Category Archives: Pharmaceutical Development

Vaccine for Meningitis Shows Some Protection Against Gonorrhea

Scientists have not been able to develop a vaccine against the sexually transmitted disease gonorrhea, despite working toward one for more than 100 years.  However, they may have stumbled onto something that could provide clues to advance the development of such a vaccine.

Decades ago, in the late 1990s, a strain of meningitis B was reaching epidemic proportions in New Zealand. A vaccine, MeNZB, was developed to protect young people who were at the highest risk of getting this particular type. It did not provide protection against any other strain.

Between 2004 and 2006, MeNZB was given to anyone under the age of 20. Babies and preschoolers were routinely immunized until 2008. People with a high medical risk continued to get the vaccine until 2011. Once the epidemic was over, the vaccination program was stopped.

However, scientists noticed that the meningitis vaccine also seemed to offer some protection against gonorrhea. A study published in the Lancet last month showed that one-third of the people who had received MeNZB did not get gonorrhea, compared to a control group who was not inoculated. The lead author noted that the bacteria causing both diseases share between 80 and 90 percent of their primary genetic sequences.

Dr. Steven Black, an infectious disease expert at Cincinnati Children’s Hospital, noted, “This is the first time it’s been shown that you could have a vaccine that would protect against gonorrhea. And if these results are confirmed in another setting, that would mean that it would be very reasonable … to go forward with developing perhaps a more targeted vaccine.” Black’s comments were published in the current issue of JAMA, the Journal of the American Medical Association.  The JAMA article concludes that ultimately, a preventive vaccine could be the only sustainable solution to a fast-changing bug that has proven adept at developing resistance.

The World Health Organization reports that gonorrhea is becoming harder, and sometimes impossible, to treat, warning that it could become incurable in the not-too-distant future. At the moment, there no new antibiotics being developed to treat this disease.

The U.S. CDC reports that gonorrhea is the second most commonly reported notifiable disease in the United States. All known cases must be reported to the CDC, but officials there estimate that they are notified of fewer than half of the 800,000 new cases each year.

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IMMUNO-ONCOLOGY: Targeting the immune system, not the tumor

Immuno-oncology is based on the concept of harnessing the body’s own immune system to fight cancer.

One of the most exciting areas of cancer research today is immuno-oncology, and while it’s an approach that scientists first considered more than 100 years ago, recent scientific discoveries and clinical advances have ushered in a truly historical time in cancer research.

Immuno-oncology Video Capture

Image Courtesy of PhRMA (Video Capture)

Recently, PhRMA released a new video highlighting immuno-oncology, which is currently being researched and developed by Bristol-Myers Squibb and several other bio-pharmaceutical companies.

Cancer is clever and has found ways to outwit the immune system. Rather than killing these cancer cells directly with traditional tools like radiation or chemotherapy, immunotherapy seeks to intensify the immune system’s power to eliminate them. Immuno-oncology is already improving outcomes and survival rates for some patients, including melanoma, kidney and lung cancer, and researchers are urgently working to gain new insights into the complex interactions between patients’ immune systems and the cancer cells growing in their bodies with the goal of markedly improving outcomes in many more tumor types.

While the science has advanced rapidly in recent years, there is more work to do. Researchers hope to replace chemotherapy as the first line treatment for many cancers and help as many patients as possible achieve long-term survival. This new treatment approach has the potential to help patients live longer, healthier lives.

Learn more about advancements in science at From Hope to Cures.

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New Drug Holds Promise Against Zika

MV-4 Based On Research That Unlocks Key to Breaking Down Viruses

A New York pharmaceutical company, TGV-Laboratories, says it has unlocked the key for breaking down the protective barriers of viruses, a breakthrough that it believes can be developed into antiviral drugs to treat Zika and host of life-threatening viruses, including Ebola, HIV and Bird Flu.  The company’s lead researchers, Victor and George Tetz, said lab tests show their discovery, called MV-4, can break down the protective barriers of both enveloped and non-enveloped viruses, indicating it can be developed into targeted synthetic antiviral drugs to kill a wide variety viruses.

ZIKA Virus WordleArtIn laboratory tests, the researchers said they were able to kill influenza viruses, HIV, Herpes viruses, polio and adenoviruses, and are eager to test their discovery against Zika, Ebola and Bird Flu.  In a letter to the CDC, the researchers outlined their findings, and offered their help in developing new treatments against these global health threats.

“We are very excited about our findings to date, and are eager to expand the testing of our drugs on these dangerous and highly contagious viruses,” said Victor Tetz, scientific head of TGV-Laboratories. The Zika virus is primarily a mosquito-transmitted infection. However, it was recently isolated from semen, and there is evidence it can be transmitted through sexual intercourse — opening new frontiers for global spreading and raising the need for an antiviral drug that can attack it at many levels. “The studies we have conducted to date show MV-4 can inactivate viruses in the outer environment, on human skin and at intravenous administration, so we believe discovery shows huge promise against Zika, Ebola and Bird flu.”

TGV Laboratories, along with its Institute of Human Microbiology, are finalizing a paper that details their discovery and what they believe to be its strong potential for safely treating a broad range of life-threatening and non-life threatening viruses. Currently, there are no broad-spectrum antiviral drugs.  MV-4 is the second broad-spectrum drug candidate developed by TGV Laboratories, whose Mul-1867 has shown potential for being developed into drugs that can treat an extensive range of antibiotic-resistant bacterial and fungal infections. TGVs subsidiary, TGV-Inhalonix, recently filed an application with the Food and Drug Administration seeking Orphan Drug Status for Mul-1867 to be tested on cystic fibrosis patients with life-threatening antimicrobial-resistant pulmonary infections.

“Developing broad-spectrum drugs that can treat viruses and the growing list of antibiotic-resistant infections is one of the most important, but elusive, goals in infectious research today,” said George Tetz, head of research for the Institute of Human Microbiology. “With MV-4 and Mul-1867, we believe we have unlocked the key for developing the next-generation of antibiotics and anti-viral drugs.”

The drugs in TGV’s pipeline were discovered as the result of years of research by Victor and George Tetz using a new concept they developed called Pangenome, (http://www.ncbi.nlm.nih.gov/pubmed/15990697), which acts like a guide that helps mark new therapeutic targets for drugs. The Institute of Human Microbiology assisted TGV with Mul-1867’s discovery.

TGV-Laboratories Group of Companies. (http://tgv-labs.com) is a research-based pharmaceutical company headquartered in New York that provides a broad range of innovative products to treat currently untreatable infectious, autoimmune and neurodegenerative diseases. TGV operates under two divisions: the Division of Drug Discovery & Development and the Division of Medical & Industrial Microbiology.

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Quick Takes: the Pharma C-Suite with John Hudak, President: Outsourcing & Strategic Partnerships

In the recent PharmaVoice Feb 2016 PV MagCover FEB16 issue highlighting Outsourcing, PV asked Pharma’s KOL’s to answer some key questions regarding the current state of Outsourcing & Strategic Partnerships.

John Hudak, President & Founder of Criterium Inc Global CRO

John Hudak, President & Founder of Criterium Inc Global CRO

John M. Hudak, President and Founder, Criterium Inc., Global CRO answered these 2 questions for them:

  • What barriers continue to limit CROs and sponsors from becoming true development partners?

Sponsors can have unrealistic expectations of CRO partners. Most partnerships work top-down at both Sponsors and CROs, and are evidenced in timelines and budgets. But top-down management may not always translate into trickle-down commitment. There are some discouraging figures out there about the high percentage of studies that don’t meet their timelines. When timelines and budgets are estimated to satisfy board members or project committees, it’s imperative to include all key factors that affect early planning stages, and be grounded in fiscal reality.

  • What are the key ingredients to a successful outsourcing partnership?

Planning needs transparency and communication, in concert. Sponsors can sometimes have impractical expectations regarding timelines and budgets. And CROs can end up having the same unrealistic expectations of study sites. Everyone wants to meet expectations and win the job – but this sometimes results in unworkable time estimates and budgets. There needs to be more open discussion among stakeholders, Sponsors, CROs and sites regarding the environments in which studies are conducted — and realistic strategies for successful completion.

About John M. Hudak, MBA, President and Founder
John has more than 40 years of experience in the pharmaceutical research and business development arena serving the pharmaceutical and contract clinical services sectors. He has extensive expertise in strategic planning and competitive analysis, market development, promotional planning and tracking, proposal design and custom-services development, international project management and clinical study completion, protocol design and implementation, electronic data capture, and market communications and has worked with drugs, biologics, generics and medical devices.

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For Cancer Patients, The Possibility Of DNA-Tailored Care

Holly Boehle looked at the expression on the face of her radiologist and knew something was wrong. Boehle had felt a lump just before the holidays and had decided to schedule a mammogram, even though she wasn’t due to start standard screening for another eight years. The mammogram was followed up with an ultrasound, and then a biopsy. Finally, she was given the formal diagnosis: invasive ductal carcinoma, the most common type of breast cancer.

The next week she traveled to Mayo Clinic’s oncology office in Rochester, Minn., where she met with clinical breast surgeon Dr. Judy Boughey who suggested Boehle consider participating in a new, ongoing study. She quickly agreed and became one of 140 women to take part in the Breast Cancer Genome-Guided Therapy Study, affectionately called “BEAUTY.”

By the end of the month, the first phase of the study was underway. Tumor tissue was taken from Boehle, and promptly brought into a lab where the sample was injected into mice with compromised immune systems. With her tumor tissue growing inside of them, the avatar mice are given different chemotherapy drugs in order to test the efficacy of treatment before they try it out on human Boehle.

BEAUTY’s Beginnings

Each patient in BEAUTY was given a biopsy, imaging, and chemotherapy treatment, followed by a second round of biopsy and imaging before they headed into the operating room. This gave researchers information on each patient’s blood and genetic makeup, and the sequencing information for their tumor before, during, and after chemotherapy treatment. The mouse avatars, known as patient xenografts, took up the patient’s individualized tumor 40 percent of the time, ultimately serving as a preview into the patient’s treatment outcome.

DNA Strand Free CCLicenseA class of chemo drug called taxanes are the standard of treatment for breast cancer, but doctors currently don’t have a genetic marker to indicate who will respond to taxane therapy and who won’t. That’s why anthracyclines and cyclophosphamide, a separate chemotherapy drug regimen, are typically given in conjunction as the first step in treatment followed by taxane therapy. However, Boughey’s team reversed the sequence for BEAUTY patients whose mice reacted well to the taxane treatment first. It turned out that those patients who responded best to the flipped treatment sequence also shared the same gene in their genetic makeup.

Based on how her mice reacted to treatment, Boehle was one of those patients who were treated with a reverse chemotherapy schedule than what the typical patient with invasive ductal carcinoma is treated with. Within six months of her diagnosis, Boehle’s tumor shrank considerably as a reaction to the chemotherapy.

Being part of the trial has long-term implications for Boehle, too. If the cancer were to recur, she says, “we would already know…what works for me and what doesn’t. It really opens up a whole new world for me and other breast cancer patients in terms of individualized medicine and knowing that I don’t need to be the person who they experiment on and say, ‘Let’s try this medication or chemotherapy and see if it works and we hope that it does.’”

Four years ago, when Boughey and her team began setting up the BEAUTY study, they wanted to be able to design a treatment plan with a relatively accessible patient population. Because breast cancer is so common among women, the research team chose to start work on individualized medicine with those patients with plans to eventually work their way to other solid tumor cancers such as prostate.

By sequencing the genome for both the tumor and the patient’s inheritable DNA, researchers are able to pull the curtain back and see what’s driving the tumor to grow, why it’s different from another tumor, and how the tumor might react to drug treatments. Harnessing the genetic sequence of a tumor in conjunction with a person’s DNA will allow doctors to expand personalized cancer treatments beyond breast cancer.

Standard of Care Tumor Sequencing

Sequencing a tumor for its complete genetic information can take as little as a few days, and as long as several weeks, depending upon the stage of cancer. Once they have the results, researchers then compare them to a patient’s individual germline cells, which contain hereditary mutations that occur during conception. Patients born with germline mutations can pass on to future generations. Somatic mutations can be caused by a number of different environmental factors and can occur spontaneously. As researchers unravel which gene mutations are responsible for causing each corresponding disease, it sets the foundation for creating individualized treatments through trial-and-error.

“From there potential drugs can be identified that act on genes and/or pathways,” Boughey and her colleague Dr. Matthew Goetz, a clinical oncologist at the Mayo Clinic, told Medical Daily in an email. “One novel aspect of the BEAUTY clinical trial is our ability to link drug response in the patients with both germline and somatic genomic information and validate using the patient derived xenografts [mouse avatars].”

Mayo Clinic’s research team is currently writing the protocol for BEAUTY 2 based on the types of tumors they identified, and the drug resistance and successes discovered in BEAUTY 1. Because they were able to prove mouse avatars, when administered the same drug that patients were treated with, mirror the drug response seen in patients, pharmaceutical companies will be involved with this next stage by designing medications based on study participants’ response to treatment.

BEAUTY, Boughey says, will “drive forward breast cancer treatments for the future.”

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Ophthalmology Devices Global Market Forecast (to 2021)

Ophthalmology is a segment of medical science associated with study of anatomy, physiology, diseases of human eye, and developing various therapeutic methods to treat eye diseases.

SUMMARY:  Some of the major eye diseases are either age-related or caused due to chronic disorders. They include various degenerative eye diseases like macular degeneration, cataract, ocular hypertension (glaucoma), and refractive errors among others. Revolution in the field of medical science has led to the rapid development of ophthalmology devices that are invented to effectively treat ocular defects and disorders.

Ophth Devices REPORTOphthalmology devices market is segmented on the basis of products type as diagnostic devices, surgical devices and vision care. Based on the applications the ophthalmology devices market is segmented into cataract, glaucoma, age-related macular degeneration, diabetes retinopathy and others (refractive errors, amblyopia, and strabismus). End-users are segmented into hospitals, academic and research laboratories, and others (private eye clinic and vision care outlets).

The global ophthalmology devices market is expected to grow at a CAGR of around 5.8% from 2015 to 2021. Increasing incidence of degenerative diseases of eyes, increasing baby boomer population, increase in R&D activities in ophthalmology key players and extensive use of high-end technologies involving use of software and computer aided devices and platforms in ophthalmology drives the market of ophthalmology devices market. Lack of ophthalmologists, economic slowdown and saturation of the market in developed countries are the factors hampering the market growth.  (Access the full report: http://www.reportlinker.com/p03086455-summary/view-report.html)

Lifestyle changes owing to increase in ocular cancer, diabetes and macular degeneration patients, early diagnosis and treatment of diseases, advancement of technology with its wide application areas shows that ophthalmology devices market has vast opportunities in the coming years.

North America accounts for the highest market share followed by Europe. Steep rise in aging population, increase in minimally invasive surgeries and favorable government policies makes U.S. the leader of ophthalmology devices market. However, Asian countries especially India and China’s are the fast growing regions with its growing demand for ophthalmology devices and increasing research investments.

Major players in ophthalmology devices market include: Abbott Medical Optics, Inc. (USA), Alcon Laboratories, Inc. (USA), Bausch & Lomb, Inc. (USA), Carl Zeiss (Germany), Essilor International SA (USA), Haag-Streit Holding AG (Switzerland), Hoya Corporation (Japan), Nidek Co. Ltd (Japan), STAAR Surgicals (USA), Topcon Corporation (Japan).

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HIV Uses Immune System’s Own Tools to Suppress Itself

Canadian scientists make a significant discovery on HIV persistence

A Canadian research team at the IRCM in Montréal, led by molecular virologist Éric A. Cohen, PhD, made a significant discovery on how HIV escapes the body’s antiviral responses. The team uncovered how an HIV viral protein known as Vpu tricks the immune system by using its own regulatory process to evade the host’s first line of defense. The findings pave the way for future HIV prevention or cure strategies.

The study’s goal was to determine how HIV manages to compromise antiviral responses in the initial period of infection, also called the acute infection stage, during which the virus establishes itself in the body. The acute infection is considered a critical period in determining the complexity, extent and progression of the disease. It is also during this stage that HIV establishes latent infection in long-lasting cellular reservoirs. These viral reservoirs, which harbor the virus out of sight from the immune system and antiviral drugs, represent the primary barrier to a cure.

“An important component in this process is a group of proteins collectively called type 1 Interferons, which are the immune system’s first line of defense against viral infections and are known to have a beneficial role in the early stages of HIV infection,” says Dr. Cohen, Director of the Human Retrovirology research unit at the IRCM. “The problem is that HIV has developed mechanisms to suppress the Interferon response and, until now, little was known about how this was achieved.”

PHIL-18143 PubDomLic SEM HIV Particles courtesy NIAID

SEM HIV Particles courtesy NIAID PHIL-18143

Most of the Interferon is produced by a very small population of immune cells called pDCs (plasmacytoid dendritic cells), responsible for providing immediate defense against infections. PDCs patrol the body to detect invaders and, when they recognize the presence of a pathogen, they secrete Interferon. The Interferon then triggers a large array of defense mechanisms in nearby cells, creating an antiviral state that prevents the dissemination and, ultimately, the expansion of the virus.

“When pDCs encounter HIV-infected cells, the production of Interferon is regulated by a protein located on the infected cell’s surface called BST2,” explains Mariana Bego, PhD, first author of the study and research associate in Dr. Cohen’s laboratory. “BST2 has the ability to bind to and activate a receptor called ILT7, found on the surface of pDCs, which, in turns, sends a signal that suppresses the production of Interferon and halts its defensive functions. Interestingly, BST2 is also responsible for restricting HIV production by trapping the virus at the cell surface before it can exit infected cells and disseminate. However, HIV uses the viral protein Vpu to counteract BST2 antiviral activity.”

“With this study, we uncovered a unique mechanism whereby HIV exploits the regulatory process between BST2 and ILT7 to limit the body’s antiviral response, which allows the virus to spread and leads to persistent infection,” adds Dr. Bego. “We found that HIV, through Vpu, takes advantage of the role played by BST2 by maintaining its ability to activate ILT7 and limit the production of Interferon, all the while counteracting its direct antiviral activity on HIV production.”

“The hope for a definitive cure and an effective vaccine has been frustrated by HIV’s endless propensity to subvert the host’s defenses and persist in small populations of long-lasting reservoirs despite antiretroviral therapy,” describes Dr. Cohen, who also leads CanCURE, a team of leading Canadian researchers working towards an HIV cure. “Our findings can provide tools to enhance antiviral responses during the early stages of infection. By blocking Vpu’s action, we could prevent early viral expansion and dissemination, while also allowing pDCs to trigger effective antiviral responses. We believe that such interventions during primary infection have the potential to limit the establishment and complexity of viral reservoirs, a condition that seems required to achieve a sustained HIV remission.”

“The discovery by Drs. Bego and Cohen, which explains how the virus can’t be held down or wiped out during early periods of infection, will bring us closer to ending HIV/AIDS,” says Robert Reinhard, CanCURE Community Liaison. “By filling an important gap in knowledge, this new study will advance research for an HIV cure.”

About the study
The research project was funded by the Canadian Institutes of Health Research (CIHR), the Canadian HIV Cure Enterprise (CanCURE) through a partnership between CIHR, the Canadian Foundation for AIDS Research (CANFAR) and the International AIDS Society (IAS), as well as by a pilot project from the FRQS AIDS and Infectious Disease Network. The study’s authors also include Édouard Côté and Johanne Mercier from the IRCM, as well as Nick Aschman and Winfried Weissenhorn from the Université Grenoble Alpes in France. For more information on the study, please refer to the article published online by PLoS Pathogens

Findings from this study presented by Dr. Cohen on July 21 at the Vancouver Convention Centre as part of the 8th IAS Conference on HIV Pathogenesis, Treatment and Prevention.

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Other Gene Tests for Cancer

Other than the BRCA1 and BRCA2 gene mutation test for breast and ovarian cancer, there are other gene-related tests available for cancer.

As an example, one such test that is available is for Lynch syndrome, a hereditary condition which comes with a family history of colon cancer and other types of cancer at a younger age. Those with the syndrome have a higher risk of colorectal and uterine (womb) cancers.

Like BRCA screening, the genetic screening test for Lynch is expensive and is recommended only for high-risk individuals with a strong family history of such cancers.

Trying to find the Lynch syndrome in the general population would be rather costly and not necessarily helpful, said Dr Lim Sheow Lei, a senior consultant at the department of gynaecological oncology at KK Women’s and Children’s Hospital.

BLOG CTA ButtonRead more about how targeted treatments and translational science research methods are creating better outcomes for patients with cancer

The field of cancer genetics is a rapidly evolving area.   But things are changing quickly.  As genomic technology advances, the cost of sequencing the human genome is plunging, said Dr Lim. Just two weeks ago, a US company announced it was offering a much cheaper and easier way for women to get tested for the BRCA1 and BRCA2 genetic mutations.

Color Genomics* has begun selling a test (USD $249) that it said could accurately analyze a saliva sample for the mutations, as well as check for 17 other genetic variants that have been associated with a somewhat increased risk for cancer of the breast or ovaries.

Cancer can sometimes appear to “run in families” even if it is not caused by an inherited mutation.  Acquired mutations are the most common cause of cancer. These mutations occur from genes damaged during a person’s life. Smoking, viruses, and aging can damage genes and cause these mutations.

One such cancer caused by an acquired mutation is cervical cancFREE MS Art - Cancer Biology Ribboner, which stem from the human papilloma virus (HPV). Last year, the US Food and Drug Administration approved the HPV DNA test, manufactured by Roche, as a first-line primary screening test in women 25 and older. The geno-typing test detects DNA from 14 high-risk HPV types and specifically identifies HPV 16 and HPV 18, which carry the highest risk of cervical cancer. It can be used to help a healthcare professional assess the need for the woman to undergo additional diagnostic testing for cervical cancer. The test can also provide information about the patient’s risk for developing cervical cancer in the future.

Women who test positive for HPV 16 or HPV 18 should have a colposcopy, an examination using a device that illuminates and magnifies the cervix so a doctor can directly observe the cervical cells. Those who test positive for one or more of the 12 other high-risk HPV types should have a PAP smear to determine the need for a colposcopy. In a United States study of more than 47,000 women that compared HPV DNA tests with PAP smear tests, up to one in three women with cervical cancer had a normal PAP smear result.

Scientists and researchers are uncovering more genetic markers for other cancers.  The challenge is to know what to do with these pieces of information, how to interpret the multitude of newly uncovered mutations, work out the associated cancer risks and to manage the risks, said Dr Lim.

(Reprinted with license and permission from The Straits Times by Ng Wan Ching)
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*Information and link for Color Genomics does not constitute our endorsement of or any liability associated with the product, company or service.  Please consult a physician in case you believe you or someone you know may have cancer.

Halozyme: Update Following Type B FDA Meeting

Recent FDA meeting allows for potential marketing application based on progression free survival in pivotal Phase 3 pancreatic cancer study

Halozyme Therapeutics, Inc.,a biotechnology company developing novel oncology and drug-delivery therapies, today announced it plans to proceed with a Phase 3 clinical study (Study 301) of its investigational new drug PEGPH20 in patients with metastatic pancreatic cancer, using a design allowing for potential marketing application based on either progression free survival (PFS) or overall survival. The use of PFS as the basis for marketing approval will be subject to the overall benefit and risk associated with PEGPH20 combined with nab-paclitaxel (ABRAXANE ®) and gemcitabine therapy, including the:

  • Magnitude of the PFS treatment effect observed;
  • Toxicity profile; and
  • Interim overall survival data.

The study will enroll patients whose tumors accumulate high levels of hyaluronan (HA) – a sugar that is sometimes more prevalent in the areas surrounding cancer cells. Halozyme recently discussed the study as part of a planned Type B meeting with the U.S. Food and Drug Administration (FDA).

bullseyeData discussed at the meeting focused on interim results from the company’s randomized Phase 2 study in pancreatic cancer, Study 202, which showed a doubling in median PFS in metastatic pancreatic cancer patients with high levels of HA who were treated with Halozyme’s investigational new drug PEGPH20 combined with nab-paclitaxel (ABRAXANE ®) and gemcitabine (9.2 months vs. 4.3 months in patients treated with nab-paclitaxel ABRAXANE ® and gemcitabine alone). The potential risk profile, including rate of thromboembolic events, was also discussed.  Additional takeaways from the meeting include:

  • Halozyme affirmed plans to enroll and evaluate high-HA patients using a companion diagnostic test; and
  • FDA provided feedback on the current companion diagnostic approach and confirmed that an approved companion diagnostic strategy is required prior to Phase 3 study initiation.

“We are encouraged by the FDA’s feedback and their willingness to consider a novel primary endpoint such as PFS given the high unmet medical need in high-HA metastatic pancreatic cancer,” said Dr. Helen Torley, President and Chief Executive Officer. “We are committed to rapidly finalizing the Phase 3 protocol and developing the companion diagnostic regulatory filings required to initiate Study 301.” Based on this FDA feedback, the company is targeting the end of first quarter 2016 to initiate the Phase 3 study.

About Study 202 & PEGPH20microscope in laboratory
Study 202 (Halo 109-202) is a Phase 2 multi-center, randomized clinical trial evaluating investigational new drug PEGPH20 as a first-line therapy for treatment of patients with stage 4 metastatic pancreatic cancer. The primary outcome of the trial is to measure improvement in progression-free survival in patients receiving investigational new drug PEGPH20 in combination with gemcitabine and nab-paclitaxel compared to gemcitabine and nab-paclitaxel alone. A second primary endpoint will assess the thromboembolic event rate in the PEGPH20 treatment arm. Secondary endpoints also include objective response rate and overall survival. More information may be found at: http://oncologytrials.halozyme.com/pancreatic/.

PEGPH20 is an investigational PEGylated form of Halozyme’s proprietary recombinant human hyaluronidase under clinical development for the systemic treatment of tumors that accumulate hyaluronan. FDA granted orphan drug designation to PEGPH20 for treatment of pancreatic cancer and fast track for PEGPH20 in combination with gemcitabine and nab-paclitaxel for the treatment of metastatic pancreatic cancer. Additionally, the European Commission, acting on the recommendation from the Committee for Orphan Medicinal Products of the European Medicines Agency, designated investigational drug PEGPH20 an orphan medicinal product for the treatment of pancreatic cancer.

About Halozyme
Halozyme Therapeutics is a biotechnology company focused on developing and commercializing novel oncology therapies that target the tumor microenvironment. Halozyme’s lead proprietary program, investigational drug PEGPH20, applies a unique approach to targeting solid tumors, allowing increased access of co-administered cancer drug therapies to the tumor.  PEGPH20 is currently in development for metastatic pancreatic cancer and non-small cell lung cancer and has potential across additional cancers in combination with different types of cancer therapies. For more information, visit www.halozyme.com.

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FDA Approves Pulmozyme with eRapid Nebulizer

CF Patients See 2 – 3 Minute Treatment Times

The eRapid Nebulizer System (eRapid) from PARI has been approved as the first electronic nebulizer by the Food and Drug Administration to deliver Genentech’s Pulmozyme for cystic fibrosis treatment.  A huge improvement for cystic fibrosis patients, eRapid is able to reduce average treatment times with Pulmozyme from 6-8 minutes down to 2-3 minutes.  Both adult and pediatric patients showed a strong preference for eRapid over conventional nebulizers in a clinical study. FDA-Logo

“We have been pleased with eRapid’s fast treatment times in the lab and are excited that patients now have access to a much faster Pulmozyme therapy.  As the first electronic nebulizer to deliver Pulmozyme, eRapid is a true breakthrough for cystic fibrosis patients who take the therapy daily, often for years,” said Lisa Cambridge, director of Medical Science and Pharmaceutical Alliances at PARI Respiratory Equipment, Inc.

“PARI was motivated to introduce eRapid to the US market based on encouragement from the Cystic Fibrosis Foundation and their input to have a general-use, electronic nebulizer that could improve therapy adherence. For many years, eRapid has been successfully distributed in Europe (as eFlow Rapid) with favorable feedback from patients with CF. In the Pulmozyme clinical trial, there was a 10:1 preference for eRapid in the pediatric group and a 20:1 preference in the adult group.  That confirmed our decision to bring eRapid to the US,” added Geoff A. Hunziker, president of PARI USA.

“After the successful results of a Phase IV study, we are confident that physicians will see that both pediatric and adult patients favor eRapid based on reduced treatment times, quiet operation, and its small, portable size.  We were also happy to see that patients were more satisfied with treatment and eRapid had a positive influence on adherence – good for their overall cystic fibrosis management,” added Lisa Cambridge.  eRapid is available today by prescription through a select group of specialty pharmacies.  Visit www.pari.com or call 1-800-FAST-NEB to learn more.

Pulmozyme is indicated for daily administration along with standard therapies for the management of cystic fibrosis to improve pulmonary function.  For more information, visit http://www.pulmozyme.com/.

About eRapid Nebulizer System eRapid is an electronic nebulizer that enables quick and efficient inhalation that can greatly reduce a patient’s treatment burden.  For more information on eRapid, please visit http://www.pari.com/.

About Cystic Fibrosis Cystic fibrosis, a life-threatening disease affecting 30,000 American patients, involves a genetic mutation that results in poorly hydrated, thickened mucus secretions in the lungs, as well as severely impaired mucociliary clearance.  Get more information about Cystic Fibrosis, visit http://www.cff.org

About PARI Respiratory Equipment, Inc. PARI is a leading, worldwide developer and manufacturer of fast and efficient aerosol delivery systems for patients with asthma, chronic lung disease, cystic fibrosis, RSV, VAP, and HAP.  PARI’s worldwide vision is to improve the lives of those affected by respiratory diseases and those who care for them. PARI is considered the gold standard for aerosol delivery for nebulizer therapies.  Featured products include the PARI LC PLUS Reusable Nebulizer, Vortex Holding Chamber, and the drug-specific eFlow Technology platform.

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