On November 12, 2019 Boston Scientific Corporation (NYSE: BSX) (the "Company") reported a public offering of €900.0 million aggregate principal amount of 0.625% Senior Notes due 2027 (the "Notes") (Press release, Boston Scientific, NOV 12, 2019, View Source [SID1234551012]). The Company has applied to list the Notes on the New York Stock Exchange.

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In addition, the Company completed its previously announced cash tender offer (the "Tender Offer") for up to $1.0 billion aggregate principal amount of certain outstanding senior notes, pursuant to which it accepted $206,429,000, $566,455,000, and $227,117,000 of tendered principal amounts of its 4.125% Senior Notes due 2023, 4.000% Senior Notes due 2028 and 3.850% Senior Notes due 2025, respectively on the early settlement date.

The Company used the net proceeds from the offering to (i) fund the Tender Offer and (ii) pay accrued and unpaid interest, premiums, fees and expenses in connection with the Tender Offer.

On November 12, 2019 Merck , a leading company in the scientific and technological sector, reported that it has signed a license agreement that provides Evotec SE with access to Merck’s base CRISPR intellectual property (Press release, Merck & Co, NOV 12, 2019, View Source [SID1234551027]). Evotec, an international biotech company based in Hamburg (Germany), will use Merck’s genome CRISPR technology to create modified cell lines for Evotec’s commercial and internal research purposes.

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Merck’s CRISPR license to Evotec is the impetus for important drug testing and discovery, which will accelerate research and lead to new therapy development
Merck’s CRISPR license to Evotec is the impetus for important drug testing and discovery, which will accelerate research and lead to new therapy development

"This CRISPR license will be an engine for important drug tests and discoveries that promise to accelerate research and generate the development of new treatments," said Udit Batra , a member of the Merck Board of Directors and CEO of Life Science. "Merck has been at the forefront of innovation in genome modification for 15 years and continues to work with the sector and the academic environment to solve complex problems through our patented CRISPR technology, in an ethical and responsible manner."

Evotec plans to use Merck’s CRISPR intellectual property portfolio to develop very precise tests to determine the biology and toxicity of possible drugs during the drug development cycle.

"Evotec is proud to be able to continue its solid collaboration with Merck by licensing its revolutionary CRISPR technology," said Craig Johnstone , director of Operations for Evotec. "Our vision and passion for innovation includes the use of CRISPR technology to test and improve the effectiveness of the new drugs we develop, in order to alleviate the suffering of patients with diseases with insufficient attention from around the world."

This new license reinforces previous collaborations between Merck and Evotec. In November 2016, the companies signed a series of agreements through which Evotec would provide selection services thanks to Merck’s set of genetic reagents, including CRISPR and ARNhc banks. The combination of access to Merck genome modification banks with Evotec’s selection knowledge offers a faster way to explore and identify new pharmacological targets.

Merck has 20 CRISPR patents worldwide in terms of methods and composition, including the fundamental technology of CRISPR Cas9 for genetic integration into mammalian cells.

Merck supports genome editing research paying close attention to ethical and legal norms. The company has created an external and independent Bioethics Advisory Body in order to offer research orientations in which the company participates, such as the work in which genome editing is studied or used. Merck has also defined a clear operational position that takes into account scientific and social problems to inform promising therapeutic approaches for use in research and applications.

All Merck press releases are distributed by email at the same time they are published on the Merck website. To register, modify your selection or unsubscribe from this service, visit www.merckgroup.com/subscribe .

On November 12, 2019 IVERIC bio, Inc. (Nasdaq: ISEE) reported further details of the clinical trial results for the Company’s Zimura (avacincaptad pegol) program in geographic atrophy (GA) secondary to dry age-related macular degeneration (AMD), reviewed the Company’s financial results for the third quarter 2019 and provided a general business update (Press release, Ophthotech, NOV 12, 2019, View Source [SID1234551043]).

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Zimura (avacincaptad pegol) Highlights

On October 28, 2019, IVERIC bio provided topline data confirming that Zimura (avacincaptad pegol), the Company’s complement factor C5 inhibitor, met its prespecified primary endpoint in reducing the mean rate of geographic atrophy (GA) growth in patients with dry age-related macular degeneration (AMD). Today, the Company provided further clinical details and the development strategy for Zimura in GA secondary to AMD. These announcements will be discussed during today’s conference call/webcast (also see the second press release issued earlier today and the press release issued on October 28, 2019.)

"We are excited to have achieved a major milestone with our recent Zimura clinical trial results in geographic atrophy secondary to dry AMD and we look forward to start enrolling patients in a second pivotal clinical trial in the first quarter of 2020. We believe these events have the potential to be a catalyst for our company," stated Glenn P. Sblendorio, Chief Executive Officer and President of IVERIC bio. "IVERIC bio is now in a strong position, with a diversified, retina-focused portfolio, including both a late stage clinical program for a large market retinal disease and early stage gene therapy programs in inherited retinal diseases. We are committed to efficiently progressing these programs with the goal of continuing to create value for our shareholders."

Gene Therapy Highlights

Orphan IRD Gene Therapy Programs

IC-100: Rhodopsin-Mediated Autosomal Dominant Retinitis Pigmentosa (RHO-adRP)
Natural history studies and IND-enabling activities for IC-100 are on track. The Company expects to initiate a Phase 1/2 clinical trial for IC-100 in patients with rhodopsin mediated adRP in the second half of 2020.

IC-200: BEST1-Related IRDs
Natural history studies and IND-enabling activities for IC-200 are on track.

The Company expects to initiate a Phase 1/2 clinical trial for IC-200 in patients with BEST1 related retinal diseases during the first half of 2021.

miniCEP290: Leber Congenital Amaurosis Type 10 (LCA10)
Encouraging results from the Company’s collaboration with the University of Massachusetts Medical School (UMass Medical School) in its miniCEP290 program led the Company to exercise its option and, in July 2019, the Company entered into an exclusive global license agreement with the University of Massachusetts for rights to develop and commercialize mutation independent novel AAV minigene therapy product candidates for the treatment of LCA10, which is due to mutations in the CEP290 gene and is the most common type of LCA. The Company plans to provide an update on the lead minigene construct in early 2020.

miniABCA4 Program for Stargardt Disease (STGD1)
The Company, through its collaborative sponsored research agreement with UMass Medical School, is evaluating several ABCA4 minigene constructs in both in vitro and in vivo experiments. The Company expects to have results from the miniABCA4 program in 2020.

miniUSH2A: USH2A-Related IRDs Including Usher Syndrome Type 2A and USH2A-Associated Nonsyndromatic Autosomal Recessive Retinitis Pigmentosa
In July 2019, the Company entered into a sponsored research agreement with UMass Medical School and an exclusive option agreement with the University of Massachusetts for rights to develop and commercialize novel AAV gene therapy product candidates utilizing a mutation independent minigene therapy approach for the treatment of USH2A-related IRDs. This group of orphan IRDs include Usher syndrome Type 2A and USH2A-associated nonsyndromatic autosomal recessive retinitis pigmentosa.

On October 29, 2019, Abraham Scaria, PhD was appointed to the position of Chief Scientific Officer. Dr. Scaria will lead the Company’s research and pre-clinical gene therapy activities. Dr. Scaria’s extensive experience includes positions at Genzyme, Sanofi and most recently at Casebia Therapeutics, leading multiple ocular gene therapy programs.

Second Quarter 2019 Financial Results
Operational Update
As of September 30, 2019, the Company had $94.9 million in cash and cash equivalents. The Company reaffirms its estimate that year-end 2019 cash and cash equivalents will range between $80 million and $85 million. With the initiation of enrollment for the Company’s second pivotal clinical trial for Zimura in GA planned for the first quarter of 2020, the Company estimates that its cash and cash equivalents will be sufficient to fund its operations and capital expenditure requirements as currently planned through the first half of 2021. These estimates are based on the Company’s current business plan, including the continuation of its current research and development programs. This estimate does not reflect any additional expenditures in the event the Company were to in-license or acquire any new product candidates or commences any new sponsored research programs.

R&D Expenses: Research and development expenses were $10.4 million for the quarter ended September 30, 2019, compared to $9.4 million for the same period in 2018. For the nine months ended September 30, 2019, research and development expenses were $28.1 million compared to $25.6 million for the same period in 2018. Research and development expenses increased primarily due to increases in costs associated with the Company’s gene therapy programs and HtrA1 inhibitor program, offset by decreases in costs associated with the Company’s Zimura programs.

G&A Expenses: General and administrative expenses were $4.7 million for the quarter ended September 30, 2019, compared to $6.0 million for the same period in 2018. For the nine months ended September 30, 2019, general and administrative expenses were $15.4 million compared to $17.9 million for the same period in 2018. General and administrative expenses decreased primarily due to decreases in costs to support the Company’s operations and infrastructure.

Net Income: The Company reported a net loss for the quarter ended September 30, 2019 of $14.4 million, or ($0.35) per diluted share, compared to a net loss of $14.7 million, or ($0.41) per diluted share, for the same period in 2018. For the nine months ended September 30, 2019, the Company reported a net loss of $41.4 million or ($1.00) per diluted share, compared to a net loss of $41.0 million or ($1.13) for the same period in 2018.

Conference Call/Web Cast Information
IVERIC bio will host a conference call/webcast to discuss the Company’s financial and operating results and provide a business update. The call is scheduled for November 12, 2019 at 8:00 a.m. Eastern Time. To participate in this conference call, dial 888-208-1711 (USA) or 323-994-2082 (International), passcode 5526863. A live, listen-only audio webcast of the conference call can be accessed on the Investors section of the IVERIC bio website at www.ivericbio.com. A replay will be available approximately two hours following the live call for two weeks. The replay number is 888-203-1112 (USA Toll Free), passcode 5526863.

On November 12, 2019 PharmaCyte Biotech (OTCQB: PMCB) reported that has reduced a long list of FDA-required items to just one last important component—release testing—before it can submit an Investigational New Drug application (IND) to the U.S. FDA (Press release, PharmaCyte Biotech, NOV 12, 2019, View Source [SID1234550944]). PharmaCyte’s IND application will request permission from the FDA to conduct a Phase 2b clinical trial in locally advanced, inoperable pancreatic cancer (LAPC). With the FDA’s approval to begin a clinical trial, PharmaCyte’s opportunities for funding improve dramatically.

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PharmaCyte has already conducted and completed a long list of tests and entered the data from those tests into the IND application. PharmaCyte’s Chief Executive Officer, Kenneth L. Waggoner, said of completing the IND for submission to the FDA, "Most of the work needed for PharmaCyte to submit an IND to the FDA has been completed."

PharmaCyte recently announced that it has encapsulated the live cells from its Master Cell Bank (MCB) into its live-cell encapsulation technology, Cell-in-a-Box, in what is the second of two staggered and back-to-back manufacturing runs. In that announcement the company’s CEO stated that after reviewing pictures of the growing cells, he is confident that those cells are growing as well as those in the first run, which ended successfully after a host of changes were implemented to improve the manufacturing process.

And, with the second manufacturing run well underway and growing as expected, this final run should end in success over the next week or so, and then the release testing for this second run combined with the release testing from the first run will complete what has been a long and arduous path to submitting the much-anticipated IND.

Now, let’s walk through each of the tests (release testing) that are required by cGMP Validation, the company that is taking responsibility for "releasing" the clinical trial product into the U.S. for use in PharmaCyte’s upcoming clinical trial. Many of these tests are also FDA-required tests that must be completed in order to obtain the data necessary to complete and submit the IND and for PharmaCyte to receive a Certificate of Analysis (CoA) from Austrianova. A CoA is a document issued by Austrianova’s Quality Assurance and Quality Control Department (QA/QC) that confirms that PharmaCyte’s product meets its product specifications. The CoA will contain the actual results obtained from the testing performed as part of the QA/QC of a representative sample of PharmaCyte’s clinical trial product.

After a manufacturing run is completed successfully, 300 Cell-in-a-Box capsules are placed into 400 of PharmaCyte’s clinical trial syringes and then frozen. A representative sample of those syringes will be thawed and undergo release testing.

All release testing related to "safety" of the encapsulated cells is being outsourced by PharmaCyte to independent third-party laboratories in Europe. They include tests for sterility, endotoxin, pH, mycoplasma and cell identity.

A sterility test confirms that all syringes are free from the presence of viable microorganisms or microbial contamination.

An endotoxin test of a biologic product is necessary to ensure the product is endotoxin free. This means that the cells do not contain bacterial toxins.

A pH test will confirm how much hydrogen is in the product and that the product meets the specified pH balance for a human being.

A mycoplasma test confirms that the syringes are free from mycoplasma. Mycoplasma are small, simple bacteria which lack a cell wall and represent one of the most prevalent and serious sources of cell line contamination.

A cell identity test confirms that the cell line used in production is positively identified as the 22P1G cell line. This is the human cell line that PharmaCyte cloned from the original cell line used in the first two clinical trials using the Cell-in-a-Box technology.

All release testing related to "functionality" of the encapsulated cells is being conducted by Austrianova at its GMP facility in Thailand. Those tests include enzymatic activity, cell viability, capsule count in each syringe, and capsule diameter.

An enzymatic activity test shows that the requisite enzymes are being produced by the cells inside the capsules to create the desired chemical reaction in the body. In PharmaCyte’s case, this means that the correct enzymes are being produced to convert the cancer-killing prodrug, ifosfamide, from its inactive form to its active form. Ifosfamide is the chemotherapy drug that PharmaCyte uses with the encapsulated cells that are implanted in a patient as close to the tumor as possible.

A cell viability test measures the number of cells that remain alive and viable after thawing a syringe of encapsulated cells.

A capsule count test confirms that each syringe contains 300 capsules. One syringe full of the encapsulated cells is implanted in a pancreatic cancer patient before ifosfamide is given intravenously to treat LAPC.

A capsule diameter test confirms that the diameter of each capsule inside a syringe of encapsulated cells meets a specified range.

Additionally, a pyrogenicity test must be completed to ensure that the capsules themselves do not produce a fever. This FDA-required test is not in the list of release tests. The pyrogenicity test is being conducted in the United States.

To learn more about PharmaCyte’s pancreatic cancer treatment and how it works inside the body to treat locally advanced inoperable pancreatic cancer, watch the company’s documentary video complete with medical animations at: View Source

On November 12, 2019 Synlogic, Inc. (Nasdaq: SYBX), a clinical stage company applying synthetic biology to beneficial microbes to develop novel, living medicines, reported its financial results for the third quarter ended September 30, 2019, and provided an update on its programs and progress (Press release, Synlogic, NOV 12, 2019, View Source [SID1234550963]).

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"We are making good progress towards our goal of evaluating the breadth of therapeutic applications of our Synthetic Biotic platform. Notably, the recent initiation of the first clinical trial of a Synthetic Biotic medicine for the treatment of cancer represents a significant step for Synlogic," said Aoife Brennan, M.B., B.Ch., Synlogic’s president and chief executive officer. "We also continue to advance our SYNB1618 program for the treatment of phenylketonuria in an ongoing bridging study to evaluate the tolerability and activity of a solid oral formulation, and we look forward to sharing those data and our plans for the next steps in this program."

Recent Highlights
Pipeline

Clearance of Investigational New Drug (IND) application and initiation of SYNB1891 immuno-oncology (IO) clinical study. Synlogic has initiated a Phase 1 open-label, multicenter, dose escalation trial of its STING-agonist producing bacterial strain, SYNB1891, for the treatment of refractory solid tumors. The study’s primary objectives are to evaluate safety and tolerability of escalating doses of intratumorally administered SYNB1891 as a monotherapy. Once a maximum tolerated dose is established, patients will receive escalating dose levels of SYNB1891 in combination with a fixed dose of the checkpoint inhibitor, Atezolizumab, to establish a recommended Phase 2 dose for the combination regimen. Synlogic expects to have data from the monotherapy arm of the study in 2020.
Presentation of positive data from Phase 1/2a Study of SYNB1618 in patients with phenylketonuria (PKU) as well as modeling work to estimate target dosing of SYNB1618. In September, clinical data from a randomized, double-blind, placebo-controlled Phase 1/2a study of SYNB1618, which is being developed for the treatment of PKU were presented at the Annual Symposium of the Society for the Study of Inborn Errors of Metabolism (SSIEM) by Jerry Vockley, M.D. Ph.D., Professor of Pediatrics and Chief of Medical Genetics, University of Pittsburgh, and a principal investigator on the trial. The study’s primary objective was to evaluate safety and tolerability and to measure biomarkers of phenylalanine-consuming activity of multiple ascending doses of a liquid formulation of SYNB1618 in healthy volunteers and a single dose level in PKU patient cohorts. SYNB1618 was well tolerated in healthy volunteers and patients with PKU and the data demonstrated equivalent phenylalanine-consumption.
Initiation of bridging study of a solid oral formulation of SYNB1618 in healthy volunteers to establish maximum tolerated dose (MTD) for efficacy study in patients with PKU. Based on data from its Phase 1/2a study of a liquid formulation of SYNB1618, Synlogic initiated a bridging study in healthy volunteers of a new solid oral formulation to establish an MTD that would potentially enable at least a 30% lowering of blood Phe levels in PKU patients based on the company’s modeling data. With supporting data from the bridging study, Synlogic expects to advance SYNB1618 into an efficacy study in patients in the first half of 2020.
Corporate

Key appointments to Synlogic’s leadership and research and development teams
Richard Riese, M.D. Ph.D., chief medical officer, joined Synlogic in September and has assumed responsibilities for all clinical and regulatory functions from current president and CEO, Aoife Brennan.
Gregg Beloff, interim chief financial officer, was appointed in October 2019. Mr. Beloff, who has more than 20 years of experience in the life sciences industry, brings significant expertise in operational management, strategic planning, corporate and business development, fundraising and mergers and acquisitions.
Third Quarter 2019 Financial Results
As of September 30, 2019, Synlogic had cash, cash equivalents, and short- and long-term investments of $138.7 million. The Company anticipates that, based on its current operating plan, its existing cash and cash equivalents will fund company operations through 2021.

For the three months ended September 30, 2019, Synlogic reported a consolidated net loss of $13.3 million, or $0.39 per share, compared to a consolidated net loss of $10.7 million, or $0.43 per share, for the corresponding period in 2018.

Research and development expenses were $10.6 million for the three months ended September 30, 2019 compared to $9.9 million for the corresponding period in 2018. The increase was primarily due to increased clinical development costs for our SYNB1618 program.

General and administrative expenses for the three months ended September 30, 2019 were $3.9 million compared to $3.4 million for the corresponding period in 2018.

Revenues were $0.3 million for the three months ended September 30, 2019, compared to $1.8 million for the corresponding period in 2018. The revenue for both periods is associated with Synlogic’s collaboration with AbbVie to develop a Synthetic Biotic medicine for the treatment of inflammatory bowel disease. The decrease in revenue was primarily the result of the achievement of a $2.0 million milestone under a September 2018 amendment to the AbbVie agreement of which $1.8 million was recognized in revenue in the quarter ended September 30, 2018.

Conference Call & Webcast Information
Synlogic will host a conference call and live webcast today at 5:00 p.m. ET today, Tuesday, November 12, 2019. To access the live webcast, please visit the "Event Calendar" page within the Investors and Media section of the Synlogic website. Alternatively, investors may listen to the call by dialing +1 (844) 815-2882 from locations in the United States or +1 (213) 660-0926 from outside the United States. The conference ID number is 3889667. For those unable to participate in the conference call or webcast, a replay will be available for 30 days on the Investors and Media section of the Synlogic website.