On February 19, 2019 Acorda Therapeutics, Inc. (Nasdaq:ACOR) reported that Andrew Hindman, Acorda’s Chief Business Officer, will present at the Leerink Global Healthcare Conference on Wednesday, February 27 at 11:00 a.m. EST (Press release, Acorda Therapeutics, FEB 19, 2019, View Source [SID1234533465]). A live audio webcast of the presentation can be accessed under "Investor Events" in the Investor section of the Acorda website at www.acorda.com, or you may use the link:

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On February 19, 2019 Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare genetic diseases, reported its financial results and corporate update for the quarter and full year ended December 31, 2018 (Press release, Ultragenyx Pharmaceutical, FEB 19, 2019, View Source [SID1234533390]).

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"This last year was important for Ultragenyx as we successfully launched two therapies internationally and validated clinical data from our gene therapy platform," said Emil D. Kakkis, M.D., Ph.D., Chief Executive Officer and President of Ultragenyx. "In 2019 we look forward to expanding the global commercial reach of our approved therapies, submitting a New Drug Application for a third, and advancing our gene therapy platform toward pivotal studies."

Fourth Quarter and Full Year 2018 Financial Results

Net Revenues

For the fourth quarter of 2018, Ultragenyx reported $16.3 million in total revenue. Ultragenyx recognized $11.6 million in total Crysvita revenue. This includes $9.9 million in collaboration revenue in the U.S. profit share territory and $1.3 million in royalty revenue in the European territory from the collaboration and license agreement with Kyowa Hakko Kirin. Net product sales for Crysvita in other regions were $0.4 million. Mepsevii (vestronidase alfa) product revenue for the fourth quarter of 2018 was $2.7 million, and UX007 named patient revenue was $0.5 million. Ultragenyx recognized $1.6 million in revenue from its research agreement with Bayer.

Net revenue for the year ended December 31, 2018 totaled $51.5 million. Since launching Crysvita on April 27, 2018 through the end of the year, Ultragenyx recognized $18.9 million in total Crysvita revenue. This includes $15.3 million in collaboration revenue in the U.S. profit share territory and $2.9 million in royalty revenue in the European territory from the collaboration and license agreement with Kyowa Hakko Kirin. Net product sales for Crysvita in other regions totaled $0.6 million. Mepsevii product revenue for the year ended December

31, 2018 was $7.9 million and UX007 named patient revenue was $1.3 million. Ultragenyx recognized $23.5 million in revenue from its research agreement with Bayer in the year ended December 31, 2018.

Operating Expenses

Total operating expenses for the fourth quarter of 2018 were $106.6 million compared with $99.2 million for the same period in 2017, including non-cash stock-based compensation of $21.1 million and $19.5 million in the fourth quarter of 2018 and 2017, respectively. Total operating expenses for the year ended December 31, 2018, were $422.9 million compared with $331.6 million for the same period in 2017, including non-cash stock-based compensation of $80.1 million and $68.0 million in the full year of 2018 and 2017, respectively. The increase in total operating expenses is due to the increase in commercial, development, and general and administrative costs as the company commercializes, grows and advances its pipeline.

For the fourth quarter of 2018, Ultragenyx reported a net loss of $87.8 million, or $1.73 per share, basic and diluted, compared with a net loss for the fourth quarter of 2017 of $81.7 million, or $1.89 per share, basic and diluted. For the year ended December 31, 2018, net loss was $197.6 million, or $3.97 per share, basic and diluted, compared with a net loss for the same period in 2017 of $302.1 million, or $7.12 per share, basic and diluted. The loss from the full year 2018 was reduced by the sale of the Mepsevii (vestronidase alfa) priority review voucher (PRV) in January 2018 for net proceeds of $130.0 million and a $40.3 million gain from Ultragenyx’s portion of the sales of the PRV received with the Crysvita (burosumab) approval. The net loss for the full year 2018 reflected cash used in operations of $290.6 million compared to $253.8 million for the same period in 2017.

Cash, Cash Equivalents and Investments

Cash, cash equivalents and investments were $459.7 million as of December 31, 2018.

Recent Updates

Crysvita in X-Linked Hypophosphatemia (XLH)

Longer-term data from the Phase 3 pediatric study demonstrated superior efficacy of Crysvita compared with conventional oral phosphate treatment. After 64 weeks of treatment, patients treated with Crysvita continued to show significantly greater improvement in healing of rickets, growth, and bowing of the legs compared with patients treated with oral phosphate and active Vitamin D, which was the standard of care for over 30 years. The 64-week safety profile was similar to that observed at 40 weeks and in other Crysvita pediatric XLH studies.

Crysvita was approved and launched in Canada for the treatment of XLH in adults and pediatric patients one year of age and older. Canada is included in the North American profit share agreement with our partner Kyowa Hakka Kirin.

UX007 in Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD)

Positive topline data from ongoing long-term extension study of UX007 for the treatment of LC-FAOD showed sustained reductions in the duration and frequency of major clinical events (MCEs), primarily hospitalizations for hypoglycemia, cardiomyopathy, and rhabdomyolysis. Patients who had previously been enrolled in the Company-sponsored Phase 2 study (n=24) demonstrated sustained reductions in MCEs after an additional 78 weeks of treatment, and 20 additional patients who had not previously received UX007 demonstrated similar substantial reductions in these MCEs with treatment, further corroborating prior results. The safety profile was consistent with what has been previously observed with UX007.

DTX401 Gene Therapy in Glycogen Storage Disease Type Ia (GSDIa)

Positive topline results from the first cohort of the Phase 1/2 clinical study of DTX401 gene therapy in GSDIa. All three patients demonstrated a clinical response, reflected by improved glucose control, increased time to hypoglycemia during fasting and reductions in necessary cornstarch dosing. Two patients demonstrated a clinically meaningful improvement in time to hypoglycemia, making it possible to sleep through the night. Typically, for GSDIa patients, cornstarch or tube feeding is required during the night to prevent severe hypoglycemia, which can cause seizures or death. There were no infusion-related adverse events and no treatment-related serious adverse events reported.

Upcoming Key Milestones

UX007 in LC-FAOD

Ultragenyx is on track to submit a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) in mid-2019. The submission will include data from the company-sponsored Phase 2 study in 29 patients, data from the long-term efficacy and safety extension study in 75 patients, a retrospective medical record review of 20 original compassionate use patients, data from 70 patients treated through expanded access, and a randomized controlled investigator-sponsored study of 32 patients showing an effect of UX007 on cardiac function.

DTX301 Gene Therapy in Ornithine Transcarbamylase (OTC) Deficiency

Enrollment is ongoing in the third-dose cohort of the Phase 1/2 study. Cohort 3 data expected in mid-2019.

DTX401 Gene Therapy in GSDIa

Enrollment is ongoing in the second-dose cohort of the Phase 1/2 study. Cohort 2 data expected in mid-2019.

Corporate

Ultragenyx plans to host an Analyst Day on Wednesday, April 17. The event will provide updates and expert commentary on commercial, clinical stage and early pipeline programs. Additional details to come.

Conference Call and Webcast Information

Ultragenyx will host a conference call today, Tuesday, February 19, 2018, at 2 p.m. PT/ 5 p.m. ET to discuss fourth quarter and full year 2018 financial results and provide a corporate update. The live and replayed webcast of the call will be available through the company’s website at View Source To participate in the live call by phone, dial (855) 797-6910 (USA) or (262) 912-6260 (international) and enter the passcode 6689186. The replay of the call will be available for one year.

On February 19, 2019 Alkermes plc (Nasdaq: ALKS) and Clovis Oncology, Inc. (Nasdaq: CLVS) reported that the companies have entered into a research collaboration to evaluate ALKS 4230, Alkermes’ investigational engineered interleukin-2 (IL-2) variant immunotherapy, in combination with rucaparib, Clovis’ marketed PARP inhibitor, and lucitanib, Clovis’ investigational tyrosine kinase inhibitor (Press release, Clovis Oncology, FEB 19, 2019, View Source [SID1234533389]). The collaboration will explore the potential anti-cancer effects of both treatment combinations in preclinical models across multiple tumor types. Results of this research may form the basis for potential future clinical studies of the novel combinations of ALKS 4230 with rucaparib and/or lucitanib.

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"Our preclinical partnership with Clovis reflects our ongoing efforts to explore the numerous combination options afforded to ALKS 4230. The collaboration will allow us to examine combinations in two areas of keen interest, PARP and tyrosine kinase inhibition pathways," said Mark Namchuk , Ph.D., Senior Vice President, Research, Pharmaceutical and Non-Clinical Development at Alkermes. "Evidence of combined benefit of ALKS 4230 with rucaparib and/or lucitanib from these preclinical studies may provide a strong rationale to advance into clinical development."

"The unique profiles of rucaparib, lucitanib and ALKS 4230 may offer the potential for complementary therapies that could represent a meaningful opportunity for the development of new anti-cancer combination treatment options," said Patrick J. Mahaffy , President and CEO of Clovis Oncology . "We are committed to exploring combinations such as these with Alkermes in order to bring improved therapeutic outcomes to patients with multiple tumor types."

Under this collaboration agreement, Alkermes and Clovis will perform preclinical studies to examine the mechanism of action and efficacy of the combinations of ALKS 4230 with rucaparib and ALKS 4230 with lucitanib in multiple tumor models. Under the terms of the agreement, the companies will share costs related to the preclinical studies, and each will contribute their respective compounds to the research collaboration.

About ALKS 4230
ALKS 4230 is an investigational, novel, engineered fusion protein designed to selectively activate tumor-killing immune cells while avoiding the expansion of immunosuppressive cells by preferentially binding to the intermediate-affinity interleukin-2 (IL-2) receptor complex. The selectivity of ALKS 4230 is designed to leverage the proven anti-tumor effects of existing IL-2 therapy while mitigating certain limitations.

About Rucaparib
Rucaparib is an oral, small molecule inhibitor of the poly (ADP-ribose) polymerase (PARP) enzymes PARP-1, PARP-2, and PARP-3, which play a role in DNA repair. Rucaparib stimulates an anti-tumor immune response through the activation of the stimulator of interferon ( STING ) pathway and tumor cell death, resulting in the infiltration of multiple immune subsets including CD8 positive T-cells. 1

Rucaparib is being developed in multiple tumor types, including ovarian, metastatic castration-resistant prostate, and bladder cancers, as monotherapy, and in combination with other anti-cancer agents. Exploratory studies in other tumor types are also underway. Clovis holds worldwide rights for rucaparib. Rucaparib is an unlicensed medical product outside of the U.S. and Europe .

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 (PDFGRα/β) and fibroblast growth factor receptors 1 through 3 (FGFR1-3).

Emerging clinical data support the combination of angiogenesis inhibitors and immunotherapy to increase effectiveness in multiple cancer indications. 2 Angiogenic factors, such as vascular endothelial growth factor (VEGF), are frequently up-regulated in tumors and create an immunosuppressive tumor microenvironment. 3 Use of antiangiogenic drugs reverses this immunosuppression and can augment response to immunotherapy.

On February 19, 2019 NEW ORLEANS & Ochsner Health System, Louisiana’s largest non-profit academic healthcare system, and Pfizer Inc (NYSE:PFE) reported that have entered into a multi-year strategic alliance to develop innovative models for clinical trials (Press release, Pfizer, FEB 19, 2019, View Source [SID1234533388]). Through this partnership, Pfizer and Ochsner — through its innovation lab, innovationOchsner (iO), in partnership with Ochsner Research — will explore ways to enhance the clinical trial experience and ease participation in clinical research for both patients and healthcare professionals.

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The alliance aims to create faster, improved access and connectivity to clinical trials for patients, with the ultimate goal of better experiences and outcomes. Participating patients will have the opportunity to test out new digital tools designed to make the clinical trial experience more inclusive and enjoyable. Participating clinicians will benefit from reduced manual data entry as a result of direct data system integration and automated study conduct tools, freeing up time and work to allow them to offer clinical research as an option to a broader range of patients. And research groups will experience efficiencies from interoperability that may lower costs while increasing capacity. These projects are designed to ensure that all data collected is secure and will only be used with patient consent, as in any clinical trial.

"As a national leader in healthcare, Ochsner is delivering on its mission to bring the most innovative ideas to our patients. We are relentless about using the latest breakthroughs in science and technology to solve some of the toughest healthcare challenges," said Dr. Richard Milani, Ochsner’s Chief Clinical Transformation Officer and Medical Director of iO. "Partnering with Pfizer allows us to transform medicine by creating a digital clinical trial experience that improves patient participation, integration, communication and accessibility."

The strategic alliance’s first project began with a "proof of value" phase in which researchers successfully transferred mock data (e.g. no actual patient data was used or shared) from Ochsner’s EHR system to Pfizer’s electronic data capture system used in Pfizer clinical trials. The experiment aimed at understanding the gaps and variances between data collected in electronic health records and patient-reported data from clinical trials.

Since the inception of electronic health records (EHRs), integrating EHR data into clinical trial databases has been a goal of research institutions, industry and regulators. Such integration would reduce the burden of manual data entry, save time, decrease cost and accelerate clinical trials. The ongoing challenge has been exchanging data between healthcare systems and clinical trial systems, because each uses different technology platforms and data standards. In recent years, Fast Healthcare Interoperability Resources, or FHIR, a standard developed by the nonprofit organization Health Level Seven International (HL7), has been introduced as a secure and open solution for healthcare data exchange to accelerate information sharing. Most health apps use FHIR data standards, but FHIR has yet to be adopted for data exchange in industry-sponsored clinical trials. As one of the first health systems to implement FHIR, Ochsner has continuously explored opportunities to use the standards in other applications, including clinical trials. The Ochsner-Pfizer alliance project aims to build a model for applying FHIR standards so that high-quality clinical trial data may be collected consistently, reliably and securely within hospital and clinic electronic systems.

"We’re pleased that we succeeded in transferring core data types collected in healthcare provider electronic health records to Pfizer’s clinical trial data capture system using FHIR standards. To the best of our knowledge, this is a first for our industry," explains Rob Goodwin, Vice President, Operations Center of Excellence, Global Product Development at Pfizer. "There is more work ahead, but this is a significant step forward in simplifying data capture for clinical trials, and the first of many pioneering solutions we hope to develop through our partnership with Ochsner."

During the next phase, Pfizer and Ochsner will continue to develop new ways to digitize the patient and clinician experience in clinical trials, with attention to patient preferences on access to and use of their health data and with an overarching goal of enhancing the quality and value of clinical research interactions for all participants.

On February 19, 2019 Pfizer Inc. (NYSE: PFE) reported the European Commission (EC) has approved ZIRABEV for the treatment of metastatic carcinoma of the colon or rectum, metastatic breast cancer, unresectable advanced, metastatic or recurrent non-small cell lung cancer (NSCLC), advanced and/or metastatic renal cell cancer and persistent, recurrent or metastatic carcinoma of the cervix.1,2 (Press release, Pfizer, FEB 19, 2019, View Source [SID1234533387])

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"Pfizer is dedicated to increasing access to biosimilars for patients suffering from serious illnesses and helping create a more sustainable healthcare system," said Andreas Penk, M.D., regional president, Oncology International Developed Markets at Pfizer. "We are proud that ZIRABEV was approved today as our second oncology biosimilar in Europe. This milestone reflects our ongoing commitment to biosimilars as we continue to bring high-quality medicines to market that may help generate cost savings for cancer care."

* Avastin is a registered trademark of Genentech

The EC approval is based on a comprehensive submission package which demonstrated biosimilarity of ZIRABEV and the originator product. This includes results from the phase 3 REFLECTIONS B739-03 clinical comparative study, which showed clinical equivalence and found no clinically meaningful differences between ZIRABEV and the originator product in patients with advanced non-squamous NSCLC.3 As part of the overall REFLECTIONS clinical trial program, ZIRABEV has been studied in approximately 400 subjects.3,4

This approval follows the positive recommendation from the Committee for Medicinal Products for Human Use in December 2018.5 ZIRABEV has also been filed for regulatory approval with the U.S. Food and Drug Administration.

Pfizer has a robust portfolio of potential biosimilar candidates in mid- to late-stage development.6 ZIRABEV is Pfizer’s fifth biosimilar approved for use in Europe.1,7, 8,9,10

About ZIRABEV (bevacizumab biosimilar)

ZIRABEV is a monoclonal antibody (mAb) biosimilar of the originator biologic medicine, Avastin, which works by inhibiting the formation of new blood cells (angiogenesis) by specifically recognizing and binding to vascular endothelial growth factor (VEGF) protein.11, 12,13 ZIRABEV has been studied in nearly 400 patients.3,4

ZIRABEV safety information

Do not use ZIRABEV if you are allergic to bevacizumab, any of its ingredients, Chinese hamster ovary (CHO) cell products, or other recombinant human or humanised antibodies, or if you are pregnant.

Before starting treatment with ZIRABEV, tell your healthcare provider if:

you have any conditions causing inflammation inside the abdomen (e.g. diverticulitis, stomach ulcers, colitis associated with chemotherapy)
you have persistent, recurrent or metastatic cervical cancer
you are going to have an operation, if you have had major surgery within the last 28 days or if you still have an unhealed wound following surgery
you had holes in the gut wall or problems with wound healing
you have high blood pressure which is not well controlled with blood pressure medicines
you are over 65 years old, if you have diabetes, or if you have had previous blood clots in your arteries
you or your family tend to suffer from bleeding problems or you are taking medicines to thin the blood for any reason
you have metastatic cancer affecting your brain
you have noticed coughing or spitting blood
you have ever received anthracyclines (for example doxorubicin, a specific type of chemotherapy used to treat some cancers) or had radiotherapy to your chest, or if you have heart disease
you have previously experienced problems after injections, such as dizziness/feeling of fainting, breathlessness, swelling or skin rash
you have headache, vision changes, confusion or seizure with or without high blood pressure
you have or have had pain in the mouth, teeth and/or jaw, swelling or sores inside the mouth, numbness or a feeling of heaviness in the jaw, or loosening of a tooth
you need to undergo an invasive dental treatment or dental surgery, in particular when you are also receiving or have received an injection of bisphosphonate into your blood
you are currently using or recently used a medicine called sunitinib malate, are receiving platinum- or taxane-based chemotherapy (medicines used to treat cancer) or are receiving or recently received radiotherapy
you have previously received any other treatment for cancer
ZIRABEV increases the risk of having protein in your urine, especially if you already have high blood pressure. ZIRABEV can also increase the risk of developing blood clots in your veins (a type of blood vessel) and may cause infections and a decreased number of your neutrophils (a type of blood cell important for your protection against bacteria).

Like all medicines, ZIRABEV can cause side effects, although not everybody gets them. If you have an allergic reaction, contact your doctor or a member of medical staff straight away. The signs may include difficulty in breathing or chest pain. You could also experience redness or flushing of the skin or a rash, chills and shivering, feeling sick (nausea) or being sick (vomiting). Very common severe side effects of treatment with ZIRABEV are:

high blood pressure
the feeling of numbness or tingling in hands or feet
a decreased number of cells in the blood, including white cells that help to fight infection (this may be accompanied by fever), and cells that help the blood to clot
feeling weak and having no energy
tiredness
diarrhoea, nausea, vomiting and abdominal pain.
Other very common side effects that are not as severe include constipation, loss of appetite, fever, problems with eyes (including increased tear production), changes in speech, changes in sense of taste, runny nose, dry skin, flaking and inflammation of skin, changes in skin color, loss of body weight and nose bleeds. You should tell your healthcare provider immediately if you notice any of the above symptoms.

Tell your healthcare provider if you are taking, have recently taken or may take any other medicines.

Tell your healthcare provider if you are pregnant, plan to become pregnant, or are breastfeeding.

Ask your healthcare provider about the risks and benefits of ZIRABEV. Only a healthcare provider can decide if ZIRABEV is right for you.

You are encouraged to report negative side effects to the European Medicines Agency. Visit View Source Please refer to the European Summary of Product Characteristics for ZIRABEV for complete safety information.