On May 05, 2016 Portola Pharmaceuticals Inc. (NASDAQ:PTLA), today provided a corporate update and reported its financial results for the first quarter ended March 31, 2016 (Press release, Portola Pharmaceuticals, MAY 5, 2016, View Source;p=RssLanding&cat=news&id=2165766 [SID:1234512036]).

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"During the first quarter of 2016, we continued to advance the development of our three product candidates. We reported acceptance of our ANDEXXA (andexanet alfa) BLA and Phase 3 topline data from the APEX trial of betrixaban, completed a successful FDA pre-approval inspection of our Generation 1 commercial manufacturing process for ANDEXXA and completed the Phase 1 cerdulatinib study," said Bill Lis, chief executive officer of Portola. "We remain focused on successfully launching ANDEXXA, an FDA-designated Breakthrough Therapy and filing an NDA for betrixaban, an FDA-designated Fast Track Therapy, this year."

Recent Achievements, Upcoming Events and Milestones

ANDEXXA (andexanet alfa) – an FDA-designated Breakthrough Therapy Factor Xa inhibitor antidote in development for reversal of anticoagulation in patients treated with a Factor Xa inhibitor who are admitted to the hospital with uncontrolled bleeding or who need urgent surgery

The FDA accepted Portola’s BLA submission for ANDEXXA for filing in February 2016
The FDA completed a successful pre-approval inspection of CMC Biologics’ Generation 1 process; the inspection addressed the Generation 1 2,500 liter scale and the 6×2,000 liter scale process and confirmed alignment with the BLA
Completed Generation 2 GMP batches at the 10,000 liter scale at Lonza and achieved target yields
Entered into collaboration agreements with all of the manufacturers of oral Factor Xa inhibitors in Japan to develop and/or commercialize andexanet alfa in that territory
Enrollment remains on track for ANNEXATM-4, a Phase 3b/4 confirmatory study
Plan to present data from the Phase 2 proof-of-concept study with betrixaban in healthy volunteers at a medical conference this year
Preparing for commercial launch shortly after the PDUFA date of August 17, 2016, if approved
Plan to submit an MAA with the EMA in the third quarter of 2016
Betrixaban – an oral Factor Xa inhibitor anticoagulant in development for the prevention of venous thromboembolism (VTE) in acute medically ill patients

Reported topline results from the pivotal Phase 3 APEX Study
Plan to present data from the APEX Study on Friday, May 27, at the International Society on Thrombosis and Haemostasis (ISTH) 62nd Annual SSC (Scientific and Standardization Committee) Meeting in Montpellier, France; will hold an investor webcast directly following the presentation to discuss the APEX Study data
Plan to meet with the FDA in the second quarter and with the European Medicines Agency (EMA) to discuss the APEX Study results and a regulatory path forward
Pending discussions, plan to submit a New Drug Application (NDA) with the FDA and a Marketing Authorization Application (MAA) with the EMA by the end of the year
Cerdulatinib – an oral, dual syk/JAK inhibitor in development to treat resistant or relapsed hematologic cancer patients

Achieved the maximum tolerated dose and completed the Phase 1 study; plan to present results in a poster presentation at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) 2016 Annual Meeting in June
Initiated a Phase 2 study and expect to enroll the first patient during the second quarter
First Quarter 2016 Financial Results
Collaboration revenue earned under Portola’s collaborations with Bristol-Myers Squibb Company and Pfizer, Bayer Pharma and Janssen Pharmaceuticals, Daiichi Sankyo and Lee’s Pharmaceutical was $8.3 million for the first quarter of 2016 compared with $2.4 million for the first quarter of 2015. The increase in revenue was primarily the result of achieving certain milestones from Portola’s Daiichi Sankyo and Bayer and Janssen clinical agreements with the filing of the ANDEXXA BLA.

Total operating expenses for the first quarter of 2016 were $73.6 million compared with $48.9 million for the same period in 2015. Total operating expenses for the first quarter of 2016 included $7.1 million in stock-based compensation expense compared with $5.2 million for the same period in 2015.

Research and development expenses were $58.8 million for the first quarter of 2016 compared with $39.9 million for the first quarter of 2015 as Portola continued to support its manufacturing scale-up of ANDEXXA in preparation for commercial launch and work on its larger-scale Generation 2 manufacturing process at Lonza, the Phase 3b/4 ANNEXA-4 study of ANDEXXA, and the Phase 1/2a clinical study of cerdulatinib.

Selling, general and administrative expenses for the first quarter of 2016 were $14.8 million compared with $9.0 million for the same period in 2015 as the Company increased headcount to support its growth and increased pre commercial launch activities, including hiring key regional sales directors and national account managers and further developing medical affairs.

For the first quarter of 2016, Portola reported a net loss of $65.0 million, or $1.15 net loss per share, compared with a net loss of $46.9 million, or $0.95 net loss per share, for the same period in 2015.

As of March 31, 2016, cash, cash equivalents and investments totaled $421.0 million compared with cash, cash equivalents and investments of $460.2 million as of December 31, 2015.

2016 Annual Financial Guidance
For the fiscal year 2016, Portola expects total pro-forma operating expenses to be between $295 million and $320 million, excluding stock-based compensation. These expenses will be primarily in support of submissions of Generation 1 and Generation 2 manufacturing scale-up of ANDEXXA in preparation for commercial launch, the ongoing ANNEXA-4 study of ANDEXXA, the U.S. launch of ANDEXXA, the submission of NDA and MAA for betrixaban, pending regulatory discussions, and the Phase 2 clinical study of cerdulatinib.

Non-GAAP Financial Projection
This press release and the reconciliation table included herein include a non-GAAP projection of 2016 operating expenses, excluding stock-based compensation. A reconciliation to projected GAAP 2016 operating expenses is provided in the accompanying table entitled "Reconciliation of GAAP to Non-GAAP Projected Operating Expenses." Portola management believes this non-GAAP information is useful for investors because it provides information about the Company’s ability to independently advance its assets.

On May 05, 2016 Celldex Therapeutics, Inc. (NASDAQ:CLDX) reported business and financial highlights for the first quarter ended March 31, 2016 (Press release, Celldex Therapeutics, MAY 5, 2016, View Source [SID:1234511977]).

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"While the recent setback of the RINTEGA program was certainly a disappointment, the Company is focused on executing across the breadth and depth of our pipeline, including six ongoing company-led clinical trials—the pivotal METRIC study in triple negative breast cancer, two Phase 2 studies across a broad range of indications and multiple Phase 1/2 studies that are actively enrolling patients," said Anthony Marucci, Co-founder, President and Chief Executive Officer of Celldex Therapeutics.

"To this end, Celldex and our collaborators presented seven posters at the recent AACR (Free AACR Whitepaper) meeting across multiple compounds in our pipeline and introduced a new preclinical program that has identified promising agonist antibodies targeting the CD40 receptor. Most importantly, we reported favorable safety and immune monitoring data from the Phase 1 study of varlilumab and Opdivo. The Phase 2 study enrolled its first patients last month and will continue to add to a wealth of data slated for presentation later in 2016 and throughout 2017, including the presentation of Phase 2 data from the glembatumumab vedotin study in metastatic melanoma later this year. With the recent realignment of our pipeline, we believe our current cash position will carry us through the first half of 2018, allowing us to read out all current ongoing studies and to initiate several new studies, as well," concluded Marucci.

Program Updates:

RINTEGA ("rindopepimut"; "rindo"; CDX-110), an EGFRvIII(v3)-specific therapeutic vaccine for glioblastoma (GBM)

In March, Celldex announced that the independent Data Safety and Monitoring Board (DSMB) determined, based on a preplanned interim analysis, that continuation of the Phase 3 ACT IV study of RINTEGA in patients with newly diagnosed EGFRvIII-positive glioblastoma would not reach statistical significance for overall survival in patients with minimal residual disease, the primary endpoint of the study, as both the RINTEGA arm and the control arm were performing on par with each other. In the ACT IV study, RINTEGA performed consistently with prior Phase 2 studies, but the control arm significantly outperformed expectations. Based on this recommendation, Celldex discontinued the study and does not anticipate incurring substantial additional costs related to RINTEGA at this time. The Company is conducting an analysis of the data and plans to present the study at a future scientific/medical meeting or in a peer-reviewed publication. All patients on the RINTEGA arm of the ACT IV study, prior Phase 2 studies and existing compassionate use recipients have been offered ongoing access to RINTEGA on a compassionate use basis.
Glembatumumab vedotin ("glemba"; CDX-011), an antibody-drug conjugate (ADC) targeting gpNMB in multiple cancers

Enrollment continues in the Company’s Phase 2b randomized study (METRIC) of glembatumumab vedotin in patients with metastatic triple negative breast cancers that overexpress gpNMB, a molecule associated with poor outcomes for triple negative breast cancer patients and the target of glembatumumab vedotin. Enrollment is open across the United States, Canada, and Australia and recently opened in the European Union, with the goal of completing enrollment by year-end 2016.
Patient enrollment is complete in the Phase 2 single-agent study of glembatumumab vedotin in metastatic melanoma. The Company is currently amending the protocol to add a second cohort of patients to a glembatumumab vedotin and varlilumab combination arm to assess the potential clinical benefit of the combination and to explore varlilumab’s potential biologic and immunologic effect when combined with an ADC. The Company expects to present data from the single-agent cohort at an appropriate medical meeting in the second half of 2016.
Celldex is also evaluating glembatumumab vedotin in other cancers in which gpNMB is expressed.
Celldex has entered into a collaborative relationship with PrECOG, LLC, which represents a research network established by the Eastern Cooperative Oncology Group (ECOG), and PrECOG, LLC is conducting a Phase 1/2 study in squamous cell lung cancer. This study opened to enrollment in April 2016.
Celldex and the National Cancer Institute (NCI) have entered into a Cooperative Research and Development Agreement (CRADA) under which the NCI is sponsoring two studies of glembatumumab vedotin—one in uveal melanoma and one in pediatric osteosarcoma. Both studies are currently open to enrollment.
Data enhancing the understanding of glembatumumab vedotin’s mechanism of action and further validation of the overexpression of its target, gpNMB, in a wide range of tumor types were presented at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2016 in April. Using a validated immunohistochemistry (IHC) assay to detect the expression of gpNMB, the Company examined tissues from multiple types of solid tumors and normal tissue. Overexpression of gpNMB in samples of tumor tissue versus normal tissue was found in squamous cell carcinoma of the lung (85%), osteosarcoma (62%), pancreatic cancer (55%), lung adenocarcinoma (45%) and squamous cell carcinoma of the head and neck (40%). These results support the potential broad applicability of gpNMB as a therapeutic target across a wide range of tumor types. In addition, in a preclinical study investigating resistance mechanisms in melanoma, glembatumumab vedotin demonstrated synergies with therapies for BRAF mutated melanoma and overcame phenotypes associated with resistance, suggesting use of glembatumumab vedotin may be particularly effective as a single-agent or in combination in this refractory patient population.
Varlilumab ("varli"; CDX-1127), a fully human monoclonal agonist antibody that binds and activates CD27, a critical co-stimulatory molecule in the immune activation cascade

The Phase 2 portion of the varlilumab and nivolumab (Opdivo) study opened to enrollment in April 2016. The study includes cohorts in advanced non-small cell lung cancer (n=35), colorectal cancer (n=18), ovarian cancer (n=18), head and neck squamous cell carcinoma (n=18), renal cell carcinoma (n=25) and glioblastoma (n=20). The study is being conducted by Celldex under a clinical trial collaboration with Bristol-Myers Squibb Company. The companies are sharing development costs.
Data were presented from the Phase 1 portion of the varlilumab and nivolumab study in a poster at the AACR (Free AACR Whitepaper) Annual Meeting in April 2016. The Phase 1 portion of the study, conducted in patients with solid tumors, has completed enrollment (n=36) and primarily enrolled patients with colorectal and ovarian cancer. The primary objective of the Phase 1 portion of the study was to evaluate the safety and tolerability of the combination.
The combination showed acceptable tolerability and safety across all dose levels without any evidence of increased autoimmunity or inappropriate immune activation.
Marked changes in the tumor microenvironment including increased infiltrating CD8+ T cells and increased PD-L1 expression, which have been shown to correlate with a greater magnitude of treatment effect from checkpoint inhibitors in other clinical studies, were observed.
Additional favorable immune biomarkers, such as increase in inflammatory chemokines and decrease in T regulatory cells, were also noted.
In a subset of patients (n=17) on study who had both pre- and post-tumor biopsies available, preliminary evidence suggest a correlation between biomarker data and stable disease or better in seven of these patients (4 ovarian cancer, 2 colorectal cancer, 1 squamous cell carcinoma of the head and neck).
The Phase 1/2 study of varlilumab and atezolizumab (anti-PDL1) is currently enrolling patients with multiple solid tumors in the dose escalation Phase 1 portion of the study. The Phase 2 portion of the study will be conducted in renal cell carcinoma. This study is being conducted by Celldex under a clinical trial collaboration with Roche. Roche is providing study drug, and Celldex is responsible for conducting and funding the study.
Additional combination studies of varlilumab continue to enroll patients including:
A Phase 1/2 safety and tolerability study examining the combination of varlilumab and sunitinib (Sutent) in patients with metastatic clear cell renal cell carcinoma (CC-RCC).
A Phase 1/2 safety and tolerability study examining the combination of varlilumab and ipilimumab (Yervoy) in patients with stage III or IV metastatic melanoma. In the Phase 2 portion of the study, patients with tumors that express NY-ESO-1 will also receive Celldex’s CDX-1401, an NY-ESO-1-antibody fusion protein for immunotherapy.
CDX-1401, an NY-ESO-1-antibody fusion protein for immunotherapy

As discussed above, a Phase 1/2 study examining the combination of varlilumab and ipilimumab continues to enroll patients with stage III or IV metastatic melanoma. In the Phase 2 portion of the study, patients with tumors that express NY-ESO-1 will also receive CDX-1401.
Celldex continues to support several external collaborations, including an NCI sponsored Phase 2 study of CDX-1401 and CDX-301 for patients with metastatic melanoma, which has completed enrollment. Based on results to date, plans for additional studies are being considered by NCI. Additionally, Roswell Park Cancer Center is conducting an investigator sponsored study evaluating CDX-1401, poly-ICLC (Hiltonol) and the IDO1 inhibitor epacadostat (INCB24360) in patients in remission with ovarian, fallopian tube or primary peritoneal cancer. Patients’ tumors must have expressed NY-ESO-1 or the LAGE-1 antigen to be eligible for the study. Celldex is providing CDX-1401 and poly-ICLC in support of this study.
CDX-301 (recombinant human Flt3L), a potent hematopoietic cytokine that uniquely expands dendritic cells and hematopoietic stem cells

The Company presented early data from the pilot study of CDX-301 alone and in combination with plerixafor (Mozobil) in hematopoietic stem cell transplantation (HSCT) in February at the annual meeting of the American Society for Blood and Marrow Transplantation (ASBMT). Data on three donor/patient pairs from the non-plerixafor treated arm showed that CDX-301 given as a single agent was well tolerated and effective at mobilizing hematopoietic stem cells in healthy donors. The stem cell graft contained notable increases in naïve lymphocytes and plasmacytoid dendritic cells consistent with preclinical data suggesting a possible better outcome. Recipients experienced successful engraftment in an expected time frame. Given that hematopoietic stem cell transplantation is outside of Celldex’s core focus, in an effort to prioritize human and capital resources, the Company has decided not to advance CDX-301 in this particular indication at this time and instead to focus near-term efforts on its potential role in combination immunotherapy.
CDX-301’s potential activity is being explored in a Phase 1/2 study of CDX-301 and poly-ICLC in combination with low-dose radiotherapy in patients with low-grade B-cell lymphomas conducted by the Icahn School of Medicine at Mount Sinai.
First Quarter 2016 Financial Highlights and Updated 2016 Guidance

Cash position: Cash, cash equivalents and marketable securities as of March 31, 2016 were $254.0 million compared to $289.9 million as of December 31, 2015. The decrease was primarily driven by our first quarter cash used in operating activities of approximately $34.9 million. As of March 31, 2016 Celldex had 98.7 million shares outstanding.

Revenues: Total revenue was $1.3 million in the first quarter of 2016, compared to $0.5 million for the comparable period in 2015. The increase in revenue was primarily due to our clinical trial collaboration with Bristol-Myers Squibb, our research and development agreement with Rockefeller University and an increase in grant revenue.

R&D Expenses: Research and development (R&D) expenses were $27.4 million in the first quarter of 2016, compared to $25.1 million for the comparable period in 2015. The increase in R&D expenses was primarily attributable to increased headcount.

G&A Expenses: General and administrative (G&A) expenses were $9.3 million in the first quarter of 2016, compared to $6.1 million for the comparable period in 2015. The increase in G&A expenses was primarily due to higher stock-based compensation of $1.1 million, increased headcount, and RINTEGA and glembatumumab vedotin commercial planning costs.

Net loss: Net loss was $34.7 million, or ($0.35) per share, for the first quarter of 2016, compared to a net loss of $30.2 million, or ($0.33) per share, for the comparable periods in 2015.

Financial guidance: Celldex expects that its cash, cash equivalents and marketable securities will be sufficient to fund our operating expenses and capital expenditure requirements through the first half of 2018.

RINTEGA is a registered trademark of Celldex Therapeutics. Opdivo and Yervoy are registered trademarks of Bristol-Myers Squibb. Sutent is a registered trademark of Pfizer. Mozobil is a registered trademark of sanofi-aventis U.S. LLC. Hiltonol is a registered trademark of Oncovir.

Caladrius Biosciences, Inc. (NASDAQ: CLBS) ("Caladrius"), a cell therapy company combining an industry-leading development and manufacturing services provider (through its subsidiary, PCT, LLC a Caladrius CompanyTM ("PCT")) with a select therapeutic development pipeline, announces financial results for the three months ended March 31, 2016.

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Financial and Business Highlights

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Achieved total revenues of $7.5 million for the first quarter of 2016, up 136% compared with $3.2 million in the first quarter of 2015 driven by higher Clinical Services revenue at PCT.
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Entered into a global collaboration and license agreement with Hitachi Chemical Co. America, Ltd. and Hitachi Chemical Co., Ltd. (collectively, "Hitachi Chemical"), selling a 19.9% equity stake in PCT for $19.4 million and licensing PCT’s cell therapy technology and know-how for certain Asian territories for $5.6 million and future royalties.
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Enrolled the first patient in The Sanford Project: T-Rex Study, a Phase 2 trial of CLBS03 (autologous expanded regulatory T cells, or Tregs) for the treatment of recent-onset type 1 diabetes (T1D) in adolescents.
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Received classification from the European Medicines Agency (EMA) of CLBS03 as an Advanced Therapeutic Medicinal Product (ATMP).
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Reached agreement with Japanese regulators on a Phase 2 development plan that could qualify for early conditional approval for CD34 cell therapy as a treatment for critical limb ischemia.

Management Commentary

"The significant revenue growth at PCT along with Hitachi Chemical’s implied valuation of our subsidiary further support our strategy to focus on growth opportunities in the emerging cell therapy manufacturing market," stated David J. Mazzo, PhD, Chief Executive Officer of Caladrius. "In addition to validating our expertise and know-how, the strategic partnership with Hitachi Chemical strengthens our financial position with $25 million in non-dilutive capital.

"We will continue to leverage the significant momentum in the regenerative medicine and cell therapy industries to grow our PCT business. We believe that the quality of PCT’s services, the increasing number of clinical trials planned and underway and the number of clinical programs nearing commercialization will provide a healthy platform for growth at PCT throughout 2016 and beyond.

"We are delighted that patients are being enrolled in the Sanford Project: T-Rex Study. Sanford Research, a leader of innovation and research in T1D, will provide and cover the costs of two initial clinical trial sites, which are expected to enroll most of if not all of the first cohort of subjects. After this first cohort has completed the three-month post-treatment visit, an interim safety analysis and early analysis of immunological biomarkers will be conducted. With positive results, more sites will be added to facilitate the timely enrollment of the second cohort of this important proof-of-concept study designed to show that CLBS03 can preserve pancreatic beta cell function and lower insulin use in adolescents with recent-onset T1D," concluded Dr. Mazzo.

First Quarter Financial Highlights

Total revenues for the first quarter of 2016 increased 136% to $7.5 million compared with $3.2 million for the first quarter of 2015. Gross margin on revenues was 17% in the first quarter of 2016, compared with gross margin of negative 6% in the first quarter of 2015.

Research and development (R&D) expenses for the first quarter of 2016 decreased 14% to $5.9 million compared with $6.8 million for the first quarter of 2015. The decrease was primarily related to lower costs subsequent to the discontinuation of Caladrius’ Intus Phase 3 clinical trial as well as decreased costs associated with our ischemic repair platform, compared to the prior year periods. These decreases were partially offset by an increase in expenses related to the initiation of The Sanford Project: T-Rex Phase 2 Study in type 1 diabetes, as well as one-time restructuring costs for severance and asset impairments.

Selling, general and administrative (SG&A) expenses decreased 42% to $6.5 million for the first quarter of 2016 compared with $11.1 million for the same period in 2015, which included expenses associated with executive management changes including one-time new hire compensation-related costs as well as separation-related costs. Equity-based compensation expenses were also significantly lower in the first quarter of 2016 compared to the prior year period.

The operating loss for the first quarter of 2016 was $11.1 million compared with an operating loss of $18.1 million for the first quarter of 2015, reflecting higher gross margin on sales, and lower R&D and SG&A expenses.

Total net loss for the first quarter of 2016 was $12.0 million, and $0.21 per share for Caladrius stockholders, compared with a net loss for the first quarter of 2015 of $19.2 million and $0.51 per share.

Balance Sheet and Cash Flow Highlights

As of March 31, 2016, Caladrius had cash and cash equivalents of $25.4 million. The cash and cash equivalents balance includes the receipt of $22.5 million from the Hitachi Chemical transaction and the payment of $7.0 million to Oxford Finance LLC for repayment of long-term debt, interest and fees. The net cash used in operating activities in the first quarter of 2016 was $8.0 million, compared with $14.2 million in the first quarter of 2015. During the current quarter, Caladrius also invested $1.0 million in capital expenditures primarily related to improvements to PCT’s Allendale, N.J. manufacturing facility.

2016 Financial Guidance

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Consolidated Revenues: exceed $30 million (greater than 30% increase compared with 2015) (guidance reaffirmed)

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Capital Improvements at PCT’s Allendale, NJ facility: ~$6 million in 2016 (guidance reaffirmed)

•
CLBS03 Phase 2 Study Costs in 2016: $6 million to $7 million (guidance reaffirmed)

•
Consolidated Annual Operating Cash Burn: $25 million to $28 million (new guidance)
(lower operating cash burn in the second half of 2016 than in the first half of the year)

On May 05, 2016 Progenics Pharmaceuticals, Inc. (Nasdaq:PGNX) reported financial and business results for the first quarter 2016 (Press release, Progenics Pharmaceuticals, MAY 5, 2016, View Source [SID:1234512037]).

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Key Business Highlights

PSMA-Targeted Prostate Cancer Pipeline

On May 2nd, the Company Announced It Had Granted Exclusive World-Wide Rights to Bayer to Develop and Commercialize Products Using Progenics’ PSMA Antibody Technology In Combination with Alpha-Emitting Radionuclides. The transaction combines Bayer’s expertise in alpha emitter radiopharmaceuticals with Progenics’ validated PSMA antibody technology to develop a new therapeutic approach for prostate cancer. Under the terms of the agreement, Progenics will receive an upfront payment of $4 million and could receive up to an additional $49 million in potential clinical and regulatory development milestones. The Company is also entitled to single digit royalties on net sales, and potential net sales milestone payments up to an aggregate total of $130 million.

Advancing Pivotal Phase 3 Study of PSMA-Targeted SPEC/CT Imaging Agent 1404 in the U.S. and Canada. The study will enroll approximately 450 patients with newly-diagnosed or low-grade prostate cancer who are candidates for active surveillance who have decided to undergo a radical prostatectomy. Progenics is planning an interim analysis during the second half of 2016 to assess futility and evaluate the need for a sample size re-estimation.

Johns Hopkins University Study Evaluating the Utility of PyL in Men with Elevated PSA Following Radical Prostatectomy to be Presented at the 2016 American Urological Association Annual Meeting, which is being held May 6-10 in San Diego. PSMA-targeted 18F-DCFPyL PET/CT (PyL) appears to be a sensitive imaging modality for detecting prostate cancer recurrence. Progenics plans to meet with the FDA in the second quarter to discuss a phase 2 study design.

Company Remains On-Track to Initiate a Phase 1 Trial of 1095 in the Second Half of 2016. The Phase 1 Study of 1095, a PSMA-Targeted Therapeutic for Metastatic Prostate Cancer, will be conducted at Memorial Sloan Kettering Cancer Center.

AZEDRA, Ultra-orphan radiotherapeutic candidate

AZEDRA Topline Results Expected Between December 2016 and March 2017. In late 2016 or early 2017, Progenics expects to report topline results from its ongoing pivotal Phase 2b study of AZEDRA. If positive, the Company expects to submit an NDA to the FDA during the first half of 2017.

RELISTOR, treatment for opioid-induced constipation (partnered with Valeant Pharmaceuticals International, Inc.)

RELISTOR Net Sales for the First Quarter 2016 Total $16.6 Million. The first quarter 2016 sales, as reported to us by our partner Valeant, translated to $2.2 million in royalty revenue, net of prior year adjustments.

PDUFA Date for Oral RELISTOR Extended to July 19, 2016. The FDA extended the action date to allow for a full review of Valeant’s responses to recent information requests from the FDA. If approved, Progenics would be entitled to a $50 million milestone payment and subsequent royalties and sales milestones from Valeant.

"The Progenics’ portfolio features multiple value-creating opportunities, and we are continuing to drive development of high-priority internal programs while leveraging the potential of our technology through strategic transactions, such as the recent Bayer license agreement," said Mark Baker, Chief Executive Officer of Progenics. "AZEDRA represents a near-term commercial candidate in an ultra-orphan indication, while our portfolio of imaging agents and therapeutics has the potential to transform how prostate cancer is detected, managed and treated. We look forward to progressing toward key milestones over the next several quarters."

First Quarter 2016 Financial Results

Net loss attributable to Progenics for the quarter was $12.7 million or $0.18 per basic and diluted share, compared to a net loss of $10.3 million or $0.15 per basic and diluted share in the 2015 period. Progenics ended the quarter with cash and cash equivalents of $65.7 million, a decrease of $8.4 million in the quarter.

First quarter revenue totaled $2.5 million, up from $0.2 million in the 2015 period, reflecting RELISTOR royalty income of $2.2 million (net of prior year adjustments) compared to $0.1 million in the prior year period, based on net sales reported by Valeant. Net sales in the first quarter of the prior year were impacted by a wholesaler inventory reduction initiative implemented in the fourth quarter of 2014 by Salix Pharmaceuticals, Inc., which was subsequently acquired by Valeant.

First quarter 2016 research and development expenses increased by $2.7 million compared to the prior year period, primarily attributable to higher clinical trial and contract manufacturing expenses for AZEDRA and 1404 and higher compensation expenses, partially offset by clinical trial expenses for PSMA ADC which were incurred in the prior year but not the current period. First quarter 2016 general and administrative expenses increased by $2.1 million compared to the prior year period, primarily attributable to incremental depreciation, which is expected to continue through August 1, 2016, as a result of a reduction in the remaining useful lives of the Company’s leasehold improvements at its Tarrytown, NY location, and higher compensation, consulting, and professional fees. The Company also recorded a non-cash charge of $0.2 million in the first quarter of 2016 related to an increase in the fair value estimate of the contingent consideration liability.

Corporate Update

Progenics also announced today that Dr. Paul Maddon, the founder of the Company, having served on the Company’s Board of Directors for three decades, will serve out his current term as director and retire from the Board as of the Company’s 2016 Annual Meeting of Stockholders on June 8. Mr. Peter Crowley, Chairman of Progenics’ Board of Directors, said, "Paul obviously has played a critical role in founding the Company and contributed greatly as a scientist, as an executive, and as a board member. We celebrate his 30 years of work and achievements, and we wish him well in the further important work he has committed himself to in the future."

"I am proud of Progenics’ scientific achievements and of the new medicines developed and being developed by Progenics to bring benefits to patients around the world. I express my profound thanks to all of my colleagues at Progenics who have been instrumental to the Company’s progress," commented Dr. Maddon.

Progenics announced that Mr. Bradley Campbell, President and Chief Operating Officer of Amicus Therapeutics, Inc., has been nominated for election to the Board of Directors of the Company at the upcoming annual meeting of stockholders. "We are pleased to recommend to the stockholders the election of Bradley Campbell to the Board," Mr. Crowley continued. "Brad brings to Progenics a deep knowledge of the ultra-orphan disease industry. As we look forward to the commercialization of AZEDRA and other products in the Company’s diverse pipeline, I know that Brad’s experience and insights will contribute greatly to the Company’s progress." Please see our 2016 proxy statement for additional information.

On May 5, 2016 Clovis Oncology, Inc. (NASDAQ:CLVS) reported financial results for its quarter ended March 31, 2016, and provided an update on the Company’s clinical development programs and regulatory outlook for the remainder of 2016 (Press release, Clovis Oncology, MAY 5, 2016, View Source;p=RssLanding&cat=news&id=2165717 [SID:1234511978]).

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"We are very disappointed in the outcome for rociletinib, as there is a need for additional options for this difficult to treat disease," said Patrick J. Mahaffy, President and CEO of Clovis Oncology. "Our focus moving forward is clear: prioritize rucaparib development activity and prepare for its potential U.S. launch, and manage our existing cash into 2018."

First Quarter 2016 Financial Results

Clovis had $445.5 million in cash, cash equivalents and available-for-sale securities as of March 31, 2016. Cash used in operating activities was $83.7 million for the first quarter of 2016, compared with $48.4 million in the first quarter of 2015. Clovis had approximately 38.4 million outstanding shares of common stock as of March 31, 2016.

Clovis reported a net loss for the first quarter of 2016 of $83.4 million, or ($2.17) per share, compared to a net loss of $63.1 million, or ($1.86) per share, for the first quarter of 2015. Net loss for the first quarter of 2016 included share-based compensation expense of $11.0 million compared to $8.7 million for the first quarter of 2015.

Research and development expenses totaled $74.6 million for the first quarter of 2016, compared to $56.8 million for the first quarter 2015. The year-over-year increase in expenses is due to the significantly expanded clinical development activities for rucaparib, increased commercial product planning costs and increased personnel-related expenses associated with the hiring of additional staff including the U.S. sales force to support the Company’s expanded activities, partially offset by lower expenses related to clinical development activities for rociletinib.

General and administrative expenses totaled $9.8 million for the first quarter of 2016, compared to $6.8 million for the first quarter 2015. The increase year over year is primarily due to higher legal expense, consulting fees and personnel costs for employees engaged in general and administrative activities.

The Company expects cash used in operating activities for 2016 will total approximately $294 – $309 million, and to end the year with approximately $220 – $235 million in cash, cash equivalents and available-for-sale securities. Clovis anticipates being able to continue to fund operations into 2018 from currently available cash, cash equivalents and available-for-sale securities.

2016 Key Milestones and Objectives

Highlights of planned or completed objectives for each product follow:

Rucaparib

During the second quarter of 2016, Clovis commenced the submission of its rolling New Drug Application (NDA) regulatory filing to the U.S. Food and Drug Administration (FDA) for rucaparib for the monotherapy treatment of patients with advanced ovarian cancer with deleterious BRCA-mutated tumors (inclusive of both germline and somatic BRCA mutations) previously treated with multiple prior therapies. Rucaparib was granted Breakthrough Therapy designation by the FDA in April 2015. Clovis agreed with the FDA that the submission would be a rolling NDA and has filed the first component for potential accelerated approval of rucaparib in the U.S. The rolling NDA allows completed portions of an NDA to be submitted and reviewed by the FDA on an ongoing basis. The Company intends to complete the NDA submission by the end of the second quarter of 2016.

Foundation Medicine, Clovis’ companion diagnostic partner, intends to file a Premarket approval application (PMA) of its diagnostic assay designed to identify both germline and somatic BRCA mutations with the FDA. The timing of the submission is expected to allow for regulatory approval of the companion diagnostic at substantially the same time that rucaparib would be approved.

In addition, the Company intends to submit its Marketing Authorization Application (MAA) for rucaparib to the European Medicines Agency for a comparable ovarian cancer treatment indication in Q4 2016.

We have completed target enrollment in the ARIEL3 pivotal maintenance study, with data expected to be available in approximately 12 months. Pending positive data, Clovis intends to follow up with supplemental NDAs for maintenance indications in tumor BRCA mutant patients and BRCA-like patients.

Clovis recently entered into a clinical trial collaboration with Genentech, a member of the Roche Group, to evaluate a novel combination therapy of Genentech’s investigational cancer immunotherapy atezolizumab (MPDL3280A; anti-PDL1) and rucaparib for the treatment of gynecological cancers, with a focus on ovarian cancer. The Phase 1b trial is planned to begin enrolling patients during the second half of 2016.

Also during the second half of 2016, the Company intends to initiate a study of rucaparib in metastatic castrate-resistant BRCA mutant (inclusive of germline and somatic) prostate cancer patients, as well as the ARIEL4 confirmatory study in advanced ovarian cancer.

Rociletinib

In a recent meeting with the FDA, Clovis was notified that it could anticipate receiving a Complete Response Letter (CRL) for the rociletinib NDA on or before the PDUFA date of June 28, 2016. The FDA issues a CRL to indicate that their review of an application is complete and that the application is not ready for approval. In anticipation of receiving the CRL, Clovis has terminated enrollment in all ongoing sponsored clinical studies of rociletinib. Clovis will continue to provide drug to patients whose clinicians recommend continuing rociletinib therapy. In addition, Clovis has withdrawn its MAA for rociletinib previously filed with European regulatory authorities. Related to terminating enrollment in all ongoing sponsored clinical studies of rociletinib, Clovis is reducing its staff, eliminating contractor positions and delaying or eliminating planned new positions. This will result in the reduction of our staff and contractor positions by 35 percent by the end of 2016, compared to year-end 2015.

However, we intend to maintain the U.S. sales force in preparation for the potential U.S. launch of rucaparib. Clovis has determined there would be effectively no cost savings in eliminating the U.S. sales force and replacing it with a contract organization to support the potential U.S. launch of rucaparib in Q4 2016 or Q1 2017. In addition, a decision to use a contract sales organization could potentially delay the timing of the U.S. launch.

Lucitanib

Enrollment was completed during the first quarter in the ongoing Phase 2 study exploring lucitanib in patients with treatment-refractory breast cancer. In parallel with Clovis’ sponsored study, a Servier-sponsored Phase 2 study of lucitanib in patients with advanced breast cancer is underway to identify the population of patients most likely to benefit from lucitanib therapy. The Company expects to make a decision regarding the future development of lucitanib by the end of 2016.

About Rucaparib

Rucaparib is an oral, potent small molecule inhibitor of PARP1-3 being developed for the treatment of ovarian cancer, specifically in patients with tumors with BRCA mutations and other DNA repair deficiencies beyond BRCA, including those with high genomic loss of heterozygosity (LOH) commonly referred to as "BRCA-like." Clovis is also exploring rucaparib in other solid tumor types with significant BRCA and BRCA-like populations, including prostate, breast and gastroesophageal cancers. Rucaparib was granted Breakthrough Therapy designation by the U.S. FDA in April 2015. Clovis holds worldwide rights for rucaparib.

About Rociletinib

Rociletinib is an oral, mutant-selective inhibitor of epidermal growth factor receptor (EGFR). Rociletinib targets the activating mutations of EGFR (L858R and Del19), while also inhibiting the dominant acquired resistance mutation, T790M. Clovis holds worldwide rights for rociletinib.

About Lucitanib

Lucitanib is an oral, potent inhibitor of the tyrosine kinase activity of vascular endothelial growth factor receptors 1 through 3 (VEGFR1-3), platelet-derived growth factor receptors alpha and beta (PDGFRα-β) and fibroblast growth factor receptors 1 through 3 (FGFR1-3). Clovis, which holds exclusive U.S. and Japanese rights, is collaborating with its development partner Les Laboratoires Servier (Servier) on the global clinical development of lucitanib outside of China, initially targeting advanced breast cancer.