Sun Pharma Launches Ready-to-Infuse Chemo Product in U.S.

INFUGEM™ (gemcitabine in sodium chloride injection) is the first and only chemotherapy product in a premixed, ready-to-infuse formulation

Use of ready-to-infuse bags eliminates steps in the complex chemotherapy preparation process, reducing exposure and mitigating inherent provider and patient safety risks

Sun Pharmaceutical Industries Ltd. (Reuters: SUN.BO, Bloomberg: SUNP IN, NSE: SUNPHARMA, BSE: 524715, “Sun Pharma” and includes its subsidiaries and/or associate companies) today announced that INFUGEM™ (gemcitabine in sodium chloride injection), for intravenous use, is now commercially available in the U.S. INFUGEM, the first chemotherapy product that comes in a premixed, ready-to-infuse formulation, was approved by the U.S. Food and Drug Administration (FDA) in July 2018 in combination with other drugs for the treatment of breast, ovarian, non-small cell lung cancers, and as a single agent for the treatment of pancreatic cancer.

INFUGEM is an alcohol-free, clear, colorless, sterile solution of 10mg/mL gemcitabine in 0.9% sodium chloride that is supplied to pharmacists in ready-to-infuse bags as a Spike & Go™ package. It involves dose banding practice, whereby standardized doses of intravenous cytotoxic drugs are used for ranges (or “bands”) of doses calculated for individual patients. INFUGEM is the only available gemcitabine formulation that does not require reconstitution and syringe withdrawal prior to intravenous administration. Eliminating these steps reduces complexity and minimizes the inherent risks of hazardous drug exposure, contamination, and medication errors.

https://www.marketscreener.com/SUN-PHARMACEUTICAL-INDUST-9058928/news/Sun-Pharmaceutical-Industries-Pharma-Launches-Ready-to-Infuse-INFUGEM-in-the-U-S-28367590/

Sanofi, Alnylam conclude research and option phase of 2014 rare disease alliance

Sanofi and Alnylam have agreed to conclude the research and option phase of the companies’ 2014 RNAi therapeutics alliance in rare genetic diseases. The material collaboration terms for patisiran, vutrisiran (ALN-TTRsc02) and fitusiran, as previously announced, will continue unchanged.

As part of this agreement, Alnylam will advance an additional investigational asset in an undisclosed rare genetic disease through the end of IND-enabling studies. Sanofi will be responsible for any potential further development or commercialization of such asset. If this product is approved, Alnylam will be eligible to receive tiered double-digit royalties on its global net sales.

“Our alliance with Alnylam has successfully brought one important medicine to patients with ATTR amyloidosis and it also has spawned a molecule that is in pivotal clinical trials for people with hemophilia,” said John Reed, Global Head of Research and Development at Sanofi. “As we wrap up this phase of our program with Alnylam, we remain committed to advancing therapies for patients with rare diseases and rare blood disorders.”

In addition, Alnylam and Sanofi have agreed to amend certain terms of the companies’ equity agreement, with Sanofi obtaining a release of its lock-up of Alnylam stock holdings, subject to certain trading restrictions, among other provisions.

https://www.marketscreener.com/SANOFI-4698/news/Sanofi-and-Alnylam-conclude-research-and-option-phase-of-2014-rare-disease-alliance-28367818/

Dallas/Ft. Worth: A “No-Brainer” for Life Science Development

Texas is such a large state—larger than France with chunks of Germany, the UK and Italy included—that it can be a bit unexpected when organizations analyzing the biopharma industry break it up into specific cities or regions.

For example, a recent report by CBRE Research analyzing U.S. life science clusters found that Houston, Texas is the third-fastest growing life science market from 2014 to 2017. Much has been written about the life science market in Austin, both the capital of Texas and the location of the University of Texas. In terms of the report’s top-ranked emerging life science clusters, Houston ranks #2, Austin ranks #3 and Dallas/Ft. Worth ranks #7.

The recent CBRE Research report, “2019 U.S. Life Sciences Clusters” has two Texas metropolitan areas in its top-10 fastest-growing life sciences market. Houston ranked third, while Dallas/Ft. Worth ranked seventh. Let’s look at Dallas/Ft. Worth.

Of the cities and regions in that category, the report notes, “exhibit an attractive combination of a substantive life science workforce, including key scientists, strong recent life sciences employment growth, ample NIH funding, top-ranked schools and medical institutions, and a sizeable high-tech workforce to support future convergence between the industries.”

Jeff Ellerman, vice chairman, CBRE, stated, “The DFW area is an attractive market for companies in the life sciences industry. There is an enormous amount of high-tech talent in the area, which helps support the cross-pollination between tech and life sciences. When you combine the growing market with available talent, it becomes a no-brainer for large companies in the pharmaceutical and medical technology sectors to Choose North Texas for their headquarters.”

Academic Institutions

The Dallas/Ft. Worth region has several major academic institutions, including Southern Methodist University, Texas Christian University and the University of Texas-Dallas. Texas has quite a number of major research institutions, including Texas Medical Center, Baylor College of Medicine, Texas A&M University Health Science Center, The University of Texas Health Science Center (UTHealth) and The University of Texas MD Anderson Cancer Center.

The area also is host to a number of nationally renowned healthcare and medical institutions, including Parkland Memorial Hospital, Baylor University Medical Center and Children’s Medical Center of Dallas.

Dallas/Ft. Worth academic institutions churned out 2,120 biological and biomedical science degrees in the 2016/2017 academic year, with 129 of these being doctorates.

In addition, Dallas/Ft. Worth institutions received $181 million in funding from the National Institutes of Health (NIH).

An Overview

According to the Fort Worth Chamber of Commerce, the region’s healthcare infrastructure is made up of more than 450 biomedical companies, 1,110 research, development and testing laboratories, and nationally-recognized healthcare systems.

“The University of North Texas (UNT) Health Science Center in Fort Worth is a major driver and collaborator in medical research and cutting-edge technologies that are gaining ground in the fight against major disease.”

Their report indicates there are 222,700 life sciences industry jobs in the DFW area.

Incentive programs in the DFW area include the Texas Enterprise Fund, used to attract new business to the state or to assist existing businesses in expanding; the Texas Emerging Technology Fund, designed to help the state create jobs and grow the economy over the long term; the Texas Enterprise Zone Program, an economic development tool for local communities to partner with the state to promote job creation and capital investment, as well as other tax incentives and workforce training funds.

Coming up on May 21, 2019, is the North Texas Life Science Career Symposium (LSCS 2019). The goal of the event is to create an annual event connecting top academic talent in North Texas with careers in the life sciences. As such, graduate students, post-doctorates, medical professionals and life science company representatives and executives are invited to attend for career exploration, networking, and job opportunities.

Keynote speakers will be John Milligan, former president and chief executive officer of Gilead Sciences, and Gaurab Chakrabarti, chief executive officer of Solugen.

The event is organized by Health Wildcatters and the Biotech Club at UT Southwestern in collaboration with Quest for Careers (QFC), Consulting Club, and the Science Policy Education and Communication (SPEaC) student organizations.

Examples

Here are just a few examples of recent DFW biopharma and life science stories.

Tricida Announces Positive Data From Kidney Disease Trial

Tricida, a biopharma company based in South San Francisco, announced that its veverimer (TRC101) was positive in treating metabolic acidosis in patients with chronic kidney disease (CKD). The TRCA-301E trial was a 40-week extension of the 12-week TRCA-301 trial.

The lead investigator for the trial, Donald E. Wesson, Professor of Medicine at Texas A&M Health Sciences Center College of Medicine in Dallas, stated, “This trial provides evidence of long-term safety and tolerability of TRC101 and that the sustained increase in blood bicarbonate led to improvements in multiple clinical outcomes that matter to both patients and physicians.”

Prelude Fertility and Inception Fertility Become Largest Fertility Services in the U.S.

New York-based Prelude Fertility has conducted a deal with Inception Infertility, which will make the Prelude Network the fastest-growing network of fertility clinics and the largest provider of comprehensive fertility services in the U.S. The Prelude Network and Inceptions’ Aspire clinics in total completed about 18,000 IVF cycles last year. TJ Farnsworth, founder of Inception Fertility, will act as chief executive officer of the new parent entity.

Inception is the parent company of Aspire Fertility, with sites at 11 locations in Texas, including Houston, San Antonio, Dallas, Austin, and McAllen, as well as one site in Atlanta.

C-Hear Will be Featured at Dallas Startup Week

C-Hear was launched in Dallas in 2015. It delivers a small digital footprint file that contains both image and sound using its Codec platform. This allows visually impaired individuals to “hear” images on the Internet. Within the healthcare industry, it is helping transition into ADA compliance. The company founder and chief executive officer, Adena Harmon, will speakThursday, April 4, 12-12:45 pm in a panel called “Startups That Change Our Lives” at the Dallas Startup Week event. Other panelists will include ob-gyn Lyndsey M. Harper, Chris Bricker, chief executive officer of MyndVR and Lara Ashmore of the University of Texas at Dallas.

United Neuroscience to Present Alzheimer’s Vaccine Data

United Neuroscience, which has offices in Dublin, Ireland and Dallas, will present Phase IIa data from its lead product, UB-311, at the 14th International Conference on Alzheimer’s and Parkinson’s Diseases in Lisbon, Portugal, from March 26-31, 2019. UB-311 is a novel UBIth active vaccine targeting the N-terminus of A-beta peptides. It is, essentially, a vaccine for Alzheimer’s. It is designed to induce high B-cell specific responses while avoiding T-cell inflammation. In a Phase I clinical trial, UB-311 vaccination safely increased anti-Abeta antibodies with “a suggestion of cognitive stabilization.”

https://www.biospace.com/article/dallas-ft-worth-a-no-brainer-for-life-science-development/

Breath Therapeutics Launches Phase 3 Trial to Bring Relief to BOS Patients

In five years it is estimated that about 25,000 people in the United States will develop bronchiolitis obliterans syndrome (BOS), which results in respiratory failure and death. But a late-stage treatment under development by Breath Therapeutics could be a possible remedy for this rare and dread disease.

Breath Therapeutics Chief Executive Officer Jens Stegemann called BOS a devastating disease among lung transplant patients for which there is currently no cure. For these patients, he likened BOS to a severe cancer diagnosis, which he said makes a treatment for BOS a severe unmet need. However, Germany-based Breath Therapeutics initiated two Phase III trials attempting to repurpose an old drug to help these lung transplant patients.

“We’re teaching an old dog new tricks,” Stegemann said of Breath’s proprietary liposomal formulation of cyclosporine A. He added that the company’s inhaled cyclosporine therapeutic may provide the first safe and effective treatment for BOS.

The company has developed the medication to be administered to patients via a drug-specific investigational eFlow nebulizer developed by the parent company of Breath, PARI Pharma. Breath launched in 2017 as a spinout of PARI Pharma, which is based in Munich. L‑CsA‑i has received orphan drug designation for the treatment of BOS from the Food and Drug Administration and European Medicines Agency.

BOS occurs when the body’s immune system attacks the small airways of the transplanted lungs and destroys the bronchioles. BOS is caused by T-cell mediated inflammation that leads to blockage of bronchioles. There are more than 30,000 lung transplantation and allogeneic hematopoietic stem cell transplantation recipients worldwide currently affected by BOS. Stegemann told BioSpace that approximately 50 percent of lung transplant patients will be affected by BOS. The immune response to the transplant is one of the main reasons why lung transplants have the poorest outcomes, Stegemann said.

“Although lung transplant survival has improved with the advancement of surgical techniques and perioperative management, and more individuals are undergoing lung transplantation, survival has not improved to reach the rate of other organ transplantations. One key factor impacting survival in lung recipients is rejection, particularly bronchiolitis obliterans syndrome (BOS), the most common form of chronic lung allograft dysfunction,” Joseph M. Pilewski, the associate chief for clinical affairs and lung transplant physician in the Division of Pulmonary, Allergy & Critical Care Medicine at the University of Pittsburgh Medical Center, said in a statement supplied to BioSpace.

The drug at the core of Breath’s therapy was initially introduced in the 1980s to benefit organ transplant patients. However, Stegemann said there were some toxicity issues associated with the medication. However, due to the inhaled formulation of cyclosporine A that Breath has developed, high amounts of the immunosuppressant drugs can be directly delivered into the lungs, which lowers concerns about toxicity.

Breath launched two Phase III trials, the BOSTON-1 trial for individuals with BOS following a single lung transplant and BOSTON-2, for double lung transplant patients. Each study will enroll 110 participants at leading lung transplant specialty centers in eight countries. When the 48-week studies are complete, the patients will be eligible to continue in the planned BOSTON-3, an open-label extension trial.

Stegemann said the launch of the two Phase III trials represents a major milestone for the two-year-old company. He said it is “indicative of the outstanding progress we Breath Therapeutics) have achieved in the past 24 months.” As they move into the trials, Stegemann said the company is “well positioned to succeed in our goal of bringing an effective and safe treatment to people with BOS.”

Data from the BOSTON 1 and BOSTON 2 trials are expected in 2021. Stegemann said in Europe, Breath will also plan to conduct pediatric trials in BOS.

https://www.biospace.com/article/exclusive-breath-therapeutics-launches-phase-iii-trial-to-bring-relief-to-bos-patients-/

FDA Action Alert: Spotlight on Merck’s Keytruda

The FDA calendar has only one PDUFA data scheduled for the next couple of weeks. On Thursday, April 11, the U.S. Food and Drug Administration (FDA) and Merck & Company have a target action date for their supplemental Biologics License Application (sBLA) for Keytruda (pembrolizumab), its anti-PD-1 checkpoint inhibitor, as a monotherapy for the first-line treatment of locally advanced or metastatic non-small cell lung cancer (NSCLC) in patients whose tumors expressed PD-L1 without EGFR or ALK genomic tumor aberrations.

The sBLA was built on data from the Phase III KEYNOTE-042 clinical trial. In that trial, Keytruda alone showed a significant improvement in overall survival compared with chemotherapy in this patient population. There was originally an earlier date, but Merck submitted additional data late in 2018, and on December 20, the company indicated the agency found it made up a major amendment and extended the target action date three months to April 11, 2019.

Keytruda is such a successful drug, and Merck has so many ongoing clinical trials, it can be difficult to actually track what’s going on with it. On March 14, 2019, the company announcedthat the European Commission had approved Keytruda in combination with carboplatin and either paclitaxel or nab-paclitaxel, for first-line treatment of adults with metastatic squamous non-small cell lung cancer (NSCLC). That approval was based on data from the Phase III KEYNOTE-407 clinical trial, showing the drug in combination with chemotherapy significantly improved overall survival (OS) in that patient population, reducing the risk of death by 36% compared to chemotherapy alone.

The company also indicated in that announcement that in NSCLC, Keytruda had been approved in Europe for first-line treatment of metastatic nonsquamous NSCLC with pemetrexed and platinum chemotherapy in adults whose tumors have no EGFR or ALK-positive mutations (KEYNOTE-189); first-line treatment of metastatic squamous or nonsquamous NSCLC as monotherapy in adults whose tumors have high PD-L1 expression with no EGFR or ALK-positive tumor mutations (KEYNOTE-024); and locally advanced or metastatic NSCLC in adults whose tumors express PD-L1 and who have received at least one previous chemotherapy treatment (KEYNOTE-010).

And on April 1, Merck announced that Keytruda had been approved by China’s National Medical Products Administration (NMPA) in combination with pemetrexed and platinum chemotherapy for first-line treatment of metastatic nonsquamous NSCLC, with no EGFR or ALK genomic tumor mutations. This was approved conditionally based on overall survival and progression-free survival (PFS) data from Phase III KEYNOTE-189 in patients regardless of PD-L1 tumor expression. Continued approval is based on verification and clinical benefit in Chinese patients in a confirmatory trial. This is the same approval as the first EC approval mentioned above.

“This approval represents a key advance in a country with a high incidence of lung cancer, and where significant progress for the first-line treatment of this devastating disease has been very limited over a number of years,” stated Roy Baynes, Senior Vice President and Head of Global Clinical Development, Chief Medical Officer, Merck Research Laboratories. “This also means that, based on the results of KEYNOTE-189, Keytruda in combination with chemotherapy has now been approved in the U.S., Europe, Japan and China, among other countries, for the first-line treatment of appropriate patients with metastatic nonsquamous non-small cell lung cancer.”

Merck indicates that Keytruda is currently in more than 900 clinical trials in a broad range of cancers and treatment settings.

At the recent American Association for Cancer Research (AACR) meeting, Merck presented results from a post-hoc analysis of patients with liver or brain cancers from the Phase III KEYNOTE-189 trial. The point of the post-hoc analysis was to evaluate outcomes of Keytruda in combination with chemotherapy in liver and brain cancers.

The company also presented pooled data from KEYNOTE-158 and Phase Ib KEYNOTE-028 evaluating Keytruda in patients with previously treated advanced small cell lung cancer (SCLC). Data from these trials supported the company’s first application in SCLC for Keytruda. The sBLA was accepted by the FDA for priority review and has a target action date of June 17, 2019.

https://www.biospace.com/article/fda-action-alert-spotlight-on-merck-s-keytruda/

SCYNEXIS Ibrexafungerp: Positive Activity in Infections Including Candida

Six presentations will provide additional evidence of the potent and broad antifungal activity of oral ibrexafungerp, including new clinical case studies from the CARES and FURI trials as well as promising preclinical data

Case studies from the CARES trial, the first study of an investigational agent to treat patients with Candida auris infections, show favorable outcomes following treatment with oral ibrexafungerp

Ibrexafungerp shows strong clinical activity in difficult-to-treat patients with resistant Candida pathogens in a variety of infections, including esophageal candidiasis and spondylodiscitis (FURI study)

SCYNEXIS, Inc. (NASDAQ: SCYX), a biotechnology company delivering innovative therapies for difficult-to-treat and often life-threatening infections, today announced data demonstrating the potential use of ibrexafungerp as an agent to address multiple serious fungal infections, including many that have shown resistance to existing therapies. The collection of data will be presented in one oral and five poster presentations at the 29^th European Congress of Clinical Microbiology and Infectious Diseases (ECCMID), April 13-16, 2019, in Amsterdam, Netherlands.

https://www.prnewswire.com/news-releases/scynexiss-ibrexafungerp-shows-favorable-clinical-activity-in-resistant-fungal-infections-including-candida-auris-cases-from-the-cares-study-in-data-to-be-presented-at-the-29th-eccmid-300823802.html

AVITA Presents on System to Treat Third-Degree Burns in Pediatric Patients

AVITA Medical (ASX: AVH, OTCQX: AVMXY), a global regenerative medicine company, announced that interim results describing clinical outcomes for pediatric patients treated using the RECELL® System were presented at the American Burn Association (ABA) 51st Annual Meeting in Las Vegas by Jeffrey Carter, MD, FACS University Medical Center New Orleans Burn Center and LSU Health New Orleans School of Medicine. Patients enrolled in the Investigational Device Exemption (IDE), Compassionate Use and Continued Access studies had mixed-depth and full-thickness (third-degree) burns and were treated with the combination of Spray-On Skin™ Cells prepared using the RECELL system and widely meshed autografts. Patients in the studies experienced excellent healing outcomes, with 98% of wounds healed four weeks after treatment. Dr. Carter’s presentation describing the treatment of pediatric patients was selected as a “Best of the Best Abstract” out of more than 500 abstract submissions to the ABA meeting.

https://www.businesswire.com/news/home/20190407005041/en/AVITA-Medical-Announces-Presentation-ABA-Meeting-Highlighting