On August 4, 2016 Five Prime Therapeutics, Inc. (Nasdaq:FPRX), a clinical-stage biotechnology company focused on discovering and developing innovative immuno-oncology protein therapeutics, reported a corporate update and reported financial results for the second quarter ended June 30, 2016 (Press release, Five Prime Therapeutics, AUG 4, 2016, View Source [SID:1234514240]).

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"We’re pleased with the progress we’ve made through the first half of the year and remain on track with our clinical programs," said Lewis T. "Rusty" Williams, M.D., Ph.D., president and chief executive officer of Five Prime. "At ASCO (Free ASCO Whitepaper) in June, we announced early but encouraging Phase 1 data for our FGFR2b antibody, FPA144, including confirmed partial responses in three of nine patients with FGFR2b+ gastric tumors and one confirmed complete response in a patient with bladder cancer. Notably, FPA144 recently received FDA Orphan Drug Designation in patients with FGFR2b+ gastric cancer. Development of our anti-CSF1R antibody, FPA008, now named cabiralizumab, is also progressing according to plan. We still expect the Phase 1b dose expansion portion of the trial combining cabiralizumab with OPDIVO (nivolumab) in multiple tumor types to begin during the second half of 2016. Also on track is our Phase 2 clinical trial of cabiralizumab in patients with pigmented villonodular synovitis (PVNS), a rare indication for which we received FDA Orphan Drug Designation."

Business Highlights and Recent Developments

Clinical Pipeline:

FPA008 (cabiralizumab): an investigational antibody that inhibits CSF1R and has been shown to block the activation and survival of monocytes and macrophages. In some cancers, macrophages suppress the immune system’s ability to kill cancer cells. In pigmented villonodular synovitis (PVNS), macrophages form the bulk of the tumor mass in joints. Cabiralizumab blocks the activation and survival of these cell types. Five Prime and Bristol-Myers Squibb (BMS) have an exclusive worldwide collaboration agreement for the development and commercialization of cabiralizumab.

Advanced Phase 1a/1b cabiralizumab/OPDIVO combination trial.
Five Prime continued dose exploration in the Phase 1a/1b clinical trial evaluating the safety, tolerability and preliminary efficacy of the immunotherapy combination of cabiralizumab with OPDIVO, BMS’s PD-1 immune checkpoint inhibitor. The trial is currently expected to enroll up to 280 patients and remains on target to move into Phase 1b during the second half of 2016.

Advanced clinical trial of cabiralizumab in patients with PVNS into Phase 2 in May.
Five Prime advanced cabiralizumab into the Phase 2 dose expansion portion of the ongoing Phase 1/2 trial in PVNS, a CSF-1 receptor-driven tumor. The Phase 2 portion is evaluating clinical measures including response rate, pain and range of motion in approximately 30 PVNS patients. The U.S. Food and Drug Administration (FDA) granted cabiralizumab Orphan Drug Designation for the treatment of PVNS. Five Prime estimates the U.S. prevalence for diffuse PVNS patients may be as high as 25,000 patients.

FPA144: an isoform-selective antibody in development as a targeted immuno-therapy for tumors that over-express FGFR2b, a splice variant of a receptor for some members of the fibroblast growth factor (FGF) family. FPA144 has been engineered for enhanced antibody-dependent cell-mediated cytotoxicity (ADCC) to increase direct tumor cell killing by recruiting natural killer (NK) cells.

In July, received FDA Orphan Drug Designation for FPA144 for the treatment of gastric cancer and cancer of the gastroesophageal junction in patients whose tumors overexpress FGFR2b.

Presented data from the ongoing Phase 1 trial of FPA144 in an oral session at the 2016 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting in June.

FPA144 monotherapy demonstrated early evidence of anti-tumor efficacy in gastric cancer patients with FGFR2b protein overexpression from Part 1b and Part 2 of the trial. These heavily pre-treated patients had received between 1 and 6 prior therapies with a median of 2 prior therapies. The highlights of the data presented at ASCO (Free ASCO Whitepaper) include:

Efficacy as of the April 1, 2016 data cutoff:
3 confirmed partial responses (PRs) out of 9 gastric cancer patients treated (33%); 1 of these 3 PRs was confirmed after the data cutoff
7 of 9 gastric cancer patients with disease control (3 PRs + 4 stable disease), disease control rate (DCR) = 77%
12-week progression-free survival (PFS) in 6 of 9 gastric cancer patients (67%)
Median duration of treatment of 112 days (range 42-182 days), with 2 of 9 gastric cancer patients still on study
A patient with metastatic bladder cancer in the dose escalation portion of the trial in solid tumors achieved a confirmed complete response (CR)

In addition to the 3 PRs in gastric cancer noted above, there was an additional unconfirmed PR in the 10th gastric cancer patient with FGFR2b protein overexpression, whose scan became available after the data cutoff.
Safety as of the April 1, 2016 data cutoff:

No dose-limiting toxicities (DLTs); maximum-tolerated dose (MTD) was not reached
No treatment-related serious adverse events (SAEs)
The most common treatment-related AEs ( > 5%) were all grades 1 or 2: fatigue (22.5%), nausea (20%) and vomiting (12.5%)

Presented preclinical data for FPA144 at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting. FPA144 was featured in two presentations at the AACR (Free AACR Whitepaper) Annual Meeting in April 2016. Five Prime demonstrated that FPA144’s enhanced ADCC mechanism drives innate and adaptive immune responses in the tumor microenvironment, recruiting NK and T cells into the tumor. Additionally, FPA144 produced an additive effect on tumor growth inhibition when combined with PD-1 blockade. These pre-clinical findings suggest the therapeutic potential for a combination of FPA144 with a checkpoint inhibitor.

FP-1039: a protein drug designed to intervene in FGF signaling. As a ligand trap, FP-1039 binds to and neutralizes FGF ligands (such as FGF2), preventing these signaling proteins from reaching FGFR1 on the surface of tumor cells.

GSK presented data in mesothelioma patients from the ongoing Phase 1b trial of FP-1039 at the 2016 ASCO (Free ASCO Whitepaper) Annual Meeting.

Although GSK has terminated its FP-1039 license from Five Prime, GSK is continuing to conduct the ongoing study of FP-1039 in combination with 1st-line pemetrexed and cisplatin in patients with untreated, unresectable mesothelioma. GSK has now capped the trial at the 25 patients enrolled at the 15 mg/kg dose.

The trial data are not sufficiently mature for Five Prime to make decisions yet on potential future development of FP-1039 in mesothelioma. Those decisions will be based on our future assessment of the response rate and durability in this trial, as well as other considerations, such as drug supply and manufacturing.
Preclinical Research and Development:

Progressed internal immuno-oncology research programs. Five Prime continues to advance multiple immuno-oncology candidates through preclinical development and expects to have two programs entering pre-IND studies before the end of 2016. The company anticipates filing one IND by the end of 2017 and to have preclinical assets sufficient to keep the pace of one IND per year for the foreseeable future.

GSK licensed intellectual property for respiratory disease target identified using Five Prime’s proprietary protein discovery platform. In June, GSK exercised its option to take an exclusive license to the intellectual property related to a target under the 2012 respiratory diseases research collaboration between the companies. This triggered a $1.5 million license payment to Five Prime, which was recognized as revenue in the second quarter. Five Prime is eligible to receive up to $92.75 million in contingent milestone payments for each product that targets the licensed protein.
Summary of Financial Results and Guidance:

Cash Position. Cash, cash equivalents and marketable securities totaled $469.2 million on June 30, 2016, compared to $517.5 million on December 31, 2015. The decrease in cash was primarily attributable to cash used in operations to advance the FPA144 clinical trial, preclinical programs and tax payments.

Revenue. Collaboration revenue for the second quarter of 2016 increased by $2.9 million to $9.2 million from $6.3 million in the second quarter of 2015. This increase was primarily due to revenue recognized under the 2015 cabiralizumab collaboration agreement with BMS, under which Five Prime is reimbursed for the immuno-oncology trial expenses.

R&D Expenses. Research and development expenses for the second quarter of 2016 increased by $8.9 million, or 66.9%, to $22.2 million from $13.3 million in the second quarter of 2015. This increase was primarily related to advancing the FPA144 clinical trial, preclinical development and immuno-oncology research programs.

G&A Expenses. General and administrative expenses for the second quarter of 2016 increased by $3.5 million, or 76.1%, to $8.1 million from $4.6 million in the second quarter of 2015. This increase was primarily due to increases in cash and stock-based compensation expenses.

Net Loss. Net loss for the second quarter of 2016 was $13.1 million, or $0.49 per basic and diluted share, compared to a net loss of $11.5 million, or $0.45 per basic and diluted share, for the second quarter of 2015.

Shares Outstanding. Total shares outstanding were 28.3 million as of June 30, 2016.

Cash Guidance. Five Prime continues to expect full-year 2016 net cash used in operating activities to be less than $120 million, comprising less than $90 million used in operations and less than $30 million used for tax payments. The company estimates ending 2016 with approximately $400 million in cash, cash equivalents and marketable securities.

On August 5, 2016 Medigene AG (MDG1, Frankfurt, Prime Standard), a clinical stage immuno-oncology company focusing on the development of T-cell immunotherapies for the treatment of cancer, reported financial results and corporate updates for the first six months of 2016 (Press release, MediGene, AUG 4, 2016, View Source [SID:1234514267]).

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Major events since the beginning of 2016:

Immunotherapies:

Phase II of Phase I/II trial with DC vaccine for the treatment of acute myeloid leukaemia (AML) initiated following positive recommendation by DSMB
Collaboration started with Max Delbrück Center and The Charité in Berlin for Germany’s first investigator-initiated clinical TCR trial
DC platform and TCR platform strengthened by new patents
Additional viral vector production capacities secured for clinical TCR trials
Company:

Expansion of management team
Capital increase by contribution in kind to fund milestone payment for the beginning of Phase II of Medigene’s Phase I/II trial with DC vaccines
Key figures in the first half of 2016:

R&D expenses increased by 23% to €5,079 k (6M 2015: €4,117 k)
Total revenue increased by 62% to €5,470 k (6M 2015: €3,372 k)
EBITDA loss reduced by 6% to €4,011 k (6M 2015: €4,251 k)
Cash and cash equivalents and time deposits increased by 4% to €48,672 k (12/31/2015: €46,759 k)
Prof. Dolores Schendel, Chief Executive Officer and CSO of Medigene AG, comments: "In recent months we have made huge advances in preparations for the clinical development of TCRs and strengthened our portfolio of patents for this highly innovative therapy. In addition, our DC trial reached a meaningful milestone with progression into Phase II. I am delighted to be able to announce such significant progress in the first five months of my term as CEO of Medigene AG."

Dave Lemus, Chief Operating Officer of Medigene AG adds: "The continued improvement in our financial position in the first six months of 2016 reflects in part the successful repositioning of Medigene as a leading player in the field of immuno-oncology. Moreover, our solid financial situation allows us to increasingly exploit the full potential of our immunotherapy capabilities by funding further research and development activities."

Key figures in first half of 2016:

In € k 6M-2016

6M-2015

Change

Results of operations
Revenue 1,385 1,363 2%
Other operating income 4,085 2,009 103%
thereof gain on sale of intangible assets, net 2,365 0 -
Total revenue 5,470 3,372 62%
Cost of sales -773 -471 64%
Gross profit 4,697 2,901 62%
Selling and general administrative expenses -4,013 -3,476 15%
Research and development expenses -5,079 -4,117 23%
Operating result -4,395 -4,692 -6%
Income from the sale of financial assets 4,242 0 -
Net profit/loss for the period -401 -6,113 -93%
EBITDA -4,011 -4,251 -6%
Earnings per share (€) -0.02 -0.44 -95%
Personnel expenses -4,536 -3,581 27%

Cash flows
Net cash used in operating activities -8,818 -4,953 78%
Net cash from/used in investing activities 10,864 -159 >-200%
Net cash from financing activities -132 195 -168%

Balance sheet data as at June 30, 2016 and December 31, 2015
Cash and cash equivalents and time deposits 48,672 46,759 4%
Total assets 105,599 113,531 -7%
Current liabilities 5,244 9,664 -46%
Non-current liabilities 12,781 13,879 -8%
Shareholders’ equity 87,574 89,988 -3%
Equity ratio (%) 83 79 5%

Employees as at June 30 80 71 13%
FTE as at June 30 73 65 12%

Medigene share as at June 30
Total number of shares outstanding 20,088,260 14,051,815 43%
Total revenue of the Company increased by 62% to €5,470 k in the reporting period (6M 2015: €3,372 k) on account of non-recurring impacts related to the sale of EndoTAG in December 2015. Income of €2,365 k was generated by this sale in the first quarter of 2016. The Company generates its revenue and other operating income from its non-core business.

Research and development expenses increased by 23% in the first six months of 2016 to €5,079 k (6M 2015: €4,117 k) as expected. The increase in these expenses is mainly due to planned increases in expenses for preclinical and clinical trials for Medigene’s immunotherapies which increased significantly by 68% to €4,345 k in the first six months of 2016 (6M 2015: €2,585 k). This increase was partially offset by the decrease in development expenses for other sold products.

Despite higher research and development expenses for Medigene’s immunotherapy programs, the EBITDA loss decreased by 6% in the first half of 2016 to €4,011 k (6M-2015: €4,251 k). Medigene’s EBITDA is derived from the net profit/loss for the period; it excludes any taxes, financial results (comprising interest income, interest expense, other financial result and income the sale of financial assets), foreign exchange gains or losses, share of results of associates, or depreciation or amortization.

Due to non-recurring effects, including the sale of EndoTAG and Immunocore shares, the net loss after taxes improved in the first six months of 2016 to just €401 k (6M 2015: €6,113 k).

The net cash used in operating activities increased to €8,818 k in the first half of 2016 (6M 2015: €4,953 k). This represents an average monthly cash outflow of €1.5 m in the first six months of 2016 (6M 2015: €0.8 m). The major part of the cash used was directed at research and development, sales and administration and an increase in working capital. The current level of cash used in operating activities is not indicative of future trends as it is significantly impacted by non-recurring payments in partner arrangements and research and development expenses, which in turn are impacted by project stage/status.

Medigene recorded a cash inflow from investing activities of €10,864 k in the first six months of 2016 in contrast to a cash outflow of €159 k in the comparable period of the prior year. The cash inflow resulted primarily from the sale of shares in Immunocore Ltd. for €6 m and from the sale of Catherex, Inc. to Amgen Inc. for €4 m.

The cash and cash equivalents and time deposits of the Company amounted to €48,672 k as at the end of the reporting period (December 31, 2015: €46,759 k). Medigene generated a cash inflow of €10,672 k from the above sales.

Financial forecast 2016:
Medigene confirms its financial guidance for the fiscal year 2016. In line with previous expectations, the Company plans to expand its clinical development programs, which will significantly increase R&D expenses in the immunotherapies segment to €9 – 11 m (2015: €5.5 m). The EBITDA loss for 2016 is anticipated to come to €10 -12 m (2015: €9.5 m).

The Company expects total Veregen revenue of €3 – 4 m in 2016 (2015: €3.1 m), and stable or increasing total revenue (2015: €6.8 m). As revenue is not generated in the Company’s core business of immunotherapies, these figures are not indicative of progress in the Company’s core business.. This financial guidance also does not include any possible revenues from potential new partnership agreements, or any exchange rate fluctuation.

The detailed 6-Months Report 2016 is available online at:
View Source

On August 4, 2016 Karyopharm Therapeutics Inc. (Nasdaq:KPTI), a clinical-stage pharmaceutical company, reported financial results for the second quarter 2016 and commented on recent accomplishments and clinical development plans for its lead, novel, oral Selective Inhibitor of Nuclear Export (SINE) compound selinexor (KPT-330), and other pipeline assets, including KPT-8602, its second-generation SINE compound, and KPT-9274, its oral, dual inhibitor of p21-activated kinase 4 (PAK4) and nicotinamide phosphoribosyltransferase (NAMPT) (Filing, Q2, Karyopharm, 2016, AUG 4, 2016, View Source [SID:1234514318])).

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"In the first half of 2016, we continued to drive rapid and meaningful progress across our development programs, including completion of enrollment in our Phase 2b STORM study in multiple myeloma (MM), the opening of a Phase 1b study arm evaluating selinexor in combination with the PD-1 inhibitor pembrolizumab in advanced solid tumors, and the advancement of KPT-9274 into the clinic," said Michael G. Kauffman, MD, PhD, Chief Executive Officer of Karyopharm. "At the 2016 European Hematology Association (EHA) (Free EHA Whitepaper) Annual Meeting, we presented exciting preliminary data from our Phase 1b STOMP study showing high response rates, along with durable activity and a favorable safety profile when selinexor is combined with standard-of-care agents in heavily pre-treated patients with MM."

Dr. Kauffman continued, "We remain on track for data readouts from several of our ongoing selinexor studies, including top-line results from the STORM and STOMP studies in relapsed/refractory MM, updated data from the SIGN study in gynecologic malignancies at the European Society of Medical Oncology (ESMO) (Free ESMO Whitepaper) 2016 Annual Meeting, and an interim analysis from the ongoing Phase 2 SOPRA study in relapsed/refractory acute myeloid leukemia (AML) in late 2016. For KPT-8602, our second generation SINE compound, we look forward to reporting top-line safety and tolerability results from the Phase 1 portion of the ongoing study in patients with relapsed/refractory MM in late 2016. All of these efforts bring us ever closer to our goal of providing novel, first-in-class medicines to patients with cancer and other major diseases. We look forward to keeping you updated on our progress."

On August 4, 2016 Genocea Biosciences, Inc. (NASDAQ:GNCA), a biopharmaceutical company developing T cell-directed vaccines and immunotherapies, reported corporate highlights and financial results for the second quarter ended June 30, 2016 (Press release, Genocea Biosciences, AUG 4, 2016, View Source [SID:1234514241]).

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"Full results from the Phase 2 dose optimization trial evaluating GEN-003, our immunotherapy for genital herpes, were showcased at ASM Microbe 2016 in June. The data show that a single course of treatment has significant and durable effects on both HSV-2 viral activity and genital herpes clinical disease for at least 12 months post dosing. Based on this clinical profile, we believe GEN-003 may offer benefits similar to a full year of daily administration of oral antivirals, giving us confidence in the potential for GEN-003 to become a cornerstone therapy for patients suffering with genital herpes," said Chip Clark, president and chief executive officer of Genocea. "We are looking forward to reporting viral shedding and six-month clinical efficacy data from our ongoing Phase 2b study of GEN-003 in September and around the end of this year, respectively."

Recent Business Highlights and Anticipated Milestones

GEN-003 – Immunotherapy for treatment of genital herpes in Phase 2 development. Greater than $1 billion potential revenue opportunity in U.S. alone

In June 2016, Genocea highlighted positive 12-month efficacy data from its Phase 2 dose optimization trial evaluating GEN-003 for the treatment of genital herpes at the American Society for Microbiology ("ASM") annual general meeting, ASM Microbe 2016. The study results, first announced in March 2016, demonstrate sustained and statistically significant reductions compared to baseline across multiple dose groups in the rate of viral shedding 12 months after treatment with GEN-003, as well as sustained efficacy at multiple dose levels across secondary endpoints measuring the impact on clinical disease. GEN-003 was well tolerated by patients, with no serious adverse events related to the vaccine in the trial.

Multiple anticipated upcoming clinical and regulatory milestones for GEN-003

Phase 2b viral shedding data expected in September
Phase 2b 6-month clinical efficacy data expected around the end of 2016
End-of-Phase 2 meeting with the U.S. Food and Drug Administration (FDA) expected in Q1 2017
Initiation of Phase 2 antiviral combination study in fourth quarter of 2016
In September, Genocea expects to report viral shedding data from an ongoing Phase 2b trial. Approximately 135 subjects have been randomized to one of three dose groups – placebo, 60 µg per protein / 50 µg of adjuvant and 60 µg per protein / 75 µg of adjuvant – and are being monitored for 12 months.

Around the end of 2016, Genocea expects to report 6-month clinical efficacy data from this Phase 2b trial. The placebo-controlled data will represent the first opportunity to measure GEN-003 against potential Phase 3 clinical endpoints at 6-months after dosing.

Following these two data readouts, Genocea expects to conduct an end-of-Phase 2 meeting with the FDA in the first quarter of 2017. During this meeting, the Company will confirm the Phase 3 program for GEN-003, which it expects to initiate in the second half of 2017.

Genocea also expects to commence a Phase 2b antiviral combination study in the fourth quarter of 2016. Six-month clinical efficacy data from this trial is expected in the second half of 2017. If GEN-003 is additive to the effect of chronic suppressive oral anti-viral therapy, the Company believes this would further strengthen GEN-003’s value proposition to patients and physicians.

Immuno-oncology collaborations and cancer vaccine strategy

Genocea continues to advance its collaborations with Memorial Sloan Kettering Cancer Center and Dana-Farber Cancer Institute and expects to announce further data from these collaborations together with an update on its internal cancer vaccine development strategy in the fourth quarter of 2016.

Expanded company leadership

John Bishop, Ph.D. joined as Senior Vice President of Pharmaceutical Sciences
Ron Cooper appointed to the Board of Directors
In May 2016, John Bishop, Ph.D. joined the company as senior vice president of pharmaceutical sciences. He was formerly at Momenta Pharmaceuticals Inc., where he was responsible for all aspects of chemistry, manufacturing, and controls across an array of development and commercial-stage products. In his new role at Genocea, Dr. Bishop will lead pharmaceutical sciences and manufacturing activities for all stages of product development and commercialization.

In June 2016, Ron Cooper joined the company’s board of directors. Mr. Cooper is currently president and chief executive officer of Albireo Ltd. (Albireo), a late-stage biopharmaceutical company developing therapeutics to treat orphan pediatric liver diseases. Prior to joining Albireo, he spent more than 25 years at Bristol-Myers Squibb, where he worked in five different countries and held positions of increasing responsibility in sales, marketing and general management, most recently as president of Europe.

Second Quarter 2016 Financial Results and Guidance

Cash Position: Cash, cash equivalents and investments as of June 30, 2016 were $86.0 million compared to $95.7 million as of March 31, 2016.
Research and Development (R&D) Expenses: R&D expenses for the quarter ending June 30, 2016 decreased $0.3 million, to $6.7 million, from the same period in 2015. The decrease was driven by the conduct of a smaller Phase 2 trial for GEN-003 in comparative quarterly periods and lower GEN-003 manufacturing costs due to the timing of activities in support of clinical trial supply. GEN-004 costs were also lower due to the Phase 2a trial which was ongoing in the second quarter of 2015 and has since been completed. These lower costs were partially offset by higher personnel and lab-related costs to advance Genocea’s preclinical product candidates and develop the ATLAS platform for immuno-oncology.
General and Administrative (G&A) Expenses: G&A expenses increased approximately $0.9 million to $4.0 million for the second quarter ending June 30, 2016 from $3.2 million for the same period in 2015. The increase was due largely to GEN-003 market research costs and higher depreciation costs from facility expansion.
Net Loss: Net loss was $11.0 million for the second quarter ended June 30, 2016, compared to a net loss of $10.3 million for the same period in 2015.
Financial Guidance: On the basis of Genocea’s current operating plans, including the planned commencement of Phase 3 trials for GEN-003 in the second half of 2017, Genocea expects that cash, cash equivalents, and investments will be sufficient to fund its operating expenses and capital expenditure requirements into the second half of 2017, assuming no receipt of proceeds from potential business development partnerships, equity financings or debt drawdowns.

On August 4, 2016 Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, reported its consolidated financial results for the second quarter 2016, and highlighted recent progress in advancing its pipeline (Press release, Alnylam, AUG 4, 2016, View Source;p=RssLanding&cat=news&id=2193147 [SID:1234514268]).

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"We continue to advance our pipeline of now ten investigational RNAi therapeutics, an entirely new and innovative class of medicines, across a broad range of diseases. We believe our two latest stage programs, patisiran and revusiran, have demonstrated encouraging progress for patients with hATTR amyloidosis," said John Maraganore, Ph.D., Chief Executive Officer of Alnylam. "We also recently presented new results in our hemophilia program showing that a once-monthly, subcutaneous regimen of fitusiran can achieve a median estimated annualized bleeding rate of zero in hemophilia A and B patients. In addition, we presented encouraging initial data in hemophilia patients with inhibitors. Through the end of the year, we are anticipating a very data rich period marked with over six planned clinical readouts – we look forward to sharing our continued progress."

Second Quarter 2016 and Recent Significant Corporate Highlights

Advanced investigational pipeline programs in Genetic Medicine Strategic Therapeutic Area (STAr).
Advanced investigational RNAi therapeutic programs for the treatment of transthyrethin (TTR)-mediated amyloidosis (ATTR amyloidosis).
Reported positive initial 24-month data from ongoing Phase 2 open-label extension (OLE) study with patisiran for the treatment of hereditary ATTR amyloidosis with polyneuropathy (hATTR-PN). Results showed that patisiran can potentially halt or improve neuropathy progression. Patisiran administration was also found to be generally well tolerated in hATTR-PN patients out to 25 months, with no drug-related serious adverse events (SAEs) reported through the data transfer date.
Presented baseline demographic data from APOLLO Phase 3 study of patisiran, revealing enrollment of a globally representative patient population with a wide range of disease severity and TTR mutations.
Reported initial 12-month results from ongoing Phase 2 OLE study with revusiran for the treatment of hereditary ATTR amyloidosis with cardiomyopathy (hATTR-CM).
Initiated Phase 1 clinical trial for ALN-TTRsc02, an Enhanced Stabilization Chemistry (ESC)-GalNAc-siRNA conjugate targeting TTR for the treatment of ATTR amyloidosis, which is expected to enable a low volume, once quarterly, subcutaneous dosing regimen.
Reported positive interim clinical results from Phase 1 study of fitusiran for the treatment of hemophilia and rare bleeding disorders (RBD).
Fitusiran achieved a median estimated annualized bleeding rate (ABR) of zero in hemophilia patients without inhibitors. In the initial low-dose cohort of patients with inhibitors, fitusiran achieved antithrombin lowering, increased thrombin generation, and preliminary evidence for reduced bleeding. Fitusiran administration was generally well tolerated in patients with and without inhibitors, with no SAEs related to study drug, and no thromboembolic events or laboratory evidence (based on D-dimer, platelet count, fibrinogen, and/or PT/INR) of pathologic clot formation through the data transfer date.
Continued dosing hemophilia patients in ongoing Phase 1/2 OLE study, with patients currently having received up to 13 months of dosing.
The Company also updated its guidance for Phase 3 initiation, and now plans to start studies in early 2017.
Reported initial clinical results in patients with paroxysmal nocturnal hemoglobinuria (PNH) from ongoing Phase 1/2 study of ALN-CC5 for the treatment of complement-mediated diseases.
Initial data support the potential for ALN-CC5 to reduce the dose and frequency of eculizumab, as well as to improve disease control in eculizumab inadequate responders.
ALN-CC5 was generally well tolerated in patients with PNH after multiple doses, with the majority of adverse events (AEs) being mild or moderate in severity. There were no drug related SAEs or discontinuations due to AEs in the study through the data transfer date.
The Company announces today that the European Medicines Agency (EMA) has granted Orphan Drug Designation to ALN-AS1 for the treatment of acute hepatic porphyrias.
The Company also announces today the publication of pre-clinical data with ALN-GO1 for the treatment of primary hyperoxaluria type 1 (PH1) in the Journal of the American Society of Nephrology (Liebow et al., J Am Soc Nephrol, 2016; doi:10.1681/ASN.2016030338).
Advanced investigational pipeline programs in Cardio-Metabolic Disease STAr.
The Medicines Company announced completion of enrollment in its Phase 2 ORION-1 study for ALN-PCSsc (also known as PCSK9si). The trial enrolled 501 patients with atherosclerotic cardiovascular disease (ASCVD), exceeding the original enrollment target of 480 patients.
Advanced investigational pipeline programs in Hepatic Infectious Disease STAr.
Initiated Phase 1/2 clinical trial with ALN-HBV for the treatment of hepatitis B virus (HBV) infection.
Broke ground on new manufacturing facility in Norton, Massachusetts. The 200,000 square foot, state-of-the-art facility is being built to support the Company’s expanding development pipeline and transition toward commercial stage.
Expanded Management Team with appointments of Adrian Dana, M.D., Vice President of Drug Safety and Pharmacovigilance; Brendan Martin, General Manager, UK and Ireland; Jeffrey Miller, Vice President, General Manager, CC5 Program; and Andrew Slugg, Vice President of Regulatory Affairs.
Upcoming Events in 2H 2016

Alnylam announces today that it plans to present clinical data at these upcoming conferences:
Complete data from Parts A and B (single and multiple ascending dose, respectively) of the ongoing Phase 1 clinical trial of ALN-AS1 in patients who are asymptomatic "high excreters" (ASHE) at the 2016 Society for the Study of Inborn Errors of Metabolism (SSIEM) Annual Symposium, being held September 6 – 9, 2016 in Rome, Italy, in an oral presentation on Wednesday, September 7, at 2:15 pm Central European Summer Time (8:15 am ET).
Initial clinical results from the ongoing Phase 1/2 study of ALN-GO1 at the 17th Congress of the International Pediatric Nephrology Association (IPNA), being held September 20 – 24, 2016 in Iguaçu, Brazil, in an oral presentation on Saturday, September 24, at 3:25 pm Brasília Time (2:25 pm ET).
Initial Phase 1/2 data for ALN-AAT at the 12th Annual Meeting of the Oligonucleotide Therapeutics Society (OTS), being held September 25 – 28, 2016 in Montreal, Canada;
Alnylam also announces today that it plans to host an R&D Day on the morning of Friday, December 16, 2016 at the Westin New York at Times Square in New York City.
In addition, in the second half of 2016, Alnylam plans to:
Complete enrollment in ENDEAVOUR Phase 3 study of revusiran;
Present additional clinical data from the fitusiran Phase 1 study and initial data from the Phase 1/2 OLE study;
Start a new Phase 2 trial with ALN-CC5 in inadequate eculizumab responder PNH patients;
Present additional clinical data from the ongoing Phase 1/2 trial of ALN-CC5 in PNH patients;
Present initial ALN-AS1 data in recurrent attack porphyria patients;
Present initial ALN-TTRsc02 Phase 1 data;
File a Clinical Trial Application for a new Genetic Medicine program; and
Consistent with guidance from The Medicines Company, present initial Phase 2 data for ALN-PCSsc.
Upcoming RNAi Roundtables

Alnylam plans to continue hosting its series of online "RNAi Roundtables" in August, September, and October. Upcoming events include:
Fitusiran for the treatment of hemophilia and rare bleeding disorders
Monday, August 22, 10:30 – 11:45 am ET
Akin Akinc, Ph.D., Vice President, General Manager, Fitusiran
Benny Sorensen, M.D., Ph.D., Senior Director, Clinical Research
Guest Speaker: Brian O’Mahony, Chief Executive, Irish Haemophilia Society Ltd. and person living with severe hemophilia B
ALN-CC5 for the treatment of complement-mediated diseases
Wednesday, August 31, 11:00 am – 12:00 pm ET
Jeff Miller, Vice President, General Manager, CC5 Program
Pushkal Garg, M.D., Senior Vice President, Clinical Development
Guest Speaker: Anita Hill, M.D., Ph.D., MRCP, FRCPath, Consultant Haematologist for Leeds Teaching Hospitals NHS Trust, UK, and Lead for the National PNH Service in England
ALN-AS1 for the treatment of acute hepatic porphyrias
Tuesday, September 13, 11:30 am – 12:45 pm ET
John Maraganore, Ph.D., Chief Executive Officer
William Querbes, Ph.D., Associate Director, Research
Guest Speaker: Ariel Lager, living with Acute Intermittent Porphyria
ALN-GO1 for the treatment of primary hyperoxaluria type 1 (PH1)
Tuesday, September 27, 10:00 – 11:00 am ET
Barry Greene, President and Chief Operating Officer
David Erbe, Ph.D., Director, Research
Guest Speaker: Sally-Anne Hulton, M.D., FRCPCH, MRCP, FCP, MBBCh, Consultant Paediatric Nephrologist and Clinical Lead, Birmingham Children’s Hospital NHS Trust
Guest Speaker: Jennifer Lawrence, M.D. (mother of George Tidmore, a PH1 patient)
ALN-HBV for the treatment of hepatitis B virus (HBV) infection
Tuesday, October 11, 9:00 am – 10:00 am ET
Barry Greene, President and Chief Operating Officer
Laura Sepp-Lorenzino, Ph.D., Vice President, Entrepreneur-in-Residence
Guest Speaker: Heiner Wedemeyer, M.D., Managing Senior Physician and Assistant Professor in the Department of Gastroenterology, Hepatology and Endocrinology at Hannover Medical School
Additional details for the RNAi Roundtables will be provided at www.alnylam.com/roundtables.

Financials

"Alnylam continues to maintain a strong balance sheet, ending the second quarter of 2016 with approximately $1.28 billion in cash," said Michael Mason, Vice President, Finance and Treasurer. "Our financial strength allows us to continue to invest in a broad pipeline of investigational RNAi therapeutics across our three STArs, aligned with achievement of our ‘Alnylam 2020′ goals. As for financial guidance this year, we remain on track to end 2016 with greater than $1.0 billion in cash, including $150.0 million in restricted investments."

Cash and Investments

At June 30, 2016, Alnylam had cash, cash equivalents and marketable securities, and restricted investments of $1.28 billion, as compared to $1.28 billion at December 31, 2015.

Credit Agreements

In April 2016, Alnylam entered into $150.0 million of term loan agreements, related to the build out of the Company’s new manufacturing facility, that mature in April 2021. Interest on the borrowings is calculated based on LIBOR plus 0.45 percent. The obligations under the term loan agreements are secured by cash collateral in an amount equal to, at any given time, at least 100 percent of the principal amount of all term loans outstanding under the agreements at such time.

GAAP Net Loss

The net loss according to accounting principles generally accepted in the U.S. (GAAP) for the second quarter of 2016 was $90.1 million, or $1.05 per share on both a basic and diluted basis (including $15.8 million, or $0.18 per share of non-cash stock-based compensation expense), as compared to a net loss of $71.8 million, or $0.85 per share on both a basic and diluted basis (including $10.2 million, or $0.12 per share of non-cash stock-based compensation expense), for the same period in the previous year.

Revenues

Revenues were $8.7 million in the second quarter of 2016 and 2015. Revenues for the second quarter of 2016 included $5.4 million from the company’s alliance with Sanofi Genzyme and $3.3 million from the company’s alliance with The Medicines Company. The company expects net revenues from collaborators to increase during the second half of 2016 due to an expected increase in expense reimbursement from Sanofi Genzyme.

Research and Development Expenses

Research and development (R&D) expenses were $83.2 million in the second quarter of 2016, which included $9.3 million of non-cash stock-based compensation, as compared to $67.0 million in the second quarter of 2015, which included $6.1 million of non-cash stock-based compensation. The increase in R&D expenses for the quarter ended June 30, 2016 as compared to the prior year period was due primarily to higher compensation and related expenses and non-cash stock-based compensation expenses resulting from a significant increase in headcount during the period as the company continues to advance and expand its development pipeline. In addition, clinical trial and manufacturing and external services expenses increased during the quarter ended June 30, 2016 as compared to the quarter ended June 30, 2015 as a result of the significant advancement of the company’s Genetic Medicine pipeline. The company expects that R&D expenses during the second half of 2016 will increase compared to the first half of 2016 as it continues to develop its pipeline and advance its product candidates into clinical trials, but that such expenses will be variable on a quarterly basis depending on the timing of manufacturing batches, clinical trial enrollment, and non-cash stock-based compensation expenses.

General and Administrative Expenses

General and administrative (G&A) expenses were $18.0 million in the second quarter of 2016, which included $6.5 million of non-cash stock-based compensation, as compared to $14.6 million in the second quarter of 2015, which included $4.0 million of non-cash stock-based compensation. The increase in G&A expenses for the quarter ended June 30, 2016 as compared to the prior year period was due primarily to an increase in compensation and related expenses and non-cash stock-based compensation expense due to an increase in headcount. The company expects that G&A expenses during the second half of 2016 will remain relatively consistent with the first half of 2016.