Shire delivers strong Q1 2016 results with double-digit growth in revenue and Non GAAP earnings per ADS

On April 29, 2016 Shire plc ("Shire") (LSE: SHP, NASDAQ: SHPG) reported unaudited results for the three months ended March 31, 2016 (Press release, Shire, APR 29, 2016, View Source [SID:1234511736]).

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Financial Highlights Q1 2016 Growth(1) Non GAAP CER(1)(2)
Product sales $1,627 million +14% +16%
Total revenues $1,709 million +15% +17%

Non GAAP operating income $797 million +17% +16%
US GAAP operating income from continuing operations $544 million +15%

Non GAAP EBITDA margin (excluding royalties & other revenues)(3) 46% 0pps(4)
US GAAP net income margin(5) 25% -3pps

Non GAAP net income $632 million +13%
US GAAP net income $419 million +2%

Non GAAP diluted earnings per ADS $3.19 +12% +12%
US GAAP diluted earnings per ADS $2.12 +2%

Non GAAP cash generation $492 million -5%
Non GAAP free cash flow $338 million +38%
US GAAP net cash provided by operating activities $390 million -31%
(1) Percentages compare to equivalent 2015 period.
(2) On a Constant Exchange Rate ("CER") basis, which is a Non GAAP measure.
(3) Non GAAP earnings before interest, tax, depreciation and amortization ("EBITDA") as a percentage of product sales, excluding royalties and other revenues.
(4) Percentage point change ("pps").
(5) US GAAP net income as a percentage of total revenues.

The Non GAAP financial measures included within this release are explained on pages 25 – 26, and are reconciled to the most directly comparable financial measures prepared in accordance with US GAAP on pages 19 – 22.

First Quarter & Recent Highlights:

Product sales growth of 14% (16% on a Non GAAP CER basis) to $1.6 billion, driven by VYVANSE, LIALDA/MEZAVANT, CINRYZE, FIRAZYR, GATTEX/REVESTIVE and NATPARA.
Rare disease products acquired from NPS Pharmaceuticals, Inc. ("NPS") continued to perform well with GATTEX/REVESTIVE sales up 247% (up 97% on a pro-forma basis(1)) to $52 million, and NATPARA sales of $16 million.
Free cash flow remained strong, impacted primarily by net payments and receipts of taxes between Q1 2015 and Q1 2016.
Lifitegrast New Drug Application ("NDA") accepted by the US Food and Drug Administration ("FDA"), with Prescription Drug User Fee Act ("PDUFA") date set for July 22, 2016.
Pipeline progression with positive topline results from SHP465 safety and efficacy study in children and adolescents with Attention Deficit Hyperactivity Disorder ("ADHD").
Completed acquisition of Dyax Corp. ("Dyax") and enrollment on track for SHP643 (formerly DX2930) Phase 3 studies for the treatment of Hereditary Angioedema ("HAE").
Patent upheld for LIALDA (mesalamine) delayed release tablets by U.S. District Court for the Southern District of Florida; the case has been appealed.
Baxalta Incorporated ("Baxalta") acquisition on track with integration progressing well; shareholder votes set for May 27 and closing anticipated in early June.
(1) Sales prior to February 21, 2015 were recorded by NPS.
Flemming Ornskov, M.D. Chief Executive Officer, commented:

"Shire is off to a strong start in 2016, delivering double-digit product sales and Non GAAP earnings per ADS growth, and advancing our innovative pipeline. We were pleased to report positive Phase 3 topline results for SHP465 in children and adolescents with ADHD, a therapeutic area with significant need for additional treatment options. We are also looking forward to hearing from the FDA by late July regarding lifitegrast, a potential new treatment for dry eye disease.

While we maintain our sharp focus on Shire’s business, we closed the acquisition of Dyax during the quarter and we are making excellent progress with the Baxalta integration planning. Our shareholder vote is scheduled for May 27 and the closing is anticipated to follow in early June. We look forward to officially welcoming our Baxalta colleagues to Shire, and creating a global biotechnology leader focused on rare diseases and other highly specialized conditions."

European Medicines Agency Issues Positive Opinion for the Use of IMBRUVICA® (ibrutinib) as a First-Line Treatment for Chronic Lymphocytic Leukemia Patients

On April 29, 2016 AbbVie (NYSE: ABBV), a global biopharmaceutical company, reported that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) issued a positive opinion in favor of the use of IMBRUVICA (ibrutinib) for the treatment of adult patients with previously-untreated chronic lymphocytic leukemia (CLL) in the European Union (EU) (Press release, AbbVie, APR 29, 2016, View Source [SID:1234511707]). The positive CHMP recommendation follows the March 4, 2016 U.S. Food and Drug Administration (FDA) approval of IMBRUVICA for the first-line treatment of patients with CLL.1 If approved by the European Commission (EC), this would be the fifth treatment indication for IMBRUVICA in the EU to date.

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CLL is a chronic disease, and the prevalence rate in Europe among men and women is approximately 5.87 and 4.01 cases per 100,000 persons per year, respectively.2 An expansion of the CLL indication in Europe would mean a larger number of CLL patients, in addition to those with the genetic mutations del 17p or TP53, could initiate their treatment with a safe and effective oral alternative option to chemotherapy, rather than having to wait until they have become relapsed or refractory on another treatment option.

IMBRUVICA is jointly developed and commercialized in the U.S. by Pharmacyclics LLC, an AbbVie company and Janssen Biotech, Inc. In Europe, Janssen-Cilag International NV (Janssen) holds the marketing authorization and its affiliates market IMBRUVICA in EMEA (Europe, Middle East, Africa), as well as the rest of the world. IMBRUVICA is already approved in Europe to treat adult patients with relapsed or refractory mantle cell lymphoma (MCL), adult patients with CLL who have received at least one prior therapy or who have del 17p or TP53 mutations, and adult patients with Waldenström’s macroglobulinemia (WM) who have received at least one prior therapy, or as a first-line treatment for WM patients unsuitable for chemo-immunotherapy.3 The EC will review the CHMP opinion and is expected to render a final decision on the use of IMBRUVICA for previously untreated patients with CLL later this year.

"If approved by the European Commission, IMBRUVICA will be available as a treatment option for all CLL patients, including those who are treatment-naïve. The ability to offer a safe and effective, oral, single-agent alternative to chemotherapy in the first-line setting is a landmark few therapies are able to reach," said Michael Severino, M.D., executive vice president, research and development and chief scientific officer, AbbVie. "The fact that IMBRUVICA has been granted this initial recommendation in the first-line setting is a testament to the dedication of those working on the compound to improve the lives of patients around the world who are suffering from CLL."

The positive CHMP opinion was based on data from the Phase 3, randomized, open-label RESONATE-2 (PCYC-1115) trial, which were presented at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting in December 2015 and simultaneously published in The New England Journal of Medicine (NEJM). After a median of 18 months of follow-up, IMBRUVICA was associated with a significant improvement in all efficacy endpoints versus chlorambucil in patients aged 65 or older with newly diagnosed CLL. Specifically, IMBRUVICA was associated with a 90% progression-free survival (PFS; the primary endpoint) rate versus 52% for chlorambucil. Moreover, IMBRUVICA significantly prolonged overall survival (OS; a key secondary endpoint), with a 24-month survival rate of 98% versus 85% for chlorambucil (HR: 0.16; 95% CI, 0.05, 0.56). The safety of IMBRUVICA in the treatment-naïve CLL patient population was consistent with previously reported studies.

About the RESONATE-2 Study
RESONATE-2 is a Pharmacyclics-sponsored study which enrolled 269 treatment-naïve patients with CLL or small lymphocytic lymphoma (SLL) aged 65 years or older in the U.S., EU and other regions. Patients were randomized to receive either IMBRUVICA 420 mg orally, once daily until progression or unacceptable toxicity, or chlorambucil on days 1 and 15 of each 28-day cycle for up to 12 cycles. The starting dose for chlorambucil in Cycle 1 was 0.5 mg/kg and was increased based on tolerability in Cycle 2 by increments of 0.1 mg/kg to a maximum of 0.8 mg/kg. The primary endpoint of the study was PFS as assessed by an Independent Review Committee (IRC) according to the International Workshop on Chronic Lymphocytic Leukemia (iWCLL) 2008 criteria, with modification for treatment-related lymphocytosis. Key secondary endpoints included overall response rate (ORR; based on the same iWCLL criteria), OS and safety.

IMBRUVICA is a first-in-class, oral, once-daily therapy that inhibits a protein called Bruton’s tyrosine kinase (BTK).1 BTK is a key signaling molecule in the B-cell receptor signaling complex that plays an important role in the survival and spread of malignant B cells.1,4 IMBRUVICA blocks signals that tell malignant B cells to multiply and spread uncontrollably.1

IMBRUVICA is approved in the U.S. to treat patients with chronic lymphocytic leukemia (CLL), patients with mantle cell lymphoma (MCL) who have received at least one prior therapy and patients with Waldenström’s macroglobulinemia (WM). Accelerated approval was granted for the MCL indication based on overall response rate. Continued approval for this indication may be contingent upon verification of clinical benefit in confirmatory trials.1

IMBRUVICA is approved in the EU to treat adult patients with relapsed or refractory MCL, adult patients with CLL who have received at least one prior therapy or who have the genetic mutations del 17p or TP53, and adult patients with WM who have received at least one prior therapy, or as a first-line treatment for WM patients unsuitable for chemo?immunotherapy. 3

IMBRUVICA was one of the first medicines to receive U.S. FDA approval via the new Breakthrough Therapy Designation pathway.

IMBRUVICA is being studied alone and in combination with other treatments in several blood and solid tumor cancers. More than 6,000 patients have been treated with IMBRUVICA in clinical trials. Currently, 14 Phase 3 trials have been initiated with IMBRUVICA and more than 90 trials are registered on



Hemorrhage – Fatal bleeding events have occurred in patients treated with IMBRUVICA. Grade 3 or higher bleeding events (intracranial hemorrhage [including subdural hematoma], gastrointestinal bleeding, hematuria, and post-procedural hemorrhage) have occurred in up to 6% of patients. Bleeding events of any grade, including bruising and petechiae, occurred in approximately half of patients treated with IMBRUVICA.

The mechanism for the bleeding events is not well understood. IMBRUVICA may increase the risk of hemorrhage in patients receiving antiplatelet or anticoagulant therapies and patients should be monitored for signs of bleeding. Consider the benefit-risk of withholding IMBRUVICA for at least 3 to 7 days pre- and postsurgery depending upon the type of surgery and the risk of bleeding.

Infections – Fatal and nonfatal infections have occurred with IMBRUVICA therapy. Grade 3 or greater infections occurred in 14% to 26% of patients. Cases of progressive multifocal leukoencephalopathy (PML) have occurred in patients treated with IMBRUVICA. Evaluate patients for fever and infections and treat appropriately.

Cytopenias – Treatment-emergent Grade 3 or 4 cytopenias including neutropenia (range, 19% to 29%), thrombocytopenia (range, 5% to 17%), and anemia (range, 0% to 9%) occurred in patients treated with IMBRUVICA. Monitor complete blood counts monthly.

Atrial Fibrillation – Atrial fibrillation and atrial flutter (range, 6% to 9%) have occurred in patients treated with IMBRUVICA, particularly in patients with cardiac risk factors, hypertension, acute infections, and a previous history of atrial fibrillation. Periodically monitor patients clinically for atrial fibrillation. Patients who develop arrhythmic symptoms (eg, palpitations, lightheadedness) or new-onset dyspnea should have an ECG performed. Atrial fibrillation should be managed appropriately and if it persists, consider the risks and benefits of IMBRUVICA treatment and dose modification.

Hypertension – Hypertension (range, 6% to 17%) has occurred in patients treated with IMBRUVICA with a median time to onset of 4.5 months (range, 0.03 to 18.40 months). Monitor patients for new-onset hypertension or hypertension that is not adequately controlled after starting IMBRUVICA. Adjust existing antihypertensive medications and/or initiate antihypertensive treatment as appropriate.

Second Primary Malignancies – Other malignancies (range, 5% to 16%) including non-skin carcinomas (range, 1% to 4%) have occurred in patients treated with IMBRUVICA. The most frequent second primary malignancy was non-melanoma skin cancer (range, 4% to 13%).

Tumor Lysis Syndrome – Tumor lysis syndrome has been infrequently reported with IMBRUVICA therapy. Assess the baseline risk (eg, high tumor burden) and take appropriate precautions. Monitor patients closely and treat as appropriate.

Embryo-Fetal Toxicity – Based on findings in animals, IMBRUVICA can cause fetal harm when administered to a pregnant woman. Advise women to avoid becoming pregnant while taking IMBRUVICA and for 1 month after cessation of therapy. If this drug is used during pregnancy or if the patient becomes pregnant while taking this drug, the patient should be apprised of the potential hazard to a fetus.

The most common adverse reactions (?20%) in patients with B-cell malignancies (MCL, CLL, WM) were thrombocytopenia* (57%, 53%, 43%), diarrhea (51%, 48%, 37%), anemia* (41%, 37%, 13%), neutropenia* (47%, 46%, 44%), musculoskeletal pain (37%, 32%†, NA‡), fatigue (41%, 29%, 21%), bruising (30%, 25%†, 16%†), nausea (31%, 24%, 21%), rash (25%, 23%†, 22%†), and upper respiratory tract infection (34%, 19%, 19%).

*Based on adverse reactions and/or laboratory measurements (noted as platelets, neutrophils, or hemoglobin decreased).
†Includes multiple ADR terms.
‡Not applicable; no associated ADRs.

The most common Grade 3 or 4 non-hematologic adverse reactions (?5%) in MCL patients were pneumonia (7%), abdominal pain (5%), atrial fibrillation (5%), diarrhea (5%), fatigue (5%), and skin infections (5%).

Approximately 4% (CLL), 14% (MCL), and 11% (WM) of patients had a dose reduction due to adverse reactions.

Approximately 4%-10% (CLL), 9% (MCL), and 6% (WM) of patients discontinued due to adverse reactions. Most frequent adverse reactions leading to discontinuation were pneumonia, subdural hematomas, and atrial fibrillation (1% each) in CLL patients and subdural hematoma (1.8%) in MCL patients.


CYP3A Inhibitors – Avoid coadministration with strong and moderate CYP3A inhibitors. If a moderate CYP3A inhibitor must be used, reduce the IMBRUVICA dose.

CYP3A Inducers – Avoid coadministration with strong CYP3A inducers.


Hepatic Impairment – Avoid use in patients with moderate or severe baseline hepatic impairment. In patients with mild impairment, reduce IMBRUVICA dose.

Please see Full Prescribing Information: View Source

NewLink Genetics Reports First Quarter 2016 Financial Results

On April 29, 2016 NewLink Genetics Corporation (NASDAQ:NLNK), a biopharmaceutical company at the forefront of discovering, developing and commercializing novel immuno-oncology product candidates, including both cellular immunotherapy and checkpoint inhibitor programs, to improve the lives of patients with cancer, reported consolidated financial results for the first quarter of 2016 (Press release, NewLink Genetics, APR 29, 2016, View Source [SID:1234511634]).

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"We had a productive quarter and made solid progress in achieving our mission of bringing patients with cancer better treatment options," said Charles J. Link, Jr., M.D., Chairman, Chief Executive Officer and Chief Scientific Officer. "The pivotal IMPRESS trial for patients with resected pancreatic cancer is moving rapidly toward final analysis. And, we are preparing for multiple updates from both our proprietary and partnered IDO pathway inhibitor clinical programs at key scientific meetings in 2016."

Nicholas Vahanian M.D., President and Chief Medical Officer, added, "With the upcoming clinical trial read outs, we anticipate that these data will bring us closer to clinical validation of our IDO pathway inhibitor program. The team at NewLink Genetics continues with confidence and excitement as our partner, Genentech begins to report on the clinical development of GDC-0919 alone and in combination studies. We look forward to Genentech reporting on the results from these studies this year. "

Program Updates:

HyperAcute Cellular Immunotherapy Program

Algenpantucel-L/IMPRESS Trial

Today, the company reported that it expects to report top-line results from the IMPRESS trial during the second quarter of 2016. Algenpantucel-L is the most advanced clinical program utilizing NewLink Genetics’ HyperAcute Cellular Immunotherapy platform technology. IMPRESS is a randomized, controlled, two-arm Phase 3 trial (n=722) for patients with resected pancreatic cancer designed to test algenpantucel-L in combination with the standard of care versus the standard of care alone.

IDO Pathway Inhibitor Programs


At the upcoming American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting from June 3 to 7, 2016 in Chicago, NewLink Genetics expects to report on clinical updates on its proprietary IDO pathway inhibitor programs. Abstracts that have been selected include:

Interim data update from a Phase 2 trial with a target enrollment of 80 patients evaluating the addition of indoximod to gemcitabine/nab-paclitaxel for patients with metastatic pancreatic cancer.
Mid-trial and safety data update from a Phase 2 trial with a target enrollment of 96 patients of indoximod and ipilimumab or PD-1 inhibitors for patients with stage 3 or 4 advanced or metastatic melanoma.
Additional data updates expected in the second half of 2016 include:

Mid-trial update from a Phase 2 trial with a target enrollment of 132 patients of indoximod for patients with refractory malignant brain tumors.
Guidance on timing of preliminary results in a randomized Phase 2 trial with a target enrollment of 154 patients of indoximod for patients with metastatic breast cancer.
GDC-0919 Small Molecule Program

The company is anticipating a clinical update from the combination study of GDC-0191. Genentech recently reported that it will update the Phase 1b study with a target enrollment of 224 patients in solid tumors at the European Society of Medical Oncology (ESMO) (Free ESMO Whitepaper) from October 7-11, 2016 in Denmark. The dose-escalation and expansion study combines GDC-0919 and atezolizumab (PD-L1 Mab).

In 2014, NewLink Genetics entered into an exclusive worldwide license agreement with Genentech, a member of the Roche Group, for the development of NLG919, NewLink Genetics’ IDO pathway inhibitor. The parties also entered into a research collaboration for the discovery of next generation IDO/TDO compounds.

Financial Results for the Three-Month Period Ended March 31, 2016

Cash Position: NewLink Genetics ended the quarter on March 31, 2016, with cash, cash equivalents, and certificates of deposit totaling $178 million compared to $197.8 million for the year ending December 31, 2015. The decrease was attributable primarily due to the increased operating expenses for R&D and pre-commercialization development, offset by amounts received under government contracts.

R&D Expenses: Research and development expenses in the first quarter of 2016 were $21.9 million compared to $18 million during the comparable period in 2015. The increase was primarily due to increases in clinical trial and manufacturing expenses related to NewLink Genetics’ broad pipeline of product candidates.

G&A Expenses: General and administrative expenses in the first quarter of 2016 were $9.2 million compared to $8.4 million during the comparable periods in 2015. The increase was primarily due to an increase in share-based compensation expense, as well as increases in travel expenses and medical affairs and marketing.

Net Income/Loss: NewLink Genetics reported a net loss of $23.7 million or a $0.82 loss per diluted share for the first quarter of 2016 compared to net income of $11.2 million or earnings of $0.35 per diluted share for the comparable period in 2015.

NewLink Genetics ended the quarter with 28,860,925 shares outstanding.

Financial Guidance and Upcoming Investor Meetings

NewLink Genetics has reiterated their goal and expectation is to finish 2016 with two years of cash on hand.

We have presented at five investor meetings since the beginning of the year and expect to present at Bank of America Healthcare Conference on May 12 and Cantor Fitzgerald Healthcare Conference on July 12 and 13.

HyperAcute Cellular Immunotherapy Program

HyperAcute Cellular Immunotherapies are unique, tumor-specific product candidates that take advantage of a pre-existing human immune response to initiate a powerful cascade, potentially educating the body’s natural defenses to identify and destroy cancer cells. Unlike certain immuno-oncology products, HyperAcute Cellular Immunotherapies do not require patient tissue or cancer cells and are designed to be easy to administer. HyperAcute Cellular Immunotherapies use allogeneic (disease-specific, not patient-specific), tumor-specific human cell lines that have been modified to express alpha-gal. Intact, whole cells are used rather than cell fragments or purified proteins, which we believe result in the stimulation of a more powerful immune response. Our most advanced clinical program utilizing this technology is for patients with pancreatic cancer. Additionally, there are ongoing clinical development programs and data on induced immune responses targeting non-small-cell lung cancer, melanoma, prostate cancer and kidney cancer.

Indoleamine 2,3-Dioxygenase (IDO) Checkpoint Inhibitor Programs

The indoleamine 2,3-dioxygenase (IDO) pathway regulates immune response by suppressing T cell function and enabling local tumor immune escape. NewLink Genetics is researching two IDO pathway inhibitors, GDC-0919 (in partnership with Genentech) and indoximod, small-molecule product candidates that have the potential to disrupt mechanisms by which tumors evade the immune system.

NewLink Genetics’ indoximod and GDC-0919 each have a distinct mechanism of action within the IDO pathway and are in Phase 1 or 2 clinical trials for a range of cancers, including breast cancer, melanoma, and other solid tumors.

Medivation’s Board of Directors Unanimously Rejects Sanofi’s Unsolicited Proposal

On April 29, 2016 Medivation, Inc. (NASDAQ: MDVN) reported that its Board of Directors, after consultation with its financial and legal advisors, unanimously determined that the unsolicited proposal from Sanofi to acquire Medivation for $52.50 per share in cash substantially undervalues Medivation and is not in the best interests of the company and its stockholders (Press release, Medivation, APR 29, 2016, View Source [SID:1234511611]).

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"Over the past several years, we have established a world class oncology franchise and a unique, diversified and highly-promising late-stage development pipeline," said David Hung, M.D., Founder, President and Chief Executive Officer of Medivation. "Further, we have a track record of delivering extraordinary value to our stockholders. Sanofi’s opportunistically-timed proposal, which comes during a period of significant market dislocation, and prior to several important near-term events for the company, is designed to seize for Sanofi value that rightly belongs to our stockholders. We believe the continued successful execution of our well-defined strategic plan will deliver greater value to Medivation’s stockholders than Sanofi’s substantially inadequate proposal."

The Medivation Board of Directors’ unanimous conclusion was based on the following:

The proposal substantially undervalues Medivation and its leading oncology franchise.

Medivation has significant scarcity value as one of the few profitable, commercial-stage oncology companies;it has brought a blockbuster product to market and is leveraging its expertise to develop and bring to market additional products.

Medivation has built XTANDI (enzalutamide) capsules into a rapidly-growing, multi-billion dollar oncology product and remains on track to achieve its 2016 U.S. net sales guidance, which implies approximately 28% growth (at the mid-point) for the year.
XTANDI is one of the most successful oncology product launches in history and just surpassed Johnson & Johnson’s Zytiga (abiraterone) in U.S. market share, despite launching sixteen months later.

XTANDI has achieved worldwide annual net sales of $2.2 billion on a run rate basis, less than four years after its initial approval.
XTANDI has significant patent life with 10+ years of remaining exclusivity.

XTANDI is poised to capitalize on a substantial, near-term commercial opportunity in urology, enabling it to serve a larger patient population of men with metastatic castration-resistant prostate cancer (mCRPC) and increasing the duration of therapy.
The PDUFA date for TERRAIN/STRIVE label expansion on October 22, 2016, is rapidly approaching and is anticipated to drive significantly greater adoption by urologists.

In April, the CHMP issued a positive opinion to include findings from the TERRAIN trial in the European label.
Medivation recently expanded its specialty salesforce to create dedicated urology and oncology selling teams that are just starting to have a promotional impact.

XTANDI has multiple opportunities beyond mCRPC, which are not reflected in the company’s current valuation.
Ongoing Phase 3 trials, i.e. PROSPER and EMBARK, are designed to move XTANDI even earlier in the prostate cancer treatment paradigm; PROSPER is on track to complete enrollment of 1,560 patients in mid-2017.

Medivation is pursuing clinical development across three major subtypes of breast cancer, a new and significant market opportunity for XTANDI, and expects to report top-line Phase 2 data in patients with ER/PR+ breast cancer, which represents 50% of all breast cancers, in the second half of 2016.

A Phase 3 trial in Triple Negative Breast Cancer, a significant unmet need, is expected to initiate later in 2016.
The company continues to explore XTANDI in other solid tumor indications and settings, e.g., in hepatocellular carcinoma and in combination with immunotherapy.

Sanofi’s proposal would deny Medivation’s stockholders the value of Medivation’s wholly-owned, innovative late-stage pipeline.

Talazoparib represents another blockbuster opportunity as a potentially best-in-class PARP inhibitor targeting a wide range of oncology indications.

Top-line data from the Phase 3 EMBRACA trial in germline BRCA mutated advanced breast cancer is expected in the first half of 2017.

Recent data reported at AACR (Free AACR Whitepaper) demonstrate talazoparib’s potential in tumors with defects in DNA repair beyond BRCA deficiency and possibly in patients without evidence of homologous recombination deficiency when used in combination with low dose chemotherapy.

The company is preparing to initiate several clinical trials in 2016, including in breast cancer beyond germline BRCA mutations, prostate cancer, ovarian cancer and small cell lung cancer, including potentially registrational trials.
Medivation recently met with the FDA to discuss clinical trial design and a potential accelerated approval pathway in prostate cancer.

Talazoparib is highly synergistic with our existing development and commercial infrastructure.
Additional clinical data demonstrating talazoparib’s potent activity is expected to be presented at a medical meeting later in the year.

Pidilizumab has the potential to be a novel immuno-oncology candidate supported by clinical efficacy and a strong safety profile in several hematological malignancies.

The execution of Medivation’s business plan will deliver value to its stockholders that is far superior to Sanofi’s proposal.

Medivation has an exceptional track record for execution.

XTANDI has achieved all development and sales milestones under Medivation’s collaboration with Astellas.

Medivation’s track record for delivering value to our stockholders is exemplified by XTANDI, which was in-licensed as a pre-clinical asset in 2005, received full FDA approval in seven years (better than industry average), and achieved $2.2 billion in worldwide annual net sales on a run rate basis in less than four years.

The company has generated a 1,440%+ total shareholder return for its stockholders since 2009.

Medivation has already achieved two years of profitability, despite only launching XTANDI in late 2012, and the company has pursued minimal, dilutive capital raises.
Sanofi’s timing is designed to benefit Sanofi – not Medivation’s stockholders.

Sanofi approached Medivation following a period of significant market dislocation in biotech and just as the market was beginning to recover.
The "private offer" was submitted to Medivation when the NBI index was almost 30% below its July 2015 high.
The proposal offer price is:
21% below Medivation’s 52-week trading high of $66.40
Less than a 14% premium to Medivation’s 12-month volume weighted average price, or VWAP
Only a 9% premium to Medivation’s VWAP over the last month
Medivation will provide additional information on its financial performance, XTANDI’s utilization and the company’s clinical development plans for talazoparib on next week’s earnings call.

Kim D. Blickenstaff, Chairman of Medivation’s Board of Directors, notes that "Medivation has a long history of producing superior growth and generating significant value for its stockholders. Since the launch of XTANDI, Medivation has achieved revenues of nearly $1 billion in just over three years. There are several exciting pipeline opportunities that will drive significant growth. The Board is determined to continue to aggressively focus on working for, and delivering value to, Medivation’s stockholders."

ImmunoGen Reports Third Quarter Fiscal Year 2016 Financial Results and Provides Corporate Update

On April 29, 2016 ImmunoGen, Inc. (Nasdaq: IMGN), a biotechnology company developing targeted cancer therapeutics using its proprietary ADC technology, reported financial results for the three-month period ended March 31, 2016 – the third quarter of the Company’s 2016 fiscal year (Press release, ImmunoGen, APR 29, 2016, View Source [SID:1234511596]). ImmunoGen also provided an update on the Company’s lead program, mirvetuximab soravtansine, and other wholly owned clinical-stage product candidates.

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"We are making important progress with our key product programs," commented Daniel Junius, President and CEO. "In early June, expanded Phase 1 findings with mirvetuximab soravtansine will be presented at ASCO (Free ASCO Whitepaper). Based on these data, we are modifying the design of our FORWARD I trial to be a Phase 3 study intended to support full marketing approval. Patient enrollment is proceeding well in our Phase 1b/2 FORWARD II trial that is assessing this novel ADC in combination regimens, and patient dosing has begun in Phase 1 testing of IMGN779, the first ADC utilizing one of our new DNA-alkylating cancer-killing agents."

Mr. Junius continued, "Our partners are also making progress. Takeda has reported preclinical information on a GCC-targeting ADC it is developing utilizing our DNA-alkylating technology, and Novartis and Sanofi recently presented preclinical data on product candidates with our maytansinoid technology. Phase 1 clinical data with Bayer’s anetumab ravtansine and Sanofi’s SAR566658 are scheduled for poster discussion at ASCO (Free ASCO Whitepaper), with data also being presented on Sanofi’s isatuximab."

ImmunoGen Product Program Updates

Mirvetuximab soravtansine – First FRα-targeting ADC; potential new treatment for FRα-positive ovarian cancer.

Data will be presented at ASCO (Free ASCO Whitepaper) from a 46-patient Phase 1 expansion cohort assessing this ADC as monotherapy for FRα-positive platinum-resistant ovarian cancer (abstract #5567). This cohort was increased from 20 patients to provide additional experience in the patient population to better inform the design of ImmunoGen’s FORWARD I trial. The data presented will be updated from the 20-patient data reported previously and from that available at the time of abstract submission.
Based on the expanded findings, ImmunoGen is modifying its FORWARD I trial from a two-stage, Phase 2 trial with response rate as the primary endpoint to a single-stage, Phase 3 trial with progression-free survival as the primary endpoint. Patients with FRα-positive (medium or high) platinum-resistant ovarian cancer treated with up to three prior regimens will be eligible for enrollment.
Patient enrollment is ongoing in the FORWARD II trial assessing mirvetuximab soravtansine in combination regimens. A cohort is being added to assess this novel ADC in combination with Merck’s anti-PD1, pembrolizumab.
IMGN779 – First-in-class CD33-targeting ADC utilizing a DNA-alkylating cancer-killing agent from ImmunoGen’s new family called IGNs.

Patient enrollment has started in the Phase 1 trial assessing this ADC for the treatment of acute myeloid leukemia.
IMGN529 and coltuximab ravtansine – CD37- and CD19-targeting, respectively, ADCs for diffuse large B-cell lymphoma (DLBCL).

Patient enrollment is expected to open shortly in a Phase 2 trial assessing IMGN529 in combination with rituximab and in 1H2017 for coltuximab ravtansine in a combination regimen.
Update on Partner Programs

Phase 1 findings with Sanofi’s SAR566658 and Bayer’s anetumab ravtansine ADCs with ImmunoGen technology have been accepted for poster discussion at ASCO (Free ASCO Whitepaper), with data also being presented on Sanofi’s isatuximab (SAR650984).
ImmunoGen, Novartis, and Sanofi had multiple ADC-related presentations at the American Association of Cancer Research (AACR) (Free AACR Whitepaper) annual meeting earlier this month. Those by ImmunoGen scientists featured new, novel technologies while those by Novartis and Sanofi related to cadherin6- and LAMP1-targeting ADCs, respectively, utilizing ImmunoGen maytansinoid ADC technology.
Takeda reported data at a scientific conference on a GCC-targeting ADC the company is developing utilizing one of ImmunoGen’s new IGN agents.
Financial Results

For the Company’s quarter ended March 31, 2016 (3QFY2016), ImmunoGen reported a net loss of $31.9 million, or $0.37 per basic and diluted share, compared to a net loss of $21.6 million, or $0.25 per basic and diluted share, for the same quarter last year (3QFY2015).

Revenues for 3QFY2016 were $19.7 million, compared to $11.4 million for 3QFY2015. The current period includes a $10 million milestone earned from Bayer with the advancement of anetumab ravtansine into a Phase 2 clinical trial designed to support product registration. License and milestone fees for the prior year period include a $5 million milestone earned from Novartis with its initiation of LOP628 Phase 1 clinical testing. Revenues in 3QFY2016 include $7.4 million of non-cash royalty revenues, compared with $5.1 million in cash royalty revenues for the prior year period. Revenues for 3QFY2016 also include $1.2 million of clinical materials revenue and $1.1 million of research and development support fees, compared with $0.7 million and $0.5 million, respectively, in the prior year period.

Operating expenses in 3QFY2016 were $47.3 million, compared to $32.7 million in 3QFY2015. Operating expenses in 3QFY2016 include research and development expenses of $36.1 million, compared to $25.7 million in 3QFY2015. This change is primarily due to increased third-party costs related to the advancement of our wholly owned product candidates, increased clinical trial costs, primarily related to our expansion of the mirvetuximab soravtansine development program, and increased personnel expenses, principally due to recent hiring. Operating expenses include general and administrative expenses of $11.2 million in 3QFY2016, compared to $7 million in 3QFY2015. This increase is primarily due to a non-cash stock compensation charge resulting from the CEO transition, as well as increased personnel expenses and professional services.

ImmunoGen had approximately $182.9 million in cash and cash equivalents as of March 31, 2016, compared with $278.1 million as of June 30, 2015, and had no debt outstanding in either period. Cash used in operations was $91.6 million in the first nine months of FY2016, compared with $26.8 million in the same period in FY2015. The prior year period benefited from $25 million in upfront payments received including $20 million in connection with the execution of the right-to-test agreement with Takeda in March 2015, as well as lower operating expenses. Capital expenditures were $8.6 million and $4.5 million for the first nine months of FY2016 and FY2015, respectively.

Financial Guidance for Fiscal Year 2016

ImmunoGen has updated its guidance for its fiscal year ending June 30, 2016. Expected revenues are now projected to be between $60 million and $70 million, compared with previous guidance of between $70 million and $80 million. The change is primarily due to changes in the expected timing of partner events and is mainly non-cash. Operating expenses are now projected to be between $180 million and $185 million, compared with previous guidance of between $175 million and $180 million. The change is primarily related to greater clinical trial costs and non-cash stock compensation charges. The Company’s guidance for its net loss is now expected to be between $135 million and $140 million, compared to its previous estimate of $120 million and $125 million with most of this change being non-cash related.

ImmunoGen now projects cash and cash equivalents at June 30, 2016 to be between $155 million and $160 million, compared to previous guidance of $165 million to $170 million. This change reflects the cash impact of less partner upfront and milestone payments. The Company’s guidance for cash used in operations is now projected to be between $110 million and $115 million, which had previously been $100 million and $105 million. The Company’s guidance for capital expenditures remains unchanged, which is between $13 million and $15 million.