Why Pharmaceutical R&D Global Spending Could Exceed $200 Billion by 2024

PALM BEACH, Florida, Dec. 11, 2019 /PRNewswire/ -- According to various industry and market publications, pharmaceutical companies spend, on average, 17% of revenues on R&D, making it one of the biggest spenders in this area. Outside of the semiconductor industry, no other industry spends more on R&D. A quick survey of other industries by Investopedia, clearly shows how much most of them are outspent in R&D by pharmaceutical companies. The overall average spending on R&D by industries engaged in developing new products is a mere 1.3% of sales revenues. The chemicals sector, one of the larger R&D sectors, spends an average of 2 to 3%. Aerospace and defense firms, although they do a great deal of research and development work, only dedicate about 4 to 5% of revenues to R&D spending.  Investopedia says that, in fact, the pharmaceutical industry's lifeblood is R&D. The success of major drug companies is almost wholly dependent upon the discovery and development of new medicines, and their allocation of capital expenditures reflects that fact. "Although the average spending is 17% of revenues, some companies spend substantially more." The report continued: "As of 2019, many of the largest pharmaceutical firms spend nearly 20% on R&D. Of the 20 largest R&D spending industries in the world, the pharmaceutical industry makes up nearly half the list… Many smaller pharma companies have lower revenue totals; so, they often spend significantly higher percentages of their budget on R&D – up to 50% for some firms.  Active biotech and pharma companies in the markets this week include CNS Pharmaceuticals, Inc. (NASDAQ: CNSP), Agios Pharmaceuticals, Inc. (NASDAQ: AGIO), Proteostasis Therapeutics, Inc. (NASDAQ: PTI), Eli Lilly and Company (NYSE: LLY), Sangamo Therapeutics, Inc. (NASDAQ: SGMO).

Another report says that: "The pharmaceutical industry's activities have significant socio-economic impacts on society in the form of investments in R&D and manufacturing. Of note, R&D is the "backbone" for success in any drug discovery program. Thus, the extent of pharma R&D spending serves as an important metric to show a company's commitment to finding new drugs. At present, the global pharmaceutical industry has the second highest R&D intensity (expenditure as a share of sales) measures of any sector…"   The report also indicates that overall R&D spending is expected to grow by 3% each year, reaching roughly $203.9 USD billion by 2024.

CNS Pharmaceuticals, Inc. (NASDAQ: CNSP) BREAKING NEWS:  CNS' Berubicin Awarded $5.8 Million Non-Dilutive Grant from EU/Polish National Center Through its Development Partner - CNS Pharmaceuticals, a biotechnology company specializing in the development of novel treatments for brain tumors, announced that its partner, WPD Pharmaceuticals ("WPD"), a Polish corporation founded by Dr. Waldemar Priebe, the founder of the Company, was awarded a $5,798,875 grant from the EU/Polish National Center for Research and Development under the Smart Growth Operational Program 2014-2020 (the "EU Grant").

The Company previously entered into a sublicense agreement with WPD, in which WPD committed to a $2.0 million minimum expenditure on the development, testing, regulatory approval, and commercialization of Berubicin over a three –year period. WPD's subsequent development project, "New approach to glioblastoma treatment addressing the critical unmet medical need" (the "WPD Project"), received the grant co-financed by the European Union under the Smart Growth Operational Program 2014-2020, Priority I: Support for Research and Development work by Enterprises, Measure 1.2: Sectorial Research and Development Programs, Sectorial Program InnoNeuroPharm. The main goal of the WPD Project is to implement the world's first multicenter pediatric Phase I clinical trial to determine maximum tolerated dose (MTD) and Phase IB and II clinical trials in adults, to assess the efficacy of Berubicin. Proceeds from the grant will be used independently by WPD Pharmaceuticals in order to fulfill its commitment arising from its sublicense agreement with the Company.  

"The EU Grant represents an important milestone for the advancement of our novel anthracycline, Berubicin, especially in pediatric settings," commented CEO of CNS, John M. Climaco. "We believe this grant will allow our partner, WPD Pharmaceuticals, to fulfill its commitment arising from its sublicense agreement with us in a manner that is capital-efficient and non-dilutive to the CNS. Additionally, this EU Grant and our partnership with WPD presents an opportunity to potentially accelerate our efforts to bring Berubicin to patients around the world."   Read this and more news for CNSP at:  https://www.financialnewsmedia.com/news-cnsp/

Other recent developments in the biotech industry include:

Agios Pharmaceuticals, Inc. (NASDAQ: AGIO), a leader in the field of cellular metabolism to treat cancer and rare genetic diseases, reported new data from the extension phase of the DRIVE PK Phase 2 study of mitapivat (AG-348) in adults with pyruvate kinase (PK) deficiency at the 2019 American Society of Hematology (ASH) Annual Meeting. Mitapivat is an investigational, first-in-class, oral, small molecule allosteric activator of wild-type and a variety of mutated pyruvate kinase-R (PKR) enzymes that directly targets the underlying metabolic defect in PK deficiency, a rare, potentially debilitating, hemolytic anemia. In addition, data was shared from the Agios-sponsored Natural History Study of PK deficiency that detailed the comorbidities and complications associated with the disease and the impact of transfusion history.

"DRIVE PK was the first clinical trial aimed at addressing the metabolic defect in PK deficiency, and demonstrated that clinically meaningful and robust increases in hemoglobin can be achieved with an oral PKR activator," said Eduard J. van Beers, M.D., Ph.D., University Medical Center Utrecht and an investigator in the study. "These new data demonstrate that chronic treatment with mitapivat is well tolerated and can lead to a reduction in hemolysis as demonstrated by sustained improvements in hemoglobin and other markers for more than three years."

Proteostasis Therapeutics, Inc. (NASDAQ: PTI), a clinical stage biopharmaceutical company dedicated to the discovery and development of groundbreaking therapies to treat cystic fibrosis (CF), today announced positive, initial ex-vivo results of PTI's proprietary cystic fibrosis transmembrane conductance regulator (CFTR) modulators, PTI-801, PTI-808, and PTI-428, in individuals with CF who are ineligible for the current standard of care CFTR modulator therapies due to their genotype. The data are part of a pan-European strategic initiative, known as HIT-CF (Human Individualized Therapy of CF), which seeks to accelerate the development of, and access to, personalized therapies for CF patients, beginning with those for whom no currently approved CFTR modulator therapy is indicated.  

HIT-CF is sponsored by the European Commission Horizon 2020 program, in which CF-Europe, a patient organization representing more than 48,000 individuals with CF, collaborates and with the European Cystic Fibrosis Society Clinical Trial Network (ECFS-CTN), which is recruiting adult CF patients into the ex- vivo ­study through its 43 clinical trial centers.  HIT-CF collects tissue samples from CF patients and develops organoids, or miniaturized organs, that are genetically identical to the patient donor, and share the same micro-anatomy as the organ from which they were derived.

Eli Lilly and Company (NYSE: LLY) today announced the opening of the LIBRETTO-431 clinical trial [NCT04194944] for selpercatinib, also known as LOXO-292, for treatment-naïve RET fusion-positive non-small cell lung cancer (NSCLC) patients. Enrolled trial participants will be randomized to receive either selpercatinib or platinum-based (carboplatin or cisplatin) and pemetrexed therapy with or without pembrolizumab as initial treatment of their advanced or metastatic RET fusion-positive NSCLC.

"Given the remarkable results of the LIBRETTO-001 trial, I am excited to open this important Phase 3 trial of selpercatinib, a highly selective and potent molecule that has previously demonstrated sustained responses with a well-tolerated safety profile," said Professor Ben Solomon, principal investigator at the Peter MacCallum Cancer Centre in Melbourne Australia. "This trial endeavors to generate outcome data that place patients with RET fusions alongside those with EGFR mutations and ALK fusions, as driver-positive populations that should be treated with targeted therapies in the first-line setting, rather than chemoimmunotherapy."

Sangamo Therapeutics, Inc. (NASDAQ: SGMO), a genomic medicine company, announced preliminary results from the first three patients treated in the Phase 1/2 THALES study evaluating investigational ST-400 ex vivo gene-edited cell therapy in transfusion-dependent beta thalassemia (TDT). The data are featured in a poster presentation on December 9, 2019 at the 61st Annual Meeting of the American Society of Hematology (ASH) in Orlando, FL. The ST-400 ASH poster will be available on Sangamo's website in the Investors and Media section under Events and Presentations at the beginning of the poster session at 10am Eastern Time.

"The prompt hematopoietic reconstitution and on-target indels in circulating white blood cells observed in the three patients treated with ST-400 indicate successful editing with zinc finger nuclease technology," said Angela Smith, MD, Associate Professor in the Division of Pediatric Blood and Marrow Transplantation at the University of Minnesota, and a Principal Investigator of the THALES study. "As is the case in other myeloablative conditioning studies of stem cell transplants for beta thalassemia patients, the full effects of the treatment may take as long as 12 to 18 months or more to manifest. Longer-term follow-up, including from additional patients, will be necessary to understand the safety profile and potential clinical benefit of ST-400 in beta thalassemia. The emerging tolerability and safety profile of ST-400, as well as the induction of fetal hemoglobin and presence of indels, suggest that further exploration of this novel gene-edited cell therapy is merited."

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