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Ultragenyx to Present at Upcoming Investor Conferences

On March 11, 2019 (GLOBE NEWSWIRE) — Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare genetic diseases, reported that it will present at the following upcoming investor conferences (Press release, Ultragenyx Pharmaceutical, MAR 11, 2019, View Source [SID1234534196]):

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Emil D. Kakkis, M.D., Ph.D., the company’s Chief Executive Officer and President, will present at the Cowen Annual Healthcare Conference on Tuesday, March 12, 2019 at 10 a.m. ET in Boston, MA.
Tom Kassberg, the company’s Chief Business Officer, will present at the Barclays Global Healthcare Conference on Wednesday, March 13, 2019 at 8 a.m. ET in Miami, FL.
The live and archived webcast of the company presentations will be accessible from the company’s website at View Source The replay of the webcast will be available for 90 days.

Ability of AVID200, a Novel TGF-beta Inhibitor, to Enhance Immune Cell Infiltration and Efficacy of Immune Checkpoint Inhibition Featured at the Keystone Symposia on Cancer Immunotherapy

On March 11, 2019 Forbius, a clinical-stage company that develops novel biologics for the treatment of fibrosis and cancer, reported a presentation of AVID200 preclinical data at the Keystone Symposia on Cancer Immunotherapy, March 10 – 14 (Press release, Forbius, MAR 11, 2019, View Source [SID1234534195]).

This presentation describes AVID200’s immuno-oncology mode of action and ability to increase T-cell-mediated cytotoxicity and immune cell infiltration, resulting in enhanced efficacy of immune checkpoint inhibitors in syngeneic mouse tumor models.

TGF-beta 1 & 3 are the main oncogenic TGF-beta isoforms expressed by many solid tumors. They are believed to play a major role in T-cell suppression, fibrosis, and resistance to anti-PD-(L)1 therapies such as nivolumab (Opdivo) and pembrolizumab (Keytruda) (Chakravarthy et al., Nature Comm., 2018; Tauriello et al., Nature, 2018; Mariathasan et al., Nature, 2018).

Athenex, Inc. Announces Fourth Quarter and Year Ended December 31, 2018 Financial Results and Provides Corporate Update

On March 11, 2019 Athenex, Inc. (NASDAQ: ATNX), a global biopharmaceutical company dedicated to the discovery, development and commercialization of novel therapies for the treatment of cancer and related conditions, reported its financial results and business highlights for the fourth quarter and year ended December 31, 2018 (Press release, Athenex, MAR 11, 2019, View Source;p=RssLanding&cat=news&id=2390797 [SID1234534194]).

"In 2018, we made substantial progress in advancing our pipeline and expanding our technology platforms in oncology as we work towards building a global biopharmaceutical business," stated Dr. Johnson Lau, Chief Executive Officer and Chairman of Athenex. "We are on track to continue this positive momentum in 2019, with the expected completion of our Phase III study of Oraxol in metastatic breast cancer and preparations underway for regulatory filings for KX2-391 in actinic keratosis. We now have a total of 8 product candidates in the clinic, with INDs on 2 further candidates scheduled by mid-year, and we are committed to continuing our strong clinical execution. On the operational front, we continue to build out our commercial infrastructure and global supply chain as we put the plans in place to launch our proprietary products."

Fourth Quarter 2018 and Recent Business Highlights:

Clinical Programs:

KX2-391 ointment in actinic keratosis: Positive topline results from two pivotal Phase III studies were featured in a late breaker session at the 2019 American Academy of Dermatology Annual Meeting
In studies KX01-AK-003 and KX01-AK-004, 44% and 54% of patients, respectively, achieved 100% AK lesion clearance at Day 57. The results were highly statistically significant.
Safety profile of KX2-391 ointment may be an important competitive advantage; adherence to treatment was greater than 99%
Oraxol Phase III studies in metastatic breast cancer: Achieved target enrollment of 360 patients. Topline results are expected to be available in mid-2019
Other Oraxol studies:
Announced positive results from the second cohort of patients in a global Phase Ib clinical trial of Oraxol plus ramucirumab in gastric cancer patients who failed previous chemotherapies. The Oraxol dose is currently being further escalated to 300 mg/m2 in the third cohort of patients and the study is ongoing.
Initiated a Phase I/II clinical study to assess the safety, tolerability and activity of Oraxol in combination with anti-PD1 antibody (pembrolizumab) in patients with advanced solid malignancies.
Initiated a Phase I clinical study of Oraxol in angiosarcomas. Oraxol received Orphan Drug Designation from the U.S. FDA for this indication. Preclinical data to be presented in a poster session at American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2019

Orascovery platform:
Licensed the rights to develop and commercialize Oradoxel (Oral Docetaxel) in Taiwan, Singapore, and Vietnam to PharmaEssentia.
Announced acceptance of an Investigational New Drug (IND) application for Eribulin ORA, Athenex’s oral version of Eribulin.

TCR-T immunotherapy: Announced positive results from pilot studies conducted in China by Xiangxue Life Sciences in end-stage cancer patients who were treated with T-cell receptor affinity enhancing specific T-cell therapy (TAEST), a form of cancer immunotherapy.
Commercial Business:

Athenex Pharmaceutical Division ("APD") currently markets a total of 28 products with 53 SKUs.
Athenex Pharma Solutions ("APS") currently markets 6 products in total with 16 SKUs.
Commercial platform is planning to launch 12 new products in 2019.
Dunkirk manufacturing facility achieved a significant construction milestone with the steel and concrete work complete and the final beam raised into place.
Financial Results for the Fourth Quarter Ended December 31, 2018

Revenue for the three months ended December 31, 2018 was $21.3 million, as compared to $14.9 million for the three months ended December 31, 2017, an increase of $6.4 million, or 43%. Product sales were $19.0 million and $14.1 million for the three months ended in December 31, 2018 and December 31, 2017, respectively. The increase was primarily attributable to an increase of $3.5 million in API revenue, $3.1 million in 503B revenue, offset by $1.8 million decrease in medical device and CMO revenue. There was a $2.0 million licensing fee revenue in the three months ended December 31, 2018, pursuant to a licensing agreement we entered into with PharmaEssentia, for rights to Oral Docetaxel in certain Asian territories.

Cost of sales for the three months ended December 31, 2018 totaled $14.3 million, as compared to $10.1 million for the three months ended December 31, 2017, an increase of $4.2 million, or 42%. The increase was primarily due to a change in our product mix. As we continue to develop our product portfolio, the gross margin might fluctuate over time.

Research and development expenses for the three months ended December 31, 2018 totaled $20.8 million. Our various clinical programs accounted for the majority of our R&D expenses. The R&D expenses for the three months ended December 31, 2018 were in line with the $20.8 million for the three months ended December 31, 2017.

Selling, general, and administrative expenses for the three months ended December 31, 2018 totaled $11.6 million, as compared to $12.3 million for the three months ended December 31, 2017, a decrease of $0.7 million, or 6%.

Net loss attributable to Athenex for the three months ended December 31, 2018 was $27.1 million, or ($0.41) per diluted share, compared to a net loss of $28.3 million, or ($0.49) per diluted share, in the same period last year.

Financial Results for the Year Ended December 31, 2018:

Revenue for the year ended December 31, 2018 was $89.1 million, as compared to $38.0 million for the year ended December 31, 2017, an increase of $51.1 million, or 134%. The increase was primarily attributable to the $30.0 million license fees related to the collaboration agreement with Almirall. Revenue from product sales also increased from $36.1 million in the year ended December 31, 2017 to $56.4 million in the year ended December 31, 2018. The increase was primarily attributable to an increase in specialty product revenue of $13.2 million, an increase in 503B revenue of $5.1 million, an increase in API revenue of $2.6 million, and an increase in medical device sales of $0.6 million, offset by a decrease in contract manufacturing revenue and other revenue of $1.7 million.

Cost of sales for the year ended December 31, 2018 totaled $47.0 million, as compared to $25.1 million for the year ended December 31, 2017, an increase of $21.9 million, or 87%. The increase in specialty product revenue, 503B revenue, and API revenue increased cost of sales by $15.2 million, $3.7 million, and $3.0 million, respectively.

Research and development expenses for the year ended December 31, 2018 totaled $119.9 million, as compared to $76.8 million for the year ended December 31, 2017, an increase of $43.1 million, or 56%. This increase was primarily due to the advancement of the Company’s clinical pipeline and additional drug licensing fees, and included the following: $18.6 million increase of clinical trial costs with the progression of the Phase 3 trials of KX2-391 Ointment and Oraxol; $15.7 million increase in drug licensing fees primarily due to a $29.5 million non-cash license fee related to the purchase of TCR-T technology in connection with the establishment of Axis Therapeutics Limited, of which $24.5 million related to the fair value of the in-process research and development (IPR&D) and $5.0 million was paid for in the Company’s common stock. This was offset by a decrease in drug licensing fees paid to Hanmi, Gland and Amphastar; a $5.4 million increase of employee salary and benefits, which was primarily attributable to hiring more research and development personnel to support our expanding research and clinical activities, including the expansion of our clinical R&D team in Taiwan; a $2.8 million increase in general product development of 503B products as they were introduced and production was scaled-up to a commercial level and product development of our proprietary products; and a $0.6 million increase in the cost of preclinical studies as research was performed on an oral formulation of Eribulin.

Selling, general, and administrative expenses for the year ended December 31, 2018 totaled $49.0 million, as compared to $46.1 million for the year ended December 31, 2017, an increase of $2.9 million, or 6%. This was primarily due to an increase in operating activities and professional fees and included the following: a $2.9 million increase in professional fees including legal fees related to the launch of 503B products and consulting fees related to the manufacturing facility in Dunkirk, NY; and a $2.2 million increase in other office expenses including property and sales taxes, insurance expenses, rent and utilities, and others. These costs were offset by a decrease in employee compensation of $1.6 million from the stock-based compensation incurred in the prior year in connection with the Company’s IPO and a decrease in marketing costs of $0.6 million.

Net loss attributable to Athenex for the year ended December 31, 2018 was $117.4 million, or ($1.82) per diluted share, compared to a net loss of $131.2 million, or ($2.63) per diluted share, for the year ended December 31, 2017.

Excluding the non-cash license fee of $24.5 million in connection with the establishment of Axis Therapeutics Limited in July 2018, the net loss attributable to Athenex for the year ended December 31, 2018 was $92.9 million, or ($1.44) per diluted share.

The Company had cash, cash equivalents and short-term investments aggregating $107.4 million at December 31, 2018, compared to $51.0 million at December 31, 2017. Based on the current operating plan, the Company expects that its cash, cash equivalents and short-term investments as of December 31, 2018, together with cash to be generated from operating activities, will enable it to fund its operating expenses and capital expenditure requirements through at least the fourth quarter of 2019.

Outlook and Upcoming Milestones:

Clinical Platforms:

Complete third cohort of patients for Oraxol with ramucirumab study in gastric cancer.
Presentation of preclinical data for Oraxol in angiosarcomas at American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting, April 3, 2019.
Expect to file INDs for TCR-T candidates and Pegtomarginase by mid-2019.
Presentations at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) 2019 Annual Meeting, May 31 – June 4, 2019
Top line results from Phase 3 trial of Oraxol in metastatic breast cancer, mid-2019
Financial Guidance:

Going forward, the company’s revenue guidance will be on product sales only, and will exclude estimates for license and collaborative fees. More importantly, we will continue to provide regular updates on our clinical progress and results, and business development activities, which remain to be the core value drivers of our Company.

We had previously provided guidance for full year 2018 revenue to be in the lower end of the guidance range, inclusive of licensing-fee revenue. In our licensing fee revenue guidance we accounted for an upfront payment of US$14.5 million from Chongqing Jingdong Pharmaceutical. As announced in early March 2019, the agreement with Chongqing Jingdong Pharmaceutical was terminated by mutual consent, leading to our 2018 reported revenue.

Athenex is forecasting that product sales in 2019 will increase by between 25% and 30% year-over-year from $56.4 million in 2018.

Conference Call and Webcast Information:

The Company will host a conference call and live audio webcast today, Monday, March 11, 2018 at 8:30 a.m. Eastern Time to discuss the financial results and provide a business update.

To participate in the call, dial 877-407-0784 (domestic) or 201-689-8560 (international) fifteen minutes before the conference call begins and reference the conference passcode 13687139.

The live conference call and replay can also be accessed via audio webcast at View Source and also on the Investor Relations section of the Company’s website, located at www.athenex.com.

A replay of the call will be accessible two hours after its completion through March 18, 2019 by dialing 844-512-2921 (domestic) or 412-317-6671 (international) and entering passcode 13687139.

MOLECULAR PARTNERS TO PRESENT AT THE COWEN AND COMPANY 39th ANNUAL HEALTHCARE CONFERENCE

On March 11, 2019. Molecular Partners AG (SIX: MOLN), a clinical-stage biotech company pioneering the use of DARPin therapeutics* to treat serious diseases, reported that it will present at the Cowen and Company 39th Annual Health Care Conference on Tuesday, March 12, 2019 at 12:00 – 12:30 PM Eastern Time (5:00 PM CET) (Press release, Molecular Partners, MAR 11, 2019, View Source [SID1234534193]). The presentation, followed by a Q&A session, will be hosted by Dr. Patrick Amstutz, CEO of Molecular Partners.

Next to highlighting the progress of the clinical DARPin compounds, the presentation will outline the evolution of the company’s therapeutic design space in oncology research, including the recent strategic partnership with Amgen on MP0310 (FAP x 4-1BB). The presentation will also outline the initiation of a research project to explore DARPin molecules targeting peptide MHC complexes supported by Gilead. The ability to target peptide MHC complexes with DARPin molecules has the potential to open the target space significantly, as these targets have been notoriously difficult to be addressed by any antibody-based approaches.

Neon Therapeutics Reports Fourth Quarter and Full Year 2018 Financial Results and Provides Business Update

On March 11, 2019 Neon Therapeutics, Inc. (Nasdaq: NTGN), a clinical-stage immuno-oncology company developing neoantigen-based therapeutics, reported financial results for the fourth quarter and full-year ended December 31, 2018 and provided a business update (Press release, Neon Therapeutics, MAR 11, 2019, View Source [SID1234534192]).

"2018 was a transformative year for Neon, highlighted by significant advances in applying our insights into neoantigen biology not only to our vaccine product candidates but also to our emerging cellular therapies. Our initial public offering in June provided us with capital to advance these important programs, which have the potential to truly change the paradigm for immunotherapy by enabling more targeted treatment," said Hugh O’Dowd, Neon’s Chief Executive Officer. "As we move into 2019, we are excited about the opportunities ahead to share data from our ongoing clinical trials of our personal neoantigen vaccine NEO-PV-01. We are also making progress to advance our personal T cell therapy program, NEO-PTC-01, into the clinic. We have adjusted timelines to enable the release of more mature data from our NT-002 study and to complete the scale-up process for our NEO-PTC-01 program."

"With regard to NEO-PV-01, we have been closely tracking multiple metrics of patients’ immune response following dosing with the vaccine, and we are looking forward to presenting a correlative analysis from our NT-001 trial with data from more than a dozen patients at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting. Later this year, we will present the more mature 52-week data set, which will include additional patients followed for a full year, a time frame that allows us to further evaluate not only their immune responses but also clinical outcomes, including progression free-survival," said Richard Gaynor, M.D., Neon’s President of Research and Development.

"We have also made important developments in our NEO-PTC-01 program, including the evaluation of additional patient sample materials, which builds upon data presented at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper)’s 33rd Annual Meeting last year. The additional evaluation of both melanoma and non-small cell lung cancer patient materials confirm that NEO-STIM, our proprietary ex vivo co-culture process using peripheral blood mononuclear cells, can reliably and reproducibly generate multiple enriched neoantigen-specific T cell populations. These findings, now replicated across multiple patient samples, support the feasibility of advancing this program into the clinic.

"Finally, we have meaningfully advanced the science behind our precision medicine approaches, which target mutations shared across patient populations known as shared neoantigens. We are leveraging our NEO-STIM induction protocol to rapidly generate T cells specific to these targets where multiple T cell receptors, or TCRs, can be sequenced and characterized. Importantly, since we can derive these TCRs from the natural repertoire of healthy donors, our TCRs show cytotoxic functionality and strong specificity for mutant targets without engineering or modification. Due to our focused efforts in this area, we now have libraries of these high-quality TCRs," concluded Dr. Gaynor.

Fourth Quarter Business Highlights

In April 2019, Neon will present data relating to both its personal neoantigen vaccine, NEO-PV-01, and its personal neoantigen T cell therapy, NEO-PTC-01, at the AACR (Free AACR Whitepaper) Annual Meeting taking place in Atlanta, GA. Data from the NEO-PV-01 program will include a correlative analysis from the melanoma cohort in NT-001, a clinical trial exploring the feasibility, safety, efficacy and immunogenicity of NEO-PV-01 dosed in combination with nivolumab in patients with advanced or metastatic melanoma, non-small cell lung cancer and bladder cancer. In addition, data from the NEO-PTC-01 program will include a detailed analysis of immune responses induced against patient-specific neoantigens using its ex vivo induction protocol, NEO-STIM.

In December 2018, Neon announced that patient dosing had commenced in NT-003, a Phase 1b combination trial of NEO-PV-01 in metastatic melanoma. This Phase 1b trial is evaluating NEO-PV-01 and nivolumab in combination with other agents, including Apexigen’s APX005M, an investigational CD40 agonist, or in combination with ipilimumab, to enhance vaccine-induced neoantigen immune responses.

In November 2018, the Company presented results related to its personal T cell product, NEO-PTC-01, at SITC (Free SITC Whitepaper). Data from two melanoma patients were presented that showed that NEO-STIM can prime, activate and expand neoantigen-specific CD8+ and CD4+ T cell responses from peripheral blood mononuclear cells. Responses were shown to be mutant-specific, polyfunctional and capable of killing antigen expressing tumor targets. Since this presentation, Neon has obtained data from additional samples from cancer patients.

In November 2018, Neon was named one of the top places to work in Massachusetts by The Boston Globe, an honor awarded based on employee feedback. The Top Places to Work awards recognize the most admired workplaces in the state as voted on by the employees of those companies. The annual survey, now in its eleventh year, measures employee opinions about their company’s direction, management, culture, pay and benefits and engagement with employees, among other factors.
Pipeline Overview and Upcoming Milestones

NEO-PV-01

NT-001: Phase 1b Clinical Trial of NEO-PV-01 in the Metastatic Setting
– Neon will present updated correlative data from its NT-001 trial at the AACR (Free AACR Whitepaper) Annual Meeting.
– Full 52-week data, including clinical outcomes, will be presented later in the first half of 2019.

NT-002: Phase 1b Clinical Trial of NEO-PV-01 in Metastatic Non-Small Cell Lung Cancer
– Neon now expects to report immune and clinical data over the course of 2020.

NT-003: Phase 1b Clinical Trial of NEO-PV-01 in Metastatic Melanoma Combinations
– Neon expects to report immune monitoring data in the first half of 2020.

NT-004: Phase 1b Clinical Trial of NEO-PV-01 in Earlier Disease Setting
– Planning ongoing.
NEO-PTC-01

Neon will present a detailed analysis of immune responses induced against patient-specific neoantigens using its ex vivo induction protocol, NEO-STIM, at the AACR (Free AACR Whitepaper) Annual Meeting.

Building on our success to date in generating both memory and de novo immune responses, Neon is in the process of completing large scale process development, which supports its plan to file a Clinical Trial Application in Europe in the second half of 2019 for the first clinical trial of this cell therapy.

This work is being performed in collaboration with the Netherlands Cancer Institute (NKI), an academic research and treatment center with leading expertise in T cell biology and treatments.
NEO-SV-01

Phase 1 Clinical Trial in Subset of ER+ Breast Cancer: Following the completion of target validation and preclinical product development work, Neon expects to submit an Investigational New Drug application to the U.S. Food and Drug Administration in the first half of 2019.
Fourth Quarter and Full Year 2018 Financial Results:

Cash Position: As of December 31, 2018, cash, cash equivalents and marketable securities were $103.3 million, as compared to cash, cash equivalents and marketable securities of $79.7 million as of December 31, 2017.

R&D Expenses: R&D expenses were $18.0 million for the fourth quarter of 2018 and $60.4 million for the year ended December 31, 2018, as compared to $10.9 million for the fourth quarter of 2017 and $37.2 million for the year ended December 31, 2017. The increase for both fourth quarter and full year 2018 was primarily driven by increased costs related to the advancement of NEO-PV-01, as well as higher personnel costs.

G&A Expenses: G&A expenses were $5.8 million for the fourth quarter of 2018 and $18.3 million for the year ended December 31, 2018, as compared to $3.7 million for the fourth quarter of 2017 and $10.9 million for the year ended December 31, 2017. The increase for both fourth quarter and full year 2018 was primarily driven by increased costs of being a public company, including professional fees and personnel costs, as well as other general and administrative costs to support Neon’s overall growth.

Net Loss Attributable to Common Stockholders: Net loss was $23.1 million for the fourth quarter of 2018 and $83.3 million for the year ended December 31, 2018, or a net loss per basic and diluted share of $(0.84) and $(5.54), respectively, as compared to a net loss of $17.4 million for the fourth quarter of 2017 and $57.9 million for the year ended December 31, 2017, or a net loss per basic and diluted share of $(8.93) and $(34.32), respectively.
Financial Guidance

Based on its current operating plan, Neon expects that its existing cash, cash equivalents and marketable securities will enable the Company to fund its anticipated operating expenses and capital expenditure requirements into at least the second quarter of 2020.

About Neon Therapeutics

Neon Therapeutics is a clinical-stage immuno-oncology company and a leader in the field of neoantigen-targeted therapies, dedicated to transforming the treatment of cancer by directing the immune system towards neoantigens. Neon is using its neoantigen platform to develop both vaccine and T cell therapies, including NEO-PV-01, a clinical-stage neoantigen vaccine for the treatment of metastatic melanoma, non-small cell lung cancer, and bladder cancer; NEO-PTC-01, a neoantigen T cell therapy for the treatment of solid tumors; and NEO-SV-01, a neoantigen vaccine for the treatment of a subset of estrogen-receptor-positive breast cancer.