely a step forward towards bringing better information about the status of people’s bones. This early information can protect the osteoporosis patients against future complications, including bone fracture, and of other hard to treat complications that are not taken care of now.
We intend to follow up on the work this firm is doing, hoping that sooner, or later,Bone Index Ltd might decide to turn public, thus opening the door for investors to benefit and for the early needed treatments to reach the patients.
Will the FDA approve Sarepta’s (SRPT) DMD Drug Eteplirsen?
If not, then which other Duchenne muscular dystrophy (DMD) product the agency believes its early approval could help the children with the disease?
Does the FDA have a drug in mind to early approve, hence satisfy the Congress demand for approval of a DMD drug, and show compassion towards DMD children and their parents’ pleads for early approval of a DMD drug that brings hope to their condemned children.
The answer to these questions will be published in Prohost Letter #396, which will be posted on Friday, May 27 after 8 PM.
In this same issue we will introduce and discuss new firms that have ambitious pipelines and agendas in addition to updating the information regarding Prohost picks.
Prohost Forward-Looking: Material presented here is for informational purposes only. Nothing in this article should be taken as a solicitation to purchase or sell securities. Before buying or selling any stock you should do your own research and reach your own conclusion. Further, these are our ‘opinions’ and we may be wrong. We may have positions in securities mentioned in this article. You should take this into consideration before acting on any advice given in this article. If this makes you uncomfortable, then do not listen to our thoughts and opinions. The contents of this article do not take into consideration your individual investment objectives so consult with your own financial adviser before making an investment decision. Investing includes certain risks including loss of principal.