On February 7, 2018 Keryx Biopharmaceuticals, Inc. (Nasdaq: KERX), a biopharmaceutical company focused on bringing innovative medicines to people with kidney disease, reported its financial results for the fourth quarter and year ended December 31, 2017 (Press release, Keryx Biopharmaceuticals, FEB 7, 2018, View Source [SID1234523774]). The company also reviewed its commercial progress with Auryxia following approval of the medicine’s second indication for the treatment of iron deficiency anemia in adults with chronic kidney disease, not on dialysis.

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"In 2017, we brought Auryxia to an increasing number of people with hyperphosphatemia, and with the recent approval of a second indication, we are confident we can bring Auryxia to even more patients living with chronic kidney disease in the coming years," said Greg Madison, president and chief executive officer of Keryx Biopharmaceuticals. "With approval of a second indication for Auryxia, we believe we are in a unique position to offer patients and their healthcare providers a medicine that can treat two distinct but related complications of chronic kidney disease. Driving growth in both indications is our focus in 2018 and we look forward to continued progress as we build a leading kidney care company."

2017 Business Highlights

• Full year 2017 net U.S. Auryxia product sales were $55.5 million, as compared to $27.2 million for the full year 2016, a 104 percent increase.
• Auryxia net U.S. product sales were $17.3 million in the fourth quarter of 2017, as compared to $8.2 million in the same quarter in 2016.
• Approximately 30,400 Auryxia prescriptions were reported in the fourth quarter of 2017, these prescriptions represent 6.5 million Auryxia tablets. This compares to approximately 8,700 prescriptions and 1.8 million Auryxia tablets in the fourth quarter of 2016.
• Auryxia received U.S. approval for the treatment of iron deficiency anemia in adults with chronic kidney disease, not on dialysis in November 2017 and was launched in the fourth quarter.
• Following formulary access expansion in 2017, Auryxia has broad formulary coverage across Medicare Part D and commercial insurance providers with no restrictions. This formulary status applies to both Auryxia indications.
Fourth Quarter and Year Ended December 31, 2017 Financial Results

"The strong prescription demand growth generated in the fourth quarter, coupled with the stabilization of the gross-to-net adjustment, led to $17.3 million of net U.S. Auryxia product sales in the fourth quarter of 2017," said Scott Holmes, senior vice president and chief financial officer of Keryx Biopharmaceuticals. "As we enter 2018, we believe we will continue to drive Auryxia growth as a chronic kidney disease treatment for both hyperphosphatemia in the dialysis setting, as well as iron deficiency anemia in the non-dialysis setting."

Total revenues for the quarter ended December 31, 2017 were approximately $18.7 million, compared with $9.5 million during the same period in 2016. Total revenues for the fourth quarter of 2017 consist of approximately $17.3 million in net U.S. Auryxia product sales, as compared to $8.2 million in the fourth quarter of 2016. Total revenues for the fourth quarter of 2017 also include $1.4 million in license revenue, as compared to $1.3 million during the same period in 2016.

For the year ended 2017, total revenues were approximately $60.6 million, as compared to $32.0 million in 2016. Total revenues for 2017 include $55.5 million of Auryxia net U.S. product sales and $5.1 million in license revenue, as compared to $27.2 million and $4.8 million, respectively, in 2016.

Cost of goods sold for the quarter ended December 31, 2017 were $7.4 million, compared with $13.4 million during the same period in 2016. For the year ended 2017, total cost of goods sold were $22.0 million, as compared to $37.8 million in 2016.

Selling, general and administrative expenses for the quarter ended December 31, 2017 were $28.8 million, as compared to $23.0 million during the same period in 2016. For the year ended December 31, 2017, total selling, general and administrative expenses were $99.6 million, as compared to $84.6 million in 2016. Selling, general and administrative expenses for the quarter and full year ended December 31, 2017 included $6.6 million and $15.6 million, respectively, in non-cash stock compensation expense, as compared to $2.8 million and $11.2 million, respectively, during the same periods in 2016.

Research and development expenses for the quarter ended December 31, 2017 were $12.6 million, as compared to $6.2 million during the same period in 2016. For the year ended December 31, 2017, total research and development expenses were $37.7 million, as compared to $29.5 million in 2016. Research and development expenses for the quarter and full year ended December 31, 2017 included $1.0 million and $2.6 million, respectively, in non-cash stock compensation expense, as compared to $0.6 million and $2.8 million, respectively, during the same periods in 2016.

Net loss for the quarter ended December 31, 2017 was $30.4 million, or $0.26 per share, as compared to a net loss of $33.8 million, or $0.32 per share, for the comparable period in 2016. For the full year 2017, net loss was $163.4 million, or $1.43 per share, as compared to $161.1 million, or $1.52 per share, in 2016.

Cash and cash equivalents as of December 31, 2017 totaled approximately $93.5 million.

Conference Call Information

Keryx Biopharmaceuticals will host an investor conference call today, February 7, 2018, at 8:00 a.m. ET to discuss financial results for the fourth quarter and full year of 2017. To participate in the conference call, please call 1-(888) 396-2320 (U.S.), 1-(774) 264-7560 (outside the U.S.), call-in ID: 3697815. The call will also be webcast with slides, which will be accessible through the Investors section of the company’s website at www.keryx.com. The audio replay will be available at View Source for approximately 15 days after the call.

About Auryxia (ferric citrate) tablets

Auryxia (ferric citrate) was approved by the U.S. Food and Drug Administration (FDA) on September 5, 2014 for the control of serum phosphorus levels in patients with chronic kidney disease on dialysis and approved by the FDA on November 6, 2017 for the treatment of iron deficiency anemia in patients with chronic kidney disease not on dialysis. Auryxia tablets were designed to contain 210 mg of ferric iron, equivalent to 1 gram of ferric citrate, and offers convenient mealtime dosing. The starting dose of Auryxia for the treatment of hyperphosphatemia for patients on dialysis is six tablets per day (two per meal) and for the treatment of iron deficiency anemia in patients not on dialysis is three tablets per day (one per meal). For more information about Auryxia and the U.S. full prescribing information, please visit www.Auryxia.com.

IMPORTANT U.S. SAFETY INFORMATION FOR AURYXIA (ferric citrate)

CONTRAINDICATION

AURYXIA (ferric citrate) is contraindicated in patients with iron overload syndromes, e.g., hemochromatosis.

WARNINGS AND PRECAUTIONS

• Iron Overload: Increases in serum ferritin and transferrin saturation (TSAT) were observed in clinical trials with AURYXIA in patients with chronic kidney disease (CKD) on dialysis treated for hyperphosphatemia, which may lead to excessive elevations in iron stores. Assess iron parameters prior to initiating AURYXIA and monitor while on therapy. Patients receiving concomitant intravenous (IV) iron may require a reduction in dose or discontinuation of IV iron therapy.
• Risk of Overdosage in Children Due to Accidental Ingestion: Accidental ingestion and resulting overdose of iron-containing products is a leading cause of fatal poisoning in children under 6 years of age. Advise patients of the risks to children and to keep AURYXIA out of the reach of children.

ADVERSE REACTIONS

Most common adverse reactions with AURYXIA were:

• Hyperphosphatemia in CKD on Dialysis: Diarrhea (21%), discolored feces (19%), nausea (11%), constipation (8%), vomiting (7%) and cough (6%)
• Iron Deficiency Anemia in CKD Not on Dialysis: Discolored feces (22%), diarrhea (21%), constipation (18%), nausea (10%), abdominal pain (5%) and hyperkalemia (5%)
SPECIFIC POPULATIONS

. Pregnancy and Lactation: There are no available data on AURYXIA use in pregnant women to inform a drug-associated risk of major birth defects and miscarriage. However, an overdose of iron in pregnant women may carry a risk for spontaneous abortion, gestational diabetes and fetal malformation. Data from rat studies have shown the transfer of iron into milk, hence, there is a possibility of infant exposure when AURYXIA is administered to a nursing woman.
To report suspected adverse reactions, contact Keryx Biopharmaceuticals at 1-844-445-3799.

Please click here to view the Full Prescribing Information for Auryxia.

On February 7, 2018 AVEO Oncology (NASDAQ: AVEO) reported that Michael Bailey, president and chief executive officer, will present at the following investor conferences (Press release, AVEO, FEB 7, 2018, View Source;p=RssLanding&cat=news&id=2330854 [SID1234523780]):

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LEERINK Partners 7th Annual Global Healthcare Conference on Wednesday, February 14, 2018 at 10:00 a.m. Eastern Time. The conference is being held at the Lotte New York Palace Hotel.

RBC Capital Markets Healthcare Conference on Wednesday, February 21, 2018 at 10:00 a.m. Eastern Time. The conference is being held at the Lotte New York Palace Hotel.

A live webcast of the presentations can be accessed by visiting the investors section of the Company’s website at www.aveooncology.com. A replay of the webcast will be archived for 30 days following the presentation date.

On February 7, 2018 Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) ("Rocket"), a leading U.S.-based multi-platform gene therapy company, reported that Gaurav Shah, M.D., Chief Executive Officer and President of Rocket, will participate in a fireside chat at the Leerink Partners 7th Annual Global Healthcare Conference in New York City (Press release, Rocket Pharmaceuticals, FEB 7, 2018, View Source [SID1234523777]). The fireside chat will take place on Wednesday, February 14, 2018, at 2:00 p.m. Eastern Time (ET).

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On February 7, 2018 BeiGene, Ltd. (NASDAQ:BGNE), a commercial-stage biopharmaceutical company focused on developing and commercializing innovative molecularly targeted and immuno-oncology drugs for the treatment of cancer, reported that the company will present at the LEERINK Partners 7th Annual Global Healthcare Conference in New York, NY (Press release, BeiGene, FEB 7, 2018, View Source;p=RssLanding&cat=news&id=2330836 [SID1234523781]). The presentation is scheduled for 9:00 AM ET on Wednesday, February 14, 2018.

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A live webcast can be accessed from the investors section of BeiGene’s website at View Source An archived replay will be available for 90 days following the event.

On February 6, 2018 Celgene Corporation (NASDAQ: CELG) reported that the Phase III, randomized, open-label, international clinical study, OPTIMISMM, achieved its primary endpoint, showing a statistically significant and clinically meaningful improvement in progression-free survival (PFS) for the pomalidomide arm versus the comparator arm (Press release, Celgene, FEB 7, 2018, View Source [SID1234523791]).

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OPTIMISMM evaluated the efficacy and safety of POMALYST/IMNOVID (pomalidomide) plus bortezomib and low-dose dexamethasone (PVd) versus bortezomib and low-dose dexamethasone in patients with relapsed/refractory multiple myeloma. It is the only phase III trial to investigate a triplet combination in patients who have all received prior lenalidomide (REVLIMID), a population for which there is a growing unmet medical need.

"The OPTIMISMM results confirm the expanding role of pomalidomide in previously treated multiple myeloma patients," said Paul Richardson, M.D., Clinical Program Leader and Director of Clinical Research, Jerome Lipper Multiple Myeloma Center at the Dana Farber Cancer Institute, RJ Corman Professor of Medicine, Harvard Medical School and principal investigator of the study. "We see the PVd combination as an important step in improving care, and especially for patients previously treated with lenalidomide in this setting."

In the study, the safety profile was consistent with previously reported data. Detailed data from OPTIMISMM will be presented at future medical meetings.

The combination of POMALYST/IMNOVID, bortezomib and low-dose dexamethasone is not currently approved for use.

About POMALYST/IMNOVID

Indication

POMALYST (pomalidomide) is a thalidomide analogue indicated, in combination with dexamethasone, for patients with multiple myeloma who have received at least two prior therapies including lenalidomide and a proteasome inhibitor and have demonstrated disease progression on or within 60 days of completion of the last therapy.

Important Safety Information

WARNING: EMBRYO-FETAL TOXICITY and VENOUS AND ARTERIAL THROMBOEMBOLISM

Embryo-Fetal Toxicity

POMALYST is contraindicated in pregnancy. POMALYST is a thalidomide analogue. Thalidomide is a known human teratogen that causes severe birth defects or embryo-fetal death. In females of reproductive potential, obtain 2 negative pregnancy tests before starting POMALYST treatment.

Females of reproductive potential must use 2 forms of contraception or continuously abstain from heterosexual sex during and for 4 weeks after stopping POMALYST treatment.

POMALYST is only available through a restricted distribution program called POMALYST REMS.

Venous and Arterial Thromboembolism

Deep venous thrombosis (DVT), pulmonary embolism (PE), myocardial infarction, and stroke occur in patients with multiple myeloma treated with POMALYST. Prophylactic antithrombotic measures were employed in clinical trials. Thromboprophylaxis is recommended, and the choice of regimen should be based on assessment of the patient’s underlying risk factors.

CONTRAINDICATIONS

Pregnancy: POMALYST can cause fetal harm and is contraindicated in females who are pregnant. If POMALYST is used during pregnancy or if the patient becomes pregnant while taking this drug, the patient should be apprised of the potential risk to a fetus.

WARNINGS AND PRECAUTIONS

Embryo-Fetal Toxicity & Females of Reproductive Potential: See Boxed WARNINGS

Males: Pomalidomide is present in the semen of patients receiving the drug. Males must always use a latex or synthetic condom during any sexual contact with females of reproductive potential while taking POMALYST and for up to 4 weeks after discontinuing POMALYST, even if they have undergone a successful vasectomy. Males must not donate sperm.

Blood Donation: Patients must not donate blood during treatment with POMALYST and for 1 month following discontinuation of POMALYST therapy because the blood might be given to a pregnant female patient whose fetus must not be exposed to POMALYST.

POMALYST REMS Program: See Boxed WARNINGS

Prescribers and pharmacies must be certified with the POMALYST REMS program by enrolling and complying with the REMS requirements; pharmacies must only dispense to patients who are authorized to receive POMALYST. Patients must sign a Patient-Physician Agreement Form and comply with REMS requirements; female patients of reproductive potential who are not pregnant must comply with the pregnancy testing and contraception requirements and males must comply with contraception requirements.

Further information about the POMALYST REMS program is available at www.CelgeneRiskManagement.com or by telephone at 1-888-423-5436.

Venous and Arterial Thromboembolism: See Boxed WARNINGS. Patients with known risk factors, including prior thrombosis, may be at greater risk, and actions should be taken to try to minimize all modifiable factors (e.g., hyperlipidemia, hypertension, smoking). Thromboprophylaxis is recommended, and the choice of regimen should be based on assessment of the patient’s underlying risk factors.

Increased Mortality with Pembrolizumab: In clinical trials in patients with multiple myeloma, the addition of pembrolizumab to a thalidomide analogue plus dexamethasone resulted in increased mortality. Treatment of patients with multiple myeloma with a PD-1 or PD-L1 blocking antibody in combination with a thalidomide analogue plus dexamethasone is not recommended outside of controlled clinical trials.

Hematologic Toxicity: Neutropenia (46%) was the most frequently reported Grade 3/4 adverse reaction in patients taking POMALYST in clinical trials, followed by anemia and thrombocytopenia. Monitor complete blood counts weekly for the first 8 weeks and monthly thereafter. Patients may require dose interruption and/or modification.

Hepatotoxicity: Hepatic failure, including fatal cases, has occurred in patients treated with POMALYST. Elevated levels of alanine aminotransferase and bilirubin have also been observed in patients treated with POMALYST. Monitor liver function tests monthly. Stop POMALYST upon elevation of liver enzymes. After return to baseline values, treatment at a lower dose may be considered.

Hypersensitivity Reactions: Angioedema and severe dermatologic reactions have been reported. Discontinue POMALYST for angioedema, skin exfoliation, bullae, or any other severe dermatologic reactions, and do not resume therapy.

Dizziness and Confusional State: In patients taking POMALYST in clinical trials, 14% experienced dizziness (1% Grade 3 or 4) and 7% a confusional state (3% Grade 3 or 4). Instruct patients to avoid situations where dizziness or confusional state may be a problem and not to take other medications that may cause dizziness or confusional state without adequate medical advice.

Neuropathy: In patients taking POMALYST in clinical trials, 18% experienced neuropathy (2% Grade 3 in one trial) and 12% peripheral neuropathy.

Second Primary Malignancies: Cases of acute myelogenous leukemia have been reported in patients receiving POMALYST as an investigational therapy outside of multiple myeloma.

Tumor Lysis Syndrome (TLS): TLS may occur in patients treated with POMALYST. Patients at risk are those with high tumor burden prior to treatment. These patients should be monitored closely and appropriate precautions taken.

ADVERSE REACTIONS

Nearly all patients treated with POMALYST + low-dose dex experienced at least one adverse reaction (99%). The most common adverse reactions (≥15%) included neutropenia (51.3%), fatigue and asthenia (46.7%), upper respiratory tract infection (31%), thrombocytopenia (29.7%), pyrexia (26.7%), dyspnea (25.3%), diarrhea (22%), constipation (21.7%), back pain (19.7%), cough (20%), pneumonia (19.3%), bone pain (18%), edema peripheral (17.3%), peripheral neuropathy (17.3%), muscle spasms (15.3%), and nausea (15%). Grade 3 or 4 adverse reactions (≥15%) included neutropenia (48.3%), thrombocytopenia (22%), and pneumonia (15.7%).

DRUG INTERACTIONS

Avoid concomitant use of POMALYST with strong inhibitors of CYP1A2. Consider alternative treatments. If a strong CYP1A2 inhibitor must be used, reduce POMALYST dose by 50%.

USE IN SPECIFIC POPULATIONS

Pregnancy: See Boxed WARNINGS. If pregnancy does occur during treatment, immediately discontinue the drug and refer patient to an obstetrician/gynecologist experienced in reproductive toxicity for further evaluation and counseling. There is a POMALYST pregnancy exposure registry that monitors pregnancy outcomes in females exposed to POMALYST during pregnancy as well as female partners of male patients who are exposed to POMALYST. This registry is also used to understand the root cause for the pregnancy. Report any suspected fetal exposure to POMALYST to the FDA via the MedWatch program at 1-800-FDA-1088 and also to Celgene Corporation at 1-888-423-5436.

Lactation: There is no information regarding the presence of pomalidomide in human milk, the effects of POMALYST on the breastfed infant, or the effects of POMALYST on milk production. Pomalidomide was excreted in the milk of lactating rats. Because many drugs are excreted in human milk and because of the potential for adverse reactions in breastfed infants from POMALYST, advise a nursing woman to discontinue breastfeeding during treatment with POMALYST.

Pediatric Use: Safety and effectiveness have not been established in pediatric patients.

Geriatric Use: No dosage adjustment is required for POMALYST based on age. Patients >65 years of age were more likely than patients ≤65 years of age to experience pneumonia.

Renal Impairment: Reduce POMALYST dose by 25% in patients with severe renal impairment requiring dialysis. Take dose of POMALYST following hemodialysis on hemodialysis days.

Hepatic Impairment: Reduce POMALYST dose by 25% in patients with mild to moderate hepatic impairment and 50% in patients with severe hepatic impairment.

Smoking Tobacco: Advise patients that smoking may reduce the efficacy of POMALYST. Cigarette smoking reduces the AUC of pomalidomide by 32% by CYP1A2 induction.