Nuvectra asked for more data by FDA on bladder condition med

Nuvectra Corporation (NVTR), a neurostimulation medical device company, today provided an update on its U.S. Food and Drug Administration (FDA) pre-market approval (PMA) application and its TÜV SÜD application for CE Mark in Europe for Virtis^TM, the Company’s Sacral Neuromodulation (SNM) System for the treatment of chronic urinary retention and the symptoms of overactive bladder.

As part of its review of the Virtis PMA original application and the amendment submitted in April 2018, the FDA recently requested that the Company provide supplemental information related to any modifications or changes to the Virtis device, labeling and manufacturing, as well as clarifications of data related to MRI.  The Company has been in active communications with the FDA and intends to promptly file comprehensive responses to address the FDA’s requests. The FDA will then have up to 180 days to review the Company’s responses.  The Company plans to work proactively with the FDA to complete the review process as soon as possible.

Also, TÜV SÜD recently informed the Company that clinical study data will be required before it can recommend approval of CE Mark for the Virtis system.  The Company is continuing its discussions with TÜV SÜD regarding its application to clarify the breadth of clinical data that may be requested.

Scott Drees, Chief Executive Officer, commented, “Our primary focus is gaining FDA approval of the Virtis system in order to both provide therapy to patients and to address the significant market opportunity for SNM in the U.S. We remain encouraged by our recent interactions with the FDA and believe that our responses will adequately address the FDA’s requests.  We are pleased that our facility and pre-PMA audit have been completed without findings. We will continue to work cooperatively with the FDA to conclude the review of our application expeditiously and look forward to entering the U.S. SNM market as soon as possible following FDA approval.”

Mr. Drees continued, “We will revisit our corporate strategy to enter the SNM market in Europe and will engage with TÜV SÜD to determine a reasonable clinical pathway to CE Mark approval.”

https://yhoo.it/2KGvzLv

Athenex Details Pipeline Plans, $100M Investment

• Collaboration with Xiangxue Pharmaceutical Expands Portfolio in to TCR-T Immunotherapy
• In-Licensing of a Biologic Product from Avalon BioMedical Management

Athenex, Inc. (ATNX), a global biopharmaceutical company dedicated to the discovery, development and commercialization of novel therapies for the treatment of cancer and related conditions, today announced that it has entered into the following agreements:

• Establishment of a joint venture with Xiangxue Life Sciences, a wholly-owned subsidiary of Guangzhou Xiangxue Pharmaceutical Co., Ltd. (Xiangxue Pharmaceutical), for the research, development and commercialization of T-cell receptor-engineered T cells (TCR-T), a cancer immunotherapy technology, based on the novel approach on high-affinity TCR developed by Xiangxue Life Sciences;
• In-licensing of worldwide rights to a pegylated genetically modified human arginase from Avalon PolyTom (HK) Limited (PolyTom), a subsidiary of Avalon Biomedical Management Limited; and
• A strategic investment of US$100 million from Perceptive Advisors, an institution dedicated to supporting the most promising technologies in healthcare.

Xiangxue Life Sciences is a wholly-owned subsidiary of Xiangxue Pharmaceutical, a long term partner of Athenex for the development of KX-02 for glioblastoma multiforme in China. Xiangxue Life Sciences is a Chinese biopharmaceutical company focused on TCR-based therapies for cancer and has developed a new generation TCR-T, named HATac, which consists of the expression of high binding affinity soluble T-cell receptors on the engineered T-cells to target HLA-antigenic peptide complex on certain types of cancer cells. Early clinical studies in China demonstrated a good safety profile in patients.

Axis Therapeutics Limited, a new joint venture to be established and owned initially as 55% by Athenex and 45% by Xiangxue Life Sciences, has in-licensed the worldwide (excluding mainland China) rights of all the intellectual properties and know-how of the TCR-T immunotherapy technology from Xiangxue Life Sciences, subject to certain closing conditions and approvals.  Pursuant to the terms of the license agreement and shareholders agreement, Athenex will issue US$5 million worth of Athenex common stock to Xiangxue Life Sciences as an upfront payment and Athenex will contribute US$30 million in cash to the joint venture. Axis Therapeutics will also pay various clinical and regulatory milestones of up to US$110 million to Xiangxue Life Sciences. Xiangxue Life Sciences will retain the mainland China rights to the technology and there will be royalties on net income payable to Axis Therapeutics upon successful commercialization in mainland China. Additional new TCR-T technologies developed by Axis will also be partnered with Xiangxue Pharmaceutical in the future for the mainland China rights.

Athenex has strategically in-licensed a pegylated genetically modified human arginase, or Pegtomarginase, which was initially developed by the Hong Kong Polytechnic University and subsequently licensed to PolyTom. Through extensive research, a single isomer of pegylated genetically modified human arginase with a favorable pharmacokinetic and potency profile was identified and preclinical work demonstrated that the molecule represents a promising clinical candidate. The anti-cancer mechanism of this biologic product lies in that some cancers do not express either ornithine transcarbamylase (OTC) and/or argininosuccinate synthetase (ASS), two enzymes critical for the synthesis of arginine in the urea cycle pathway. As cancer cells will not be able to synthesize arginine, they will have to depend on external supply of arginine for tumor survival and growth. This treatment approach shows specificity as the depletion of circulating arginine will starve such OTC-/ASS- deficient cancer cells, while normal cells can synthesize arginine intracellularly through urea cycle for their survival.

Perceptive Advisors, an investment manager with a deep history in supporting biotechnology companies, will provide a combination of equity and debt financing to support Athenex’s operations and continued development, including pipeline expansion initiatives. Perceptive Advisors will invest US$100 million in Athenex, comprised of US$50 million in equity and US$50 million in debt.

Dr. Johnson Lau, Chairman and CEO of Athenex, commented, “Given our confidence in our two Phase III programs in Oraxol for metastatic breast cancer and KX-01 ointment for actinic keratosis, we are delighted to have this opportunity to further expand our product pipeline. The additions of TCR-T based cancer immunotherapy in collaboration with our long term business partner, Xiangxue Pharmaceutical and its subsidiary Xiangxue Life Sciences, and of an anti-cancer biologic will further complement and enhance our Oncology Innovation Platform. We are particularly excited about the potential synergies that can be created between the new therapies and our existing pipeline. We have developed a business strategy to fully explore the potential of all these platforms. We are also grateful to have Perceptive Advisors as our financial partner.”

https://yhoo.it/2MJ45FM

BioLife preannounces revenue up >100%

– BioLife Solutions, Inc. (BLFS) (“BioLife”), the leading developer, manufacturer and marketer of proprietary clinical grade cell and tissue hypothermic storage and cryopreservation freeze media, today reported preliminary revenue for the three months ended June 30, 2018 and increased its revenue guidance for 2018.

Second quarter 2018 preliminary revenue from sales of BioLife’s biopreservation media products reached a record $5.2 million.  This represents a 103% increase from the second quarter of 2017 and a 36% increase from the first quarter of 2018.

BioLife CEO Mike Rice commented, “Strong product demand during the second quarter from our cell and gene therapy customers in the regenerative medicine market segment resulted in more than 100% revenue growth over the prior-year period. Given our performance for the first half of 2018, increased adoption of our products in cell therapy clinical trials, and our outlook on product demand for the remainder of the year, we are significantly increasing our revenue guidance for the full year 2018.”

Management estimates that BioLife’s proprietary biopreservation media products have been used in nearly 300 customer clinical applications, including dozens of CAR T-cell and other T cell immunotherapies targeting blood cancers and solid tumors as well as other cell types targeting debilitating diseases and disorders.

Roderick de Greef, Chief Financial Officer, remarked, “Based primarily on additional warrant exercises during the period, we ended the second quarter with more than $14 million in cash, up from $7 million at March 31, 2018.”

Management is raising its revenue guidance for the full year 2018 as follows:

• Biopreservation media revenue is now expected to be in a range of $18.5 million to $20 million, representing growth of approximately 68% to 82% over 2017. This is an increase from the Company’s prior revenue guidance issued in April 2018 of a range of $14.5 million to $15.5 million.

Management is affirming the remainder of its financial guidance for 2018 as follows:

• Gross margin is expected to be between 63% and 65%, up from 61% for 2017.
• Operating expenses are expected to range from $9.0 million to $9.5 million, compared with $7.8 million for 2017.
• Full year GAAP operating profit with proportional increases in adjusted EBITDA and cash flow from operations.

https://yhoo.it/2lP5fnJ

Neurotrope Has Additional Phase 2 Data at Alzheimer’s Association Conference

Neurotrope Inc. (NASDAQ: NTRP) a clinical-stage biopharmaceutical company developing novel therapies for neurodegenerative diseases, including Alzheimer’s disease (AD), is announcing the acceptance of a presentation at the Alzheimer’s Association International Conference 2018 (AAIC) being held in Chicago, from July 22-26, 2018.

    Title Significant Cognitive Improvement with Bryostatin
          for Advanced Alzheimer's

          Patients in the Absence of Memantine (Poster
          presentation Abstract #27295)

    Developing Topics Session: P4-199:

    Date/Time                  Wednesday, July 25, 2018: 9:30 AM-
                                4:15 PM

    Location Hall              F1 - McCormick Place

“The primary mechanisms of action (MOAs) of bryostatin, Neurotrope’s lead compound to treat Alzheimer’s disease (AD) neurodegeneration, are synaptogenesis and anti-apoptosis: the generation of new, mature synaptic connections and the prevention of neuronal death. This has been demonstrated in pre-clinical models because our target PKC epsilon activates synaptic growth factors like BDNF, NGF and IGF-1. Our most recent comprehensive data analyses, to be reported at AAIC 2018, have demonstrated that advanced AD patients show improvement (> 6.0 points vs. placebo and baseline) in the Severe Impairment Battery (SIB) even 30 days after all drug dosing has been completed in patients not on memantine. The data indicate that PKC’s synaptogenesis efficacy cannot occur when the NMDA receptor is blocked. Data from many other laboratories over the years implicating NMDA in memory processing could now take on new meaning from Neurotrope’s clinical results with bryostatin – in the absence of memantine. We are excited to be presenting this new data at this prestigious international forum” stated Dr. Daniel Alkon, President and Chief Scientific Officer of Neurotrope.

https://bit.ly/2KFP9KU

Merck to Present New Data from Studies of Investigational HIV Therapy

Merck (NYSE: MRK), known as MSD outside the United States and Canada, today announced that new data from the company are scheduled to be presented at the 22^nd International AIDS Conference (AIDS 2018) taking place July 23-27 in Amsterdam. Presentations include Week 96 data from the Phase 3 DRIVE-FORWARD clinical trial for doravirine (DOR) and additional analyses for DOR and investigational therapy MK-8591. DOR is a non-nucleoside reverse transcriptase inhibitor (NNRTI) under investigation as a single tablet for use in combination with other antiretroviral (ARV) agents and as a fixed-dose combination with lamivudine (3TC) and tenofovir disoproxil fumarate (TDF) for the treatment of HIV-1 infection in adult patients with no prior ARV treatment history (treatment-naïve). MK-8591 is an investigational nucleoside reverse transcriptase translocation inhibitor (NRTTI) currently being evaluated in clinical trials for the treatment of HIV infection.

“While enormous progress has been made in the fight against HIV/AIDS, continued scientific innovation is needed given the unmet need that continues to exist in HIV,” said Dr. George Hanna, vice president and therapeutic area head of infectious diseases, global clinical development, Merck Research Laboratories. “Merck looks forward to presenting these new data from our HIV pipeline at AIDS 2018.”

Presentations include a late-breaking poster of the Week 96 data from the pivotal Phase 3 DRIVE-FORWARD clinical trial evaluating the safety and efficacy of once-daily DOR compared to once-daily ritonavir-boosted darunavir (DRV+r), each administered with either emtricitabine/tenofovir disoproxil fumarate (FTC/TDF) or abacavir/lamivudine (ABC/3TC), in treatment-naïve adults with HIV-1 infection. In addition, analyses of the resistance profiles of DOR and MK-8591 will be presented.

Data to be presented include:

• Doravirine Versus Ritonavir-Boosted Darunavir: 96-Week Results of the Randomized, Double-Blind, Phase 3 DRIVE-FORWARD Noninferiority Trial. Abstract LBPEB017. K. Squires. Late-breaking Poster Exhibition: Wednesday, July 25, 12:30-14:30 CET, Poster Exhibition Area, Hall 1
• Understanding the Resistance Profile of the HIV-1 NNRTI Doravirine in Combination with the Novel NRTTI MK-8591. Abstract THPEB068. D. Hazuda. Poster Exhibition: Thursday, July 26, 12:30-14:30 CET, Poster Exhibition Area, Hall 1
• Characterization of Doravirine-Selected Resistance Patterns from Participants in Treatment-Naïve Phase 3 Clinical Trials. Abstract THPDB0101. M. Lai. Poster Discussion: Thursday, July 26, 13:00-14:00 CET, Emerald Room

Earlier this year, the U.S. Food and Drug Administration (FDA) accepted for review New Drug Applications for DOR and DOR/3TC/TDF for the treatment of HIV-1 infection in treatment-naïve adults. The FDA has set a target action date of October 23, 2018 for both applications.

https://bit.ly/2Nk8JLh

Wave Life Sciences gets positive Euro opinion for Duchenne med orphan tag

Wave Life Sciences announced that the European Medicines Agency Committee for Orphan Medical Products issued a positive opinion recommending WVE-210201 for designation as an orphan medicinal product for the treatment of Duchenne muscular dystrophy. To be considered for Orphan Drug Designation in the EU, companies must provide data that demonstrate the plausibility for use of the investigational therapy in the treatment of the disease and establish that the drug has the potential to provide relevant advantages or a major contribution to patient care over existing therapies. The positive opinion issued by COMP is anticipated to be adopted by the European Commission at the end of July 2018. Orphan Drug Designation by the EC provides regulatory and financial incentives for companies to develop and market therapies that treat a life-threatening or chronically debilitating condition affecting no more than five in 10,000 persons in the European Union, and where no satisfactory treatment is available. Among the incentives available to medicines designated as orphan drugs by the EC are ten-year market exclusivity in the EU after product approval, eligibility for conditional marketing authorization, protocol assistance from the EMA at reduced fees during the product development phase and direct access to centralized marketing authorization in the EU. WVE-210201 is currently being studied in an ongoing global, multicenter, double-blind, placebo-controlled Phase 1 clinical trial designed to evaluate the safety, tolerability and plasma concentrations of single ascending doses of WVE-210201 administered intravenously in DMD patients with gene mutations amenable to exon 51 skipping. The trial is expected to enroll up to 40 patients, including ambulatory and non-ambulatory patients, between the ages of 5 and 18 years. Safety data from the trial are expected in the third quarter of 2018.

https://bit.ly/2Nk20kn

Express Scripts most exposed to mail order market disruption, says Barclays

Barclays analyst Steve Valiquette believes Express Scripts (ESRX) is most exposed to Amazon’s (AMZN) acquisition of PillPack. The analyst estimates Express Scripts has 35%-40% share of the U.S. prescription mail order market. The mail order prescription market is the most immediate area for possible disruption from Amazon/PillPack given the nature of PillPack’s business and Amazon’s existing mail-order logistical infrastructure, Valiquette tells investors in a research note. The analyst assumes PillPack captures 1% share of the U.S. mail-order Rx market in 2019, and improves its market share further by 1% each year afterwards. Valiquette calculates a “hypothetical” 13c negative earnings per share impact for Express Scripts in 2019. He believes the negative earnings impact could reach 45c by 2021, but thinks Express Scripts could undertake expense cuts to offset some of this. For Walgreens Boots Alliance (WBA), the analyst calculates a negative earnings per share impact of 15c in 2020, or 2% of his current estimate assuming no additional cost-cutting. Valiquette has Equal Weight ratings on both Express Scripts and Walgreens.

https://bit.ly/2NgNie4