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Wednesday, April 3, 2019

Liquidia Presents Phase 3 Trial Data for LIQ861 at ISHLT 39th Annual Meeting

Liquidia Technologies, Inc. (Nasdaq: LQDA) (“Liquidia”), a late-stage clinical biopharmaceutical company focused on the development and commercialization of LIQ861, announced today additional detailed safety and exploratory endpoint findings at the two-month timepoint for the Phase 3 INSPIRE trial. Nicholas Hill, MD, Chief Pulmonary, Critical Care & Sleep Division and Professor of Medicine at Tufts University School of Medicine and INSPIRE Principal Investigator, presented the data at the opening plenary session of the 39th International Society for Heart & Lung Transplantation (ISHLT) Meeting and Scientific Sessions in Orlando, Florida. Liquidia is pursuing approval of LIQ861, an inhalation dry powder formulation of treprostinil that is produced using its PRINT® Technology, as an alternative to current inhaled treprostinil therapy for the treatment of patients with pulmonary arterial hypertension (PAH) (WHO Group 1).
“The two-month results of the INSPIRE trial are promising for patients with PAH. Inhaled therapy offers the benefit of getting drug directly to the lungs and we are encouraged that the safety, tolerability and quality of life metrics suggest that LIQ861 is an attractive and more convenient therapy versus the currently available inhaled therapies,” said Dr. Hill.
In March 2019, Liquidia reported two-month top-line results of the Phase 3 INSPIRE study, which indicated that the study had met its primary endpoint of safety and tolerability of LIQ861 in (PAH) patients. The ISHLT presentation, “INSPIRE: A Phase 3 Open-Label, Multicenter Study to Evaluate the Safety and Tolerability of LIQ861 in PAH,” provides detailed results on the treatment emergent adverse event (TEAE) profile, duration of treatment by  group (including those adding LIQ861 to non-prostacyclin oral therapy and those transitioning from Tyvaso®*) along with the performance of LIQ861 against exploratory endpoints such as patient quality of life metrics.
“We are continuing to demonstrate the potential benefits of LIQ861 as we present additional data from the INSPIRE trial,” said Neal Fowler, Chief Executive Officer of Liquidia. “LIQ861 is a convenient inhaled therapy that effectively manages PAH with excellent safety and tolerability. It’s our belief that LIQ861 can help PAH patients more easily manage their disease, and more importantly, the quality of their lives.”

Agbiotech Evogene Starts Med Cannabis Unit, CEO Sees ‘Significant Impact’

It’s not unusual these days to see agri-tech and biotech stocks jumping on the cannabis bandwagon to capitalize on the opportunity presented by the geographically expanding legalization of pot.

What Happened

Israeli biotech Evogene Ltd NASDAQEVGN, which is engaged in research and development for improving crop quality and productivity, announced a new subsidiary Wednesday in a foray into the medical cannabis market: Canonic Ltd.
The company said it will initially focus on creating improved cannabis varieties by tackling problems surrounding yield, stability and specific metabolite composition.
Evogene said the development will be based on the utilization of its Computational Predictive Biology, or CPB, platform.

Why It’s Important

Evogene, with its CPB platform and leadership position in the area of plant genomics, has a significant competitive advantage for the development of next-gen products that address limitations in cannabis cultivation and production, the company said in the Wednesday release.
“Evogene’s cutting-edge genomic technology, alongside our multidisciplinary team of professionals, uniquely positions Canonic to bring to market the most advanced, stable and consistent next-generation medical cannabis products, and I am confident we can make a significant impact in the world of medical grade cannabis,” Canonic CEO Arnon Heyman said in a statement.

What’s Next

Canonic, according to Evogene, will focus on three main product types: high metabolite yield cannabis varieties, stable varieties with consistent metabolite performance and cannabis varieties with a unique metabolite profile tailored to specific medical indications such as post-traumatic stress disorder, severe chronic pain and cancer.

FDA Slates 1st Public Hearing On CBD, Sends Warning Letters To 3 Companies

In what could potentially be a breakthrough for the U.S. CBD market, the Food and Drug Administration announced the first public hearing to obtain more data about CBD products. The announcement comes as the FDA and FTC is sending warning letters to three companies that sell CBD products, accusing them of false advertising.

What Happened

The May 31 hearing is set to obtain scientific data and information regarding the safety, manufacturing, product quality, marketing, labeling and sale of products containing cannabis or cannabis-derived compounds.
The regulator plans to obtain information related to health and safety risks of CBD; manufacturing standards; methods of testing of products containing CBD; how these products are labeled; and how consumers are warned about risks associated with them.
On Tuesday, the FDA and FTC sent joint warning letters to three companies: Nutra Pure, PotNetwork Holdings Inc POTN 1.92% and Advanced Spine and Pain.
The agencies said the companies are falsely advertising CBD supplements, claiming they can treat diseases such as cancer and Alzheimer’s. The companies in question have 15 days to address the issues and notify the FTC.

Why It’s Important

The 2018 Farm Bill signed into law in December legalized hemp and hemp-derived CBD, but also authorized the FDA to regulate CBD products.
This creates a problem because CBD is an active ingredient in an approved drug — GW Pharmaceuticals plc GWPH 0.25%‘s Epidiolex — which means that CBD products violate the FD&C Act and could potentially undermine the drug approval process.
Nevertheless, CBD products have been steadily making their way to the shelves of retailers like DSW IncDSWWalgreens Boots Alliance Inc WBA 0.99%, and CVS Health Corp CVS 1.3%.
Establishing a clear set of regulations for CBD products could remove many uncertainties and incentivize more retailers to start offering CBD products, while consumer packaged goods companies could warm to the space and develop new products.
“We think this is a huge step forward for consumers that are clearly very excited about having CBD included in everything from topicals to infused foods and beverages. You are seeing a surge of businesses coming out with products,” said Tim Moxey, founder of the CBD and cannabis edible company botanicaGLOBAL.
“Having guidelines and rules from the FDA would allow the industry leaders to seriously commit to introducing hemp-derived products that consumers are already familiar with in states that have cannabis existing laws.”

What’s Next

The FDA said it does not intend for the May 31 hearing to result in any decisions or regulatory action, but expects the event to be an “important step” in the agency’s continued evaluation of cannabis and cannabinoids in FDA-regulated products.
“I anticipate that the FDA will decide to stop throwing up the red flags about using CBD in food due to safety concerns,” Celeste Miranda, CEO of MACE Media and CBD Expo Tour, told Benzinga.
“I think they will hit hard on testing and proper labeling as well as rigid standards on making no claims.”

Houston: One of the Fastest-Growing Life Science Communities

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. One doesn’t typically think of Texas, let alone Houston, as being a center for the life sciences. Oil, energy and tech, yes, but not life sciences.
But the state has several strong academic institutions, such as the University of Texas MD Anderson Cancer Center in Houston and UT Southwestern Medical Center in Dallas. And Austin, home to the University of Texas as well as the state’s capital, has more than 200 life science companies.
Houston Details
TribTalk, a publication of The Texas Tribune, wrote in December 2018, that Texas has approximately 4,000 life science companies and 100,000 biotech workers. And in 2017, the state ranked seventh for research funded by the National Institutes of Health (NIH), to the tune of $1.16 billion.
TribTalk noted, “Academic medical centers have become major employers in our biggest cities, generating economic activity that returns net tax revenue to the state. In San Antonio, one of every six workers are employed in the city’s flourishing bioscience and healthcare sector, anchored by UT Health San Antonio, where the Department of Defense has funded research into treating PTSD among war veterans. Employment in biotech research and development in the Dallas-Forth/Worth Metroplex jumped by 34 percent between 2010 and 2016. The Houston area lost biotech R&D jobs during that time period but welcomed an increase in pharmaceutical and medicine manufacturing jobs.”
The CBRE report lists Houston as #2 in emerging life sciences clusters. All of these, 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.”

From 2014 to 2017, the report indicates, the city’s life sciences workforce grew by slightly under 14 percent. The city has approximately 3,100 biomedical engineers, biochemists, biophysicists and chemists working there.
Houston ranks thirteenth for top-20 markets for new life sciences talent, according to the CBRE report. In 2016-2017, 1,282 total biological and biomedical science degrees were granted, with 255 of them being doctorates. Three of its biological sciences programs are ranked in the top 100.
Major Houston-Area Life Science Initiatives and Organizations
BioHouston is a non-profit tax-exempt corporation founded by Houston area academic and research institutions. The goal is to establish the Houston region as a vigorous global competitor in life science and biotechnology commercialization.
Although BioHouston sponsors several events, one coming up on May 1-3 is the 2019 Texas Life Science CEO Summit. Registration for the event is exclusively for CEOs of life science companies that are developing a product.
TMC3 refers to a collaborative 30-acre biomedical research campus being developed jointly by the 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.
TMC3 will include collaborative research space and shared core laboratories. The centerpiece is the double helix-shaped multi-story facility that will house shared core labs, retail and commercial space. There will be 1.5 million square feet of collaborative campus, 250,000 square feet of core labs and amenities, including 125,000 square feet of commercial and retail space, and 125,000 square feet of shared research facilities. There will also be a 410-room TMC Hotel and Conference Center with 50,000 square feet of conference space.
The project was announced in April 2018, with groundbreaking expected in 2019 and the project to open in 2022. It is expected to generate a $5.2 billion stimulus to Texas and create 30,000 new jobs.
“The new 30-acre research campus will provide our top medical minds with the resources to remain at the forefront of the health care industry,” Governor Greg Abbott stated at the time of the April announcement. “This cutting-edge facility will not only generate thousands of jobs, but it will solidify Texas’ position as an international leader in biomedical research.”
Greater Houston Partnership (GHP)
Noting that Houston’s Texas Medical Center is the largest medical complex in the world, the Greater Houston Partnership isn’t specific to the life sciences, but does point out that that TMC’s presence is a major driving force in the broader region’s life sciences sector. Houston hosts more than 1,760 life science companies, hospitals, health facilities and research institutions with more than 360,000 people in those areas.
A June 2018 report on Houston’s Biotech and Life Sciences notes that in 2017 there were almost 4,200 clinical trials ongoing in the Houston region, or about 13.4 percent of all clinical trials in the U.S.
“In the field of health and medical technology, the Texas Medical Center’s Innovation Institute coordinates the efforts of several life science-focused incubator and accelerator programs,” GHP’s website notes. “The organization aims to help startup companies commercialize new medical technologies by providing access to the vast resources of the world’s largest medical center.”
The Innovation Institute’s accelerator, TMCx, focuses on early-stage companies in the area of medical devices, digital health care services, diagnostics and therapeutics. TMCx+, a related program, is an incubator targeting seed-stage companies in those same sectors. So far, 84 companies have enrolled in the TMCx program and a total of $157 million in funding was raised.
The TMC also launched the TMC Venture Fund in 2017, which has a goal of investing $25 million in early-stage health care tech companies in Houston.
TMC Innovation has also partnered with Johnson & Johnson’s JLABs @TMC and with the AT&T Foundry, which supports companies in the area of digital health services.

Houston Life Science News
Here’s just a taste of recent life science news coming out of Houston.
Saranas, a Houston-based medical device company, announced on March 5 that its Early Bird Bleed Monitoring System had been granted de novo classification by the U.S. Food and Drug Administration (FDA). The Early Bird Bleed Monitoring System includes a vascular access sheath with embedded bioimpedance sensors designed to detect and monitor bleeding from vessel injury that could occur during endovascular procedures, such as a transcatheter aortic valve replacement (TAVR), large bore hemodynamic support device placement or other procedures.
On February 25, Marker Therapeutics, headquartered in Houston, updated data from four clinical trials that are using its multi-antigen targeted T-cell (MultiTAA) therapies. One of the highlights was data from an ongoing clinical trial in patients with acute myeloid leukemia (AML).
“We continue to be highly encouraged by the clinical results we’ve seen to date with our MultiTAA therapies,” stated Peter L. Hoang, president and chief executive officer of Marker Therapeutics. “In AML, we believe we are seeing increasing evidence of meaningful therapeutic benefit for patients with limited treatment alternatives. Our multiTAA therapy appears to be safe and well-tolerated with the potential to mediate a meaningful anti-tumor effect, in addition to demonstrating a compelling correlation between therapeutic responses, with superior in vivo expansion of our T-cells.”
On February 21, Houston-based Nanospectra Biosciences was the featured innovationsuccess at the Congressional Healthcare Innovations Summit in Houston, held at Rice University. Nanospectra is developing a novel use of nanomedicine for selective thermal ablation. Its AuroLase ultra-focal therapy for prostate cancer was highlighted.
“Nanospectra is honored to be highlighted as an example of healthcare innovation in the Houston area, and more specifically, for the continued development of its underlying technology originally developed locally at Rice University,” stated state Congressman Dan Crenshaw at the keynote address.
Moleculin Biotech, based in Houston, announced on January 9 that it had begun recruiting patients in Poland for its second clinical trial of Annamycin to treat relapsed and refractory acute myeloid leukemia. Moleculin’s drug candidates came out of license agreements with The University of Texas System on behalf of the MD Anderson Cancer Center.
“We are encouraged to see ready access to qualified patients in Poland,” stated Walter Klemp, Moleculin’s chairman and chief executive officer. “Having now cleared the unique European approval process to ship Annamycin, which had delayed the start of the trial in Poland, clinical supplies are now in country and ready to treat patients.”
Houston once had the motto, “Space City: A space of Infinite Possibilities.” And maybe it still is for a fast-growing life science industry.

Fusion Secures $105 Million, Will Double Headcount

Fusion Pharmaceuticals will forge ahead with a Phase I trial for its lead radiopharmaceutical asset bolstered by a $105 million Series B financing round supported by Varian, Johnson & Johnson Innovation and OrbiMed.
Fusion Chief Executive Officer John Valiant told BioSpace that radiopharmaceutical therapeutics targeting solid tumors is an effective and more precise way to deliver an agent straight to the tumor and kill the cancer without increasing toxicity concerns. The antibody molecules in the process are paired with potent alpha emitting medical isotopes that target the cancer cells and “precisely and effectively induce cancer cell death.” Unlike external radiation treatments that have been widely used for years as a way to target tumors, Valiant said the method utilized by Fusion delivers the radiation directly to the tumor itself.
“It’s like a bomb that goes off inside the cancer cell. It’s a really effective localized kill,” Valiant said in a telephone interview.
Valiant noted that since alpha particles travel only a short distance, they localize the radiation to the tumor, which helps reduce concerns about toxicity or other problems associated with the radiation.

QIAGEN, LabCorp to Provide Day-One Access at the Time of Drug Approvals

QIAGEN N.V. (NYSE: QGEN; Frankfurt Prime Standard: QIA) today announced a master service agreement with LabCorp®(NYSE: LH) to further accelerate the access of cancer patients to QIAGEN’s companion diagnostic products following regulatory approvals of drugs and their associated tests.
As a new participant in QIAGEN’s Day-One Lab Readiness program, LabCorp will be able to provide physicians and patients with faster access to new, genomically targeted drugs that are becoming increasingly important therapies for a growing variety of cancers. LabCorp is the latest company to join the program, and its expertise in the commercial use of companion diagnostics will help a broader range of patients gain access to innovative tools for precision medicine.
Building on the U.S. Food and Drug Administration’s modernized regulatory approach, QIAGEN´s Day-One Lab Readiness program enables molecular diagnostic labs to begin implementing the activities necessary to prepare for commercial launch of new drugs and IVD tests once FDA approval is obtained.
LabCorp’s participation in QIAGEN´s network of laboratory partners will contribute to ensuring testing readiness for a portfolio of new companion diagnostics, including tests based on next generation sequencing (NGS) and qualitative polymerase chain reaction (qPCR), being prepared for launches in 2019 and 2020 in several countries around the world.
As more biomarkers and new technologies are used to develop in vitro diagnostics (IVD), companion diagnostic testing is becoming increasingly complex. LabCorp’s extensive experience in these areas is a strong complement to QIAGEN’s personalized health collaborations with leading pharmaceutical companies. Assays for multiple indications, including novel companion diagnostics across a range of cancers including lung, breast, colorectal, bladder and eventually pan-tumor disease areas, are currently in LabCorp’s Day-One Lab Readiness pipeline.
“Our Day-One Lab Readiness program covers all the steps in being able to begin serving patients with access to companion diagnostic results immediately upon approval of new targeted therapies and the related companion diagnostics. Our Day-One program includes pre-approval preparation of workflow implementation, training, assay verification, forecasting, medical communication and reimbursement to ensure immediate readiness,” said Thierry Bernard, Senior Vice President and Head of QIAGEN’s Molecular Diagnostics Business Area. “LabCorp has a broad global presence and deep involvement in clinical testing, and we have a long history of working together. Day-One Readiness marks a new level of our successful collaboration with LabCorp, which already ensures patient access to several precision diagnostic tests, including therascreen® assays for oncology and also NGS solutions. By aligning the timelines of LabCorp and our partners, we can bring new treatment options to market earlier.”
“LabCorp’s expanded collaboration with QIAGEN builds on our leading position in companion diagnostics, and it allows us to make those precision tests and new targeted drugs available sooner to physicians and patients,” said Marcia Eisenberg, Ph.D., Chief Scientific Officer of LabCorp Diagnostics. “This is perfectly aligned with LabCorp’s mission to improve health and improve lives by delivering world-class diagnostics, bringing innovative medicines to patients faster, and using technology to improve the delivery of care. LabCorp Diagnostics and our Covance Drug Development business already work closely with QIAGEN and its pharma partners on multiple oncology biomarker and clinical trial programs, and we are pleased to join the Day-One Lab Readiness program.”

Editas and BlueRock Forge Agreement to Develop Novel Engineered Cell Medicines

Cambridge, Mass.-based Editas Medicine and privately-held BlueRock Therapeutics have teamed up and combined their genome editing and cell therapy technologies to discover, develop and manufacture novel engineered cell medicines.
Financial terms of the deal were not disclosed. But, the companies said the research collaboration and cross-licensing agreement will create a platform for the discovery of “off-the-shelf engineered cell medicines with transformative potential.” Editas and BlueRock said the research collaboration will include a focus on the creation of novel, allogenic pluripotent stem cell (PSC) lines utilizing a combination of Editas Medicine’s CRISPR genome editing technology and BlueRock’s induced pluripotent stem cell (iPSC) platform.
The companies said the collaboration will enable the creation of engineered, differentiated, off-the-shelf cell medicines in the respective fields of oncology, including solid tumors and blood cancers, for Editas Medicine and neurology, cardiology, and immunology for BlueRock. BlueRock gains non-exclusive rights to Editas Medicine’s CRISPR technology and intellectual property and Editas Medicine gains non-exclusive rights to BlueRock’s iPSC and cell differentiation technology and intellectual property, in each of their respective fields.
Under terms of the agreement between the two companies, Editas and BlueRock are both responsible for the development, regulatory, and commercial milestones to their respective partner for any licensed engineered cell medicine developed in their respective field as well as royalties on global net product sales.
Cindy Collins, the interim chief executive officer of Editas Medicine, said that combining CRISPR-based genome editing with cell therapy has the potential to “deliver game-changing allogeneic medicines.” The collaboration with BlueRock provides the potential to “enable and accelerate” the development of therapies for people with different diseases, Collins said.
Collins took over the reins of Editas last month after CEO Katrine Bosley abruptly announced resignation from her role at the company. Collins, who most recently served as CEO of Human Longevity Inc., as well as CEO of General Electric’s Healthcare Cell Therapy Business, has been a director of Editas Medicine since December.

Emile Nuwaysir, CEO of BlueRock, noted that the two companies share a common belief in the “disruptive potential of utilizing an engineered cell as a therapeutic.”
“We are equally committed and passionate about bringing these new treatment options to those living with diseases where the unmet medical need is high and growing. We are thrilled to partner with the world-class scientific team at Editas and believe that this new collaboration will allow both companies to meaningfully expand our pipelines in a manner that further positions both as leaders in our respective fields,” Nuwaysir said in a statement.
The deal with BlueRock isn’t the only one Editas has made in recent months. In February, Editas and Gamida Cell struck a deal to evaluate the Editas’ CRISPR technology to edit NAM-NK cells, which are natural killer cells that have been expanded using Gamida Cell’s proprietary nicotinamide-based technology. Through this agreement, the companies aim to discover optimized NAM-NK cells that could be used to improve the treatment of hematologic malignancies and solid tumors.