Quoin Pharmaceuticals offers value-added drug development through therapies against rare and orphan diseases


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New York, New York – (Newsfile Corp. – December 14, 2021) – PCG Digital – Drug development is complex, time consuming and expensive, and not without risk. It takes years to bring a drug to market, with costs quickly reaching hundreds of millions of dollars, and no promise of success in the end.

Rare and orphan indications constitute an “sweet spot” of R&D for biopharmaceutical companies pursuing drug development. Drugs for rare diseases have a high success rate in obtaining regulatory approval, with around 26% approval, compared to 11% for all other drugs.

Rare diseases affect fewer than 200,000 people and of more than 7,000 rare or orphan diseases, 90% do not involve any treatment approved by the FDA. Medicines entering the market have defined and concentrated patient populations and little or no competition from established therapies. Orphan drug development represents a lucrative opportunity for drug developers and can often be more cost effective than non-orphan therapies.

On the fast track to the FDA

The rare disease market is robust. In 2019, almost half of all FDA approvals (44%) were for orphan drugs. Early and sometimes more frequent discussions with the FDA can help reduce uncertainty about how applications will be received, helping to plan and manage expectations both inside and outside the organization. clinical.

Quoin Pharmaceuticals (NASDAQ: QNRX), a specialty pharmaceutical company that develops treatments for rare skin disorders, is a prime example of a company that has found this sweet spot for R&D. Quoin’s management team has received very constructive feedback from the FDA regarding its lead candidate, QRX003, for Netherton syndrome, and now has a clear path for approval. The favorable position of the FDA has resulted in the possibility of a lower endpoint for a successful clinical outcome. Additionally, QRX003 is potentially eligible for the Rare Pediatric Disease designation and freely redeemable priority review voucher upon approval, which could result in a net cash inflow of approximately $ 100 million. The product also qualifies for a number of expedited approval paths.

Pricing power

Although rare and orphan disease patient populations are smaller in scale, they can be well defined and are often geographically concentrated. Due to the rarity of these conditions, a small number of medical specialists often treat entire groups of patients, and these can be more easily identified and targeted for marketing. As the only treatment available and approved for a specific condition, orphan drugs have higher pricing power and can be very profitable despite smaller markets. The average orphan drug cost per patient of America’s top 100 orphan drugs is nearly 4.5 times the cost of non-orphan drugs, and orphan drug sales are expected to reach $ 217 billion in 2024.

Quoin Pharmaceuticals positions QRX003 as a ‘whole body, life time’ therapy, which means that patients will apply the lotion topically once daily to their entire skin surface for the rest of their lives, providing an opportunity long-term market for the company. Quoin also intends to continue the development of QRX003 for related rare skin disorders for which there is currently no approved treatment or therapy, such as SAM syndrome and peeling skin syndrome.

Provide value

For investors and shareholders, growth, progress and value are essential. Strong FDA commitment, incentive drug development programs, and weak market competition make for an attractive value proposition for investors. With a cash trail of $ 25 million, Quoin has added value beyond drug development, entering into licensing and distribution partnerships to ensure its products are marketed more widely beyond core US markets. and Europeans of society.

Quoin recently entered into an exclusive license and distribution agreement with AFT Pharmaceuticals for the commercialization of QRX003 in Australia and New Zealand. It has also signed an exclusive agreement with GenPharm, a pharmaceutical company specializing in rare diseases, for the MENA region. Other licensing opportunities are actively sought by the company.

Quoin is targeting regulatory approvals for its therapeutic products pipeline in the United States and Europe in 2024, 2025 and 2026 and plans to establish its own business infrastructure for sales and distribution in both geographies.

Finally, Quoin demonstrated its commitment to longer-term R&D opportunities by licensing a new protein from the Queensland University of Technology in Australia.

Market-creating impact

Since 1983, when the Orphan Drug Act changed the face of drug development, rare and orphan drugs have had a market-making impact. Beyond the life-changing, and often saving, attributes of these treatments, the orphan drug designation has a positive financial impact on small drug developers. Tax credits for R&D costs, grants, and market exclusivity all provide an attractive business opportunity for drug developers and create value for investors.

Warning

This communication was produced by PCG Digital Holdings, LLC, a subsidiary of PCG Advisory Inc., (collectively “PCG”). PCG is not a registered or licensed broker or investment advisor. Nothing contained in this communication constitutes an offer to sell, a solicitation of an offer to buy or a recommendation of any security. PCG may be remunerated by respective clients for the publication of information relating to the securities of its clients. See www.pcgadvisory.com/disclosures.

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