On November 7, 2017 Five Prime Therapeutics, Inc. (NASDAQ: FPRX), a clinical-stage biotechnology company focused on discovering and developing innovative immuno-oncology protein therapeutics, reported that it will host a conference call and live audio webcast on Wednesday, November 8, 2017, at 8:00 a.m. (ET) to review data from the Phase 1a/1b clinical trial evaluating the immunotherapy combination of its CSF-1R antibody, cabiralizumab (FPA008), with Opdivo (nivolumab), Bristol-Myers Squibb’s PD-1 immune checkpoint inhibitor. An abstract of the data has been selected for a late-breaking oral presentation on Saturday, November 11, 2017, at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) 32nd Annual Meeting in National Harbor, Maryland. The late-breaking abstracts were published today by SITC (Free SITC Whitepaper) (Press release, Five Prime Therapeutics, NOV 7, 2017, View Source [SID1234521676]).

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Five Prime originally intended to host the call and webcast on November 7, 2017, but an extended GlobeNewswire outage prevented a press release from being issued. In advance of the event tomorrow, Five Prime will file a Current Report on Form 8-K with the Securities and Exchange Commission that will include the slides to accompany the conference call presentation and discussion. Five Prime’s management will provide important additional details during the call.

The live audio webcast may be accessed through the "Events & Presentations" page in the "Investors" section of the company’s website at www.fiveprime.com. Alternatively, participants may dial (877) 878-2269 (domestic) or (253) 237-1188 (international) and refer to conference ID 8687899.

The archived conference call will be available on Five Prime’s website beginning approximately two hours after the event and will be archived and available for replay for at least 30 days after the event.

On November 7, 2017 Halozyme Therapeutics, Inc. (NASDAQ: HALO), a biotechnology company developing novel oncology and drug-delivery therapies, reported financial results and recent highlights for the third quarter ended September 30 (Press release, Halozyme, NOV 7, 2017, View Source/investors/news-releases/news-release-details/2017/Halozyme-Reports-Third-Quarter-2017-Results/default.aspx" target="_blank" title="View Source/investors/news-releases/news-release-details/2017/Halozyme-Reports-Third-Quarter-2017-Results/default.aspx" rel="nofollow">View Source [SID1234521677]).

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"During the third quarter, we realized clear benefits from strategies we have been executing against in both the ENHANZE and PEGPH20 pillars of our business," said Dr. Helen Torley, president and chief executive officer. "On ENHANZE we significantly increased 2017 revenue and the potential future value of the technology with the signing of the landmark collaboration agreement with Bristol-Myers Squibb for development of up to 11 immuno-oncology targets, and with the expansion of our collaboration agreement with Roche. In addition, our partner Janssen took an important step towards the commercialization of a subcutaneous formulation of Darzalex with the initiation of a Phase 3 study.

"In our PEGPH20 pillar, investigator interest remains strong in our HALO-301 study, resulting in continued progress with enrollment. An interim analysis will be conducted for the first primary endpoint of progression-free survival when we achieve the target number of events, which we project will occur in late Q4 2018."

Third Quarter 2017 and Recent Highlights include:

Announcing a Global Collaboration and License Agreement with Bristol-Myers Squibb to develop subcutaneously administered Bristol-Myers Squibb immuno-oncology medicines using Halozyme’s ENHANZE drug-delivery technology. The agreement is the largest in company history including a $105 million upfront payment and $160 million in potential milestones for each of 11 immuno-oncology targets, including the initial target selection of programmed death 1 (PD-1).
Roche licensing a new ENHANZE target in exchange for a $30 million upfront payment and up to $160 million in potential development, regulatory and sales-based milestones. The agreement serves as an extension to the original collaboration between the companies, under which Roche has developed two subcutaneous formulations of cancer drugs for markets worldwide.
Janssen initiating the first of three planned Phase 3 studies of the subcutaneous formulation of DARZALEX (daratumumab). Halozyme’s ENHANZE technology has the potential to enable a 15 ml injection to be delivered in five minutes or less, with no requirement for an intravenous loading dose. Data informing this decision from the Phase 1 PAVO study in patients with relapsed or refractory multiple myeloma were accepted for presentation at the 2017 American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition. Upon the dosing of the third patient in the recently initiated study, Halozyme will earn a $15 million milestone payment.
Genentech launching RITUXAN HYCELA (rituximab/hyaluronidase human) for subcutaneous injection, a combination of rituximab and Halozyme’s hyaluronidase human ENHANZE technology, for patients with follicular lymphoma, diffuse large B-cell lymphoma and chronic lymphocytic leukemia.
Continued progress screening and enrolling patients in the HALO-301 study of PEGPH20 in combination with ABRAXANE (nab-paclitaxel) and gemcitabine in first line metastatic pancreas cancer patients with high levels of tumor hyaluronan (HA-High). An interim analysis will be conducted for the first primary endpoint of progression-free survival when the target number of events has been reached, which the company projects will be in late Q4 2018. At that time, Halozyme projects approximately 500 patients will have been enrolled in the study.
Initiating multiple trials in collaboration with Genentech to evaluate PEGPH20 in combination with TECENTRIQ (atezolizumab) in four tumor types. Studies include a Halozyme-sponsored randomized clinical trial in patients with previously untreated, unresectable, locally advanced, or metastatic cholangiocarcinoma and gallbladder adenocarcinoma and two Genentech-funded and operated, Phase 1b/2 multi-arm clinical studies evaluating patients with previously treated metastatic pancreatic ductal adenocarcinoma and previously treated locally advanced unresectable or metastatic gastric cancer. The studies are part of a clinical collaboration agreement announced in 2016 to evaluate PEGPH20 and atezolizumab in up to eight tumor types.
Third Quarter 2017 Financial Highlights

Revenue for the third quarter was $63.7 million compared to $31.9 million for the third quarter of 2016. The year-over-year increase was driven by a $30 million upfront payment from Roche and growth in royalties from partner sales of Herceptin (trastuzumab) SC, MabThera (rituximab) SC and HYQVIA (Immune Globulin Infusion 10% (Human) with Recombinant Human Hyaluronidase) offset by a decrease in research and development reimbursements and license payments from ENHANZE partners. Revenue for the third quarter included $17.1 million in royalties, an increase of 31 percent from the prior-year period, $9.8 million in sales of bulk rHuPH20 primarily for use in manufacturing collaboration products and $3.8 million in HYLENEX recombinant (hyaluronidase human injection) product sales.
Research and development expenses for the third quarter were $34.0 million, compared to $33.9 million for the third quarter of 2016. The increase was primarily due to a ramp in spending associated with the HALO-301 study.
Selling, general and administrative expenses for the third quarter were $13.3 million, compared to $11.6 million for the third quarter of 2016. The increase was primarily due to personnel expenses, including stock compensation, for the period.
Net income for the third quarter was $2.7 million, or $0.02 per share, compared to net loss in the third quarter of 2016 of $28.9 million, or $0.23 per share.
Cash, cash equivalents and marketable securities were $316.9 million at September 30, 2017, compared to $297.5 million at June 30, 2017.
Financial Outlook for 2017

Halozyme updated year-end guidance, now expecting:

Net revenue increasing from the prior range of $245 million to $260 million announced on Sept. 14 to $265 million to $280 million, driven by stronger product sales, royalties, and sponsored research;
Operating expenses decreasing from the prior range of $240 million to $250 million to $230 million to $240 million;
Positive operating cash flow increasing from the prior range of $50 million to $60 million to $70 million to $85 million;
Year-end cash balance increasing from the prior range of $380 million to $395 million to $400 million to $415 million.
Webcast and Conference Call

Halozyme will webcast its Quarterly Update Conference Call for the third quarter of 2017 today, Tuesday, November 7 at 4:30 p.m. ET/1:30 p.m. PT. Dr. Helen Torley, president and chief executive officer, will lead the call. The call will be webcast live through the "Investors" section of Halozyme’s corporate website and a recording will be made available following the close of the call. To access the webcast and additional documents related to the call, please visit View Source approximately fifteen minutes prior to the call to register, download and install any necessary audio software. The call may also be accessed at (877) 410-5657 (domestic callers) (334) 323-7224 (international callers) using passcode 769890. A telephone replay will be available after the call by dialing (877) 919-4059 (domestic callers) or (334) 323-0140 (international callers) using replay ID number 19320711.

On November 7, 2017 Jazz Pharmaceuticals plc (Nasdaq: JAZZ) reported financial results for the third quarter of 2017 and updated financial guidance for 2017 (Press release, Jazz Pharmaceuticals, NOV 7, 2017, View Source;p=RssLanding&cat=news&id=2315108 [SID1234521680]).

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"The third quarter of 2017 was highlighted by the approval and strong launch of Vyxeos in the U.S. for the treatment of adult patients with newly-diagnosed high-risk AML, leading to our increase in 2017 Vyxeos sales guidance," said Bruce Cozadd, chairman and chief executive officer of Jazz Pharmaceuticals. "While Xyrem has experienced lower than expected growth in 2017, and we are slightly decreasing our annual sales guidance accordingly, we remain confident in our ability to address the key drivers impacting Xyrem to position the product for solid future growth."

GAAP net income for the third quarter of 2017 was $63.5 million, or $1.03 per diluted share, compared to $89.8 million, or $1.45 per diluted share, for the third quarter of 2016. GAAP net income for the third quarter of 2017 included an upfront payment of $75.0 million to ImmunoGen, Inc. related to a collaboration and option agreement.

Adjusted net income for the third quarter of 2017 was $197.6 million, or $3.22 per diluted share, compared to $161.2 million, or $2.61 per diluted share, for the third quarter of 2016.

The tax provision and the effective tax rate for the third quarter of 2017 on both a GAAP and non-GAAP basis were favorably impacted by certain tax benefits. For further information, see "Operating Expenses and Income Tax Provision" below. Reconciliations of applicable GAAP reported to non-GAAP adjusted information are included at the end of this press release.

Financial Highlights

Three Months Ended
September 30,

Nine Months Ended
September 30,

(In thousands, except per share amounts and percentages)
2017

2016

Change

2017

2016

Change
Total revenues
$
411,855

$
374,181

10
%

$
1,182,294

$
1,091,352

8
%
GAAP net income
$
63,526

$
89,828

(29)
%

$
255,641

$
280,142

(9)
%
Adjusted net income
$
197,649

$
161,153

23
%

$
496,225

$
461,525

8
%
GAAP EPS
$
1.03

$
1.45

(29)
%

$
4.17

$
4.51

(8)
%
Adjusted EPS
$
3.22

$
2.61

23
%

$
8.09

$
7.43

9
%
Total Revenues

Three Months Ended
September 30,

Nine Months Ended
September 30,
(In thousands)
2017

2016

2017

2016
Xyrem (sodium oxybate) oral solution
$
303,870

$
285,907

$
874,222

$
816,412

Erwinaze / Erwinase (asparaginase Erwinia chrysanthemi)
49,173

42,986

149,585

143,907

Defitelio (defibrotide sodium) / defibrotide
31,213

28,137

97,351

79,280

VyxeosTM (daunorubicin and cytarabine) liposome for injection
9,719

—

9,719

—

Prialt (ziconotide) intrathecal infusion
7,930

8,783

21,303

23,065

Other
6,066

5,808

19,124

21,983

Product sales, net
407,971

371,621

1,171,304

1,084,647

Royalties and contract revenues
3,884

2,560

10,990

6,705

Total revenues
$
411,855

$
374,181

$
1,182,294

$
1,091,352

Net product sales increased 10% in the third quarter of 2017 compared to the same period in 2016 primarily due to an increase in net product sales of our lead marketed products.

Xyrem net product sales increased 6% in the third quarter of 2017 compared to the same period in 2016. Xyrem net product sales growth in the 2017 period was negatively impacted by payer mix, one fewer shipping day, and operational changes that delayed some prescription fulfillment.

Erwinaze/Erwinase net product sales increased 14% in the third quarter of 2017 compared to the same period in 2016. The company experienced supply disruptions during both periods; however, net product sales were higher in the third quarter of 2017 compared to the same period in 2016 due to the timing of product availability. The company expects that additional supply disruptions may occur in 2017 and into 2018.

Defitelio/defibrotide net product sales increased 11% in the third quarter of 2017 compared to the same period in 2016 primarily due to an increase in U.S. net product sales. The company expects continued inter-quarter variability in Defitelio net sales given that veno-occlusive disease is an ultra-rare disease.

Vyxeos net product sales in the third quarter of 2017 were $9.7 million. Vyxeos launched in the U.S. on August 11, 2017.

Operating Expenses and Income Tax Provision

Three Months Ended
September 30,

Nine Months Ended
September 30,
(In thousands, except percentages)
2017

2016

2017

2016
GAAP:

Cost of product sales
$
31,203

$
24,311

$
84,940

$
71,730

Gross margin
92.4
%

93.5
%

92.7
%

93.4
%
Selling, general and administrative
$
124,523

$
124,368

$
401,106

$
375,751

% of total revenues
30.2
%

33.2
%

33.9
%

34.4
%
Research and development
$
47,362

$
47,796

$
132,447

$
118,139

% of total revenues
11.5
%

12.8
%

11.2
%

10.8
%
Acquired in-process research and development
$
75,000

$
15,000

$
77,000

$
23,750

Income tax provision
$
1,239

$
26,437

$
65,914

$
100,888

Effective tax rate
1.9
%

22.7
%

20.5
%

26.5
%

Three Months Ended
September 30,

Nine Months Ended
September 30,
(In thousands, except percentages)
2017

2016

2017

2016
Non-GAAP adjusted:

Cost of product sales
$
29,630

$
22,963

$
80,594

$
68,620

Gross margin
92.7
%

93.8
%

93.1
%

93.7
%
Selling, general and administrative
$
103,620

$
94,534

$
333,524

$
296,633

% of total revenues
25.2
%

25.3
%

28.2
%

27.2
%
Research and development
$
42,712

$
43,323

$
118,796

$
106,847

% of total revenues
10.4
%

11.6
%

10.0
%

9.8
%
Income tax provision
$
24,410

$
38,500

$
104,307

$
129,663

Effective tax rate
11.0
%

19.3
%

17.4
%

21.9
%
Operating expenses changed over the prior year period primarily due to the following:

Selling, general and administrative (SG&A) expenses increased in the third quarter of 2017 compared to the same period in 2016 on a GAAP and on a non-GAAP adjusted basis due to higher headcount and other expenses resulting from the expansion of the company’s business, including the launch of Vyxeos in the U.S. SG&A expenses in the third quarter of 2016 on a GAAP basis included transaction and integration costs of $10.3 million.
Research and development (R&D) expenses were consistent on a GAAP and on a non-GAAP adjusted basis in the third quarter of 2017 compared to the same period in 2016. R&D expenses in the third quarter of 2017 reflected an increase in expenses related to the company’s ongoing clinical development programs and regulatory activities, including an increase in headcount, and a decrease in JZP-110 costs following the completion of three Phase 3 studies this year.
The tax provision and the effective tax rate for the third quarter of 2017 on both a GAAP and non-GAAP basis were favorably impacted by the release of a valuation allowance held against certain foreign net operating losses and the release of reserves related to uncertain tax positions upon the expiration of a statute of limitation.

Cash Flow and Balance Sheet
As of September 30, 2017, cash, cash equivalents and investments were $452.6 million, and the outstanding principal balance of the company’s long-term debt was $1.8 billion. In the third quarter of 2017, the company sold $575.0 million aggregate principal amount of 1.50% exchangeable senior notes due 2024 and used the net proceeds to repay $500.0 million of outstanding borrowings under the company’s revolving credit facility. During the nine months ended September 30, 2017, the company repaid a total of $850.0 million of borrowings under the company’s revolving credit facility, made an upfront payment of $75.0 million to ImmunoGen, Inc. and used $56.4 million to repurchase approximately 398,000 ordinary shares under the company’s share repurchase program at an average cost of $141.73 per ordinary share.

Recent Developments

In August 2017, the company and ImmunoGen, Inc. entered into a collaboration and option agreement granting the company rights to opt into exclusive, worldwide licenses to develop and commercialize two early-stage, hematology-related antibody-drug conjugate (ADC) programs, as well as an additional program to be designated during the term of the agreement. The programs covered under the agreement include IMGN779, a CD33-targeted ADC for the treatment of acute myeloid leukemia (AML) in Phase 1 testing, and IMGN632, a CD123-targeted ADC for hematological malignancies expected to enter clinical testing before the end of the year.
In November 2017, the company submitted a Marketing Authorization Application (MAA) for Vyxeos to the European Medicines Authority (EMA) for the treatment of high-risk AML patients. Separately, the EMA granted Vyxeos an accelerated assessment review and the UK Medicines and Healthcare Products Regulatory Agency granted Vyxeos the Promising Innovative Medicine designation.
2017 Financial Guidance
Jazz Pharmaceuticals is updating its full year 2017 financial guidance as follows (in millions, except per share amounts and percentages):

Revenues
$1,600-$1,650
Total net product sales
$1,590-$1,630
-Xyrem net sales
$1,180-$1,200
-Erwinaze/Erwinase net sales
$200-$215
-Defitelio/defibrotide net sales
$130-$150
-Vyxeos net sales
$20-$30
GAAP gross margin %
93%
Non-GAAP adjusted gross margin %1,4
93%
GAAP SG&A expenses
$521-$551
Non-GAAP adjusted SG&A expenses2,4
$440-$460
GAAP R&D expenses
$180-$200
Non-GAAP adjusted R&D expenses3,4
$165-$180
GAAP net income per diluted share
$5.30-$6.30
Non-GAAP adjusted net income per diluted share4
$10.70-$11.20

1.
Excludes $5 million of share-based compensation expense from estimated GAAP gross margin.
2.
Excludes $75-$85 million of share-based compensation expense and $6 million of expenses related to certain legal proceedings and restructuring from estimated GAAP SG&A expenses.
3.
Excludes $15-$20 million of share-based compensation expense from estimated GAAP R&D expenses.
4.
See "Non-GAAP Financial Measures" below. Reconciliations of non-GAAP adjusted guidance measures are included above and in the table titled "Reconciliation of GAAP to Non-GAAP Adjusted 2017 Net Income Guidance" at the end of this press release.
Conference Call Details
Jazz Pharmaceuticals will host an investor conference call and live audio webcast today at 4:30 p.m. EST (9:30 p.m. GMT) to provide a business and financial update and discuss its 2017 third quarter results. The live webcast may be accessed from the Investors section of the company’s website at www.jazzpharmaceuticals.com. Please connect to the website prior to the start of the conference call to ensure adequate time for any software downloads that may be necessary. Investors may participate in the conference call by dialing +1 855 353 7924 in the U.S., or +1 503 343 6056 outside the U.S., and entering passcode 95499424.

A replay of the conference call will be available through November 14, 2017 by dialing +1 855 859 2056 in the U.S., or +1 404 537 3406 outside the U.S., and entering passcode 95499424. An archived version of the webcast will be available for at least one week in the Investors section of the company’s website at www.jazzpharmaceuticals.com.

On November 7, 2017 Kura Oncology, Inc., (Nasdaq:KURA) a clinical-stage biopharmaceutical company focused on the development of precision medicines for oncology, reported third quarter 2017 financial results and provided a corporate update (Press release, Kura Oncology, NOV 7, 2017, View Source;p=RssLanding&cat=news&id=2315104 [SID1234521681]).

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"Over the last several months, Kura has continued to deliver important advances against our pipeline priorities," said Troy Wilson, Ph.D., J.D., President and CEO of Kura Oncology. "We recently achieved a key milestone with positive Phase 2 results for our lead product candidate, tipifarnib, to treat relapsed or refractory head and neck squamous cell carcinomas (HNSCC) with HRAS mutations. This positive study validates the potential of our precision medicine approach to generate evidence of clinical activity in a well-defined, difficult-to-treat patient population that we believe justifies continued and rapid drug development. Based on these very encouraging clinical results and, subject to further input from regulatory authorities, we plan to initiate a registration-enabling study of tipifarnib in HRAS mutant HNSCC in 2018.

"In addition, we continue to evaluate tipifarnib in our ongoing Phase 2 trials in peripheral T-cell lymphoma (PTCL), myelodysplastic syndromes (MDS) and chronic myelomonocytic leukemia (CMML), as well as advance our pipeline programs, KO-947 and KO-539, and we look forward to providing additional updates on these programs over the next several quarters. Finally, we expect the proceeds from our capital raise in August, coupled with our existing cash on hand, will provide the resources necessary to support our pipeline of precision medicines through a series of significant milestones anticipated in 2018."

Recent Operational Highlights

Positive Phase 2 study of tipifarnib in HRAS mutant HNSCC – In September, Kura announced positive topline results from a Phase 2 trial of tipifarnib in patients with HRAS mutant HNSCC. The trial achieved its primary endpoint prior to the completion of patient enrollment. Updated results from this study presented at the AACR (Free AACR Whitepaper)-NCI-EORTC International Conference in October showed that confirmed partial responses were observed in four out of six patients with HRAS mutant HNSCC, and tipifarnib demonstrated rapid and durable responses, with partial responses observed beyond one year in duration.

KO-539 shows robust anti-tumor activity in preclinical models of AML – In October, Kura presented preclinical data for KO-539, an inhibitor of the menin-MLL interaction, which supports the potential clinical utility of KO-539 in NPM1- and DNMT3A-mutant acute myeloid leukemia (AML). The data, presented at the AACR (Free AACR Whitepaper)-NCI-EORTC International Conference, suggest KO-539 drives robust and persistent responses in preclinical models of AML and has the potential to be active in subtypes representing approximately half of patients with AML.

Completed follow-on offering – In August, Kura completed an underwritten public offering resulting in net proceeds to the company of approximately $53.5 million.
Upcoming Potential Milestones and Expectations for Clinical Programs

Presentation of preliminary results from the ongoing Phase 2 trial of tipifarnib in CMML at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting in December 2017

Presentation on the CXCL12/CXCR4 Pathway as a potential target of tipifarnib in AML and MDS at ASH (Free ASH Whitepaper) in December 2017

Presentation on the CXCL12/CXCR4 Pathway as a potential target of tipifarnib from the ongoing Phase 2 trial of tipifarnib in PTCL at ASH (Free ASH Whitepaper) in December 2017

Presentation of updated results from the Phase 2 trial of tipifarnib in HRAS mutant HNSCC at the Multidisciplinary Head and Neck Cancers Symposium in February 2018

Additional data from the Phase 2 trials of tipifarnib in PTCL, CMML and MDS in 2018

Data from the KO-947 Phase 1 trial in non-hematological malignancies in 2018

Initiation of a registration-enabling trial of tipifarnib in HRAS mutant HNSCC in 2018
Financial Results for the Third Quarter 2017

Cash, cash equivalents and short-term investments totaled $100.8 million as of September 30, 2017, compared with $53.2 million as of June 30, 2017. Cash, cash equivalents and short-term investments at the end of September includes net proceeds of approximately $53.5 million from the follow-on offering in August. Management expects that current cash, cash equivalents and short-term investments will be sufficient to fund current operations into 2019.

Research and development expenses for the third quarter of 2017 were $7.1 million, compared to $5.3 million for the third quarter of 2016.

General and administrative expenses for the third quarter of 2017 were $2.4 million, compared to $1.7 million for the third quarter of 2016.

Net loss for the third quarter of 2017 was $9.3 million, or $0.38 per share, compared to a net loss of $6.9 million, or $0.37 per share, for the third quarter of 2016.
Conference Call and Webcast

Kura’s management will host a webcast and conference call regarding this announcement at 1:30 p.m. PT/4:30 p.m. ET today. The live call may be accessed by dialing (877) 516-3514 for domestic callers and (281) 973-6129 for international callers and using conference ID # 2685708. A live webcast of the call will be available from the investor relations section of the company website at www.kuraoncology.com, and will be archived there for 30 days. A telephone replay of the call will be available by dialing (855) 859-2056 for domestic callers, or (404) 537-3406 for international callers, and entering the conference ID # 2685708.

On November 7, 2017 Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, reported its consolidated financial results for the third quarter 2017, and highlighted recent progress in advancing its pipeline (Press release, Alnylam, NOV 7, 2017, View Source [SID1234521669]).

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"In our view, 2017 has proven to be a remarkable year for RNAi therapeutics, for Alnylam, and, most importantly, for the patients that we serve. With patisiran, our recent APOLLO Phase 3 study results demonstrate what we believe to be the transformative potential for RNAi therapeutics as a new class of innovative medicines. With these data, we expect to submit our first regulatory filings in the coming months, and are planning for the possibility of having regulatory approval for patisiran in mid-2018," said John Maraganore, Ph.D., Chief Executive Officer of Alnylam. "We have also made significant progress across our other programs, including the initiation of our ENVISION Phase 3 program for givosiran in acute hepatic porphyrias and, with our partners at The Medicines Company, the ORION Phase 3 program in hypercholesterolemia. Additionally, we aim to resume dosing in all fitusiran studies, including the ATLAS Phase 3 program, as soon as possible. These milestones position Alnylam with multiple late-stage clinical assets, while we concurrently transition into a fully integrated commercial company with the goal of delivering innovative medicines to patients around the world."

Third Quarter 2017 and Recent Significant Corporate Highlights

Advanced patisiran, an investigational RNAi therapeutic in development for the treatment of patients with hereditary ATTR (hATTR) amyloidosis, with positive results from the APOLLO Phase 3 study (N=225).
Patisiran met its primary endpoint (p = 9.26 x 10-24) with a 34.0 point mean difference relative to placebo and a negative 6.0 point mean change (improvement) relative to baseline in the modified neuropathy impairment score (mNIS+7) at 18 months, as well as all secondary endpoints (p less than 0.001), including a 21.1 point mean difference relative to placebo and a negative 6.7 point mean change (improvement) relative to baseline in the NORFOLK Quality of Life-Diabetic Neuropathy score (NORFOLK QOL-DN) (p = 1.10 x 10-10), at 18 months.
Patisiran also demonstrated a favorable safety and tolerability profile relative to placebo. The most commonly reported adverse events (AEs) for patisiran were generally mild to moderate and included peripheral edema (29.7 percent) and infusion-related reactions (IRRs) (18.9 percent), and the frequency of deaths and serious adverse events (SAEs) was similar in the patisiran and placebo groups. No deaths were considered drug-related.
Specifically, the Company believes that these data support a potentially "best-in-class" product profile, with significant benefit relative to placebo, negative mean and median values (improvement) for mNIS+7 and QOL measures relative to baseline, and encouraging safety and tolerability.
In addition, patisiran achieved significant effects in the study’s cardiac subpopulation, including on disease biomarker, echocardiographic, and functional parameters.
The Company believes the totality of the APOLLO data are consistent with a clinically meaningful impact for patisiran on hATTR amyloidosis, and plans to submit an NDA for patisiran by the end of 2017 and a Marketing Authorisation Application (MAA) shortly thereafter.
Advanced ALN-TTRsc02, a subcutaneously administered investigational RNAi therapeutic in development for the treatment of ATTR amyloidosis.
Presented updated Phase 1 data showing up to 95% transthyretin (TTR) knockdown with a single 50 mg dose, with durability supportive of a once quarterly and, possibly, bi-annual subcutaneous dose regimen.
Reaffirmed guidance to initiate a Phase 3 program for ALN-TTRsc02 in 2018.
Advanced givosiran, an investigational RNAi therapeutic in development for the treatment of acute hepatic porphyrias (AHPs), with initiation of the ENVISION Phase 3 study.
The Company reached alignment with the U.S. Food and Drug Administration (FDA) on the design of ENVISION, including an interim analysis based on reduction of urinary aminolevulinic acid (ALA), a biomarker that the FDA considers to be reasonably likely to predict clinical benefit.
The Company has also reached alignment on the ENVISION Phase 3 study design with the European Medicines Agency (EMA).
The Company is guiding that it expects interim analysis results in mid-2018 and, pending FDA review of the program at the time of interim analysis and assuming positive results, it expects to submit an NDA at or around year-end 2018.
Advanced fitusiran, an investigational RNAi therapeutic in development for the treatment of hemophilia A and B with or without inhibitors, with new positive data from the Phase 2 open-label extension (OLE) study presented at the International Society on Thrombosis and Haemostasis 2017 Congress.
Results from the Phase 1 study were published in The New England Journal of Medicine in a paper titled, "Targeting of Antithrombin in Hemophilia A or B with RNAi Therapy."
Announced the initiation of the ATLAS Phase 3 program, a global, multicenter clinical program designed to evaluate the safety and efficacy of fitusiran in patients with hemophilia A and B with or without inhibitors.
The Company temporarily suspended dosing in all ongoing studies of fitusiran following the observation of a fatal thrombotic SAE that occurred in a patient with hemophilia A without inhibitors who was receiving fitusiran in the Phase 2 OLE study. Alnylam and fitusiran study investigators have aligned on a risk management plan for further advancement of fitusiran and are now conferring with global regulators with the goal of resuming dosing as soon as possible, potentially by the end of 2017.
Alnylam and The Medicines Company announced initiation of the ORION-11 Phase 3 study of inclisiran, an investigational RNAi therapeutic targeting PCSK9 in development for the treatment of hypercholesterolemia, in patients with atherosclerotic cardiovascular disease (ASCVD).
The companies announced new positive data from the ORION-1 Phase 2 study of inclisiran at the European Society of Cardiology Congress 2017.
Advanced cemdisiran (formerly known as ALN-CC5), a subcutaneously administered investigational RNAi therapeutic targeting complement component C5 for the treatment of complement-mediated diseases, with the initiation of a Phase 2 clinical study in patients with atypical hemolytic-uremic syndrome (aHUS).
Advanced lumasiran (formerly known as ALN-GO1), an investigational RNAi therapeutic in development for the treatment of primary hyperoxaluria type 1 (PH1), with new positive data from the Phase 1/2 study presented at the American Society of Nephrology Kidney Week 2017 Annual Meeting.
Alnylam announced a licensing agreement with Vir Biotechnology for the development and commercialization of RNAi therapeutics for infectious diseases, including hepatitis B.
Advanced RNAi platform technology with new pre-clinical data on Alnylam’s next generation "Enhanced Stabilization Chemistry Plus" (ESC+) GalNAc-siRNA conjugate platform presented at the 13th Annual Meeting of the Oligonucleotide Therapeutics Society.
Upcoming Events

Alnylam announces today that Alnylam scientists and collaborators will present new results from multiple pipeline programs at the 59th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting, being held December 9 – 12, 2017 in Atlanta, Georgia. Presentations include:
Explore: A Prospective, Multinational History Study of Patients with Acute Hepatic Porphyrias (AHP) with Recurrent Attacks
Session: 102. Regulation of Iron Metabolism: Poster II
Date/Time: Sunday, December 10, 6:00 – 8:00 p.m. ET
Perioperative Management in Patients with Hemophilia Receiving Fitusiran, an Investigational RNAi Therapeutic Targeting Antithrombin for the Treatment of Hemophilia
Session: 322. Disorders of Coagulation or Fibrinolysis: Poster II
Date/Time: Sunday, December 10, 6:00 – 8:00 p.m. ET
In Silico Modeling of the Impact of Antithrombin Lowering on Thrombin Generation in Rare Bleeding Disorders
Session: 321. Blood Coagulation and Fibrinolytic Factors: Poster III
Date/Time: Monday, December 11, 6:00 – 8:00 p.m. ET
Alnylam plans to file its first NDA for patisiran with the FDA by the end of 2017, followed by an MAA in the EU in early 2018.
Alnylam plans to meet with global regulatory authorities with the goal of reaching agreement on a risk mitigation plan and resumption of dosing in fitusiran clinical studies.
Financial results for the quarter ended September 30, 2017

"Alnylam’s strong balance sheet and overall financial position allow us to build our commercial capabilities in preparation for anticipated product launches in the U.S. and Western Europe during 2018, assuming regulatory approvals in the U.S. and EU," said Manmeet Soni, Chief Financial Officer of Alnylam. "Additionally, we continue to invest in our broad pipeline of investigational RNAi therapeutics, advancing our four late-stage programs as well as the early-stage pipeline programs."

Cash and Investments
At September 30, 2017, Alnylam had cash, cash equivalents and fixed income marketable securities, and restricted investments of $1.15 billion, as compared to $1.09 billion at December 31, 2016.

GAAP and Non-GAAP Net Loss
The net loss according to accounting principles generally accepted in the U.S. (GAAP) for the third quarter of 2017 was $122.9 million, or $1.34 per share on both a basic and diluted basis, as compared to a net loss of $104.1 million, or $1.21 per share on both a basic and diluted basis, for the same period in the previous year.

The non-GAAP net loss for the third quarter of 2017 was $97.0 million, or $1.06 per share on both a basic and diluted basis, as compared to a non-GAAP net loss of $88.5 million, or $1.03 per share on both a basic and diluted basis for the same period in the previous year.

The non-GAAP net loss excludes stock-based compensation expense. See "Use of Non-GAAP Financial Measures" below for a description of non-GAAP financial measures and a reconciliation between GAAP and non-GAAP net loss appearing later in this press release.

Revenues
Revenues were $17.1 million in the third quarter of 2017, as compared to $13.7 million in the third quarter of 2016. Revenues for the third quarter of 2017 included $14.6 million from the Company’s alliance with Sanofi Genzyme, $2.3 million from the Company’s alliance with The Medicines Company and $0.2 million from other sources.

GAAP and Non-GAAP Research and Development Expenses
GAAP research and development (R&D) expenses were $95.3 million in the third quarter of 2017 as compared to $97.9 million in the third quarter of 2016.

Non-GAAP R&D expenses were $80.2 million in the third quarter of 2017 as compared to $88.6 million in the third quarter of 2016. Non-GAAP R&D expenses exclude stock-based compensation expense. A reconciliation between GAAP and non-GAAP R&D expenses appears later in this press release.

GAAP and Non-GAAP General and Administrative Expenses
GAAP general and administrative (G&A) expenses were $47.6 million in the third quarter of 2017 as compared to $22.4 million in the third quarter of 2016.

Non-GAAP G&A expenses were $36.8 million in the third quarter of 2017 as compared to $16.2 million in the third quarter of 2016. Non-GAAP G&A expenses exclude stock-based compensation expense. A reconciliation between GAAP and non-GAAP G&A expenses appears later in this press release.

Financial Guidance
Alnylam remains on track to end 2017 with greater than $1.0 billion in cash, cash equivalents and fixed income marketable securities including $150.0 million in restricted investments.

Conference Call Information
Management will provide an update on the Company and discuss third quarter 2017 results as well as expectations for the future via conference call on Tuesday, November 7, 2017 at 4:30 p.m. ET. To access the call, please dial 877-312-7507 (domestic) or 631-813-4828 (international) five minutes prior to the start time and refer to conference ID 2432127. A replay of the call will be available beginning at 7:30 p.m. ET on the day of the call. To access the replay, please dial 855-859-2056 (domestic) or 404-537-3406 (international), and refer to conference ID 2432127.

Alnylam – Sanofi Genzyme Alliance
In January 2014, Alnylam and Sanofi Genzyme, the specialty care global business unit of Sanofi, formed an alliance to accelerate the advancement of RNAi therapeutics as a potential new class of innovative medicines for patients around the world with rare genetic diseases. The alliance enables Sanofi Genzyme to expand its rare disease pipeline with Alnylam’s novel RNAi technology and provides access to Alnylam’s R&D engine, while Alnylam benefits from Sanofi Genzyme’s proven global capabilities to advance late-stage development and, upon commercialization, accelerate market access for these promising genetic medicine products.

In the case of patisiran, Alnylam will advance the product in the United States, Canada and Western Europe, while Sanofi Genzyme will advance the product in the rest of the world. In November 2016, Sanofi Genzyme elected to co-develop (through Sanofi R&D) and co-commercialize fitusiran in the United States, Canada and Western Europe, in addition to commercializing fitusiran in its rest of world territories. Sanofi Genzyme has the right to opt in to develop and commercialize lumasiran in territories outside of the United States, Canada and Western Europe and could elect to exercise its one right to a global license for lumasiran. In the case of ALN-TTRsc02, Sanofi Genzyme has the right to opt into the program with co-development/co-commercialization rights.

About RNAi
RNAi (RNA interference) is a natural cellular process of gene silencing that represents one of the most promising and rapidly advancing frontiers in biology and drug development today. Its discovery has been heralded as "a major scientific breakthrough that happens once every decade or so," and was recognized with the award of the 2006 Nobel Prize for Physiology or Medicine. By harnessing the natural biological process of RNAi occurring in our cells, a major new class of medicines, known as RNAi therapeutics, is on the horizon. Small interfering RNA (siRNA), the molecules that mediate RNAi and comprise Alnylam’s RNAi therapeutic platform, function upstream of today’s medicines by potently silencing messenger RNA (mRNA) – the genetic precursors – that encode for disease-causing proteins, thus preventing them from being made. This is a revolutionary approach with the potential to transform the care of patients with genetic and other diseases.

About LNP Technology
Alnylam has licenses to Arbutus LNP intellectual property for use in RNAi therapeutic products using LNP technology.