Developing Drugs Is Expensive: How Biopharmas Hope to Change That

Perhaps unsurprisingly, the average cost of developing new drugs is very high. For pharmaceutical companies that are developing multiple drugs, the R&D costs can average around $5 billion. This is concerning for pharma companies, especially considering that returns on R&D are the lowest they’ve been in the past nine years.

However, there are more investments needed for drug development other than pure cash, and these investments need to be taken into account as well. Time, money, and the quality of development are the three things that every biopharma company must balance in order to stay profitable. Luckily, there may be some new advancements and changes on the horizon that could help with this careful balance by improving overall quality and reducing costs.

• Big data

The term “big data” simply refers to very large sets of data that requires a data-processing application to analyze fully. Once this data is analyzed, it can reveal many interesting statistical patterns and associations that would be helpful for those in biopharma R&D.

Through the right use of big data analyzation, biopharma companies could see not only more efficient drug development, but also more effective treatments that are personalized to target specific sections of the population rather than developing a single drug to treat everyone.

Personalized medicine, in particular, has become a big focus for those in the biopharma industry the past few years as these treatments are incredibly effective and lead to better outcomes in patients, thus meaning more successful drugs can be developed.

Along with personalized treatments, big data can help with almost every facet of drug R&D. The benefits include finding new targets for treatment, validating mechanisms of action, providing more information on safety, predicting the effects the medicine will have, and much more.

• Automation

One of the biggest things making the news as of late is how automation is going to drastically and permanently alter all kinds of industries. This will be no different for biopharma, and in the not-too-distant future, the drug R&D landscape may be forever changed.

Specifically, automation will make drug development much faster and more efficient by freeing up researchers’ time and automating a lot of the regular processes and menial routines in the lab. Robots will be able to handle and transport liquids and run tests, and scientists will have more free time to focus on design and deciphering test results. This is something that many biopharma companies are already investing in and they will only increase this investment in the coming years as automation grows more sophisticated.

According to the CEO of HighRes Biosolutions, Peter Harris, automation not only helps cut costs and speeds up drug development, but the data that it captures is more accurate and higher quality than with humans doing the work. This translates to fewer mistakes made during the research process and better results overall.

• Patient engagement

One of the biggest changes throughout the field of medical research as of late has been patient engagement. Like other parts of medicine, patient engagement has already started making big changes for biopharma companies.

There is even an entire movement around patient engagement that is spearheaded by patients like Dave deBronkart, along with many physicians, researchers, and others in the medical field who also are in favor of more patient engagement.

So, it’s clearly a big deal, but how is patient engagement changing the way biopharma approaches research?

By implementing more and more patient engagements, not only do researchers help improve trust and involvement with patients, but they also streamline the R&D process in multiple ways. This includes making patients more eager to join clinical trials and give feedback, getting a better insight into the full effects of the drug and how it is impacting the patient, and discovering relevant designs for medication that patients would find useful. All of these help speed up the drug discovery and development process as well as reduce costs by improving the clinical trial process.

Despite all of these advantages, patient engagement is still a low priority during clinical trials and biopharma companies are taking a while to pick up on this trend. However, companies are still slowly adapting to it and it may not be long until it’s a standard procedure for every company’s R&D.

https://www.biospace.com/article/developing-drugs-is-an-expensive-process-how-do-biopharma-companies-hope-to-change-that-/

Sanofi granted U.K. marketing authorization for TIV High Dose

U.K. marketing authorization has been granted for Sanofi Pasteur’s Trivalent Influenza Vaccine High Dose, which is indicated for active immunisation of people 65 years of age and older against influenza disease. The decision means the U.K. is the only European country where TIV High Dose is approved, Sanofi said in a statement. TIV High Dose is included in the Joint Committee on Vaccination and Immunisation advice on influenza vaccines for the 2019/20 influenza season. TIV High Dose is already approved for people 65 years of age and older in the United States, Canada, Australia and Brazil.

https://thefly.com/landingPageNews.php?id=2851247

ResMed 1st Top-of-Head CPAP Mask, AirFit N30i, Now Available across U.S.

• AirFit N30i adds a top-of-head mask-to-tube connection to ResMed’s expanding CPAP mask portfolio, helps the wearer move and sleep in any position
• New “cradle” cushion sits under the nose, designed to reduce facial markings and irritation

ResMed (NYSE: RMD, ASX: RMD), today introduced AirFit N30i, its first top-of-head-connected nasal CPAP mask, across the United States, with a newly designed nasal cradle cushion that sits just under the wearer’s nose.

The top-of-head connection keeps tubing out of the wearer’s way, letting them move and sleep in any position. The nasal cradle cushion – ResMed’s first – is designed to reduce facial markings and irritation.

AirFit N30i is also convenient for equipment providers and sleep labs – it fits 96 percent of PAP users with just two frame sizes and four cushion sizes.

In side-by-side comparisons, three out of four users preferred AirFit N30i over the other nasal mask with a top-of-head connection. A majority stated they loved AirFit N30i and would take it home as their mask of choice, with many claiming it was “very comfortable,” it’s “easy to use,”or that it “caused little disruption to their bed partner.”

“AirFit N30i is a great choice for nasal mask wearers who move when they sleep,” said Jim Hollingshead, president of ResMed’s Sleep business. “It’s all about freedom – users are free to sleep however they want and enjoy a comfortable, reliable seal. And AirFit N30i is so easy to fit, sleep professionals are going to be freed up to serve more people.”

AirFit N30i is also available in Canada, Australia, New Zealand, and most of Europe, with other countries planned to follow later this year.

https://www.marketscreener.com/RESMED-14259/news/ResMed-s-First-Top-of-Head-CPAP-Mask-AirFit-N30i-Now-Available-across-U-S-27894363/

Kura Reports Clinical Activity of Med in Pancreatic Cancer

Kura Oncology, Inc. (Nasdaq: KURA), a clinical-stage biopharmaceutical company focused on the development of precision medicines for oncology, reported new findings identifying a potential association between CXCL12 expression and clinical benefit in patients with pancreatic cancer treated with tipifarnib.

These findings are being presented today at the 2019 Gastrointestinal Cancers Symposium in San Francisco. A copy of the poster is available on Kura’s website at http://www.kuraoncology.com.

Tipifarnib has been shown to downregulate CXCL12, and previously reported data support tipifarnib as a CXCL12/CXCR4 pathway inhibitor. Elevated CXCL12 expression is known to be a poor prognosis factor in patients with pancreatic, Iung and esophageal-gastric cancers. In the specific context of pancreatic cancer, high CXCL12 expressing tumors may evade early diagnosis by decreasing abdominal pain through the attraction of pain-suppressing Schwann cells.

To investigate the potential for association between CXCL12 expression and clinical benefit in pancreatic cancer patients, Kura conducted a retrospective analysis of study INT-11, a randomized, placebo-controlled Phase 3 study of gemcitabine plus tipifarnib compared with gemcitabine plus placebo in patients with advanced pancreatic cancer.

A total of 688 pancreatic cancer patients were enrolled in study INT-11, of whom 155 reported no abdominal pain at study entry. Although no differences in survival were observed in the overall study, the absence of patient-reported abdominal pain at study entry was associated with higher median survival in the tipifarnib plus gemcitabine arm (10.2 months vs. 5.9 months, HR=0.52, p

https://www.marketscreener.com/KURA-ONCOLOGY-INC-24827079/news/Kura-Oncology-Reports-Clinical-Activity-of-Tipifarnib-in-Subsets-of-Pancreatic-Cancer-Associated-w-27894656/

Editas Publishes Data on Congenital Condition Therapy Progress

Editas Medicine, Inc.(Nasdaq: EDIT), a leading genome editing company, today announced the journal Nature Medicine published the comprehensive, pre-clinical data demonstrating the pharmacology and specificity of EDIT-101, an experimental, CRISPR genome editing medicine being investigated for the treatment of Leber congenital amaurosis 10 (LCA10), a genetically-driven form of blindness. EDIT-101 is set to be the first in vivo, or editing inside the body, CRISPR-based medicine administered to people anywhere in the world.

“We are thrilled Nature Medicine published our paper sharing the comprehensive set of pre-clinical data for EDIT-101 and our approach to creating CRISPR-based genome editing medicines,” said Charles Albright, Ph.D., Chief Scientific Officer, Editas Medicine. “The research presented in this important manuscript provided the foundation for our recently accepted Investigational New Drug (IND) application for EDIT-101 to treat LCA10, bringing us closer to the clinic and our goal of realizing the full potential of genome editing. We look forward to initiating the Phase 1/2 study to further evaluate the safety, tolerability, and efficacy of EDIT-101 for people living with LCA10, a disease currently with no treatment options.”

Published results detail the development of EDIT-101, an experimental genome editing medicine designed to remove the abnormal splice donor created by the IVS26 mutation in the CEP290 gene found in LCA10 patients and restore normal CEP290 expression. The paper summarizes in vitro experiments in human cells and retinal explants demonstrating the molecular mechanism of action and nuclease specificity.

Subretinal delivery of EDIT-101 in humanized CEP290 mice showed rapid and sustained CEP290 gene editing. A comparable surrogate non-human primate (NHP) vector also achieved productive editing of the NHP CEP290 gene at levels that met the target therapeutic threshold and demonstrated the ability of CRISPR/Cas9 to edit somatic primate cells in vivo. The results presented support further development of EDIT-101 for the treatment of patients with LCA10, as well as the application of genome editing approaches to treat a wide variety of inherited retinal diseases.

In the Phase 1/2 clinical trial, Editas Medicine and Allergan Pharmaceuticals International Limited (Allergan) plan to initiate patient screening mid-year and begin patient dosing in the second half of 2019, enrolling 10-20 patients in the U.S. and Europe.

https://www.marketscreener.com/EDITAS-MEDICINE-INC-26123639/news/Editas-Medicine-Announces-Publication-in-Nature-Medicine-of-Data-Supporting-the-Development-of-EDI-27894982/

Private equity firms circling Nestle’s skin health business

Private equity firms Cinven and Advent have teamed up to bid in an auction that could value Nestle’s skin health business at about 7 billion Swiss francs ($7 billion), three sources familiar with the matter said.

Private equity companies Blackstone, KKR, Carlyle, CVC, EQT and Partners Group are also expected to bid and might look for partners, according to the sources, who declined to be identified as the process is private.

Nestle launched a review of the unit in September, as the maker of Nescafe coffee and Perrier water ditches underperforming businesses and fends off criticism from an activist investor who wants an overhaul.

The fragmented consumer health sector is attractive for deals because of aging populations in many industrialized nations, increased interest in health and rising incomes, analysts say.

Information memorandums on the skin health business sale, being run by Credit Suisse and Evercore, are expected to be sent by the end of January and first-round bids are likely to be submitted in early March, the sources said.

Nestle and all the funds declined to comment.

Merz Pharma, a private German company that sells wrinkle fillers and other skin treatments, has reached out to several private equity bidders to find a partner, the sources said.

The move by Merz shows the strength of private equity in the auction, given they want the whole Nestle unit. Industry rivals are only expected to bid for parts of the business.

Nestle Skin Health, which sells Cetaphil and Proactiv skin care products, Restylane wrinkle fillers and prescription dermatology medicines, had sales of 2.7 billion Swiss francs last year, accounting for about 3 percent of Nestle’s total.

The sources said industry players which might take part in the auction included Beiersdorf, Allergan, Henkel, Johnson & Johnson (J&J), L’Oreal, Pfizer and Unilever.

Sources previously estimated the unit could fetch 6 billion to 8 billion francs.

On Monday, one source put the range at 7.5 billion to 8.5 billion francs, based on 2018 operating earnings of 500 million to 550 million francs. Another source said the price tag could reach more than 9 billion francs if the unit was broken up.

Nestle, the world’s largest packaged food maker, created the Skin Health unit in 2014 when it bought out L’Oreal’s stake in their Galderma joint venture.

Under CEO Paul Bulcke, skin treatments were part of Nestle’s push into higher-growth health products to counter a slowdown in its traditional food business. But the unit has performed poorly, leading to one-off costs and restructuring.

https://www.marketscreener.com/news/Exclusive-Private-equity-firms-circling-Nestle-s-skin-health-business-sources–27895017/?countview=0

BerGenBio Starts Phase I Trial of Novel Therapy for Advanced Solid Tumours

BerGenBio ASA (OSE:BGBIO), announces that the first patient has been dosed in a phase I clinical trial evaluating the safety, tolerability, pharmacokinetics and anti-tumour efficacy of ADCT-601, an AXL-targeting antibody drug conjugate (ADC), in patients with advanced solid tumours.

ADCT-601 is composed of a humanised monoclonal antibody against human AXL (BGB601) discovered by BerGenBio, conjugated to a pyrrolobenzodiazepine (PBD) dimer toxin. BGB601 was out-licensed for ADC development to ADC Therapeutics SA (ADCT). In preclinical studies, ADCT-601 has demonstrated potent and specific anti-tumour activity in multiple in vivo models and was stable and well tolerated, as reported by ADCT at the American Association of Cancer Research (AACR) annual congress in 2018.

The open-label, multi-centre, single arm phase I trial will enrol approximately 75 patients with selected tumour types and will be managed and sponsored by license partner ADC Therapeutics. For more information see https://clinicaltrials.gov (accessing trial identifier NCT03700294).

https://www.biospace.com/article/releases/bergenbio-announces-start-of-phase-i-trial-evaluating-adct-601-a-novel-anti-axl-adc-in-patients-with-advanced-solid-tumours/