Since John Steele, John Richardson, and Jerzy Olszewski first described Progressive Supranuclear Palsy (PSP) in 1964, the neuroscience community has been on the lookout to find a “miraclemolecule” or an “elixir” that can cure patients diagnosed with this debilitating disease. With more than 2,000 research studies published so far, the domain has garnered significant attention from researchers, but there is no cure for terminal illness, so far. As per our research, nearly 15 clinical-stage PSP investigational programs (highlighted below), have been discontinued, in the last two decades. In 2019, owing to the failure to meet the desired endpoints, two big pharma players, Abbvie and Biogen announced their decision to terminate the development of their pipeline anti-tau antibodies for PSP, namely ABBV-8E12 and gosuranemab, respectively.
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Since majority of the trials evaluating potential Progressive Supranuclear Palsy therapeutics candidates were discontinued because of endpoint failure, in-depth research is needed to identify the endpoints that should be measured in these trials. Owing to the high failure rates of PSP to prove efficacy, and the consequent delay in commercialization of a curative therapy, physicians have no option, but to prescribe less effective generic medications, such as antiparkinsonian and anti-depressants, along with non-pharmacological therapies. Driven by the non-availability of a disease modifying therapy, higher number of hospitalizations, outpatient visits, and prescriptions, mean total cost per PSP patient amounts to more than USD 35,000, annually. As a result, the healthcare industry is faced with a pressing need for accurate diagnostic (predictive) tests, and efficient treatment options that have the capability to cure or slow the progression of this neurodegenerative disease. Sponsor companies should follow best practices for disseminating the experience and data resulting from terminated trials in order to facilitate maximum benefit from the investments of trial participants and others who are involved in the clinical trial study.
Nearly 25 investigational therapeutic compounds are currently being evaluated by close to 20 industry and non-industry players
According to our study, the market is still in its infancy with only 20% of the therapies being evaluated in phase II studies, the most advanced stage of development. Owing to their ability to cross the blood-brain barrier, small molecule drugs represent ~70% of the PSP pipeline; this is followed by anti-tau antibodies and antisense oligonucleotide molecules. While majority of the PSP therapy developers are small (with less than 50 employees), this niche market has witnessed the engagement of few large players, including Eisai and Novartis, that are leading the development of phase I clinical programs.
More than 100 clinical trials, related to PSP, have been registered in the last two decades
Stakeholders are actively investigating therapeutic interventions (molecules / devices) and diagnostics for this rare and complex disorder; nearly 50% of these studies are in phase I trials. UCB Biopharma and Molecular Neuroimaging emerged as the key players, that have sponsored multiple trials for evaluating a curative PSP treatment and a diagnostic radiotracer ligand for positron emission tomography (PET), respectively.
Nearly 25 partnerships were established by stakeholders in this domain, since 2015
Maximum deals were inked by PSP therapy developers within the US; these deals were primarily focused on the discovery of therapies targeting neurological disorders.
The PSP therapies market is anticipated to grow at an annualized rate of over 35%, till 2030
Growth in this domain is anticipated to be driven by the lack of competition from generics and consequent steering adoption of high-priced curative treatments. Owing to the greater prevalence of PSP and high prices of therapies in North America, the region is likely to capture a relatively higher market share (68%) in 2030. Within Europe, UK and Germany are likely to emerge as the key markets for PSP therapies.
It is interesting to note that in view of the high unmet patient need, FDA and EMA have fast tracked the approval process of six therapies by granting them orphan designation, which 7-year marketing exclusivity to sponsors of the approved orphan products. The key players that are likely to receive approval of their therapies, in the next decade, include AlzProtect, Retrotope, Woolsey Pharmaceuticals, EmeraMed and Lundbeck. The company that is able to crack the tough nutby navigating through the regulatory check posts, is likely to have the “first-mover” advantage in the PSP therapies market, enabling it to establish a strong brand position and sustain profits over a long period of time.
For detailed insights about this domain, check out our report here.