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Tuesday, October 2, 2018

Clovis Gets Breakthrough Therapy Tag for Prostate Cancer Med


 Clovis Oncology, Inc. (NASDAQ: CLVS) announced today that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation for Rubraca® (rucaparib) as a monotherapy treatment of adult patients with BRCA1/2-mutated mCRPC who have received at least one prior androgen receptor (AR)-directed therapy and taxane-based chemotherapy.
Breakthrough Therapy designation is granted by the FDA to investigational agents intended to treat a serious or life-threatening disease or condition and whose preliminary clinical evidence may demonstrate substantial improvement on at least one clinically significant endpoint over available therapy. The FDA previously granted Breakthrough Therapy designation to Rubraca for the monotherapy treatment of certain advanced ovarian cancer patients and then in December 2016 approved Rubraca for the treatment of certain adult patients with deleterious BRCA mutation (germline and/or somatic) associated epithelial ovarian, fallopian tube, or primary peritoneal cancer who have been treated with two or more chemotherapies. The FDA subsequently approved Rubraca in a second indication, the maintenance treatment of adult patients with recurrent epithelial ovarian, fallopian tube, or primary peritoneal cancer who are in a complete or partial response to platinum-based chemotherapy, in April 2018.
“We are committed to the rapid development of Rubraca in mCRPC and we are obviously pleased to receive Breakthrough Therapy designation. We look forward to presenting the data that served as the basis of our BTD application at the ESMO conference later this month,” said Patrick J. Mahaffy, President and CEO of Clovis Oncology. “We hope the decision by the FDA to grant this Breakthrough Therapy designation for Rubraca offers encouragement to the prostate cancer community, and we will do our best to make Rubraca available to eligible prostate cancer patients as quickly as possible.”
This most recent Breakthrough Therapy designation was granted to Rubraca based on initial efficacy and safety results from TRITON2, the Phase 2 study of Rubraca in men with advanced prostate cancer with BRCA 1/2 mutations (germline or somatic) and deleterious mutations of other homologous recombination (HR) repair genes, in the metastatic castration-resistant setting.
Initial data from the TRITON2 clinical study, which served as the basis for BTD, will be presented for the first time at the 2018 European Society for Medical Oncology (ESMO) Congress taking place October 19-23, 2018, in Munich, Germany.
“We are pleased the FDA has granted Breakthrough Therapy designation to Rubraca in mCRPC,” said Howard R. Soule, Ph.D., Executive Vice President and Chief Scientific Officer of the Prostate Cancer Foundation. “There is tremendous need for new therapeutic options in advanced prostate cancer. In particular, we are enthusiastic about the potential for targeted therapies that may provide more meaningful benefit to patients with specific genetic mutations.”
Data from the TRITON2 clinical study will also be presented in an oral presentation at the 25thAnnual Prostate Cancer Foundation Scientific Retreat, taking place October 26-28, 2018, in Carlsbad, CA.
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Reduce Snoring by Playing a Smartphone App


In partnership with the University of Minnesota’s Sleep Centers and the Medical Devices Center, researchers recently demonstrated the ability to reduce snoring through a noninvasive therapy facilitated by a smartphone game.
Soundly addresses the root cause of snoring through the smartphone-based delivery of oropharyngeal exercises. (The screen shown is from actual gameplay.)
Snoring occurs when the upper airway muscles relax, obstructing a patient’s airflow, which causes noisy tissue vibrations during inhalation. In their research paper, “Smartphone-based delivery of oropharyngeal exercises for treatment of snoring,” Brian Krohn, PhD., Adam Black, PhD., and Dr. Umesh Goswami, MD, prove that these noisy vibrations can be reduced by performing an oral therapy designed to strengthen and tone specific muscles within the upper airway.
Snoring continues to be the most common complaint in sleep medicine clinics, affecting 35 percent of all adults. In addition to negatively impacting sleep partners, snoring may be a predictor of obstructive sleep apnea, which can lead to high blood pressure, inflammation, and numerous other health risks.
Existing snoring treatments are often uncomfortable and require expensive devices that need to be worn during sleep. They tend only to address the symptoms of snoring, not the root cause.
Recognizing the universal need for a simpler solution to snoring, Dr. Krohn enlisted the help of sleep physicians, speech pathologists, and video game developers to create Soundly, the first app-based therapy clinically proven to reduce snoring. Soundly accomplishes this by presenting a visually stimulating, gamified therapy in which an onscreen character responds to scientifically optimized vocal commands dictated by the user.
“The ‘ee’ sound moves the tongue to the front of the mouth and the ‘aw’ sound moves the tongue to the back of the mouth,” explains Dr. Krohn, Soundly’s founder and CEO. “Meanwhile, the ‘ng’ sound engages the muscles of the soft palate. By saying ‘nee’ and ‘naw’ in succession, the user is able to move their in-game character while doing the perfect pushup for their oropharyngeal muscles.”
With 100 percent of test subjects’ bed partners reporting a reduction in snoring and support from the medical sleep community, Soundly continues to focus its app on delivering a science-driven sleep therapy through the vehicle of a gamified smartphone app. The newly updated Soundly app is available in the iOS App Storeand will debut at Somnex, The Sleep Show in London, Oct. 12-14. Get details here.
About Soundly: 
Soundly is a Minnesota-based medical technology company founded in 2017. Research for Soundly is supported by the National Institute of Health (NIH) and the National Science Foundation (NSF) in conjunction with a 2017 sleep study. Research partners include the Medical Sleep Clinic, Fairview Sleep Centers, and Medical Devices Center.
To download a free trial of Soundly, visit the app store from any iPhone or iOS device.
For more information on Soundly, visit www.sleepsound.ly or contactcoreybrenner@soundlyapp.com 
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FDA OKs Paratek-Allergan Acne Treatment


Paratek Pharmaceuticals, Inc. (Nasdaq: PRTK), a biopharmaceutical company focused on the development and commercialization of therapies based upon tetracycline chemistry, today announced that the U.S. Food and Drug Administration (FDA) has approved SEYSARA™ (sarecycline) for the treatment of inflammatory lesions of non-nodular moderate to severe acne vulgaris in patients 9 years of age and older. Paratek has exclusively licensed U.S. development and commercialization rights of SEYSARA for the treatment of acne to Allergan PLC, who has assigned such rights to Almirall SA. Paratek retains development and commercialization rights in the rest of the world. SEYSARA (sarecycline) is a once-daily, oral, narrow spectrum tetracycline-derived antibiotic with anti-inflammatory properties for the potential treatment of moderate to severe acne in the community setting. Under the parties’ agreement, Paratek earned a $12 million milestone payment upon FDA approval and is now entitled to receive tiered royalties at rates ranging from high-single to low double digits on net sales of SEYSARA.
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Avrobio Hit by Mixed Data from Fabry Disease Gene Therapy Trial


Shares of Cambridge, Mass.-based AVROBIO plunged more than 50 percent on Monday after the company provided an updated look at its gene therapy for Fabry disease that showed promise for the treatment, but also raised some concern for investors.
Citing interim data, Avrobio announced that the first two patients in its Phase I study of its AVR-RD-01 gene therapy treatment continued to “demonstrate AGA enzyme activity above the diagnostic range for classic Fabry disease 18 months and six months.” The company said that the first patient is no longer receiving enzyme replacement therapy. For the patients on the treatment, that was the good news.
Geoff MacKay, president and chief executive officer of Avrobio, touted the results of the treatment for the first two patients.
“We are encouraged by the AGA enzyme activity we are seeing after treatment with AVR-RD-01 in the first two patients with Fabry disease in the Phase 1 study. Both of these patients have AGA activity that remains above the diagnostic range for males with classic Fabry disease, and all patients will continue to be followed for assessment of long-term durable response. We are especially pleased that patient 1 was taken off ERT in mid-July and remains off,” MacKay said in a statement.
Avrobio’s AVR‑RD‑01 is an ex vivo lentiviral gene therapy that is designed as a one-and-done treatment. The therapy works by inserting the GLA gene that encodes functional α‑galactosidase A (AGA), the enzyme that is deficient in Fabry disease. By inserting the GLA gene, the therapy should enable continued AGA production and distribution to tissues and organs. From the initial data, that appears to be happening with AVR‑RD‑01.
However, what has investors concerned is not the results of the AGA enzyme activity, rather it was the fact that four patients saw a drop in vector copy number in peripheral blood. VCN refers to the average number of copies of the lentiviral-vector inserted gene that are integrated into the genome of a cell. While Patient 1 has been taken off the enzyme replacement therapy, his VCN was 0.1. Patient 1 began the study with a VCN of .7. Patient 2 had a VCN of .4 and began with a 1.4. The other two patients also saw a similar decline. That’s what caused share prices to plunge on Monday – wiping out significant gains the company’s stock has made since its initial public offering this past spring when it brought in in $99.7 million.
On Monday, shares fell from Friday’s closing price of $51.80 per share to $25.13.
In separate news, Avrobio announced that it will initiate a Phase I/II trial in Canada to test its lentiviral gene therapy treatment AVR-RD-02 for Gaucher disease. This therapy is designed to permanently integrate the GBA gene that encodes functional glucocerebrosidase, the enzyme that is deficient in Gaucher disease, to enable continuous GBA production and distribution to tissues and organs.
Additionally, the company reported first-patient information from a Phase II trial testing AVR-RD-01, data from the first patient shows AGA plasma enzyme activity was 2.74, but the VVCN was .5.
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Cannabis-Based Med Firms Hope Epidiolex Paves Way for More Approvals


Following the U.S. Drug Enforcement Agency’s rescheduling of GW Pharmaceuticals‘ Epidiolex from a Schedule I to a Schedule V, other cannabis-based drug development companies believe there is momentum for their medications to gain regulatory approval.
In June the U.S. Food and Drug Administration (FDA) approved Epidiolex as a treatment for seizures associated with two rare forms of epilepsy, Lennox-Gastaut syndrome (LGS) and Dravet syndrome. The approval of Epidiolex marked the first time the FDA approved a drug that contains a purified drug substance derived from marijuana.
This morning Las Vegas-based GB Sciences, Inc. said the rescheduling and approval of Epidiolex “validates the commercial potential” of the company’s assets. Like U.K.-based GW Pharmaceuticals, GB Sciences is developing cannabinoid therapies for a number of medical conditions. GB’s lead asset is focused on Parkinson’s disease. GB Sciences noted that a significant consequence of the DEA’s rescheduling of Epidiolex, a treatment for a rare form of epilepsy, established that Epidiolex can be distributed to patients through standard pharmaceutical channels. That means, the company said, it will be easier for future cannabis-based medicines to get to patients should they pass regulatory muster.
Despite the DEA’s rescheduling of Epidiolex, other non-FDA-approved CBD preparations remain in Schedule I. That is a sore point for some other CBD drugmakers like GB Sciences.
John Poss, chief executive officer of GB Sciences said GB Sciences is not a “cannabis company that dabbles in science,” rather it is a “biopharmaceutical company that happens to grow cannabis.”
“Although the recent ruling by the DEA is a positive development, it falls short of giving researchers the tools required to rapidly advance cannabis-based drug research and development,” Poss said in a statement.
The hope for GW Pharma’s medication to pave the way for future CBD drugs to gain approval comes days after GB Sciences struck a deal with U.K.-based Catalent Pharma Solutions to provide oral delivery systems, formulation development, and clinical-scale oral dose manufacturing of GB Sciences’ CBD treatments, GBS101, GBS102, and GBS103, for Parkinson’s disease. The company’s assets are complex mixtures containing up to nine cannabinoid-containing complex mixtures. It’s that combination of components that the company said are “more potent than would have been predicted by combining the activity of each individual compound.”
Andrea Small-Howard, chief science officer of GB Sciences, said she was hopeful about the rescheduling of CBD medications to Schedule V, particularly in light of the announcement that Catalent will be formulating the cannabis-based Parkinson’s disease formulations in preparation for the company’s planned exploratory investigational new drug-filing
“This may be the best time in history to be developing cannabis-based medicines,” Small-Howard said in a statement.
To date, GB Sciences said it has filed four patent applications for its cannabinoid-containing complex mixtures for use in four disease categories: neurodegenerative disorders, cardiovascular diseases, inflammatory disorders, and chronic and neuropathic pain. The company said more than 60 potential medical conditions can be addressed by its cannabis-based formulations. CBD is a therapeutic component in about 20 percent of these formulations, the company said. Additionally, the company noted that less than 10 percent of its formulations contain delta-nine tetrahydrocannabinol (THC), the psychoactive component of cannabis.
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5-Member GO Therapeutics Inks $195 Million Deal with Roche


GO Therapeutics, headquartered in Cambridge, Mass., signed a licensing deal with Swiss-based Roche to develop antibodies for cancer.
Under the terms of the deal, GO will receive upfront and near-term milestones of $9 million. GO is also eligible for up to $186 million in milestone payments in addition to mid-single-digit to low double-digit royalties on any products that might come out of the agreement.
GO Therapeutics focuses on advances in glycobiology to develop cancer drugs that are both more potent and less toxic. It develops cancer-specific antibodies that can be used as antibody-drug conjugates (ADC), bispecific T-cell engagers and immune-based cell therapies.
“We are excited about this collaboration to develop an innovative immune-redirected therapy to potentially improve the lives of patients suffering from cancer in the future,” said Constantine Theodoropulos, GO Therapeutics’ chief executive officer, in a statement. “GO’s glycoprotein targeting platform opens an exciting class of tumor-specific antigens that can help widen the therapeutic window for cancer therapies such as T-cell bispecific antibodies, CAR-T and ADCs (Antibody Drug Conjugates). Preclinical data show GO’s approach can provide superior specificity in targeting solid tumors over normal tissue and demonstrate clean in-vivo toxicity profiles in the context of potent immunotherapies.”
GO Therapeutics is a tiny company, which makes the size of the deal, relatively small for the industry, look huge in comparison. GO Therapeutics has a staff of five. It is operating out of shared laboratory space at LabCentral in Cambridge.
The company’s technology is designed to be more targeted, which would make the drugs more effective with fewer side effects since they have less impact on healthy cells. The company was founded by Theodoropulos, Hans Wandall and Thayer White.
Theodoropulos has been a business consultant in the life sciences and technology for about 20 years. Before GO, he was founder and president of Base Pair Group, a consultancy. He was head of strategic marketing for EBD Group, a life science partnering events company, where he worked on corporate strategy, product development and marketing. EBD Group was sold to Informa in 2014.
Wandall is the co-director of the center for glycobiology at the University of Copenhagen. His research focuses on the genetics of glycan function, creating epithelial tissue models used to dissect glycan functions in cancer and viral infection.
White earned his doctorate at the University of Washington. He has worked at The Pacific Northwest Research Foundation, The Fred Hutchinson Cancer Research Center, Imre Corporation, The Biomembrane Research Institute, and Cell Therapeutics. He was also the vice president of Research and Development at ZymeQuest.
About a year ago, Salubris Pharma, based in Shenzhen, China, invested $5 million into the company. At the time, Sam Murphy, vice president and head of International Business Development for Salubris, joined GO’s board of directors. “Novel, cancer-specific targets are imperative to widening the therapeutic window for powerful, cutting-edge cancer therapeutic modalities such as T-cell engagement and ADCs that offer tremendous promise for patients around the world,” Murphy stated at the time. “GO Therapeutics has the opportunity to generate significant value by opening an exciting new class of cancer-specific targets that will underpin transformative cancer therapeutics.”
The company expects to add on a couple key staffers, and is contemplating a Series B financing toward the end of the year, based on how close they feel they are to taking any compounds into the clinic.
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Janssen Terminates Collaboration Deal with Aduro Biotech


Janssen Biotech, a Johnson & Johnson company, informed Aduro Biotech that it was terminating its research and license deals related to Aduro’s Listeria treatment for cancers.
The original deal was launched on October 13, 2014 and later amended on November 11, 2015. Under the deal, Aduro gave Janssen an exclusive, worldwide license to research, develop, manufacture, sell and “otherwise exploit” products that have ADU-214, ADU-741 and GVAX Prostate. The terms of the agreement provided Aduro with up to $365 million in upfront license fees and milestone payments, some of which have been paid out. The deal had a potential value of $766 million with high single-digit to low teen royalties. So far, Aduro had received a $30 million upfront payment and a $21 million milestone payment.
Aduro’s LADD platform uses live-attenuated double-deleted Listeria monocytogenes strains of bacteria engineered to stimulate an innate immune response and to express tumor-associated antigens that stimulate tumor-specific T cell-mediated immunity. Its GVAX is a family of vaccines made up of human cancer cell lines genetically modified to secrete granulocyte-macrophage colony-stimulating factor (GM-CSF), an immune-stimulatory cytokine. The GVAX portfolio includes vaccines for pancreatic, prostate, colon and breast cancers in addition to multiple myeloma.
Overall, Aduro has three specific tech platforms, all designed to exploit the body’s natural immune system. They are LADD, TING and the B-select monoclonal antibody platform.
On June 7, Aduro initiated a Phase Ib clinical trial of ADU-214 in combination with Opdivo (nivolumab) to treat advanced lung cancer. ADU-214 is based on the LADD tech platform. Janssen was conducting the global trial.
The trial was designed to evaluate the safety and efficacy of ADU-214 in combination with Bristol-Myers Squibb’s PD-1 immune checkpoint inhibitor, Opdivo (nivolumab). The trial was focused on patients with mesothelin-positive, relapsed/refractory Stage IIIB or Stage IV adenocarcinoma of the lung.
At the time, Stephen T. Isaacs, chairman, president and chief executive officer of Aduro stated, “Based on single agent data from a Phase I dose-escalation study in patients with advanced-stage relapsed or refractory non-small cell lung presented at the 2017 International Association for the Study of Lung Cancer’s World Conference, Janssen made the decision to advance ADU-214 in combination with an anti-PD-1 checkpoint inhibitor. The initial data demonstrated that five out of nine patients treated with single-agent ADU-214 achieved a best response of stable disease, with one patient having received 25 cycles of treatment at the time of data cut off.”
According to Aduro’s 8-K filing with the United States Securities and Exchange Commission (SEC), the termination will be effective on December 24, 2018.
Although the Janssen partnership appears to be over, Aduro still has existing deals with Novartis, Merck, Johns Hopkins University and the University of California, Berkeley (CAL)for its products targeting the STING pathway. STING is a central mediator of innate immunity. When stimulated, it causes the expression of type I interferon, cytokines and T cell recruitment factors, which then active macrophages and dendritic cells, natural killer (NK) cells, and prime tumor-specific T cells.
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