On April 28, 2016 Alkermes plc (NASDAQ: ALKS) reported financial results for the first quarter of 2016 (Press release, Alkermes, APR 28, 2016, View Source;p=RssLanding&cat=news&id=2162613 [SID:1234511537]).

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"Our solid first quarter performance was highlighted by the robust growth in VIVITROL sales, the launch of ARISTADA, and continued strength of our base royalty and manufacturing business. The launch of ARISTADA continues to gain traction, and we are pleased with the progress that we are making with reimbursement discussions and physician awareness," commented James Frates, Chief Financial Officer of Alkermes. "With our strong financial position and growing commercial portfolio, we are well positioned to invest in our advancing pipeline, and today we are reiterating our financial expectations for 2016."

"We have built a differentiated and resilient business. Our portfolio of innovative products, including VIVITROL and ARISTADA, is growing rapidly and represents a significant opportunity in the years ahead," said Richard Pops, Chief Executive Officer of Alkermes. "Our pipeline has a number of exciting late-stage programs, and we are on the threshold of numerous development milestones. With three drug candidates in pivotal studies, each representing an important and differentiated treatment option in its therapeutic space, and two new candidates beginning clinical studies, the medical importance and potential economic value of our pipeline is substantial and growing."

Quarter Ended March 31, 2016 Highlights

Total revenues for the quarter were $156.8 million. This compared to $161.2 million for the same period in the prior year, or $142.0 million excluding $19.2 million of revenue from the products associated with the Gainesville manufacturing facility that was divested in April 2015 (the "Gainesville Divestiture").

Net loss according to generally accepted accounting principles in the U.S. (GAAP) was $77.4 million, or a basic and diluted GAAP loss per share of $0.51, for the quarter and reflected increased investment in the company’s advancing late-stage pipeline and commercial infrastructure. This compared to GAAP net loss of $30.7 million, or a basic and diluted GAAP loss per share of $0.21 for the same period in the prior year, or GAAP net loss of $34.9 million, or a basic and diluted loss per share of $0.24, excluding $4.2 million of GAAP net income related to the Gainesville Divestiture.

Non-GAAP net loss was $24.6 million, or a non-GAAP basic and diluted loss per share of $0.16 for the quarter. This compared to non-GAAP net income of $9.2 million, or a non-GAAP diluted earnings per share (EPS) of $0.06, for the same period in the prior year, or non-GAAP net income of $1.9 million, or basic and diluted EPS of $0.01, excluding $7.3 million of non-GAAP net income related to the Gainesville Divestiture.

Quarter Ended March 31, 2016 Financial Results

Revenues

Net sales of VIVITROL were $43.8 million, compared to $31.1 million for the same period in the prior year, representing an increase of approximately 41%.

Net sales of ARISTADA were $5.5 million, following its launch in October 2015.

Manufacturing and royalty revenues from RISPERDAL CONSTA and INVEGA SUSTENNA/XEPLION and INVEGA TRINZA were $54.7 million, compared to $46.9 million for the same period in the prior year.

Manufacturing and royalty revenues from AMPYRA/FAMPYRA1 were $28.2 million, compared to $36.5 million for the same period in the prior year, reflecting the timing of shipments.

Royalty revenue from BYDUREON was $10.5 million, compared to $9.8 million for the same period in the prior year.
Costs and Expenses

Operating expenses were $233.7 million, reflecting increased investment in the company’s development pipeline, the continued launch of ARISTADA and a $10.0 million upfront payment to Reset Therapeutics, Inc. related to a collaboration on their novel orexin modulators, which was recorded as research and development expense. Operating expenses for the quarter ended March 31, 2015 were $188.5 million, or $173.5 million excluding $15.0 million of operating expenses related to the Gainesville Divestiture.
Balance Sheet

At March 31, 2016, Alkermes had cash and total investments of $719.4 million, compared to $798.8 million at Dec. 31, 2015. At March 31, 2016, the company’s total debt outstanding was $348.5 million.

Financial Expectations

Alkermes reiterates all of its financial expectations for 2016 set forth in its press release dated Feb. 25, 2016.

On April 28, 2016 Alexion Pharmaceuticals, Inc. (NASDAQ: ALXN) reported financial results for the first quarter of 2016 (Press release, Alexion, APR 28, 2016, View Source [SID:1234511536]). Total revenues grew to $701 million, a 17 percent increase, compared to $600 million for the same period in 2015. In the first quarter, the negative impact of currency on total revenue was 5 percent or $30 million, net of hedging activities, compared to the same quarter last year. First quarter revenue growth was further negatively impacted by increased macroeconomic weakness in Latin American countries, primarily Brazil and Argentina. Non-GAAP diluted earnings per share (EPS) for the first quarter of 2016 was $1.11 per share, compared to $1.28 per share in the first quarter of 2015. On a GAAP basis, diluted EPS for the first quarter of 2016 was $0.41 per share, compared to $0.45 per share in the first quarter of 2015.

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"In Q1 2016, we grew our core Soliris business by serving a steady number of new patients with PNH and aHUS in the U.S., Europe and Japan, partially offset by the increased impact of macroeconomic weakness in Latin America. We are very pleased with the strong start to the Strensiq launches in initial countries and have now commenced the U.S. launch of Kanuma," said David Hallal, Chief Executive Officer of Alexion. "We look forward to 2016 being another transformative year for Alexion as we serve an increasing number of patients with four devastating, ultra-rare diseases and progress multiple milestones in our robust rare disease pipeline."

First Quarter 2016 Financial Highlights

Soliris (eculizumab) net product sales were $665 million compared to $600 million in Q1 2015. Net product sales increased 11 percent year-on-year, despite continued currency headwinds as well as increased macroeconomic weakness in Latin American countries, primarily Brazil and Argentina. Soliris volume increased 18 percent year-on-year.

Strensiq (asfotase alfa) net product sales were $33 million.

Kanuma (sebelipase alfa) net product sales were $2.5 million.

Non-GAAP R&D expense was $158 million, compared to $97 million in the same quarter last year. GAAP R&D expense was $176 million, compared to $221 million in the same quarter last year.

Non-GAAP SG&A expense was $194 million, compared to $157 million in the same quarter last year. GAAP SG&A expense was $233 million, compared to $187 million in the same quarter last year.

Non-GAAP diluted EPS was $1.11 per share, compared to $1.28 per share in the same quarter last year. On a GAAP basis, diluted EPS was $0.41 per share, compared to $0.45 per share in the same quarter last year.
Product and Pipeline Updates

Complement Portfolio

Eculizumab—Generalized Myasthenia Gravis (gMG): Enrollment is complete in the REGAIN study, a single, multinational, placebo-controlled registration trial of eculizumab in refractory gMG, and data are expected in mid-2016.

Eculizumab—Neuromyelitis Optica Spectrum Disorder (NMOSD): Alexion expects to complete enrollment this year in the PREVENT study, a single, multinational, placebo-controlled registration trial of eculizumab in patients with relapsing NMOSD.

Eculizumab—Delayed Graft Function (DGF): Enrollment is complete in the PROTECT study, a single, multinational, placebo-controlled registration trial of eculizumab in the prevention of DGF, and data are expected in the second half of 2016.

ALXN1210: Alexion exceeded target enrollment in both a Phase 1/2 study and a Phase 2 study of ALXN1210, our highly innovative longer-acting C5 antibody, in patients with paroxysmal nocturnal hemoglobinuria (PNH), and we expect data from the Phase 1/2 study to be presented in mid-2016. Alexion also expects to initiate a clinical program in patients with atypical hemolytic uremic syndrome (aHUS) later this year.

ALXN1007: Alexion is continuing to advance the development of ALXN1007, a complement inhibitor that targets C5a, in patients with graft-versus-host disease involving the lower gastrointestinal tract (GI-GVHD). Interim Phase 2 data reported in the fourth quarter of 2015 support the evaluation of higher doses of ALXN1007 in additional patients with acute GI-GVHD.
Metabolic Portfolio

Strensiq: New long-term data presented at the Endocrine Society’s 98th Annual Meeting and Expo (ENDO) in April showed sustained improvements in survival rates, bone healing, respiratory support, and growth and mobility in children with HPP treated with Strensiq. In addition, the data presented at ENDO showed that adolescent and adult patients treated with Strensiq reduced or eliminated their need of ambulatory assistive devices and had improvements in physical function as measured by the Six Minute Walk Test.

Kanuma: Kanuma received marketing approval from Japan’s Ministry of Health, Labour and Welfare on March 28, 2016. Additionally, new data presented by researchers at the WORLDSymposium meeting in March showed a substantial survival benefit beyond 2 years of age in infants with LAL-D treated with Kanuma.

SBC-103: Alexion has commenced the planned dose escalation in the Phase 1/2 trial of SBC-103, a recombinant form of the NAGLU enzyme, in patients with mucopolysaccharidosis IIIB, or MPS IIIB. Patients are now being randomized to either a 5 mg/kg or 10 mg/kg dose. Six-month data presented at the WORLDSymposium meeting in March showed continued reductions in heparan sulfate cerebrospinal fluid with a mean reduction of 26% in the highest dose studied, 3 mg/kg.

cPMP Replacement Therapy (ALXN1101): Alexion is progressing a pivotal study to evaluate ALXN1101 in neonates with Molybdenum Cofactor Deficiency (MoCD) Type A. Alexion received Breakthrough Therapy designation for its cPMP replacement therapy.
Preclinical Portfolio

Alexion has more than 30 diverse preclinical programs across a range of therapeutic modalities, with four of these programs expected to enter the clinic in 2016.
2016 Financial Guidance

Alexion expects 2016 total revenues to be at the low end of our previously guided range of $3,050 million to $3,100 million, primarily due to increased macroeconomic weakness in Latin America, partially offset by an increase in Strensiq revenues and the strengthening of foreign currencies.

R&D and SG&A expenses are expected to be at the high end of guidance primarily due to continued investment in key programs in our R&D pipeline and the commercial launches of Strensiq and Kanuma, as well as the strengthening of foreign currencies.

Alexion expects 2016 non-GAAP EPS to be at the low end of the previously guided range of $5.00 to $5.20 per share.

Updated 2016 non-GAAP financial guidance is as follows:

Updated Guidance (1) Prior Guidance (1)
Total revenues Low end of $3,050 to $3,100 million $3,050 to $3,100 million
Soliris revenues $2,835 to $2,875 million $2,900 to $2,925 million
Metabolic revenues $180 to $200 million $150 to $175 million
Cost of sales 8% to 9% 8% to 9%
Research and development expense High end of $650 to $680 million $650 to $680 million
Selling, general and administrative expense High end of $760 to $790 million $760 to $790 million
Interest expense $100 million $100 million
Effective tax rate 7% to 8% 7% to 8%
Earnings per share Low end of $5.00 to $5.20 $5.00 to $5.20
Diluted shares outstanding 230 million 230 million

(1) Financial guidance is based on forecasted results at current spot rates net of hedging activities.

On April 28, 2016 Oxford BioMedica plc (LSE: OXB), ("OXB" or "the Group"), a leading gene and cell therapy group, reported its preliminary financial results for the twelve months ended 31 December 2015 (Press release, Oxford BioMedica, APR 28, 2016, View Source [SID:1234511533]).

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OPERATIONAL HIGHLIGHTS (including post-period end):

Strong progress from LentiVector delivery platform

Portfolio review in Q1 2016: focus on OXB-102, OXB-202 and OXB-302

OXB-102: On track for Phase I/II study in Parkinson’s disease

OXB-202: Phase I/II study preparations continued; CTA filing planned for 2016 in corneal graft rejection

OXB-302: pre-clinical data demonstrates efficacy in tumour challenge model (CAR-T 5T4)

OXB-201 safety, tolerability, dose responsive protein expression in eye

Lentiviral vector production volumes increased by 71%

Investment in people, facilities and plant

Headcount increased from 134 to 231

New Yarnton facility operational

Harrow House extension and Windrush Court laboratories currently being validated

Partnerships broadened

Novartis extend beyond CTL019 with second CAR-T product

Immune Design LV305 collaboration extended and new IP licence

GSK acquired IP licence for two rare disease product candidates

Board strengthened

Dr Lorenzo Tallarigo joined as Chairman and Stuart Henderson joins as non-executive Director and Chair of Audit Committee in February 2016 and June 2016, respectively.

FINANCIAL HIGHLIGHTS(1):

28% growth in gross income (2) from £14.7 million to £18.8 million

72% growth in income from process development and bioprocessing from £7.2 million to £12.4 million

Loss and total comprehensive expense for the year £13.0 million (2014: £8.7 million)

£14.9 million cash used in operations (2014: £7.4 million)

£16.7 million capital expenditure (2014: £5.6 million)

£9.4 million cash at 31 December 2015 (2014: £14.2 million); £7.6 million net proceeds from placing in February 2016

Audited financial results

Aggregate of Revenue and Other operating income

Commenting on the financial results, John Dawson, Chief Executive officer of Oxford BioMedica, said:"Oxford BioMedica is ideally placed to capitalise on the rapid progress that is ongoing across the gene and cell therapy sector. We have a unique lentiviral vector delivery platform, LentiVector, based on our intellectual property, expert staff, and state-of-the-art facilities and equipment. This platform is at the core of our business, enabling us to build a leading gene and cell therapy presence with both our own proprietary product candidates and, as the partner-of-choice for other companies operating in the sector, a long term economic interest in an increasing number of partners’ products.

"The outlook for the business is excellent and I look forward to further success in 2016 as we advance our focused in-house pipeline, look forward to progress with our partners’ programmes, and secure further partnerships."

On April 28, 2016 Kite Pharma, Inc., (Nasdaq:KITE) ("Kite") a clinical-stage biopharmaceutical company focused on developing engineered autologous T cell therapy (eACT) products for the treatment of cancer, reported four presentations to be delivered at the upcoming American Society of Gene & Cell Therapy (ASGCT) (Free ASGCT Whitepaper) Annual Meeting (Press release, Kite Pharma, APR 28, 2016, View Source [SID:1234511531]). The presentations will address KTE-C19, Kite’s lead chimeric antigen receptor (CAR) product candidate, and, separately, a fully-human anti-CD19 CAR product candidate for the treatment of B-cell lymphomas and leukemias. The fully-human anti-CD19 CAR product candidate is currently being studied in an ongoing Phase 1 clinical trial as part of a Cooperative Research and Development Agreement (CRADA) between Kite and the National Cancer Institute (NCI). Under this CRADA, Kite collaborates with James (Jim) N. Kochenderfer, M.D., an investigator in the Experimental Transplantation and Immunology Branch of the NCI.

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Oral Presentations:

The Impact of Different Hinge and Transmembrane Components on the Function of a Novel Fully-Human Anti-CD19 Chimeric Antigen Receptor

Date: Wednesday, May 4, 2016 4:45-5:00PM Eastern Time
Session: Cancer-Targeted Gene and Cell Therapy (3:30-5:30PM Eastern Time)
Abstract Number: 74
Location: Washington 1-2, Marriott Wardman Park
Presenter: Leah Alabanza, Ph.D., Experimental Transplantation and Immunology Branch, National Cancer Institute, Bethesda, MD

Production of KTE-C19 (Anti-CD19 CAR T Cells) for ZUMA-1: A Phase 1/2 Multi-Center Study Evaluating Safety and Efficacy in Subjects with Refractory Aggressive Non-Hodgkin Lymphoma (NHL)

Date: Thursday, May 5, 2016 4:15-4:30PM Eastern Time
Session: Vector and Cell Engineering/Manufacturing (4:00-5:45PM Eastern Time)
Abstract Number: 287
Location: Washington 1-2, Marriott Wardman Park
Presenter: Marc Better, Ph.D., Kite Pharma, Santa Monica, CA

Updated Phase 1 Results from ZUMA-1: A Phase 1/2 Multi-Center Study Evaluating the Safety and Efficacy of KTE-C19 (Anti-CD19 CAR T Cells) in Subjects with Refractory Aggressive Non-Hodgkin Lymphoma (NHL)

Date: Saturday, May 7, 2016 10:15-10:30AM Eastern Time
Session: Cancer-Immunotherapy, Cancer Vaccines III (10:15AM-12:15PM Eastern Time)
Abstract Number: 745
Location: Thurgood Marshall North, Marriott Wardman Park
Presenter: Frederick Locke, M.D., Moffitt Cancer Center, Tampa, FL

Poster Presentation:

Development of a Manufacturing Process Using Monte Carlo Simulations to Support KTE-C19 (Anti-CD19 CAR T Cells) Studies in Leukemia

Date: Friday, May 6, 2016 6:00-8:00PM Eastern Time
Session: Cancer-Immunotherapy, Cancer Vaccines III
Abstract Number: 650
Location: Exhibit Hall C & B South, Marriott Wardman Park
Presenter: Kenny Choi, Kite Pharma, Santa Monica, CA

On April 28, 2016 Varian Medical Systems reported that it will demonstrate its full range of radiotherapy delivery systems at the 35th ESTRO (European Society for Radiotherapy and Oncology) meeting, taking place here in Turin from April 29th-May 3rd (Press release, InfiMed, APR 28, 2016, View Source [SID:1234511530]). The Varian booth features the company’s technology and products for radiotherapy, radiosurgery, brachytherapy, and proton therapy, and will offer the European clinical community its first glimpse of High Definition Radiotherapy (HDRT).

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Visitors to the Varian booth (No. 5500) can learn about the company’s four pi (4π) non-coplanar treatment technology*, which exploits specific capabilities of the TrueBeam platform and is designed to deliver more compact radiation doses that may fully saturate a targeted tumor and "fall off" sharply outside the target zone, potentially minimizing dose to specific organs requiring more protection.

"We believe this technology could enable High Definition Radiotherapy — the next big advance in radiation oncology, rivaling the development of IMRT in the 1990s, IGRT and volumetric modulated arc therapy (VMAT) in the 2000s, and linac-based radiosurgery in more recent years," says Kolleen Kennedy, president of Varian’s Oncology Systems group. "This has the potential to make a big difference in the treatment of cancer patients."

Also on display on the Varian (NYSE: VAR) booth is the company’s TrueBeam platform for radiotherapy and radiosurgery, along with the RapidArc image-guided intensity-modulated radiotherapy system, the PerfectPitch six-degrees-of-freedom couch, and the Calypso ‘GPS for the Body’ system, all of which are aimed at helping clinicians to deliver treatments with both precision and speed. Varian will also exhibit its powerful family of oncology software products, including RapidPlan software for improving the quality and speed of treatment planning.

Additionally, the Varian booth is spotlighting the company’s ProBeam system, a fully-integrated image-guided IMPT (intensity-modulated proton therapy) solution, which incorporates pencil-beam scanning technology to optimize the dose applied to every point within the area being treated. Varian was recently selected to equip national proton therapy centers in Holland, Denmark and the UK with the ProBeam system.

Varian is hosting an ESTRO symposium entitled ‘Moving Radiotherapy towards the Horizon’ at 1.10pm on Sunday May 1st. This event will be chaired by Dr. Patrick Kupelian, Varian’s vice president of clinical affairs, and will feature presentations from Dr. Clive Peedell from South Tees Hospital in Middlesbrough, UK, and Dr. Max Dahele of VU University Medical Center in Amsterdam. These presentations will focus on technological advances and challenges in accessing new technology.