PTC Therapeutics to Present at the RBC Capital Markets Global Healthcare Conference

On February 9, 2018 PTC Therapeutics, Inc. (NASDAQ: PTCT) reported that the company will present a company overview at the upcoming 2018 RBC Capital Markets Global Healthcare Conference on Thursday, February 22nd at 2:35 pm ET (Press release, PTC Therapeutics, FEB 9, 2018, View Source [SID1234523882]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

The presentation will be webcast live on the Events and Presentations page under the investor relations section of PTC Therapeutics’ website at www.ptcbio.com. The presentation will be archived for two weeks following the presentation. It is recommended that users connect to PTC’s website several minutes prior to the start of the webcast to ensure a timely connection.

BeiGene Presents Preliminary Phase 1 Data on Tislelizumab in Patients with Urothelial Carcinoma at 2018 Genitourinary Cancers Symposium

On February 9, 2018 BeiGene, Ltd. (NASDAQ:BGNE), a commercial-stage biopharmaceutical company focused on developing and commercializing innovative molecularly targeted and immuno-oncology drugs for the treatment of cancer, reported preliminary clinical data from patients with urothelial carcinoma (UC) enrolled in an ongoing Phase 1 clinical trial of tislelizumab, an investigational anti-PD-1 antibody, at the 2018 Genitourinary Cancers Symposium in San Francisco (Press release, BeiGene, FEB 9, 2018, View Source;p=RssLanding&cat=news&id=2331663 [SID1234523892]). The preliminary Phase 1 data suggest that tislelizumab was generally well tolerated and exhibited objective responses in patients with UC.

"Tislelizumab administration resulted in objective responses, including a complete response, and a disease-control rate of 53 percent. Tislelizumab was generally well-tolerated in patients with urothelial carcinoma," said Shahneen Sandhu, M.D., medical oncologist at the Peter MacCallum Cancer Center in Melbourne, Australia and lead author. "We are highly encouraged by these results and that further study of tislelizumab may lead to a new treatment for patients with urothelial cancer."

"Tislelizumab is currently being evaluated in five pivotal trials globally and in China, including a pivotal trial in patients with previously treated, PD-L1-positive, locally advanced or metastatic urothelial carcinoma in China. This is the first presentation of tislelizumab data in the population with urothelial cancer, an area of unmet need. We are pleased by these preliminary results, which we believe provide an important foundation for our clinical understanding of tislelizumab’s efficacy and safety in specific patient populations both as a single agent and in combination," commented Amy Peterson, M.D., Chief Medical Officer, Immuno-Oncology, at BeiGene.

Summary of Results from the Ongoing Phase 1 Trial

The multi-center, open-label Phase 1 trial (NCT02407990) of tislelizumab as monotherapy in advanced solid tumors is being conducted in Australia, New Zealand, the United States, Taiwan, and South Korea and consists of dose escalation, schedule expansion, fixed dose expansion, and indication expansion in disease-specific cohorts.

Data presented at the Genitourinary Cancers Symposium included 16 patients with urothelial carcinoma. Of these, 12 had one or more prior systemic anticancer treatment for metastatic disease and the remaining four had progressed after receiving platinum-based regimen in the neoadjuvant or adjuvant setting. In addition, five patients had prior radiotherapy. At the time of the data cutoff on August 28, 2017, median treatment duration was 4.3 months (range of 0.7 to 18.3 months). A total of six patients remained on treatment.

Adverse events (AEs) assessed by the investigator to be related to treatment occurred in 14 patients (88%). Of those, fatigue (31%), rash (19%), infusion related reactions (13%), nausea (13%), pain in extremity (13%), and proteinuria (13%) occurred in more than one patient. All of the treatment-related AEs were grade 1 or 2 except one case each of fatigue, hyperglycemia, and diabetes mellitus. One adverse event of muscle weakness, which was associated with disease progression and occurred more than one month after the last dose of the study drug, had a fatal outcome; this event was considered not related to treatment.

At the time of the data cutoff, 15 patients were evaluable, defined as having a measurable baseline tumor assessment and at least one evaluable post-baseline tumor response assessment, or had progressed or died prior to the initial tumor assessment. One patient had a confirmed complete response (CR), four achieved a confirmed partial response (PR), and three achieved stable disease (SD). Nine evaluable patients had PD-L1 status determined. There was one CR, two PR and one SD among six PD-L1 high patients, and one PR among three PD-L1 low or negative patients.

About Urothelial Carcinoma

Cancer that begins in cells that line the urethra, bladder, ureters, renal pelvis, and some other organs are referred to as urothelial carcinoma.i Urothelial carcinoma is the most common type of bladder cancer and the fifth most common cancer in the United States.ii In 2017, it was estimated that there were 79,030 new cases of bladder cancer and 16,870 deaths.iii

About Tislelizumab

Tislelizumab is an investigational humanized monoclonal antibody that belongs to a class of immuno-oncology agents known as immune checkpoint inhibitors. It is designed to bind to PD-1, a cell surface receptor that plays an important role in downregulating the immune system by preventing the activation of T-cells. Tislelizumab has demonstrated high affinity and specificity for PD-1. It is potentially differentiated from the currently approved PD-1 antibodies in an engineered Fc region, which is believed to minimize potentially negative interactions with other immune cells, based on preclinical data. Tislelizumab is being developed as a monotherapy and in combination with other therapies for the treatment of a broad array of both solid tumor and hematologic cancers. BeiGene and Celgene Corporation have a global strategic collaboration for the development of tislelizumab in solid tumor cancers outside of Asia (except Japan).

Arrowhead Pharmaceuticals Reports Fiscal 2018 First Quarter Results

On February 9, 2018 Arrowhead Pharmaceuticals Inc. (NASDAQ: ARWR) reported financial results for its fiscal 2018 first quarter ended December 31, 2017 (Press release, Arrowhead Research Corporation, FEB 9, 2018, View Source [SID1234523890]). The company is hosting a conference call at 4:30 p.m. EST to discuss results.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Conference Call and Webcast Details

Investors may access a live audio webcast on the Company’s website at View Source For analysts that wish to participate in the conference call, please dial 855-215-6159 or 315-625-6887 and provide Conference ID 4788374.

A replay of the webcast will be available on the company’s website approximately two hours after the conclusion of the call and will remain available for 90 days. An audio replay will also be available approximately two hours after the conclusion of the call and will be available for 3 days. To access the audio replay, dial 855-859-2056 or 404-537-3406 and provide Conference ID 4788374.

Selected Fiscal 2018 First Quarter and Recent Events

Filed a Clinical Trial Application for ARO-AAT, a second generation subcutaneously administered clinical candidate for the treatment of alpha-1 antitrypsin deficiency liver disease

Filed a Clinical Trial Application for ARO-HBV, a third generation subcutaneously administered clinical candidate for the treatment of chronic hepatitis B virus infection

Presented new clinical data at HEP DART 2017 demonstrating up to 5.0 log10 reduction in HBV s-antigen and a Sustained Host Response in 50% of hepatitis B patients following RNAi therapy, ARC-520, in the 2001 open-label extension study

Made continued progress on a two-product cardiovascular collaboration with Amgen, in which one that was previously called ARO-LPA against the target lipoprotein(a) has been formally nominated as a potential clinical candidate and which is now referred to as AMG 890 by Amgen

Expanded Arrowhead’s cardiometabolic pipeline, which now includes ARO-APOC3, targeting apolipoprotein C-III, and ARO-ANG3, targeting angiopoietin-like protein 3 (ANGPTL3); with CTA filings planned around the end of 2018

Achieved continued progress with the Company’s extra-hepatic platform and pipeline, including:

ARO-Lung1, Arrowhead’s first candidate against an undisclosed gene target in the lung, which achieved nearly 90% target knockdown following inhaled administration in rodents

ARO-HIF2, the Company’s candidate targeting renal cell carcinoma, which achieved 85% target gene knockdown in a rodent tumor model

Cambrex Reports Fourth Quarter And Full Year 2017 Financial Results

On February 8, 2018 Cambrex Corporation (NYSE: CBM), a leading manufacturer of small molecule innovator and generic Active Pharmaceutical Ingredients (APIs), reported results for the fourth quarter and full year ended December 31, 2017 (Press release, Cambrex, FEB 8, 2018, View Source [SID1234523822]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Highlights

Net revenue increased 2.5% to $182.3 million compared to $177.9 million in the same quarter last year. Excluding the impact of foreign exchange, net revenue increased 1%. Full year net revenue increased 9% to $534.5 million, compared to $490.6 million in the full year 2016.
GAAP Diluted EPS from continuing operations increased 4% to $1.20 per share from $1.15 per share in the same quarter last year. Full year GAAP Diluted EPS from continuing operations increased 17% to $3.10 per share from $2.65 per share in the full year 2016.
EBITDA increased to $64.7 million compared to $63.6 million in the same quarter last year. Full year Adjusted EBITDA increased 13% to $174.6 million from $154.2 million in the full year 2016 (see table at the end of this release).
Net cash was $183.3 million at the end of the year, an increase of $64.9 million during the quarter and $109.1 million during the year.
The Company continued to execute on its strategic growth plan, investing in new manufacturing capacity and analytical laboratory space at its facilities in Charles City, Iowa, High Point, North Carolina, Karlskoga, Sweden and Milan, Italy.
The Company expects full year 2018 Adjusted net revenue, excluding the impact of foreign currency and change in accounting principle, to be between -2% and 2% compared to 2017 and Adjusted EBITDA to be between $150 and $160 million. (see Financial Expectations – Continuing Operations section below for related explanations and additional financial guidance).
"We are pleased with our strong financial performance in 2017, our seventh straight year of solid growth. The fourth quarter was a record revenue quarter with Net revenue up 2.5% compared to a record fourth quarter last year. Strong performance at our manufacturing facilities resulted in higher profit margins versus last year," commented Steven M. Klosk, President and Chief Executive Officer of Cambrex.

"With our recently completed large scale manufacturing capacity projects and the on-going investments in R&D and pilot plant capabilities, we are well positioned to ensure our facilities are able to keep up with the strong market demand."

Basis of Reporting
The Company has provided a reconciliation of GAAP amounts to adjusted (i.e. Non-GAAP) amounts at the end of this press release. Cambrex management believes that the adjusted amounts provide useful information to investors due to the magnitude and nature of certain expenses recorded in the GAAP amounts.

Fourth Quarter 2017 Operating Results – Continuing Operations
Net revenue was $182.3 million, an increase of $4.4 million, or 2.5%, compared to the fourth quarter of 2016. Excluding a 1.5% favorable impact of foreign exchange compared to the fourth quarter of 2016, net revenue increased 1%. The increase primarily reflects higher volumes in the generic and controlled substance product categories, partially offset by lower volumes and pricing of certain branded APIs.

Gross margin decreased to 43% from 45% compared to the same quarter last year. Excluding a 1% unfavorable impact of foreign exchange compared to the fourth quarter of 2016, margins were relatively flat year over year.

Selling, general and administrative expenses were $18.7 million, compared to $18.2 million in the same quarter last year. This increase was primarily due to higher expenses related to consulting for an operational excellence initiative.

Research and development expenses were $4.3 million, compared to $3.5 million in the same quarter last year. This increase was primarily driven by costs to develop new generic drug products and higher personnel related expenses.

Operating profit decreased to $55.9 million from $56.7 million in the same quarter last year. This decrease was primarily the result of higher operating expenses as described above.

Adjusted EBITDA was $64.7 million compared to $64.4 million in the same quarter last year (see table at the end of this press release).

Income tax expense was $15.6 million resulting in an effective tax rate of 28% compared to $18.4 million and an effective tax rate of 33% in the same quarter last year. The favorable impact of immediately recognizing certain effects of share-based compensation as required by a recently adopted accounting standard and the unfavorable impact of U.S. tax reform legislation enacted in December 2017, was negligible.

Income from continuing operations was $40.2 million or $1.20 per share compared to $37.9 million or $1.15 per share in the same quarter last year. Adjusted income from continuing operations was $42.4 million or $1.27 per share, compared to $40.8 million or $1.23 per share in the same quarter last year (see table at the end of this press release).

Capital expenditures and depreciation were $13.2 million and $8.3 million, respectively, compared to $11.9 million and $6.6 million, respectively, in the same quarter last year.

Net cash was $183.3 million at the end of the fourth quarter, an increase of $64.9 million during the quarter.

Financial Expectations – Continuing Operations
The following table shows the Company’s current expectations for its full year 2018 financial performance:

Expectations
Adjusted net revenue -2% – 2%
Adjusted EBITDA $150 – $160 million
Adjusted income from continuing operations per share $2.80 – $3.03
Free cash flow $35 – $45 million
Capital expenditures $70 – $80 million
Depreciation and amortization $33 – $37 million
Adjusted effective tax rate 23% – 25%
Consistent with the Company’s usual guidance practices, these financial expectations are for continuing operations and exclude the impact of any potential acquisitions, divestitures, restructuring activities, outcomes of tax disputes and the adoption of the new revenue recognition guidance effective January 1, 2018. Adjusted net revenue expectations exclude the impact of foreign exchange and change in accounting principle. EBITDA, Adjusted EBITDA and Adjusted income from continuing operations per share for 2018 will be computed on a basis consistent with the reconciliation of the 2017 financial results in the tables at the end of this press release. Free cash flow is defined as the change in debt, net of cash during the year. Adjusted effective tax rate excludes certain effects of share-based payments that were possibly deferred under the previous guidance. The tax rate will be sensitive to the Company’s geographic mix of income, changes in the tax laws or rates within the countries in which the Company operates and the effects of certain share-based payments.

The financial information contained in this press release is unaudited, subject to revision and should not be considered final until the Company’s 2017 Form 10-K is filed with the SEC.

Conference Call and Webcast
A conference call to discuss the Company’s fourth quarter and full year 2017 results will begin at 8:30 a.m. Eastern Time on February 8, 2018 and can be accessed by calling 1-888-394-8218 for domestic and +1-323-701-0226 for international. Please use the passcode 9709552 and call approximately 10 minutes prior to the start time. A webcast will be available in the Investors section on the Cambrex website located at www.cambrex.com. A telephone replay of the conference call will be available through February 15, 2018 by calling 1-888-203-1112 for domestic and +1-719-457-0820 for international. Please use the passcode 9709552 to access the replay.

Radius Health to Present at Leerink Partners 7th Annual Global Healthcare Conference

On February 8, 2018 Radius Health, Inc. (Nasdaq:RDUS), a science-driven fully integrated biopharmaceutical company that is committed to developing and commercializing innovative therapeutics in the areas of osteoporosis, oncology and endocrine diseases, reported that Jesper Høiland, President and CEO of the Company, will present a corporate update at the Leerink Partners 7th Annual Global Healthcare Conference on Thursday, February 15, 2018 (Press release, Radius, FEB 8, 2018, View Source [SID1234523864]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Information on the presentation is as follows:

Event: Leerink Partners 7th Annual Global Healthcare Conference
Date: Thursday, February 15, 2018
Time: 2:00 p.m. ET
Location: Holmes I/II (4th fl.), Lotte New York Palace Hotel, New York, NY
A live webcast of the presentation will be available by visiting the Investors section of Radius’ website at View Source A replay of the webcast will be archived on Radius’ website for 30 days following the presentation.