On February 19, 2019 Apexigen, Inc., a clinical-stage biopharmaceutical company, reported that Xiaodong Yang, M.D., Ph.D., President and Chief Executive Officer, will present at the following upcoming investor conferences (Press release, Apexigen, FEB 19, 2019, View Source [SID1234590998]):

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Citi’s 7th Annual Immuno-oncology Leadership Summit
New York, NY
Tuesday, February 19, 2019 at 3:30 pm ET

SVB Leerink’s 8th Annual Global Healthcare Conference
New York, NY
Wednesday, February 27, 2019 at 2:10 pm ET

Cowen’s 39th Annual Healthcare Conference
Boston, MA
Tuesday, March 12, 2019 at 4:00 pm ET

William Blair’s IO Conference
New York, NY
Thursday, March 21, 2019 at 2:05 pm ET

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 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 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 18, 2019 Xspray Pharma AB reported the results of a clinical Phase I pilot study with its HyNap-Sora drug candidate (Press release, Xspray, FEB 18, 2019, View Source [SID1234649548]). The study examined HyNap-Sora’s bioavailability compared to the sorafenib cancer drug, currently marketed as Nexavar for the treatment of unresectable hepatocellular carcinoma and advanced renal cell carcinoma. Both investigated HyNap compositions demonstrated significantly increased bioavailability. The variability between subjects in AUC and Cmax was reduced to half compared to Nexavar for one of the HyNap-Sora formulations.
In the completed Phase I clinical trial in 14 healthy subjects, bioavailability of two different formulations of HyNap-Sora was assessed in comparison with Nexavar. The results are positive and the study achieved its primary purpose to show increased bioavailability compared to the originator product. In addition, and as shown in earlier studies with Xspray’s lead product candidate, HyNap-Dasa, the study also showed reduced between subject variability.

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"Sorafenib has a very different pharmacokinetic profile than our product candidates based on nilotinib and dasatinib. Therefore, I’m very pleased that we achieved the primary purpose of this study with sorafenib which enables continuation of the development program. It confirms and strengthens our belief that HyNap formulations of PKIs have the potential to improve both present and upcoming products with poor solubility, improving efficacy and safety enhancing patients’ quality of life during this type of therapy," says Per Andersson, CEO of Xspray.

The clinical results in summary:

• The HyNap formulations were administered at half the dose compared to Nexavar to account for expected increase in bioavailability

• Area under the curve (AUC) was approximately 80 % for the HyNap-Sora formulations compared to Nexavar, indicating that the bioavailability of Sorafenib from HyNap-Sora was 1.6-fold higher

• The maximum concentration of Sorafenib in plasma (Cmax) was approximately 95 % compared to Nexavar.

• The variability between subjects in AUC and Cmax was reduced to half compared to Nexavar for one of the HyNap-Sora formulations.

Sorafenib is a compound with low aqueous solubility and low bioavailability from the sorafenib tablet (Nexavar). The present study demonstrates that the HyNap technology can increase the extent of absorption of sorafenib and reduce between subject variability by improving the solubility.