Invenra and WARF Initiate a Collaboration to Discover and Develop Novel Therapeutics to Fight Neuroblastoma in Children

On January 31, 2019 Invenra and WARF (Wisconsin Alumni Research Foundation) reported they have entered into a collaboration to discover and develop a bispecific antibody therapeutic for the treatment of neuroblastoma, a cancer that is the third most common childhood cancer, after leukemia and brain tumors (Press release, Invenra, JAN 31, 2019, View Source [SID1234532990]). The collaboration will use Invenra’s proprietary SNIPERTM technology to allow for precise targeting of the cancer cells while sparing normal nerve cells, and potentially alleviating unwanted toxicities and pain related side effects. The idea for the therapeutic candidate came out of a collaboration between Invenra and University of Wisconsin, Madison Professor, Dr. Paul Sondel, a pediatric oncologist who treats these children in his practice.

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"Invenra’s SNIPERTM technology should enable creation of antibody-based therapies that are specifically able to target tumor cells while not binding to normal tissues. We are excited to be working with Invenra to initiate this approach by directing it towards improving treatment for pediatric neuroblastoma," notes Sondel.

Roland Green, Ph.D., Chief Executive Officer and Co-Founder of Invenra states, "We are very excited about this program and the potential it has for helping these children with neuroblastoma. This program also has the potential to treat patients with a variety of other cancers such as glioblastoma (a form of brain cancer), melanoma and small cell lung cancer (SCLC). This collaboration is a good example of the benefits that can come from partnerships between the UW-Madison and industry."

Agios to Webcast Conference Call of Fourth Quarter and Full Year 2018 Financial Results on February 14, 2019

On January 31, 2019 Agios Pharmaceuticals, Inc. (NASDAQ: AGIO), a leader in the field of cellular metabolism to treat cancer and rare genetic diseases, reported that the company will host a conference call and live webcast on Thursday, February 14, 2019 at 8:00 a.m. ET to report its fourth quarter and full year 2018 financial results and other business highlights (Press release, Agios Pharmaceuticals, JAN 31, 2019, http://investor.agios.com/news-releases/news-release-details/agios-webcast-conference-call-fourth-quarter-and-full-year-2018 [SID1234533007]).

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A live webcast can be accessed under "Events & Presentations" in the Investors section of the company’s website at www.agios.com. The conference call can be accessed by dialing 1-877-377-7098 (domestic) or 1-631-291-4547 (international) and referring to conference ID 9886713. The webcast will be archived and made available for replay on the company’s website beginning approximately two hours after the event.

Phyton Biotech Partners with PellePharm to Develop Renewable Supply of Cyclopamine for Investigational Topical Cancer Therapy

On January 31, 2019 Phyton Biotech and PellePharm reported an exclusive partnership to develop a plant cell culture-based process to enable the future renewable production of the raw material for PellePharm’s patidegib, an investigational topical therapy for Gorlin Syndrome and High Frequency Basal Cell Carcinoma (BCC) (Press release, Phyton Biotech, JAN 31, 2019, View Source [SID1234532991]). PellePharm is currently investigating the safety and efficacy of patidegib topical gel for the reduction of surgically-eligible BCCs in Gorlin Syndrome patients in a Phase 3 trial.

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The foundation for this joint initiative is Phyton Biotech’s proprietary Plant Cell Fermentation (PCF) technology, a unique platform that enables renewable, reliable and scalable supply of plant-derived compounds like cyclopamine, the key building block in the synthesis of patidegib. Under its agreement with PellePharm, Phyton Biotech will begin development of the new process immediately.

"We are incredibly excited to partner with PellePharm as pioneers in precision medicine to advance this topical skin cancer treatment to the next level of excellence with our patented PCF technology," said Colin Marr, president of Phyton Biotech. "This application of our ‘green chemistry’ process is just the latest example of how Phyton Biotech is advancing traditional manufacturing approaches for high-value phytochemicals and markedly improving patient care."

Currently, cyclopamine is sourced from the perennial herb Veratrum californicum (corn lily), a member of the Liliaceae plant family found in remote mountainous parts of western North America.

"PellePharm’s partnership with Phyton Biotech underscores our commitment to bring a new therapeutic option to patients with Gorlin Syndrome and other skin cancers," said Sanuj Ravindran, president and chief executive officer of PellePharm. "As we begin our pivotal Phase 3 clinical trial of patidegib topical gel and prepare for commercial-scale production of patidegib if approved, we anticipate the demand for cyclopamine will grow, spurring the need for a supplemental and more scalable source."

Gorlin Syndrome, also known as Basal Cell Carcinoma Nevus Syndrome, is a rare, genetic disease that leads to the chronic formation of multiple basal cell carcinomas, often on the face. With no FDA-approved drugs currently available for Gorlin Syndrome, the standard of care is surgery. People with severe Gorlin Syndrome may have as many as 30 or more surgeries per year, which can be repetitive, scarring and disfiguring.

The partnership with PellePharm follows Phyton Biotech’s successful demonstration of its ability to express cyclopamine directly from Veratrum californicum plant cell cultures via PCF. Further development and full scale-up into commercial-size bioreactors of up to 75 m³ in volume, operated in Phyton Biotech’s facility in Germany, is anticipated to follow the current program.

In addition to this project with PellePharm, Phyton Biotech is working on expanding its patent for the renewable production of alkaloids by Liliaceae cell culture to Europe as it is already granted in the United States and other jurisdictions.

Phyton Biotech’s award-winning green chemistry model focuses on plant cell culture development and offers superior quality, reliability, environmental and cost advantages over other methods of production of complex small molecules, plant-based actives and recombinant proteins.

McKesson Reports Fiscal 2019 Third-Quarter Results

On January 31, 2019 McKesson Corporation (NYSE:MCK) reported that revenues for the third quarter ended December 31, 2018, were $56.2 billion, up 5% compared to $53.6 billion a year ago, and also up 5% on a constant currency basis (Press release, McKesson, JAN 31, 2019, View Source [SID1234532992]). On the basis of U.S. generally accepted accounting principles ("GAAP"), third-quarter earnings per diluted share from continuing operations was $2.41, compared to earnings per diluted share of $4.32 a year ago. Prior year GAAP earnings per diluted share included a net tax benefit of approximately $1.78 per diluted share driven by the Tax Cuts and Jobs Act of 2017.

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Third-quarter Adjusted Earnings per diluted share was $3.40, flat compared to $3.41 a year ago, primarily driven by a lower share count and growth in our Medical-Surgical business, offset by a higher tax rate and lower profit contribution from our U.S. Pharmaceutical business, which includes a $60 million, or approximately $0.23 cents per diluted share, charge related to a customer bankruptcy and previously announced customer losses.

For the first nine months of the fiscal year, McKesson generated cash from operations of $141 million, and invested $405 million internally, resulting in negative free cash flow of $264 million, which was in line with the company’s expectations. During the first nine months of the fiscal year, McKesson also paid $866 million for acquisitions, repurchased approximately $1.4 billion of its common stock, paid $216 million in dividends and the company ended the quarter with cash and cash equivalents of $1.8 billion.

"Our third-quarter results reflect solid adjusted operating profit performance, particularly in our Medical-Surgical and McKesson Prescription Technology Solutions businesses, and we are pleased with the progress we are seeing in our U.S. Pharmaceutical and Canadian businesses as we work to offset headwinds we discussed when providing our fiscal year outlook in May," said John H. Hammergren, chairman and chief executive officer, McKesson Corporation. "Our year-to-date results provide momentum heading into our fiscal fourth quarter, positioning the company well as Brian Tyler assumes the role of chief executive officer on April 1, 2019."

Segment Results

U.S. Pharmaceutical and Specialty Solutions revenues were $44.3 billion for the quarter, up 6%, driven primarily by market growth and acquisitions, partially offset by previously announced customer losses and branded to generic conversions. Segment GAAP operating profit was $671 million and GAAP operating margin was 1.52%. Segment adjusted operating profit was $593 million and adjusted operating margin was 1.34%.

European Pharmaceutical Solutions revenues were $6.9 billion for the quarter, down 1% on a reported basis and up 2% on a constant currency basis, driven primarily by market growth, partially offset by the previously disclosed reduction in owned retail pharmacies and a challenging operating environment in the U.K. Segment GAAP operating profit was $26 million and GAAP operating margin was 0.38%. Segment adjusted operating profit was $69 million and adjusted operating margin was 1.00%. On a constant currency basis, adjusted operating profit was $71 million and adjusted operating margin was 0.99%.

Medical-Surgical Solutions revenues were $2.0 billion for the quarter, up 19%, driven primarily by an acquisition and market growth. Segment GAAP operating profit was $136 million and GAAP operating margin was 6.76%. Segment adjusted operating profit was $170 million and adjusted operating margin was 8.45%.

Revenues included in Other were $3.0 billion for the quarter, up 1% on a reported basis and up 5% on a constant currency basis, driven primarily by market growth. Other GAAP operating profit was $74 million and adjusted operating profit was $224 million. On a constant currency basis, adjusted operating profit was $226 million.

Fiscal Year 2019 Outlook

McKesson now expects Adjusted Earnings per diluted share of $13.45 to $13.65 for the fiscal year ending March 31, 2019, from the previous range of $13.20 to $13.80 per diluted share.

McKesson does not provide forward-looking guidance on a GAAP basis as the company is unable to provide a quantitative reconciliation of this forward-looking non-GAAP measure to the most directly comparable forward-looking GAAP measure without unreasonable effort, as items are inherently uncertain and depend on various factors, many of which are beyond the company’s control.

Dividend Declaration

The company’s Board of Directors yesterday declared a regular dividend of thirty-nine cents per share of common stock. The dividend will be payable on April 1, 2019, to stockholders of record on March 1, 2019.

Conference Call Details

The company has scheduled a conference call for today, Thursday, January 31st, at 5:00 PM ET. The dial-in number for individuals wishing to participate on the call is 323-794-2588. Craig Mercer, senior vice president, Investor Relations, is the leader of the call, and the password to join the call is ‘McKesson’. A telephonic replay of this conference call will be available for five calendar days. For individuals wishing to listen to the replay, the dial-in number is 719-457-0820 and the pass code is 9871695. An archive of the conference call will also be available on the company’s Investor Relations website at View Source

Upcoming Investor Events

McKesson management will be participating in the following investor conference:

8th Annual Leerink Partners Global Healthcare Conference, February 28, 2019, New York, NY.
Audio webcasts will be available live and archived on the company’s Investor Relations website at View Source A complete listing of upcoming events for the investment community is available on the company’s Investor Relations website.

Adjusted Earnings

McKesson separately reports financial results on the basis of Adjusted Earnings. Adjusted Earnings is a non-GAAP financial measure defined as GAAP income from continuing operations, excluding amortization of acquisition-related intangible assets, acquisition-related expenses and adjustments, LIFO inventory-related adjustments, gains from antitrust legal settlements, restructuring and asset impairment charges, and other adjustments. A reconciliation of McKesson’s GAAP financial results to Adjusted Earnings is provided in Schedules 2 and 3 of the financial statement tables included with this release.

The company does not provide forward-looking guidance on a GAAP basis prospectively as McKesson is unable to provide a quantitative reconciliation of this forward-looking non-GAAP measure to the most directly comparable forward-looking GAAP measure, without unreasonable effort, because McKesson cannot reliably forecast LIFO inventory-related adjustments, gains from antitrust legal settlements, restructuring and asset impairment charges, and other adjustments, which are difficult to predict and estimate. These items are inherently uncertain and depend on various factors, many of which are beyond the company’s control, and as such, any associated estimate and its impact on GAAP performance could vary materially.

Constant Currency

McKesson also presents its financial results on a constant currency basis. The company conducts business worldwide in local currencies, including the Euro, British pound and Canadian dollar. As a result, the comparability of the financial results reported in U.S. dollars can be affected by changes in foreign currency exchange rates. Constant currency information is presented to provide a framework for assessing how the company’s business performed excluding the effect of foreign currency exchange rate fluctuations. The supplemental constant currency information of the company’s GAAP financial results and Adjusted Earnings (Non-GAAP) is provided in Schedule 3 of the financial statement tables included with this release.

Free Cash Flow

McKesson also provides free cash flow, a non-GAAP measure. Free cash flow is defined as net cash provided by operating activities less property acquisitions and capitalized software expenditures, as outlined in the company’s condensed consolidated statements of cash flows.

Risk Factors

Except for historical information contained in this press release, matters discussed may constitute "forward-looking statements" within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934, as amended, that involve risks and uncertainties that could cause actual results to differ materially from those projected, anticipated or implied. These statements may be identified by their use of forward-looking terminology such as "believes", "expects", "anticipates", "may", "will", "should", "seeks", "approximately", "intends", "plans", "estimates" or the negative of these words or other comparable terminology. The discussion of financial trends, strategy, plans or intentions may also include forward-looking statements. It is not possible to predict or identify all such risks and uncertainties; however, the most significant of these risks and uncertainties are described in the company’s Form 10-K, Form 10-Q and Form 8-K reports filed with the Securities and Exchange Commission and include, but are not limited to: changes in the U.S. healthcare industry and regulatory environment; managing foreign expansion, including the related operating, economic, political and regulatory risks; changes in the Canadian healthcare industry and regulatory environment; exposure to European economic conditions, including recent austerity measures taken by certain European governments; changes in the European regulatory environment with respect to privacy and data protection regulations; fluctuations in foreign currency exchange rates; the company’s ability to successfully identify, consummate, finance and integrate acquisitions; the performance of the company’s investment in Change Healthcare; the company’s ability to manage and complete divestitures; material adverse resolution of pending legal proceedings; competition and industry consolidation; substantial defaults in payment or a material reduction in purchases by, or the loss of, a large customer or group purchasing organization; the loss of government contracts as a result of compliance or funding challenges; public health issues in the U.S. or abroad; cyberattack, natural disaster, or malfunction of sophisticated internal computer systems to perform as designed; the adequacy of insurance to cover property loss or liability claims; the company’s proprietary products and services may not be adequately protected, and its products and solutions may be found to infringe on the rights of others; system errors or failure of our technology products or services to conform to specifications; disaster or other event causing interruption of customer access to data residing in our service centers; changes in circumstances that could impair our goodwill or intangible assets; new or revised tax legislation or challenges to our tax positions; general economic conditions, including changes in the financial markets that may affect the availability and cost of credit to the company, its customers or suppliers; changes in accounting principles generally accepted in the United States of America; withdrawal from participation in multiemployer pension plans or if such plans are reported to have underfunded liabilities; inability to realize the expected benefits from the company’s restructuring and business process initiatives; difficulties with outsourcing and similar third party relationships; risks associated with the company’s retail expansion; and the company’s inability to keep existing retail store locations or open new retail locations in desirable places. The reader should not place undue reliance on forward-looking statements, which speak only as of the date they are first made. Except to the extent required by law, the company undertakes no obligation to publicly release the result of any revisions to these forward-looking statements to reflect events or circumstances after the date hereof, or to reflect the occurrence of unanticipated events.

Shareholders are encouraged to review the company’s filings with the Securities and Exchange Commission.

Geron Announces Expansion of Leadership Team with Appointment of Chief Medical Officer

On January 31, 2019 Geron Corporation (Nasdaq: GERN) reported an expansion of its leadership team with the appointment of a new chief medical officer to support the Company’s clinical drug development efforts in hematology-oncology (Press release, Geron, JAN 31, 2019, View Source [SID1234533009]).

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"As we transition to a late-stage development company, we are very pleased to welcome Dr. Aleksandra Rizo to Geron as Chief Medical Officer," said John A. Scarlett, M.D., Chairman and Chief Executive Officer. "With Aleksandra’s prior experience as the clinical lead for the imetelstat program for more than three years at Janssen, as well as her wealth of leadership experience with other hematology-oncology development projects at both Janssen and Celgene, we believe she is uniquely suited to build and lead our clinical development team."

"I am excited to be joining Geron at this pivotal time," said Dr. Rizo. "I strongly believe that imetelstat has great promise in hematology-oncology given my experience with the drug at Janssen and the recent data reported at the ASH (Free ASH Whitepaper) meeting in December, and look forward to the initiation of imetelstat’s Phase 3 trial in lower risk myelodysplastic syndromes."

New Chief Medical Officer Appointed

Aleksandra Rizo, M.D., Ph.D., was appointed Chief Medical Officer as of January 30, 2019 and will be a member of the Company’s Executive Management Committee. Dr. Rizo will be responsible for directing imetelstat’s clinical development strategy, including designing a product development plan for current and potential future indications. Functions under Dr. Rizo’s oversight include clinical science, clinical operations, data management, biostatistics, clinical pharmacology, translational research and medical affairs.

Dr. Rizo has more than 10 years of experience in hematology-oncology clinical development, leading teams through the entire drug development process from Phase 1 through Phase 3 clinical trials and regulatory submissions. Most recently, she was Executive Director, Strategy and Clinical Lead at Celgene Corporation, working across the myeloid portfolio. While there, she led submission activities and participated in strategic and business development initiatives. Prior to that, Dr. Rizo was a Senior Director, Compound Development Team Leader at Janssen Research and Development, LLC (Janssen) for all Phase 1 myeloid assets, and Global Clinical Leader for all late-stage myeloid assets, including imetelstat from 2014-2018. In these roles, she had oversight and leadership responsibilities for overall clinical development strategy, study designs, execution and data interpretation for all related programs. In addition, Dr. Rizo was a core member of Janssen’s Hematology Strategy Team, and in this role, participated and led diligence projects in hematology. Previously, Dr. Rizo was Global Clinical Leader for the ibrutinib mantle cell lymphoma (MCL) program and was responsible for all MCL studies led by Janssen. During her initial tenure with Janssen, Dr. Rizo worked on a variety of Velcade clinical trials in lymphoma and multiple myeloma.

Dr. Rizo holds an M.D. from the University Ss Cyril and Methodius, Skopje, Macedonia, where she also completed a residency in internal medicine/hematology. She also has a Ph.D. in human leukemic stem cell biology from the University of Groningen, Groningen, Netherlands, and a Ph.D. in mouse stem cell biology from the University of Tokyo, Tokyo, Japan.

Additional Office to be Opened in New Jersey

The Company will open an additional office in northern New Jersey in order to enhance its ability to attract talented employees from local biopharmaceutical companies with late-stage clinical drug development expertise, as well as provide support for future global clinical trials. Other corporate functions also expected to be managed from the New Jersey office include business development and, assuming imetelstat is approved, future commercial operations.

Other Executive Leadership Appointments and Responsibilities

Andrew J. Grethlein, Ph.D., has been appointed Chief Operating Officer and will be responsible for global regulatory affairs, pharmacovigilance and drug safety, manufacturing, quality, program management, human resources and information technology.

Melissa A. Kelly Behrs has been appointed Chief Business Officer and will be responsible for business development, portfolio management, alliance management, and strategic market assessment and planning.

Stephen N. Rosenfield has been appointed Chief Legal Officer and will be responsible for legal affairs, corporate compliance, intellectual property and corporate governance.

Preliminary 2019 Financial Guidance

The Company expects its operating expenses to increase as it assumes full responsibility for the development and potential commercialization of imetelstat. For fiscal year 2019, the Company expects its operating expense burn to range from $65 to $70 million, of which approximately $10 to $15 million represent one-time costs, such as imetelstat program transition activities, including the transfer of the investigational new drug (IND) sponsorship from Janssen to Geron, and purchase of raw materials and other supplies in preparation for new drug manufacturing. In addition to the one-time costs, the preliminary 2019 operating expense guidance includes costs for the expansion of the internal development team, the global Phase 3 clinical trial in lower risk myelodysplastic syndromes (MDS) and the opening of a New Jersey office. According to current hiring plans, the Company expects the total number of full-time employees to grow to be approximately 30 to 40 by year-end 2019, with half being research and development personnel. As of December 31, 2018, the Company had approximately $182 million in cash and marketable securities, which is expected to be sufficient to support its plans to initiate the Phase 3 clinical trial of imetelstat in lower risk MDS by mid-year 2019.

Inducement Grants Under Nasdaq Listing Rule 5635(c)(4)

In connection with the commencement of Dr. Rizo’s employment with the Company, the Company granted her a non-statutory stock option to purchase 750,000 shares of Geron common stock on January 30, 2019 which vests over four years, with 12.5% of the shares underlying the option vesting on the six-month anniversary of commencement of employment and the remaining shares vesting over the following 42 months in equal installments of whole shares, subject to Dr. Rizo’s continued employment with Geron. In addition, Dr. Rizo was granted non-statutory stock options to purchase an aggregate of 750,000 shares of Geron common stock on January 30, 2019 with vesting conditioned on the achievement of certain regulatory milestones for imetelstat, subject to Dr. Rizo’s continued employment with Geron on the vesting dates. All of Dr. Rizo’s stock options have a 10-year term and an exercise price of $1.03 per share, which is equal to the closing price of Geron common stock on the date of grant. The stock options were granted as a material inducement to Dr. Rizo’s employment in accordance with Nasdaq Listing Rule 5635(c)(4) and are subject to the terms and conditions of stock option agreements covering the grants and Geron’s 2018 Inducement Award Plan, which was adopted December 14, 2018 and provides for the granting of stock options to new employees.

About Imetelstat

Imetelstat is a novel, first-in-class telomerase inhibitor exclusively owned by Geron and being developed in hematologic myeloid malignancies. Early clinical data suggest imetelstat may have disease-modifying activity through the suppression of malignant progenitor cell clone proliferation, which allows potential recovery of normal hematopoiesis. Ongoing clinical studies of imetelstat include a Phase 2/3 trial called IMerge in lower risk myelodysplastic syndromes (MDS) and a Phase 2 trial called IMbark in Intermediate-2 or High-risk myelofibrosis. Imetelstat has been granted Fast Track designation by the United States Food and Drug Administration for the treatment of patients with transfusion-dependent anemia due to lower risk MDS who are non-del(5q) and refractory or resistant to an erythroid stimulating agent