U.S. FDA Approves Pfizer’s Oncology Biosimilar TRAZIMERA™ (trastuzumab-qyyp), a Biosimilar to Herceptin®1

On March 11, 2019 Pfizer Inc. (NYSE: PFE) reported that the United States (U.S.) Food and Drug Administration (FDA) has approved TRAZIMERA (trastuzumab-qyyp), a biosimilar to Herceptin (trastuzumab),1 for the treatment of human epidermal growth factor receptor-2 (HER2) overexpressing breast cancer and HER2 overexpressing metastatic gastric or gastroesophageal junction adenocarcinoma (Press release, Pfizer, MAR 11, 2019, View Source [SID1234534205]).

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The FDA approval was based on review of a comprehensive data package, which demonstrated a high degree of similarity between TRAZIMERA and the originator product. This includes results from the REFLECTIONS B327-02 clinical comparative study that was recently published in the British Journal of Cancer, which showed clinical equivalence, finding a high degree of similarity and no clinically meaningful differences between TRAZIMERA and the originator product in patients with first line HER2 overexpressing metastatic breast cancer.3

"Approximately 15-30% of breast cancers and 10-30% of gastric cancers are HER2-positive, which is associated with aggressive disease and poor prognoses for patients," said Dr. Mark Pegram, associate director for clinical research at the Stanford Comprehensive Cancer Institute, and director of the Breast Oncology Program at the Stanford Women’s Cancer Center.4 "With the availability of biosimilars like TRAZIMERA in the U.S., oncologists will have additional treatment options to choose from, which may help provide patients with greater access to the medicines they need."

Pfizer has a robust portfolio of potential biosimilar candidates in mid- to late-stage development.5 TRAZIMERA is Pfizer’s first oncology monoclonal antibody (mAb) biosimilar and Pfizer’s fifth biosimilar to be approved by the FDA.2,6,7,8,9 TRAZIMERA was also approved for use in the EU in July 2018 for the treatment of HER2 overexpressing breast cancer and HER2 overexpressing metastatic gastric or gastroesophageal junction adenocarcinoma.10

About TRAZIMERA (trastuzumab-qyyp)

TRAZIMERA is a monoclonal antibody (mAb) biosimilar of the originator biologic medicine, Herceptin, which targets HER2, a protein found on the surface of some cancer cells which can stimulate the cells to divide and grow.11 TRAZIMERA locks on to the HER2 protein and blocks the receptors, stopping cell division and growth.10

As part of the REFLECTIONS clinical trial program, TRAZIMERA has been studied in nearly 500 patients and across more than 20 countries to date.12,13,14,15,16

TRAZIMERA IMPORTANT SAFETY INFORMATION

Possible Serious Side Effects With TRAZIMERA (trastuzumab–qyyp)

Not all people have serious side effects, but side effects with TRAZIMERA therapy are common.

Although some people may have a life-threatening side effect, most do not.

Your doctor will stop treatment if any serious side effects occur.

TRAZIMERA is not for everyone. Be sure to contact your doctor if you are experiencing any of the following:

HEART PROBLEMS

These include heart problems—such as congestive heart failure or reduced heart function—with or without symptoms. The risk for and seriousness of these heart problems were highest in people who received both trastuzumab and a certain type of chemotherapy (anthracycline). In a study of adjuvant (early) breast cancer, one patient died of significantly weakened heart muscle. Your doctor will check for signs of heart problems before, during, and after treatment with TRAZIMERA.

INFUSION REACTIONS, including:

Fever and chills
Feeling sick to your stomach (nausea)
Throwing up (vomiting)
Pain (in some cases at tumor sites)
Headache
Dizziness
Shortness of breath
These signs usually happen within 24 hours after receiving TRAZIMERA.

Be sure to contact your doctor if you:

Are a woman who could become pregnant, or may be pregnant

TRAZIMERA may result in the death of an unborn baby or birth defects. Contraception should be used while receiving TRAZIMERA and after your last dose of TRAZIMERA. If you are exposed to TRAZIMERA during pregnancy or within 7 months of becoming pregnant, you are encouraged to report TRAZIMERA exposure to Pfizer at 1-800-438-1985.

Have any signs of SEVERE LUNG PROBLEMS, including:

Severe shortness of breath
Fluid in or around the lungs
Weakening of the valve between the heart and the lungs
Not enough oxygen in the body
Swelling of the lungs
Scarring of the lungs
Your doctor may check for signs of severe lung problems when he or she examines you.

Have LOW WHITE BLOOD CELL COUNTS

Low white blood cell counts can be life threatening. Low white blood cell counts were seen more often in patients receiving trastuzumab plus chemotherapy than in patients receiving chemotherapy alone.

Your doctor may check for signs of low white blood cell counts when he or she examines you.

Side Effects Seen Most Often With trastuzumab

Some patients receiving trastuzumab for breast cancer had the following side effects:

Fever
Feeling sick to your stomach (nausea)
Throwing up (vomiting)
Infusion reactions
Diarrhea
Infections
Increased cough
Headache
Feeling tired
Shortness of breath
Rash
Low white and red blood cell counts
Muscle pain
Some patients receiving trastuzumab for metastatic stomach cancer had the following side effects:

Low white blood cell counts
Diarrhea
Feeling tired
Low red blood cell counts
Swelling of the mouth lining
Weight loss
Upper respiratory tract infections
Fever
Low platelet counts
Swelling of the mucous membranes
Swelling of the nose and throat
Change in taste

You should contact your doctor immediately if you have any of the side effects listed above.

Nymox to Host Investor Teleconference

On March 11, 2019 Nymox Pharmaceutical Corporation (NASDAQ:NYMX) reported that it will hold a teleconference for shareholders on March 25, 2019 (Press release, Nymox, MAR 11, 2019, View Source [SID1234534198]).

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Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

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Management will discuss the Company’s current business progress with an update on its regulatory submission activities in the US and EU. Further details re time and coordinates and information for the call will be provided prior to the teleconference.

For more information please contact [email protected] or 800-936-9669.

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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Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

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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]).

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Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

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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]).

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"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.