On November 8, 2018 Progenics Pharmaceuticals, Inc. (Nasdaq: PGNX) reported financial results and provided a business update for the third quarter of 2018 (Press release, Progenics Pharmaceuticals, NOV 8, 2018, View Source [SID1234531172]).

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"The third quarter was marked by multiple successes on the clinical and regulatory front, including the approval and launch of AZEDRA, the first-ever U.S. approved treatment for advanced or metastatic pheochromocytoma and paraganglioma. The broader medical community has recognized the importance of AZEDRA in treating these deadly tumors, and we are pleased with the high level of interest from across the entire spectrum of the healthcare system and well as the rapid addition of AZEDRA to the NCCN guidelines," commented Mark Baker, Chief Executive Officer of Progenics.

Mr. Baker continued, "We are pleased that clinicians and pharmaceutical industry leaders increasingly recognize the value of radiopharmaceuticals to address a range of unmet needs in oncology. Consistent with our strategy to maximize the full value of our PSMA-targeted radio pharmaceutical candidates, we are advancing our prostate cancer pipeline. Following encouraging data from our PyL Phase 2/3 OSPREY study, we are on track to initiate a Phase 3 study this year. In addition, we are moving forward a Phase 2 study for 1095 in patients with metastatic castration-resistant prostate cancer (mCRPC) in early 2019."

Third Quarter and Recent Key Business Highlights

AZEDRA (iobenguane I 131) 555 MBq/mL injection for intravenous use, Ultra-orphan Radiotherapeutic

AZEDRA Launch Progressing Following FDA Approval in July

In July, Progenics received U.S. Food and Drug Administration (FDA) approval of its New Drug Application for AZEDRA. AZEDRA is the first and only approved therapy in the U.S. for the treatment of adult and pediatric patients 12 years and older with iobenguane scan positive, unresectable, locally advanced or metastatic pheochromocytoma or paraganglioma who require systemic anticancer therapy.

AZEDRA Added to NCCN Guidelines and Four CMS-Recognized Drug Compendia

In September 2018, AZEDRA was added to the National Comprehensive Cancer Network (NCCN) Clinical Practice Guidelines in Oncology for Neuroendocrine and Adrenal Tumors v 3.2018. NCCN Guidelines are widely recognized and used as the standard for clinical policy in oncology by clinicians and payors. Since AZEDRA’s approval by the FDA, it has also been added to four drug compendia: Clinical Pharmacology©; DRUGDEX; Lexi-Drugs; and NCCN. These compendia are recognized by private and public payers, including Centers for Medicare and Medicaid Services (CMS) as authoritative sources to be considered in determining drug reimbursement.

Progenics Announces Third Quarter 2018 Financial Results Page 2

Pivotal trial of AZEDRA published in The Journal of Nuclear Medicine

In October 2018, the study entitled, "Efficacy and Safety of High-Specific-Activity I-131 MIBG Therapy in Patients with Advanced Pheochromocytoma or Paraganglioma," was published in The Journal of Nuclear Medicine. This article reported the complete results of the pivotal study of AZEDRA, which was the largest multicenter, prospective trial to evaluate the safety and efficacy of any therapy in patients with pheochromocytoma and paraganglioma and formed the basis of AZEDRA’s approval by the FDA.

AZEDRA Safety and Tolerability Data to be presented at a Special Interest Session at RSNA

In November, Progenics will present updated safety and tolerability data for High-Specific-Activity MIBG (AZEDRA) at the Special Interest Session: High Impact Clinical Trials the 104th Scientific Assembly and Annual Meeting of the Radiological Society of North America (RSNA).

PSMA-Targeted Prostate Cancer Pipeline

Advancing 1095 Therapeutic into Phase 2 Development

In October 2018, following discussions with the FDA, Progenics announced plans for a Phase 2 study of 1095 in combination with enzalutamide in chemo-naïve patients with metastatic castration-resistant prostate cancer (mCRPC). 1095 is a small molecule radiotherapeutic designed to selectively bind to the extracellular domain of prostate specific membrane antigen (PSMA), a protein that is highly expressed on prostate cancer cells. It is believed that once 1095 binds to the prostate cancer cells, the drug is internalized and the beta radiation kills the tumor cells. Enrollment is expected to begin in early 2019 and PyL, Progenics’ PET/CT imaging agent, will be used to select patients most likely to benefit from 1095 treatment.

Company decides to focus PSMA-Targeted Imaging Agent Efforts on PyL, Not Investing in Additional 1404 Trials

After review of the results of its Phase 3 study of 1404, a PSMA-targeted small molecule SPECT/CT imaging agent to visualize cancer; and an assessment of the PSMA-targeted imaging agent commercial landscape, the Company has decided to focus its efforts on PyL, its PSMA-targeted PET/CT imaging agent, and will not further invest in 1404.

Phase 3 Study of PyL Imaging Agent to Begin by Year End

In September 2018, the Company announced topline results of its Phase 2/3 OSPREY study evaluating the diagnostic accuracy of its PSMA-targeted PET/CT imaging agent, PyL, in prostate cancer. The study data highlights the strong positive predictive values of PyL to detect prostate cancer in pelvic lymph nodes and metastatic lesions and supports continued development of PyL. Progenics expects to initiate a Phase 3 study of PyL for the detection of biochemical recurrence of prostate cancer by the end of 2018.

Initiation of Phase 1 Study for PSMA-TTC by Bayer Expected in 2018

Progenics expects its partner Bayer to initiate a Phase 1 study of PSMA-Targeted Thorium Conjugate (PSMA-TTC) in patients with mCRPC by year end 2018. Bayer was previously granted exclusive worldwide rights to develop and commercialize products using Progenics’ PSMA antibody technology in combination with Bayer’s alpha-emitting radionuclides.

Progenics Announces Third Quarter 2018 Financial Results Page 3

Digital Technology Portfolio

Artificial Intelligence Imaging Analysis Technology to Use 1404 and PyL Data

Progenics’ imaging analysis technology uses artificial intelligence and machine learning to quantify and automate the reading of PSMA targeted imaging. Data from the company’s pipeline of PSMA-targeted imaging agents (1404 and PyL) is being used to develop deep learning algorithms delivered through compliant medical device software for the automatic detection and quantification of prostate cancer images. The use of AI applications can boost performance, increases objectivity and repeatability, and makes complex but clinically relevant assessments available to physicians in the interpretation of large and complex image data.

RELISTOR, Treatment for Opioid-Induced Constipation (partnered with Bausch Health Companies Inc.)

Third Quarter 2018 RELISTOR Net Sales of $34.5 Million

The third quarter 2018 net sales of RELISTOR, as reported to Progenics by its partner Bausch Health Companies, Inc. (formerly known as Valeant Pharmaceuticals, Inc.), translated to $5.2 million in royalty revenue for Progenics for the quarter.

Third Quarter 2018 Financial Results

Third quarter revenue totaled $5.3 million, up from $2.7 million in the third quarter of 2017, reflecting RELISTOR royalty income of $5.2 million compared to $2.6 million in the corresponding period of 2017.

Third quarter research and development expenses decreased by $2.3 million compared to the corresponding prior year period, resulting primarily from lower external costs associated with the completion of the Phase 2 study for AZEDRA and the Phase 3 trial for 1404. Third quarter selling, general and administrative expenses increased by $1.1 million compared to the corresponding prior year period, primarily attributable to higher costs associated with the commercial launch of AZEDRA. Progenics also recorded a net non-cash charge of $15.2 million in the third quarter 2018, resulting from changes in estimated fair values of intangible assets and contingent consideration liability, primarily related to 1404. For the three months ended September 30, 2018, Progenics recognized interest expense of $1.2 million related to the RELISTOR royalty-backed loan and $1.5 million income tax benefit associated with the non-cash charge recorded as a result of the change in estimated fair value of 1404 intangible assets mentioned above.

Net loss for the third quarter was $24.4 million, or $0.30 per diluted share, compared to net loss of $15.4 million, or $0.22 per diluted share, in the corresponding 2017 period.

Progenics ended the third quarter with cash and cash equivalents of $148.9 million, an increase of $58.2 million compared to cash and cash equivalents as of December 31, 2017. During the quarter, the Company raised net proceeds of $70.0 million in an underwritten public offering of 9.1 million shares of common stock at a public offering price of $8.25 per share and an additional $4.8 million in at-the-market ("ATM") transactions of 0.6 million shares of common stock at an average selling price of $8.36 per share. Progenics intends to use the proceeds to support the launch of AZEDRA, to advance its pipeline and for potential business development opportunities.

Conference Call and Webcast

Progenics will review third quarter financial results in a conference call today at 8:30 a.m. ET. To participate, please dial (877) 250-8889 (domestic) or (720) 545-0001 (international) and reference conference ID 6409609. A live webcast will be available in the Media Center of the Progenics website, www.progenics.com, and a replay will be available for two weeks.

Indication

AZEDRA (iobenguane I 131) is indicated for the treatment of adult and pediatric patients 12 years and older with iobenguane scan positive, unresectable, locally advanced or metastatic pheochromocytoma or paraganglioma who require systemic anticancer therapy.

Important Safety Information

Warnings and Precautions:

Risk from Radiation Exposure: AZEDRA contributes to a patient’s overall long-term radiation exposure. Long-term cumulative radiation exposure is associated with an increased risk for cancer. These risks of radiation associated with the use of AZEDRA are greater in pediatric patients than in adults. Minimize radiation exposure to patients, medical personnel, and household contacts during and after treatment with AZEDRA consistent with institutional good radiation safety practices and patient management procedures.

Myelosuppression: Among the 88 patients who received a therapeutic dose of AZEDRA, 33% experienced Grade 4 thrombocytopenia, 16% experienced Grade 4 neutropenia, and 7% experienced Grade 4 anemia. Five percent of patients experienced febrile neutropenia. Monitor blood cell counts weekly for up to 12 weeks or until levels return to baseline or the normal range. Withhold and dose reduce AZEDRA as recommended in the prescribing information based on severity of the cytopenia.

Secondary myelodysplastic syndrome, leukemia, and other malignancies: Myelodysplastic syndrome (MDS) and acute leukemias were reported in 6.8% of the 88 patients who received a therapeutic dose of AZEDRA. The time to development of MDS or acute leukemia ranged from 12 months to 7 years. Two of the 88 patients developed a non-hematological malignancy.

Hypothyroidism: Hypothyroidism was reported in 3.4% of the 88 patients who received a therapeutic dose of AZEDRA. Initiate thyroid-blocking medications starting at least 1 day before and continuing for 10 days after each AZEDRA dose to reduce the risk of hypothyroidism or thyroid neoplasia. Evaluate for clinical evidence of hypothyroidism and measure thyroid-stimulating hormone (TSH) levels prior to initiating AZEDRA and annually thereafter.

Elevations in blood pressure: Eleven percent of the 88 patients who received a therapeutic dose of AZEDRA experienced a worsening of pre-existing hypertension defined as an increase in systolic blood pressure to ≥160 mmHg with an increase of 20 mmHg or an increase in diastolic blood pressure to ≥ 100 mmHg with an increase of 10 mmHg. All changes in blood pressure occurred within the first 24 hours post infusion. Monitor blood pressure frequently during the first 24 hours after each therapeutic dose of AZEDRA.

Renal toxicity: Of the 88 patients who received a therapeutic dose of AZEDRA, 9% developed renal failure or acute kidney injury and 22% demonstrated a clinically significant decrease in glomerular filtration rate (GFR) measured at 6 or 12 months. Monitor renal function during and after treatment with AZEDRA. Patients with baseline renal impairment may be at greater risk of toxicity; perform more frequent assessments of renal function in patients with mild or moderate impairment. AZEDRA has not been studied in patients with severe renal impairment.

Pneumonitis: Fatal pneumonitis occurred 9 weeks after a single dose in one patient in the expanded access program. Monitor patients for signs and symptoms of pneumonitis and treat appropriately.

Progenics Announces Third Quarter 2018 Financial Results Page 6

Embryo-fetal toxicity: Based on its mechanism of action, AZEDRA can cause fetal harm. Verify pregnancy status in females of reproductive potential prior to initiating AZEDRA. Advise females and males of reproductive potential of the potential risk to a fetus and to use effective contraception during treatment with AZEDRA and for 7 months after the final dose. Advise males with female partners of reproductive potential to use effective contraception during treatment and for 4 months after the final dose.

Risk of infertility: Radiation exposure associated with AZEDRA may cause infertility in males and females. Radiation absorbed by testes and ovaries from the recommended cumulative dose of AZEDRA is within the range where temporary or permanent infertility can be expected following external beam radiotherapy.

Adverse Reactions:

The most common severe (Grade 3–4) adverse reactions observed in AZEDRA clinical trials (≥ 10%) were lymphopenia (78%), neutropenia (59%), thrombocytopenia (50%), fatigue (26%), anemia (24%), increased international normalized ratio (18%), nausea (16%), dizziness (13%), hypertension (11%), and vomiting (10%). Twelve percent of patients discontinued treatment due to adverse reactions (thrombocytopenia, anemia, lymphopenia, nausea and vomiting, multiple hematologic adverse reactions).

Drug Interactions:

Based on the mechanism of action of iobenguane, drugs that reduce catecholamine uptake or that deplete catecholamine stores may interfere with iobenguane uptake into cells and therefore interfere with dosimetry calculations or the efficacy of AZEDRA. These drugs were not permitted in clinical trials that assessed the safety and efficacy of AZEDRA. Discontinue the drugs listed in the prescribing information for at least 5 half-lives before administration of either the dosimetry dose or a therapeutic dose of AZEDRA. Do not administer these drugs until at least 7 days after each AZEDRA dose.

For important risk and use information about AZEDRA, please see Full Prescribing Information.

To report suspected adverse reactions, contact Progenics Pharmaceuticals, Inc. at 844-668-3950 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

Reference:

AZEDRA prescribing information. New York, NY: Progenics Pharmaceuticals, Inc.; 08 2018.

Reference:

AZEDRA prescribing information. New York, NY: Progenics Pharmaceuticals, Inc.; 07 2018.

About RELISTOR

Progenics has exclusively licensed development and commercialization rights for its first commercial product, RELISTOR, to Bausch Health Companies, Inc. RELISTOR Tablets (450 mg once daily) are approved in the United States for the treatment of opioid-induced constipation (OIC) in patients with chronic non-cancer pain. RELISTOR Subcutaneous Injection (12 mg and 8 mg) is a treatment for OIC approved in the United States and worldwide for patients with advanced illness and chronic non-cancer pain.

IMPORTANT SAFETY INFORMATION – RELISTOR (methylnaltrexone bromide) tablets, for oral use and RELISTOR (methylnaltrexone bromide) injection, for subcutaneous use

RELISTOR tablets and injection are contraindicated in patients with known or suspected gastrointestinal obstruction and patients at increased risk of recurrent obstruction, due to the potential for gastrointestinal perforation.

Progenics Announces Third Quarter 2018 Financial Results Page 7

Cases of gastrointestinal perforation have been reported in adult patients with opioid-induced constipation and advanced illness with conditions that may be associated with localized or diffuse reduction of structural integrity in the wall of the gastrointestinal tract (e.g., peptic ulcer disease, Ogilvie’s syndrome, diverticular disease, infiltrative gastrointestinal tract malignancies or peritoneal metastases). Take into account the overall risk-benefit profile when using RELISTOR in patients with these conditions or other conditions which might result in impaired integrity of the gastrointestinal tract wall (e.g., Crohn’s disease). Monitor for the development of severe, persistent, or worsening abdominal pain; discontinue RELISTOR in patients who develop this symptom.

If severe or persistent diarrhea occurs during treatment, advise patients to discontinue therapy with RELISTOR and consult their healthcare provider.

Symptoms consistent with opioid withdrawal, including hyperhidrosis, chills, diarrhea, abdominal pain, anxiety, and yawning have occurred in patients treated with RELISTOR. Patients having disruptions to the blood-brain barrier may be at increased risk for opioid withdrawal and/or reduced analgesia and should be monitored for adequacy of analgesia and symptoms of opioid withdrawal.

Avoid concomitant use of RELISTOR with other opioid antagonists because of the potential for additive effects of opioid receptor antagonism and increased risk of opioid withdrawal.

The use of RELISTOR during pregnancy may precipitate opioid withdrawal in a fetus due to the immature fetal blood brain barrier and should be used during pregnancy only if the potential benefit justifies the potential risk to the fetus. Because of the potential for serious adverse reactions, including opioid withdrawal, in breastfed infants, advise women that breastfeeding is not recommended during treatment with RELISTOR. In nursing mothers, a decision should be made to discontinue nursing or discontinue the drug, taking into account the importance of the drug to the mother.

A dosage reduction of RELISTOR tablets and RELISTOR injection is recommended in patients with moderate and severe renal impairment (creatinine clearance less than 60 mL/minute as estimated by Cockcroft-Gault). No dosage adjustment of RELISTOR tablets or RELISTOR injection is needed in patients with mild renal impairment.

A dosage reduction of RELISTOR tablets is recommended in patients with moderate (Child-Pugh Class B) or severe (Child-Pugh Class C) hepatic impairment. No dosage adjustment of RELISTOR tablets is needed in patients with mild hepatic impairment (Child-Pugh Class A). No dosage adjustment of RELISTOR injection is needed for patients with mild or moderate hepatic impairment. In patients with severe hepatic impairment, monitor for methylnaltrexone-related adverse reactions.

In the clinical studies, the most common adverse reactions were:

OIC in adult patients with chronic non-cancer pain

RELISTOR tablets (≥ 2% of RELISTOR patients and at a greater incidence than placebo): abdominal pain (14%), diarrhea (5%), headache (4%), abdominal distention (4%), vomiting (3%), hyperhidrosis (3%), anxiety (2%), muscle spasms (2%), rhinorrhea (2%), and chills (2%).

RELISTOR injection (≥ 1% of RELISTOR patients and at a greater incidence than placebo): abdominal pain (21%), nausea (9%), diarrhea (6%), hyperhidrosis (6%), hot flush (3%), tremor (1%), and chills (1%).

OIC in adult patients with advanced illness

RELISTOR injection (≥ 5% of RELISTOR patients and at a greater incidence than placebo): abdominal pain (29%) flatulence (13%), nausea (12%), dizziness (7%), and diarrhea (6%).

Please see complete Prescribing Information for RELISTOR at www.bauschhealth.com. For more information about RELISTOR, please visit www.RELISTOR.com.

On November 8, 2018 Loxo Oncology, Inc. (Nasdaq:LOXO), a biopharmaceutical company developing highly selective medicines for patients with genomically defined cancers, reported third quarter 2018 financial results (Press release, Loxo Oncology, NOV 8, 2018, View Source [SID1234530925]).

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"In the third quarter we made significant progress across our pipeline," said Josh Bilenker, M.D., chief executive officer of Loxo Oncology. "At ESMO (Free ESMO Whitepaper), larotrectinib investigators provided a comprehensive program update that included durability and additional response data in patients with TRK fusion cancers. Medical meeting updates for LOXO-292 in September and October showed encouraging ongoing durability of response for patients with RET-driven cancers. Taken together, these data have increased our conviction around a foundational company thesis—that selective, purpose-built medicines offer the best opportunity for durable efficacy and a manageable safety profile. In the fourth quarter, we look forward to LOXO-305, our fourth program, entering the clinic."

Recent Highlights

Larotrectinib

Conference presentations
European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) 2018 Congress: On October 21, 2018, updated clinical data for larotrectinib were presented at ESMO (Free ESMO Whitepaper). The oral presentation provided approximately one year of additional follow-up for the primary dataset, the 55 patients with TRK fusion cancer described in the larotrectinib New England Journal of Medicine publication from February 2018. In addition, the update included data for a supplementary dataset, an additional 67 patients with TRK fusion cancer who were subsequently enrolled across the larotrectinib development program. Response evaluations were based on investigator assessment. As of a data cut-off date of July 30, 2018, in the primary dataset (n=55), the overall response rate (ORR) was 80% (44/55) (95% CI: 67-90%) and in the supplementary dataset (n=67), the ORR was 81% (44/54) (95% CI: 69-91%). Across both datasets, the ORR was 81% (88/109) (95% CI: 72-88%). The ORR analyses for the supplementary and integrated datasets included nine patients with unconfirmed partial responses awaiting confirmatory response assessments, but did not include 13 patients who were awaiting an initial response assessment and continuing on study. Median duration of response (DOR) had not been reached in either the primary dataset or supplementary dataset, with median follow-up of 17.6 months and 7.4 months, respectively. Larotrectinib was well tolerated, with the majority of adverse events recorded as grade 1 or 2. The most common treatment-emergent adverse events occurring in 15% or more of patients in the trial were fatigue, dizziness, nausea, constipation, anemia, increased alanine aminotransferase (ALT), increased aspartate aminotransferase (AST), cough, diarrhea, vomiting, pyrexia, dyspnea, headache, myalgia and peripheral oedema. See the presented data here.
Annual Meeting of the American Thyroid Association (ATA): On October 4, 2018, clinical data for patients with TRK fusion thyroid cancer enrolled in the larotrectinib development program were presented in an oral presentation at ATA. See the presented data here.
International Association for the Study of Lung Cancer (IASLC) 19th World Conference on Lung Cancer: On September 24, 2018, clinical data for patients with TRK fusion non-small cell lung cancer (NSCLC) enrolled in the larotrectinib development program were presented in a poster presentation at the IASLC World Conference on Lung Cancer. The poster can be found here.
Molecular Analysis for Personalised Therapy 2018 Congress: On September 15, 2018, Ventana Medical Systems, Inc., a member of the Roche Group, and Loxo Oncology, presented a co-authored poster on the analytical validation of Ventana’s pan-TRK IHC assay at the Molecular Analysis for Personalised Therapy 2018 Congress. These data, in addition to other recently published evidence, suggest an annual incidence of approximately 2,500 to 3,000 cases of TRK fusion cancer in the United States. The poster can be found here.
Integrative Therapies Program for Children with Cancer (ITPCC): On September 13, 2018, clinical data for children and adolescents with TRK fusion metastatic thyroid carcinoma enrolled in the larotrectinib development program were presented at the ITPCC conference. The poster can be found here.
Publications
Targeted Oncology Publication: On October 2, 2018, a manuscript was published online in Targeted Oncology detailing the potential effectiveness of TRK inhibition, including larotrectinib treatment, in patients with tumors harboring NTRK gene fusions, and the need for effective testing strategies. The publication can be found here.
British Journal of Cancer Publication: On September 17, 2018, a case report was published in the British Journal of Cancer detailing a patient with TRK fusion high-grade glioma treated with larotrectinib. The publication can be found here.
Cancer Publication: On September 11, 2018, a manuscript was published online in Cancer detailing the treatment of children with locally advanced TRK fusion sarcoma who were treated preoperatively with larotrectinib and underwent subsequent surgical resection. The publication can be found here.
JCO Precision Oncology Publication: On August 2, 2018, a case report was published in JCO Precision Oncology detailing an adolescent patient with a TRK fusion undifferentiated sarcoma treated with larotrectinib. The publication can be found here.
European Marketing Authorization Application (MAA): On August 27, 2018, Loxo Oncology and Bayer announced that Bayer had submitted an MAA for larotrectinib to the European Medicines Agency (EMA). More information can be found here.
LOXO-195

LOXO-195 Orphan Drug Designation (ODD): In October, the U.S. Food and Drug Administration (FDA) granted ODD to LOXO-195 for the treatment of solid tumors with neurotrophic tyrosine receptor kinase (NTRK)-fusion proteins that have developed acquired resistance to prior TRK inhibitor therapy. The FDA’s Office of Orphan Drug Products grants orphan drug designation to support the development of medicines for underserved patient populations, or rare disorders, that affect fewer than 200,000 people in the United States. Orphan drug designation provides to Loxo Oncology certain benefits, including market exclusivity upon regulatory approval if received, exemption of FDA application fees and tax credits for qualified clinical trials.
LOXO-292

Annual Meeting of the ATA: On October 6, 2018, updated interim clinical data for LOXO-292 from the global Phase 1/2 LIBRETTO-001 trial in patients with RET-mutant medullary thyroid cancer (MTC) and RET fusion-positive thyroid cancer were presented at the Annual Meeting of the ATA. The data presented were based on a July 19, 2018 data cut-off date and included the 29 patients with RET-mutant MTC and the nine patients with RET fusion-positive thyroid cancer who were included in the LOXO-292 presentation at the 2018 ASCO (Free ASCO Whitepaper) Annual Meeting. With 3.5 months of additional follow-up since the ASCO (Free ASCO Whitepaper) presentation, LOXO-292 demonstrated encouraging, early evidence of durable activity. Sixteen of 17 (94%) responding RET-mutant MTC patients remained on therapy and in response (median follow-up of 7.6 months for all 29 patients; median follow-up of 8.4 months for responding patients). Seven of seven (100%) responding RET fusion-positive thyroid remained on therapy and in response (median follow-up of 7.6 months for all nine patients; median follow-up of 8.5 months for responding patients). In RET-mutant MTC, the overall response rate was 59% (17/29) (95% CI: 39-77%) and the confirmed overall response rate was 56% (15/27) (95% CI: 35-75%). Of nine patients with RET fusion-positive thyroid cancer, the confirmed overall response rate was 78% (7/9) (95% CI: 40-97%). Of the 82 patients in the safety analysis, most treatment-emergent adverse events were Grade 1 in severity and judged by the investigator as not related to LOXO-292. See the presented data here.
IASLC 19th World Conference on Lung Cancer: On September 25, 2018, updated interim clinical data for LOXO-292 from the global Phase 1/2 LIBRETTO-001 trial in patients with RET fusion-positive NSCLC were presented at the IASLC World Conference on Lung Cancer. The data presented were based on a July 19, 2018 data cut-off date and included the 38 patients with RET fusion-positive NSCLC who were initially included in the LOXO-292 presentation at the 2018 ASCO (Free ASCO Whitepaper) Annual Meeting. With 3.5 months of additional follow-up since the ASCO (Free ASCO Whitepaper) presentation, LOXO-292 demonstrated encouraging, early evidence of durable activity, with 25 of 26 (96%) responding RET fusion-positive NSCLC patients remaining on therapy and 24 of 26 (92%) remaining in response (median follow-up of 8.5 months for all 38 patients; median follow-up of 9.5 months for responding patients). The overall response rate was 68% (26/38) (95% CI: 51-83%) and the confirmed overall response rate was 68% (25/37) (95% CI: 50-82%). Of the 82 patients in the safety analysis, most treatment-emergent adverse events were Grade 1 in severity and judged by the investigator as not related to LOXO-292. See the presented data here.
LOXO-292 Breakthrough Therapy Designations: The FDA granted three Breakthrough Therapy Designations to LOXO-292:
for the treatment of patients with metastatic RET fusion-positive non-small cell lung cancer who require systemic therapy and have progressed following platinum-based chemotherapy and an anti-PD-1 or anti-PD-L1 therapy;
for the treatment of patients with RET-mutant medullary thyroid cancer who require systemic therapy, have progressed following prior treatment and have no acceptable alternative treatment options; and for
for the treatment of patients with advanced RET fusion-positive thyroid cancer who require systemic therapy, have progressed following prior treatment and have no acceptable alternative treatment options.
More information can be found here and here.
LOXO-292 Orphan Drug Designation: In October, the FDA granted ODD to LOXO-292 for the treatment of pancreatic cancer. The FDA’s Office of Orphan Drug Products grants orphan drug designation to support the development of medicines for underserved patient populations, or rare disorders, that affect fewer than 200,000 people in the United States. Orphan drug designation provides to Loxo Oncology certain benefits, including market exclusivity upon regulatory approval if received, exemption of FDA application fees and tax credits for qualified clinical trials.
LOXO-305

Society of Hematologic Oncology (SOHO) Annual Meeting: On September 12, 2018, preclinical characterization data for LOXO-305 were presented at the SOHO Annual Meeting. The poster can be found here.
Third Quarter 2018 Financial Results

As of September 30, 2018, Loxo Oncology had aggregate cash, cash equivalents and investments of $647.6 million, compared to $626.2 million as of December 31, 2017.

Revenue from the collaboration agreement was $42.5 million for the third quarter of 2018, compared to none for the third quarter of 2017. This represents $52.9 million in revenue recognized from the $400.0 million upfront payment from the Bayer collaboration offset by $10.5 million, Loxo Oncology’s share of the joint larotrectinib co-promotion costs in the same period.

Revenue from the collaboration agreement was $123.5 million for the nine months ended September 30, 2018, compared to none for the nine months ended September 30, 2017. This represents $147.0 million in revenue recognized from the $400.0 million upfront payment from the Bayer collaboration offset by $23.5 million, Loxo Oncology’s share of the joint larotrectinib co-promotion costs in the same period. Loxo Oncology recognizes revenue from the upfront payment on a proportional performance basis utilizing a calculation based on quarterly research and development spending associated with larotrectinib and LOXO-195, relative to cumulative and forecasted research and development spending on larotrectinib and LOXO-195 over the course of the collaboration agreement. As a result, the quarterly revenue recognized for the upfront payment varies from quarter to quarter. A supporting schedule that shows the different components of revenue from the collaboration agreement is included with the attached financial statements.

Research and development expenses were $56.9 million for the third quarter of 2018 compared to $64.8 million for the third quarter of 2017. This decrease was primarily due to a non-recurring charge related to the $40.0 million asset acquisition of the BTK inhibitor program from Redx in the third quarter of 2017, offset by expanded development expenses across the LOXO-292 and LOXO-305 programs and higher employment costs primarily due to increased headcount. These numbers are net of 50/50 cost-sharing with Bayer for larotrectinib and LOXO-195 development costs. Loxo Oncology recognized research and development-related stock-based compensation expense of $4.2 million during the third quarter of 2018 as compared to $2.1 million for the third quarter of 2017.

Research and development expenses were $130.5 million for the nine months ended September 30, 2018 compared to $109.3 million for the nine months ended September 30, 2017. This increase was primarily due to expanded development expenses across the LOXO-292 and LOXO-305 programs and higher employment costs primarily due to increased headcount. These numbers are net of 50/50 cost-sharing with Bayer for larotrectinib and LOXO-195 development costs. Loxo Oncology recognized research and development-related stock-based compensation expense of $14.3 million during the nine months ended September 30, 2018 as compared to $8.0 million for the nine months ended September 30, 2017.

General and administrative expenses were $15.9 million for the third quarter of 2018 compared to $9.7 million for the third quarter of 2017. The increase was primarily due to additional headcount, associated employment costs, and general and administrative professional fees. Loxo Oncology recognized general and administrative-related stock-based compensation expense of $6.9 million during the third quarter of 2018 compared to $3.1 million for the third quarter of 2017.

General and administrative expenses were $43.8 million for the nine months ended September 30, 2018 compared to $21.0 million for the nine months ended September 30, 2017. The increase was primarily due to additional headcount, associated employment costs, and general and administrative professional fees. Loxo Oncology recognized general and administrative-related stock-based compensation expense of $18.8 million during the nine months ended September 30, 2018 compared to $6.7 million for the nine months ended September 30, 2017.

Net loss was $27.1 million and $42.3 million for the three and nine months ended September 30, 2018, respectively, compared to $73.3 million and $128.2 million for the three and nine months ended September 30, 2017, respectively. This decrease in net loss was primarily driven by the revenue recognized from the $400.0 million upfront payment from the Bayer collaboration, the larotrectinib and LOXO-195 development reimbursement from the Bayer collaboration, offset by increases in operating expenses.

Non-GAAP net loss was $68.8 million and $156.2 million for the three and nine months ended September 30, 2018, respectively, compared to $28.1 million and $73.6 million for the three and nine months ended September 30, 2017, respectively. This non-GAAP net loss measure, more fully described below under "Non-GAAP Financial Measures," excludes the recognition of collaboration revenue related to the Bayer upfront payment and share-based compensation expenses. A reconciliation of the GAAP financial results to non-GAAP financial results is included with the attached financial statements.

Earnings Conference Call and Webcast Information
Loxo Oncology will host a conference call today at 8:00 a.m. ET to discuss the third quarter 2018 financial results and company updates. A live webcast can be accessed under "Events & Presentations" in the Investors & Media section of the company’s website at www.loxooncology.com. The conference call can be accessed by dialing (877) 930-8065 (domestic) or (253) 336-8041 (international) and referring to conference ID 8379404. The webcast will be archived and made available for replay on the company’s website beginning approximately two hours after the event.

On November 8, 2018 Acorda Therapeutics, Inc. (Nasdaq:ACOR) reported that Ron Cohen, M.D., Acorda’s President and CEO, will present at the Stifel 2018 Healthcare Conference on Tuesday, November 13 at 2:00pm EST (Press release, Acorda Therapeutics, NOV 8, 2018, View Source [SID1234531024]). 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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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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On November 8, 2018 SELLAS Life Sciences Group, Inc. (Nasdaq:SLS) ("SELLAS" or the "Company"), a clinical-stage biopharmaceutical company focused on the development of novel cancer immunotherapies for a broad range of cancer indications, reported that the Company will host a conference call and live audio webcast on Thursday, November 15, 2018, at 8:00 a.m. ET to report third quarter 2018 financial results and provide a corporate update (Press release, Sellas Life Sciences, NOV 8, 2018, View Source [SID1234531040]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

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

                  Schedule Your 30 min Free Demo!

To participate in the conference call, please dial (866) 416-7995 (domestic) or (409) 217-8225 (international) and refer to conference ID 7038536. A live webcast of the call can be accessed under "Events & Presentations" in the Investors section of the Company’s website at www.sellaslifesciences.com. An archived webcast recording will be available on the SELLAS website beginning approximately two hours after the call.

On November 8, 2018 vTv Therapeutics Inc. (Nasdaq: VTVT) reported financial results for the third quarter that ended September 30, 2018, and provided an update on recent achievements and upcoming events (Press release, vTv Therapeutics, NOV 8, 2018, View Source;p=irol-newsArticle&ID=2376419 [SID1234531087]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

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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"We continue to believe in the therapeutic potential of azeliragon and are committed to finding the optimal development pathway forward for the program," said Steve Holcombe, chief executive officer, vTv Therapeutics. "We are also making progress with our other programs, either internally in the case of our GKA program, or through our licensing partners for our GLP-1R agonist, PPAR-delta, and PDE4 programs. We hope to see milestones achieved for each of these programs during 2019."

Recent Achievements and Outlook

Presentation of data at 11th Clinical Trials on Alzheimer’s Disease. Presented positive post-hoc subgroup data indicating a potential benefit of treatment with azeliragon in Alzheimer’s disease patients with type 2 diabetes. This subgroup included 55 patients with glycosylated hemoglobin (HbA1c) of greater than 6.5% at baseline (HbA1c greater than 7.7% was an exclusion criterion at screening) and a clinical diagnosis of Alzheimer’s disease in the combined A-Study and B-Study of the STEADFAST trial. The azeliragon-treated group in the A-Study (n=18) demonstrated a 6.1 point benefit on ADAS-cog relative to the placebo group (n=8), which was nominally statistically significant (p = 0.005), and a 1.7 point benefit on CDR-sb relative to placebo (p = 0.08) after 18 months of treatment.
Meeting with European Medicines Agency. Met with the Scientific Advice Working Party (SAWP) of the EMA on October 30, 2018, to discuss future development requirements in support of seeking regulatory approval of azeliragon in the European Union. We expect to receive formal guidance from the SAWP during the fourth quarter of 2018.
SimplicT-1 Study enrolling patients with type 1 diabetes. The adaptive Phase 1/2 SimplicT-1 Study continues to enroll patients with type 1 diabetes in a 12-week study to evaluate TTP399 as an add-on to insulin therapy. We expect to have an interim read-out of the results of this study in early 2019. TTP399 has previously demonstrated statistically significant reductions in HbA1c levels in the AGATA Study, a phase 2 study in type 2 diabetes.
Upcoming Events

vTv will participate in the following upcoming investor conferences:

Piper Jaffray 30th Annual Healthcare Conference, November 27-29, New York, NY
J.P. Morgan 37th Annual Healthcare Conference, January 7-10, San Francisco, CA
Second Quarter 2018 Financial Results

Cash Position: Cash and cash equivalents as of September 30, 2018, were $3.8 million compared to $1.2 million as of June 30, 2018.
R&D Expenses: Research and development expenses were $2.7 million in the third quarter of 2018, compared to $8.6 million in the second quarter of 2018. The decrease in research and development expenses was primarily driven by the termination of the STEADFAST and open label extension studies during the second quarter of 2018.
G&A Expenses: General and administrative expenses were $2.2 million and $2.7 million, for the third quarter of 2018 and the second quarter of 2018, respectively. General and administrative expenses were lower due to reduced share-based compensation expense and professional service fees.
Net Loss Before Non-Controlling Interest: Net loss before non-controlling interest was $2.0 million for the third quarter of 2018 compared to net loss before non-controlling interest of $9.6 million for the second quarter of 2018.
Net Loss Per Share: GAAP net loss per share was $0.06 and $0.31 for the three months ended September 30, 2018 and June 30, 2018, respectively, based on weighted-average shares of 12.3 million and 10.0 million for the three month periods ended September 30, 2018 and June 30, 2018, respectively. Non-GAAP net loss per fully exchanged share was $0.06 and $0.29 for the three months ended September 30, 2018 and June 30, 2018, respectively, based on non-GAAP fully exchanged weighted-average shares of 35.4 million and 33.1 million for the three months ended September 30, 2018 and June 30, 2018, respectively.