On May 2, 2016 Sangamo BioSciences, Inc. (NASDAQ: SGMO), the leader in therapeutic genome editing, reported its first quarter 2016 financial results and accomplishments (Press release, Sangamo BioSciences, MAY 2, 2016, View Source [SID:1234511807]).

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Sangamo BioSciences, Inc. (PRNewsFoto/Sangamo BioSciences, Inc.)
"During the first quarter of 2016 we focused on activities to enable initiation of the first clinical trials of our IVPRP approach in hemophilia B and MPS I, and I am pleased to report that we will meet our stated timelines for the opening of both trials," said Edward Lanphier, Sangamo’s president and chief executive officer. "We look forward to generating and presenting clinical data that support the application of this highly leverageable platform while continuing to progress our programs in hemophilia A and several lysosomal storage disorders toward clinical development."

Recent Highlights

Presentation of new data from Sangamo’s proprietary In Vivo Protein Replacement Platform (IVPRP) programs for MPS I and MPS II at the 12TH Annual WORLDSymposium Meeting. Sangamo scientists and academic collaborators from the University of Minnesota presented new preclinical data in disease models of the Company’s IVPRP-based MPS I (Hurler syndrome) and MPS II (Hunter syndrome) programs at the 2016 WORLDSymposium Annual Meeting. In animal models of disease, the data demonstrated the production of stable, therapeutic levels of replacement enzyme from the liver into the circulation and secondary tissues, including the brain, resulting in significant reduction in biomarkers of the disease, and notably, statistically significant improvements in cognitive function in treated animals.
Publication in the New England Journal of Medicine (NEJM) highlighting Sangamo’s IVPRP approach for hemophilia B. In March, NEJM published a review authored by Dr. Amit Nathwani, a key opinion leader in the field of gene therapy approaches to hemophilia, highlighting Sangamo’s IVPRP-based SB-FIX program for the one-time, permanent treatment of hemophilia B. The article details the significant advantages of Sangamo’s ZFN-mediated, targeted integration of the Factor IX (FIX) gene at the albumin locus over conventional non-integrating gene therapy approaches that use adeno-associated virus (AAV) to deliver the FIX gene and other therapeutic genes to liver cells.
Sangamo augments clinical expertise with the appointment of Matthew Spear, M.D. as Vice President and Head of Clinical Development. Dr. Spear joins Sangamo with more than 20 years of experience in all stages of biopharmaceutical research and development and has led the development of over 15 therapeutic products. Most recently, Dr. Spear served as a Vice President in Clinical Development at Incyte Corp.
Presentation of immunological data from Sangamo’s SB-728-T Phase 2 clinical program in HIV/AIDS at the 2016 Annual Conference on Retroviral and Opportunistic Infections (CROI 2016). Sangamo’s collaborators from Case Western Reserve University presented analyses of immunological data from the Company’s clinical trials of SB-728-T, a ZFP Therapeutic designed to provide functional control of HIV/AIDS. The presentation outlined potentially interrelated mechanisms for viral load (VL) control in SB-728-T-treated subjects during a treatment interruption (TI) from their antiretroviral therapy. This suggests a model mechanism of action for SB-728-T and enables identification of patients who will most benefit from Sangamo’s ZFN-mediated CCR5 knock-out approach.
Upcoming Events in 2016

Initiation of IVPRP-based Phase 1/2 clinical trials SB-FIX-1501 (hemophilia B) and SB-318-1502 (MPS I / Hurler syndrome) by the end of the second quarter and mid-2016, respectively.
Presentation of data from several ZFP Therapeutic programs by Sangamo scientists and collaborators at the upcoming 19TH Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT) (Free ASGCT Whitepaper), which will be held in Washington D.C. from May 4-7, 2016.
Submission of investigational new drug (IND) applications in the first half of 2016 for Sangamo’s IVPRP-based SB-913 program for MPS II (Hunter syndrome), and beta-thalassemia program in collaboration with Biogen Inc. (Biogen).
Presentation of new clinical data from Cohort 3* of Sangamo’s SB-728-1101 Phase 1/2 trial in T-cells for the functional control of HIV/AIDS in the second half of 2016.
First Quarter 2016 Results
For the first quarter ended March 31, 2016, Sangamo reported a consolidated net loss of $16.5 million, or $0.23 per share, compared to a net loss of $5.3 million, or $0.08 per share, for the same period in 2015. As of March 31, 2016, the Company had cash, cash equivalents, marketable securities and interest receivable of $189.0 million.

Revenues for the first quarter of 2016 were $3.9 million, compared to $13.5 million for the same period in 2015. First quarter 2016 revenues were generated from the Company’s collaboration agreements with Biogen and Shire International GmbH (Shire), enabling technology agreements and research grants. The revenues recognized for the first quarter of 2016 consisted of $3.7 million from collaboration agreements and $0.2 million from research grants, compared to $12.7 million and $0.8 million, respectively, for the same period in 2015.

The decrease in collaboration agreement revenues was primarily a result of an amendment to the Company’s collaboration and license agreement with Shire in the third quarter of 2015, which returned the rights to the hemophilia programs to Sangamo, and a decrease in revenue under the Company’s collaboration agreement with Sigma.

In the first quarter of 2016, Sangamo recognized $2.0 million of revenues related to research services performed under the collaboration agreement with Biogen, and $0.4 million of revenues related to research services performed under the collaboration agreement with Shire. In addition, Sangamo received upfront payments of $13.0 million and $20.0 million pursuant to the agreements entered into with Shire in 2012 and Biogen in 2014, respectively. The Shire payment is being recognized as revenue on a straight-line basis over the initial six-year research term. Beginning in January 2016, the Biogen payment will be recognized over approximately 42 months which reflects the revised service period related to Sangamo’s deliverables under the Biogen agreement. The Company recognized $0.5 million of the Shire upfront payment and $0.6 million of the Biogen upfront payment as revenue for the first quarter of 2016.

Research and development expenses were $15.3 million for the first quarter of 2016, compared to $15.0 million for the same period in 2015. Research and development expenses were primarily comprised of manufacturing expenses, research expenses associated with Sangamo’s clinical and preclinical programs, and personnel-related expenses, including stock-based compensation.

General and administrative expenses were $5.4 million for the first quarter of 2016, compared to $4.7 million for the same period in 2015.

Total operating expenses for the first quarter of 2016 were $20.6 million, compared to $19.7 million for the same period in 2015.

Financial Guidance for 2016
The Company reiterates its earlier guidance as follows:

Cash and Investments: Sangamo expects that its cash, cash equivalents and marketable securities will be at least $150 million at the end of 2016, inclusive of research funding from existing collaborators but exclusive of funds arising from any additional new collaborations or partnerships, equity financings or other new sources.
Revenues: Sangamo expects that revenues will be in the range of $20 million to $25 million in 2016, inclusive of research funding from existing collaborations.
Operating Expenses: Sangamo expects that operating expenses will be in the range of $85 million to $95 million for 2016

On May 01, 2016 Cempra, Inc. (Nasdaq:CEMP), a clinical-stage pharmaceutical company focused on developing antibiotics to meet critical medical needs in the treatment of bacterial infectious diseases, reported financial results for the quarter ended March 31, 2016 and provided an update on recent corporate developments (Press release, Cempra, MAY 1, 2016, View Source [SID:1234511732]). The company will host a webcast and conference call tomorrow at 8:00 a.m. ET.

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First Quarter 2016 and Recent Corporate Highlights

In April, Cempra completed its rolling submission of two New Drug Applications (NDA) to the U.S. Food and Drug Administration (FDA) for solithromycin (intravenous and capsules) in community-acquired bacterial pneumonia (CABP). Having been granted qualified infectious diseases product (QIDP) designation in 2013, solithromycin’s NDAs qualify for an eight month priority review. Subject to approval by the FDA, Cempra plans to launch solithromycin in the U.S. in the first quarter of 2017.

In March, Cempra received authorization under its existing contract with the Biomedical Advanced Research and Development Authority (BARDA), part of the Office of the Assistant Secretary for Preparedness and Response within the U.S. Department of Health and Human Services, to receive funding of $25.5 million through mid-2018 for a Phase 2/3 pivotal clinical study of solithromycin in pediatric patients. Cempra is responsible for an additional designated portion of the cost of the planned Phase 2/3 study. Three dosage formulations of solithromycin, intravenous, capsule and oral suspension, will be tested in the trial and may provide pediatricians with greater dosing flexibility.

In January, Cempra completed a public offering of 4,166,667 shares at a price of $24.00 per share. Net proceeds after underwriting discounts and commissions and expenses of the offering were approximately $93.8 million. Cempra intends to use the funds for the commercial launch of solithromycin in CABP in the U.S., subject to the drug receiving FDA approval, and on research and development activities, working capital and general corporate and administrative expenses.

The Taksta (fusidic acid) acute bacterial skin and skin structure infection (ABSSSI) Phase 3 trial is enrolling patients, which is expected to be completed during the first half of 2017.
"I am truly delighted that Cempra is able to mark the tenth anniversary of its founding with our submission to the FDA of two NDAs for solithromycin in community-acquired bacterial pneumonia," said Prabhavathi Fernandes, Ph.D., president and chief executive officer of Cempra. "As a new chemical entity, we expect that solithromycin will be subject to an advisory committee review, however given the compelling data package that we have assembled, we look forward to working with the agency during the review process to bring this important new macrolide antibiotic to patients with CABP and the physicians who treat them. Our development programs for solithromycin for pediatric patients and urogenital gonorrhea, as well our development program for Taksta, are continuing to move forward."

Upcoming Clinical Development Milestones

Solithromycin

Solithromycin pediatric: patient enrollment for Phase 1b trial continues.
Enrollment in a Phase 2/3 pivotal trial with solithromycin for bacterial infections in pediatric patients is expected to initiate in Q2 2016.
Phase 3 trial for solithromycin in urogenital gonorrhea is ongoing.
Phase 2 trial in chronic obstructive pulmonary disease (COPD) is ongoing.
Phase 2 trial in nonalcoholic steatohepatitis (NASH) is ongoing.
Completion of the EMA submission for solithromycin in the treatment of CABP is expected by the end of the first half of 2016.
Taksta

Phase 3 trial in ABSSSI is ongoing.
An exploratory trial for Taksta in patients with refractory bone or joint infections is ongoing.
Financial Results for the Three Months Ended March 31, 2016

For the quarter ended March 31, 2016, Cempra reported a net loss of $29.4 million, or $0.61 per share, compared to a net loss of $17.4 million, or $0.41 per share for the first quarter in 2015. Research and development expense in the first quarter of 2016 was $23.5 million, a decrease of 10% compared to the same quarter in 2015. The lower R&D expense was primarily due to the timing of payments for the order of active pharmaceutical ingredient (API) necessary to support the launch of solithromycin as the company begins its commercial readiness activities and a decrease in clinical expenses as the Phase 3 Oral and Phase 3 IV-to-Oral studies are complete. General and administrative expense was $8.3 million, a 79% increase compared to the quarter ended March 31, 2015, driven primarily by commercial readiness activities and increased headcount as the company begins to plan for commercialization.

As of March 31, 2016, Cempra had cash and equivalents of $223.6 million and 48.2 million shares outstanding.

Financial Guidance

The company’s current cash and equivalents are expected to be sufficient to fund ongoing operations into the second quarter of 2017, assuming continued timely receipts under the BARDA contract and receipt of expected milestone payments from Toyama. This projection does not include any funds from new financings or partnerships.

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."

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.

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."