CRDL:CC - Surging Biotech M&A Reaffirms Massive Opportunity In Rare Disease Drug Development
(NewsDirect)
Compared to theirnon-orphan counterparts, orphan drugs have consistently outpacedgrowth and have become a major part of pharma’s mainstream business.The U.S. FDA's orphan drug designation, established under theOrphan Drug Act of 1983, encourages the development of treatments forrare diseases affecting fewer than 200,000 people in the U.S. byproviding incentives to offset the small market size.
Last year, sales fromorphan drugs soared to $168 billion, accounting for about 17% of theindustry’s total and just shy of the entire oncology therapeuticcategory at $194 billion. This year, these drugs are on track togenerate an impressive $185 billion and are projected to hit around$270 billion by 2028.
For such a large cohort of drugs to still be growing at adouble-digit pace is impressive and shows that continued investmentinto the unmet need of rare diseases is warranted.
That explains why therehas been a surge in M&A activity among orphan drug developers overthe past couple of years.
For instance, when novel cannabidiol-based drugEpidiolex successfully treated rare forms of child onset epilepsy, GWPharmaceuticals more than doubled its market value. The 120-patienttrial showed patients taking Epidiolex achieved a median reduction inmonthly convulsive seizures of 39% compared with a reduction onplacebo of 13%.
GW Pharmaceuticals went on to be acquired by JazzPharmaceuticals for $7.2 billion in 2021, representing a premium ofapproximately 50% over GW's closing stock price at the time.
Clearly, there is asignificant opportunity in orphan drug development, which is why thesefour stocks have been generating substantial investor interest.
Cardiol Therapeutics(NASDAQ:CRDL) is a Canadian biotech drug developer focused ondeveloping their novel ultrapure cannabidiol formulation,CardiolRx™, for rare inflammatory heart diseases, specificallyrecurrent pericarditis and acute myocarditis.
Pericarditis ischaracterized as inflammation of the pericardium, layers of tissuethat surround the heart, that affects about 165,000 people in the U.S.annually and has no first-line FDA-approved treatment. Among thosetreated for acute pericarditis, 15% to 30% may experience recurrentpericarditis.
Currently, anti-inflammatory drugs such as colchicine areprescribed for pericarditis treatment in cases of chronic or recurrentpericarditis. If the patient with pericarditis disease does notrespond to anti-inflammatory medications, corticosteroids such asprednisone are prescribed.
More recently, Kiniksa Pharmaceuticals’ARCALYST® (rilonacept) became the first and only FDA-approvedtreatment indicated for the treatment of recurrent pericarditis andthe reduction in risk of recurrence in adults and children 12 yearsand older.
ARCALYST is a weekly subcutaneous injection, whereasCardiolRx™ is an oral drug taken twice daily which is a majoradvantage as the idea of weekly injections could not be as appealingto patients.
Cardiol Therapeutics’ (NASDAQ:CRDL) pre-clinicalmodels showed that by inhibiting activation of the NLRP3 inflammasomepathway, cannabidiol may help resolve the symptoms ofpericarditis.
This approach has already demonstrated incredible potential, asthe company was granted Orphan Drug Designation (ODD) by the U.S. FDAfor the treatment of pericarditis, which includes recurrentpericarditis.
Cardiol Therapeutics followed this major achievement withcompleting enrollment in its Phase 2, open-label MAvERIC-Pilot studyinvestigating the tolerance, safety, and effect of CardiolRx™ inpatients with recurrent pericarditis. The primary endpoint of thetrial is the change in patient-reported pericarditis pain using an11-point numeric rating scale (NRS) from baseline to week 8 withtopline results expected in early June.
Not only that, Cardiol Therapeutics recentlypresented its concurrent Phase II ARCHER trial design at the annualcongress of the Heart Failure Association of the European Society ofCardiology. This demonstrated the high level of interest from thecardiology community in novel approaches to treat acute myocarditis,for which there are currently no therapies approved by European and USregulatory authorities.
The ARCHER trial is expected to enroll 100 patients atpre-eminent cardiovascular research centers in the United States,Canada, France, Brazil, and Israel. Patient recruitment has beenaccelerating due in large part to the growing global awareness ofmyocarditis, and ARCHER has already exceeded 85% of targetenrollment.
Cardiol Therapeutics (NASDAQ:CRDL) expects that resultsfrom the ARCHER trial will assist in furthering its understanding ofthe therapeutic potential of CardiolRx™ and will complement the moreadvanced MAvERIC-Pilot Phase II study in patients with recurrentpericarditis.
Just to put the opportunity here in context, Future MarketInsights expects that the worldwide pericarditis treatment market islikely to be worth $ 6 billion by 2032. ARCALYST’s sales furtherreaffirm this considering that the drug’s 2023 net revenue grew 90%year-over-year to $233.1 million.
Several analysts have maintained their positiveoutlook on the company and have high hopes that the June data willfurther prove CardiolRx™ efficacy in treating recurrentpericarditis. In fact, Joe Gantoss of Chimera Research Group notes,“I won’t be surprised to see the price breaking out the 3-yearhigh at $4.96 if the recurrent-pericarditis data show a clear successand open the path to move to the next stage with Phase-3 trial,”while analysts at H.C Wainwright & Co. issued a $9 price targetfor the stock.
Novo Nordisk (NYSE:NVO) announced that it was buyingPhase II-stage heart failure drug developer Cardior Pharmaceuticals(private) for up to $1.11 billion as it moves to strengthen itspipeline of drugs to treat cardiovascular disease and expand intoareas outside of its core diabetes and weight-loss markets.
The Denmark-basedpharmaceutical giant is enjoying blockbuster success for its Wegovyand Ozempic obesity and diabetes treatments, which aren’t onlyproving highly effective at regulating blood sugar and helpingpatients lose weight, but are also yielding extra health benefits suchas cutting the risk of stroke and heart attacks and slowing theprogression of kidney disease.
Based on these findings, the company recentlyunveiled plans to build a portfolio of therapies in areas likecardiovascular diseases and emerging-therapy areas while strengtheningits progress in the rare-disease pipeline.
CDR132L, Cardior’s lead compound, isdesigned to halt and reverse detrimental cardiac remodeling followingmyocardial infarction (MI) as the first-ever ncRNA-based therapy toenter clinical trials in heart disease. It is currently beinginvestigated in the Phase 2 HF-REVERT clinical trial in 280 post-MIpatients with cardiac dysfunction at more than 60 locations acrossEurope.
AstraZeneca (NASDAQ:AZN) also announced that it hasentered into a definitive agreement to acquire Amolyt Pharma, aclinical-stage biotechnology company focused on developing noveltreatments for rare endocrine diseases for $800 million. The deal alsofeatures $250 million in milestones tied to a regulatory event.
The proposedacquisition will bolster the Alexion, AstraZeneca Rare Diseaselate-stage pipeline and expand on its bone metabolism franchise withthe notable addition of eneboparatide (AZP-3601), a Phase 3investigational therapeutic peptide with a novel mechanism of actiondesigned to treat hypoparathyroidism.
In patients with hypoparathyroidism, adeficiency in parathyroid hormone (PTH) production results insignificant dysregulation of calcium and phosphate, which can lead tolife-altering symptoms and complications, including chronic kidneydisease. It is one of the largest known rare diseases, affecting anestimated 115,000 people in the US and 107,000 people in the EU,approximately 80% of whom are women.
Eneboparatide is designed to produce sustained andstable levels of calcium, which falls to low levels in patients withhypoparathyroidism, while preventing kidney disease and restoring boneturnover.
Sanofi (NASDAQ:SNY) earlier this year agreed to buy theU.S. biotech Inhibrx Inc. for as much as $2.2 billion, giving theFrench drugmaker a potential therapy for a genetic disorder thataffects the lungs and liver.
Inhibrx shareholders will get $30 per share as partof the deal, along with the right to receive $5 in cash if theexperimental drug meets a regulatory milestone.
“The addition ofINBRX-101 as a high potential asset to our rare disease portfolioreinforces our strategy to commit to differentiated and potentialbest-in-class products,” said Houman Ashrafian, Sanofi’s head ofresearch and development. “With our expertise in rare diseases andgrowing presence in immune-mediated respiratory conditions, INBRX-101will complement our approach to deploy R&D efforts in key areas offocus.”
Disclaimers: RazorPitch Inc. "RazorPitch" isnot operated by a licensed broker, a dealer, or a registeredinvestment adviser. This content is for informational purposes onlyand is not intended to be investment advice. The Private SecuritiesLitigation Reform Act of 1995 provides investors a safe harbor inregard to forward-looking statements. Any statements that express orinvolve discussions with respect to predictions, expectations,beliefs, plans, projections, objectives, goals, assumptions, or futureevents or performance are not statements of historical fact may beforward looking statements. Forward looking statements are based onexpectations, estimates, and projections at the time the statementsare made that involve a number of risks and uncertainties which couldcause actual results or events to differ materially from thosepresently anticipated. Forward looking statements in this action maybe identified through use of words such as projects, foresee, expects,will, anticipates, estimates, believes, understands, or that bystatements indicating certain actions & quote; may, could, ormight occur. Understand there is no guarantee past performance will beindicative of future results. Investing in micro-cap and growthsecurities is highly speculative and carries an extremely high degreeof risk. It is possible that an investors investment may be lost orimpaired due to the speculative nature of the companies profiled.RazorPitch has been retained and compensated by Cardiol Therapeuticsto assist in the production and distribution of content related toCRDL. RazorPitch is responsible for the production and distribution ofthis content. It should be expressly understood that under nocircumstances does any information published herein represent arecommendation to buy or sell a security. This content is forinformational purposes only, you should not construe any suchinformation or other material as legal, tax, investment, financial, orother advice. Nothing contained in this article constitutes asolicitation, recommendation, endorsement, or offer by RazorPitch orany third party service provider to buy or sell any securities orother financial instruments. All content in this article isinformation of a general nature and does not address the circumstancesof any particular individual or entity. Nothing in this articleconstitutes professional and/or financial advice, nor does anyinformation in the article constitute a comprehensive or completestatement of the matters discussed or the law relating thereto.RazorPitch is not a fiduciary by virtue of any persons use of oraccess to this content.
ContactDetails
RazorPitch Inc
Mark McKelvie
+1585-301-7700
CompanyWebsite
Copyright (c) 2024 TheNewswire - All rights reserved.