On May 9, 2018 Progenics Pharmaceuticals, Inc. (Nasdaq: PGNX) reported financial results and provided a business update for the first quarter of 2018 (Press release, Progenics Pharmaceuticals, MAY 9, 2018, View Source [SID1234526318]).

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"We continue with our preparations for the potential launch of AZEDRA in anticipation of our July 30th PDUFA Action Date, while also executing the clinical development strategy for our portfolio of PSMA-targeted radiopharmaceuticals," commented Mark Baker, Chief Executive Officer of Progenics. "Based on the efficacy and safety data from our pivotal Phase 2 trial, we believe AZEDRA represents a significant new therapy for patients with malignant pheochromocytoma and paraganglioma, indications for which there are currently no approved treatment options available in the U.S. We have also continued to advance our clinical programs for 1404, PyL and 1095, each of which has the potential to transform the prostate cancer treatment landscape. We look forward to releasing top line data from our Phase 3 trial for 1404 and expect to complete our Phase 2/3 trial for PyL in the third quarter of 2018."

First Quarter and Recent Key Business Highlights

AZEDRA, Ultra-Orphan Radiotherapeutic Candidate

AZEDRA New Drug Application (NDA) FDA Action Date Set for July 30th

In March 2018, Progenics announced a three-month extension of the review period for the NDA for AZEDRA in patients with malignant, recurrent, and/or unresectable pheochromocytoma and paraganglioma, rare neuroendocrine tumors for which there are currently no approved treatment options in the U.S. AZEDRA holds Breakthrough Therapy designation, Orphan Drug status, as well as Fast Track designation.

Progenics Announces First Quarter 2018 Financial Results

Page 2

Data from Pivotal Phase 2 AZEDRA Study Presented at Major Medical Meetings

In March 2018, Progenics presented biochemical tumor marker data from its open-label pivotal Phase 2 study evaluating AZEDRA at the Endocrine Society (ENDO) Annual Meeting. Progenics also reported that updated survival and safety data from this study will be presented during an oral presentation at the upcoming American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting in June 2018.

PSMA-Targeted Prostate Cancer Pipeline

Enrollment Complete in Phase 3 Study of 1404

In January 2018, Progenics announced the completion of enrollment in its Phase 3 study of 1404, a PSMA-targeted small molecule SPECT/CT imaging agent designed to visualize prostate cancer. The study enrolled approximately 450 patients in the U.S. and Canada with newly-diagnosed or low-grade prostate cancer, whose biopsy indicates a histopathologic Gleason grade of ≤ 3+4 severity and/or are candidates for active surveillance. Top-line results are expected in the third quarter of 2018.

Enrollment Expected to Complete in Phase 2/3 Trial for PSMA-Targeted PET/CT Imaging Agent, PyL, in Q3’18

Progenics continues to enroll patients in the Phase 2/3 study of PyL, a PSMA-targeted PET/CT imaging agent, evaluating diagnostic accuracy in patients with recurrent and/or metastatic prostate cancer. The Company expects to complete enrollment of this study in the third quarter of 2018 and initiate a second Phase 3 study in patients with biochemical recurrence of prostate cancer by year end. The Company will present a clinical trial in progress poster at the upcoming ASCO (Free ASCO Whitepaper) Annual Meeting in June 2018.

Phase 1 Trial for PSMA-Targeted Small Molecule 1095 Ongoing

The Phase 1 clinical trial for the Company’s 1095 a small molecule radiotherapeutic that selectively binds to PSMA, is ongoing. The Phase 1 open-label dose escalation study is evaluating 1095 in patients with metastatic castration-resistant prostate cancer (mCRPC) who have demonstrated tumor avidity to 1095.

Phase 1 Study for PSMA-TTC Expected to Initiate 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 has exclusive worldwide rights to develop and commercialize products using Progenics’s PSMA antibody technology in combination with Bayer’s alpha-emitting radionuclides.

RELISTOR, Treatment for Opioid-Induced Constipation (partnered with Valeant Pharmaceuticals International, Inc.)

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First Quarter 2018 RELISTOR Net Sales of $20.4 Million

The first quarter 2018 sales, as reported to Progenics by its partner Valeant, translated to $3.1 million in royalty revenue for Progenics for the quarter. Total first quarter 2018 RELISTOR U.S. net sales increased 50% over the first quarter of 2017.

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Summary Judgment Granted Upholding the Validity of Formulation Patent Protecting RELISTOR Injection from Generic Competition Until 2024

The U.S. District Court for the District of New Jersey has granted a motion for partial summary judgment of validity of a formulation patent for RELISTOR (methylnaltrexone bromide) Injection. The ruling prevents generic competition in the United States until 2024.

Progenics Announces First Quarter 2018 Financial Results Page 3

First Quarter 2018 Financial Results

First quarter revenue totaled $3.2 million, up from $2.3 million in the first quarter of 2017, reflecting RELISTOR royalty income of $3.1 million compared to $2.1 million in the corresponding period of 2017.

First quarter research and development expenses decreased by $1.9 million compared to the corresponding prior year period, resulting primarily from lower clinical trial expenses for AZEDRA. First quarter general and administrative expenses increased by $1.0 million compared to the corresponding prior year period, primarily attributable to higher costs associated with building commercial capabilities in preparation for a potential AZEDRA approval and launch. Progenics also recorded non-cash adjustments of $0.8 million in the first quarter 2018, related to changes in the fair value estimate of the contingent consideration liability. For the three months ended March 31, 2018, Progenics recognized interest expense of $1.2 million related to the RELISTOR royalty-backed loan.

Net loss for the first quarter was $13.4 million, or $0.19 per diluted share, compared to net loss of $16.4 million, or $0.23 per diluted share, in the corresponding 2017 period.

Progenics ended the first quarter with cash and cash equivalents of $83.4 million, a decrease of $7.2 million compared to cash and cash equivalents as of December 31, 2017. In order to continue to maintain a strong financial position, the Company raised $17.0 million in net proceeds from sales of its common stock under its "at-the-market" (ATM) facility in January through April 2018, with $7.5 million received in April.

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 3564709. 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.

About RELISTOR

Progenics has exclusively licensed development and commercialization rights for its first commercial product, RELISTOR, to Valeant. 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.

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.

Progenics Announces First Quarter 2018 Financial Results

Page 6

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.valeant.com. For more information about RELISTOR, please visit www.RELISTOR.com.

On May 9, 2018 Pain Therapeutics, Inc. (Nasdaq:PTIE), a drug development company, reported financial results for the first quarter ended March 31, 2018 (Press release, Pain Therapeutics, MAY 9, 2018, View Source [SID1234526317]). Net loss was $2.2 million, or $0.33 per share. This compared to a net loss of $2.7 million, or $0.42 per share, for the same period in the prior year. Cash and cash equivalents were $10.7 million as of March 31, 2018.

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"FDA’s acceptance of the NDA filing for REMOXY ER in Q1 2018 is a milestone," said Remi Barbier, Chairman, President & CEO. "Our focus is now on REMOXY ER’s potential to receive marketing clearance later this year. As part of this focus, we are preparing a strategic and thorough presentation of our key data for an upcoming FDA Advisory Committee Meeting for REMOXY ER, scheduled for June 26th in Washington, DC. We also continue to maintain tight fiscal discipline, and to advance our early-stage programs with non-dilutive funding."

Financial Highlights for First Quarter 2018

Net loss for the quarter was $2.2 million, or a net loss per share of $0.33. This compared to a net loss of $2.7 million, or $0.42 per share, for the same period in the prior year, representing a 19% decrease.

Cash and cash equivalents were $10.7 million. This compared to $10.5 million in the prior quarter, representing a 2% increase. Cash and cash equivalents for the quarter includes $1.9 million of net proceeds raised through issuance of shares of our common stock under a Capital on DemandTM program with JonesTrading Institutional Services LLC.

We have no debt.

We believe existing capital resources are sufficient to meet our projected operating requirements for at least the next 12 months.

We received $0.4 million in research grant funding from the National Institutes of Health, recorded as a reduction in research and development expenses (R&D).

R&D expenses were $1.1 million. This compared to $1.4 million for the same period in the prior year, representing a 23% decrease. R&D expenses included non-cash stock related compensation costs of $0.4 million, versus $0.3 million for the same period in the prior year.

General and administrative (G&A) expenses were $1.1 million. This compared to $1.4 million for the same period in the prior year, representing a 20% decrease. G&A expenses included non-cash stock-related compensation costs of $0.5 million, versus $0.5 million for the same period in the prior year.

About REMOXY ER (extended-release oxycodone capsules CII)

REMOXY ER is in registration with the US Food and Drug Administration (FDA) as a new type of abuse-deterrent, twice-daily, capsule gel formulation of oral oxycodone, a strong opioid drug. REMOXY ER has physical/chemical properties intended to deter abuse and still provide 12 hours of steady pain relief when properly prescribed by physician and used appropriately by patients.

Studies were extensive. The clinical efficacy of REMOXY ER was established in a Phase III study conducted under a Special Protocol Assessment. In total, over 2,400 subjects were exposed to REMOXY ER in 30 clinical studies. 9,000 unique data points were generated from 11 lab studies. The assessment of REMOXY ER’s abuse deterrence is supported by data from FDA Category 1 (lab), Category 2 (pharmacokinetic) and Category 3 (human abuse potential) studies. In addition, in November 2017 the Company and FDA held a pre-NDA meeting to confirm the sufficiency of data included in the REMOXY ER NDA resubmission.

REMOXY ER has a thick, sticky, high viscosity, hydrophobic, gel formulation that abusers cannot cut, grate or divide into smaller discrete particle sizes. The gel formulation resists syringe-ability, injection, and rapid extraction in ingestible solvents. REMOXY ER’s high viscosity and adhesive properties also cause it to stick to tools and equipment used for abuse. When exposed to heat, REMOXY ER releases an irritant to the eyes and lungs. REMOXY ER resists dose-dumping when challenged by alcohol and common physical and chemical manipulations.

We are requesting marketing approval of REMOXY ER as an analgesic drug with properties that can be expected to deter against injection, snorting and inhalation/smoking routes of abuse.

REMOXY ER intends to address the public health epidemic related to prescription opioids by advancing the science of abuse deterrence, providing an additional treatment option for physicians and patients, and increasing the range of available abuse deterrent technologies.

About Opioid Abuse
Opioid drugs such as oxycodone are an important treatment option for patients with severe chronic pain. However, oxycodone abuse and diversion remain serious, persistent problems. Opioid overdose deaths exceeded 64,000 in 2016, according to the Center for Disease Control (CDC). For over a decade, Pain Therapeutics has pioneered Abuse-Deterrent Formulations (ADFs) to help in the fight against prescription drug abuse. ADFs attempt to raise the bar on prescription drug abuse by making it more difficult, longer or aversive to tamper with long-acting opioid formulations, recognizing that no drug can be made abuse-proof.

Our Pipeline of Drug Assets also Includes:
FENROCK (transdermal fentanyl patch system) – This is a proprietary, abuse-deterrent skin patch for severe pain. FENROCK is an early-stage program, substantially funded by a research grant award from National Institute on Drug Abuse (NIDA).

PTI-125 – This proprietary, small molecule drug candidate is aimed at the treatment of Alzheimer’s disease. PTI-125 is a Phase I clinical-stage program, substantially funded by a research grant award from the National Institutes of Health (NIH).

PTI-125DX – This is a proprietary blood-based test to detect Alzheimer’s disease. PTI-125DX is an early-stage program, substantially funded by a research grant award from the NIH

On May 9, 2018 Ophthotech Corporation (Nasdaq:OPHT) reported financial and operating results for the first quarter ended March 31, 2018 and provided a business update (Press release, Ophthotech, MAY 9, 2018, View Source [SID1234526316]).

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"We continue to build on our Zimura program with on-going clinical trials in wet age related macular degeneration (AMD) with patient recruitment completed and topline data expected by the end of 2018, in geographic atrophy secondary to dry AMD where recruitment is on-track for topline data in the second half of 2019 and in autosomal recessive Stargardt disease which began enrolling patients earlier in the year," stated Glenn P. Sblendorio, Chief Executive Officer and President of Ophthotech. "In parallel, we are excited to have initiated our first innovative gene therapy program in collaboration with Horae Gene Therapy Center at the University of Massachusetts Medical School to treat orphan degenerative retinal diseases. A key aspect of our strategy is to continue to build on this momentum to uncover novel and differentiating technologies and product candidates through collaborations with leading companies and academic institutions from around the world."

Recent Key Highlights

Zimura Complement Factor C5 Inhibitor Program

In April 2018, the Company completed patient recruitment in its dose-ranging, open-label, multi-center Phase 2a clinical trial of Zimura (avacincaptad pegol) in combination with the anti-vascular endothelial growth factor (anti-VEGF) agent Lucentis (ranibizumab) in patients with wet age-related macular degeneration (AMD) who have not been previously treated with any anti-VEGF agents. This uncontrolled trial is designed to assess safety at different dosages and to detect a potential efficacy signal. The Company will evaluate data at month six.
The Company’s ongoing Zimura clinical trial for the treatment of geographic atrophy secondary to dry AMD is on track for initial top-line data to be available during the second half of 2019.
In January 2018, the first patient was enrolled in the Company’s Phase 2b randomized, double-masked, sham-controlled clinical trial assessing the efficacy and safety of Zimura in patients with autosomal recessive Stargardt disease (STGD1). Initial top-line data is expected to be available in 2020.
Scientific details for two of the Company’s ongoing Zimura clinical trials were presented at medical conferences:
The scientific details of the geographic atrophy secondary to dry AMD clinical trial were presented at the 41st Annual Macula Society Meeting in Beverly Hills, California, February 21-24, 2018.
The scientific details of the STGD1 clinical trial were presented at the 2018 Annual Meeting of the Association for Research in Vision and Ophthalmology in Honolulu, Hawaii, April 29-May 3, 2018 and at the International Symposium on Ocular Pharmacology and Therapeutics in Tel-Aviv, Israel, March 1-3, 2018.
Gene Therapy Program

In February 2018, the Company initiated an innovative gene therapy program focused on applying novel gene therapy technology to discover and develop new therapies for ocular diseases.
In February 2018, the Company announced its first gene therapy collaboration, as it entered into a series of sponsored research agreements with the University of Massachusetts Medical School (UMMS) and its Horae Gene Therapy Center to utilize their "minigene" therapy approach and other novel gene delivery methods to target retinal diseases. UMMS has granted Ophthotech an option to obtain an exclusive license to any patent or patent applications that result from this research.
Corporate Highlight

In January 2018, the Company announced the election of Jane PritchettHenderson, Chief Financial Officer and Senior Vice President of Corporate Development at Voyager Therapeutics, to its Board of Directors. Ms. Henderson has also been elected the Chair of the Ophthotech Audit Committee.

2018 Operational Update

As of March 31, 2018, the Company had $155 million in cash and cash equivalents.

The Company’s estimates its year end 2018 cash and cash equivalents will range between $112 million and $117 million based on its current 2018 business plan and planned capital expenditures. This estimate includes continuation of the Company’s development programs for Zimura and the initiation of its collaborative gene therapy research programs as currently planned. This estimate does not reflect any additional expenditures resulting from the potential in-licensing or acquisition of additional product candidates or technologies or associated development that the Company may pursue.

First Quarter 2018 Financial Highlights

Revenues: Collaboration revenue was $0 for the quarter ended March 31, 2018, compared to $1.7 million for the same period in 2017. Collaboration revenue decreased due to the completion of the Company’s licensing and commercialization agreement with Novartis Pharma AG and the recognition of all associated deferred revenue during the third quarter of 2017.
R&D Expenses: Research and development expenses were $7.7 million for the quarter ended March 31, 2018, compared to $32 million for the same period in 2017. As the Company pursues its ongoing and planned Zimura development programs, research and development expenses decreased primarily due to decreases in expenses related to the discontinuation of the Company’s FovistaPhase 3 clinical program and decreases in costs associated with the Company’s 2017 reduction in personnel program.
G&A Expenses: General and administrative expenses were $5.6 million for the quarter ended March 31, 2018, compared to $13.2 million for the same period in 2017. General and administrative expenses decreased primarily due to decreases in costs to support the Company’s operations and infrastructure and decreases in costs associated with its 2017 reduction in personnel program, which included facilities lease termination expenses incurred during the first quarter of 2017.
Net Loss: The Company reported a net loss for the quarter ended March 31, 2018 of $13.1 million, or ($.36) per diluted share, compared to a net loss of $43.1 million, or ($1.20) per diluted share, for the same period in 2017.
Conference Call/Web Cast Information

Ophthotech will host a conference call/webcast to discuss the Company’s financial and operating results and provide a business update. The call is scheduled for May 9, 2018 at 8:00 a.m. Eastern Time. To participate in this conference call, dial 800-239-9838 (USA) or 323-794-2551 (International), passcode 2075643. A live, listen-only audio webcast of the conference call can be accessed on the Investor Relations section of the Ophthotech website at: www.ophthotech.com. A replay will be available approximately two hours following the live call for two weeks. The replay number is 888-203-1112 (USA Toll Free), passcode 2075643.

On May 9, 2018 Idera Pharmaceuticals, Inc. ("Idera") (NASDAQ:IDRA), a clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of novel nucleic acid-based therapeutics for oncology and rare diseases, reported its financial and operational results for the first quarter ended March 31, 2018 (Press release, Idera Pharmaceuticals, MAY 9, 2018, View Source [SID1234526315]).

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"Our company continues to make significant progress advancing our two lead TLR modulating clinical development candidates, tilsotolimod and IMO-8400," stated Vincent Milano, Idera’s chief executive officer. Milano continued, "As we now advance through the second quarter, we are continuing to enroll patients in the ILLUMINATE oncology trials, with the next planned data from ILLUMINATE-204 in PD-1 refractory metastatic melanoma to be presented at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) ("ASCO") Annual Meeting and completion of enrollment expected by year end. For IMO-8400, we plan to report top-line data from our Phase 2 trial in dermatomyositis in June. As it pertains to our nucleic acid chemistry research group, we completed our data analysis for IDRA-008 which has led us to a decision to not advance that program into the clinic."

"In January of this year, we announced our proposed merger with BioCryst Pharmaceuticals, Inc. that we believe will build greater and more sustainable value for the benefit of stockholders as well as patients with rare diseases beyond what we could achieve alone. The Idera Board determined this combination was compelling from both a strategic and financial perspective following a careful evaluation of a range of strategies to enhance long-term stockholder value. The transaction will create a leading rare disease company with a robust pipeline including two promising Phase 3 rare disease programs and combines synergistic discovery engines that will not only expand the number of rare diseases we can target but create meaningful opportunities for differentiation in the market through joint small molecule and oligo treatments. Importantly, joining with BioCryst will also enable us to achieve cost synergies and increase our financial strength and flexibility. Subject to shareholder approval, we expect to close the transaction in the third quarter," Milano expressed.

Clinical Development Program Updates:
TLR Modulation Technology Development Candidates

ILLUMINATE (tilsotolimod) Clinical Development

ILLUMINATE 301 – Randomized phase 3 trial of tilsotolimod in combination with ipilimumab versus ipilimumab alone in patients with PD-1 refractory metastatic melanoma:

Trial initiated in Q1 2018;
Approximately 80 sites planned for trial participation across 12 countries;
Planned enrollment of approximately 300 patients with Overall Response Rate ("ORR") and Overall Survival as primary endpoints; and
U.S. Food and Drug Administration granted Fast Track Designation for tilsotolimod in combination with ipilimumab for treatment of PD-1 refractory metastatic melanoma in fourth quarter of 2017.
ILLUMINATE 204 – Phase 1/2 trial of intratumoral tilsotolimod in combination with ipilimumab or pembrolizumab in patients with PD-1 refractory metastatic melanoma:

Ipilimumab Combination Arm – Phase 2 Expansion Ongoing at RP2D of 8mg

Enrollment (60 patients) completion expected by year end 2018;
5 of the first 10 evaluable patients at the 8 mg dose of tilsotolimod were responders (50% ORR);
Additional data from the Phase 2 expansion of ILLUMINATE-204 selected for presentation at upcoming ASCO (Free ASCO Whitepaper) meeting in Chicago, IL.
Melanoma/Skin Cancers poster discussion session on June 4, 2018 at 4:45 PM CT; and
Investor/Analyst Event featuring lead ILLUMINATE-204 investigator, Adi Diab, MD from the University of Texas, MD Anderson Cancer Center to be held at 6:30 PM CT, also on June 4, 2018. As a convenience to those unable to attend, this event will be webcast.
Pembrolizumab Combination Arm – Phase 1 Dose Escalation Ongoing

Enrollment into the last dosing cohort (32 mg) ongoing;
The previously reported partial response (PR) in 1 of the first 6 patients in the 16 mg cohort of intratumoral tilsotolimod in combination with pembrolizumab has evolved into a confirmed complete response (CR).
ILLUMINATE 101 – Phase 1b trial of intratumoral tilsotolimod monotherapy in patients with refractory solid tumors:

Completed enrollment in first two cohorts (11 patients treated with 8 mg dose of tilsotolimod, 8 patients treated with 16 mg dose of tilsotolimod);
Two patients in cohort 1 (8 mg) continue in follow-up; 2 patients in cohort 2 (16 mg) continue tilsotolimod monotherapy and two patients continue in follow-up; and
6 of 8 planned patients for cohort 3 (23 mg) enrolled.
(IMO-8400) Development Activities

PIONEER-211 – Randomized placebo controlled Phase 2 trial of IMO-8400 in adult patients with dermatomyositis:

Enrollment concluded during Q3 2017 (30 patients); and
Topline phase 2 trial data expected in June 2018.
Nucleic Acid Chemistry Research Group

We are developing our nucleic acid chemistry technology to "turn off" the mRNA associated with disease causing genes. Our focus is on creating candidates targeted to specific genes to treat cancer and rare diseases.
We had selected IDRA-008 as our first nucleic acid chemistry research program candidate. IDRA-008 targets the Apolipoprotein C-III (APOC-III) gene and was being developed for the treatment of Familial Chylomicronemia Syndrome (FCS) and Familial Partial Lipodystrophy (FPL) which had available pre-clinical animal models and well-known clinical endpoints.
During the first quarter of 2018, we completed our pre-clinical analysis for IDRA-008 and based upon the outcome of pre-clinical pharmacology studies, including a comparative pharmacology study with the competitive development asset volanesorsen, and IND-enabling safety evaluation, we made a data-driven decision to not advance IDRA-008 into clinical development.
We are currently conducting analysis throughout our research portfolio to identify other candidates for future clinical development based on our nucleic acid technology expertise and potential strategic commercial opportunity.
Financial Results
First Quarter Results
Net loss applicable to common stockholders for the three months ended March 31, 2018 was $20.1 million, or $0.10 per basic and diluted share, compared to net loss applicable to common stockholders of $15.1 million, or $0.10 per basic and diluted share, for the same period in 2017. Revenue in each of the three months ended March 31, 2018 and 2017 was nominal. Research and development expenses for the three months ended March 31, 2018 totaled $13.6 million compared to $11.5 million for the same period in 2017. General and administrative expense for the three months ended March 31, 2018 totaled $7.0 million compared to $4.1 million for the same period in 2017.

During the three months ended March 31, 2018, holders of warrants, including Baker Brothers, exercised warrants to purchase shares of the Idera’s common stock which generated $9.6 million in cash proceeds. As of March 31, 2018, our cash and cash equivalents totaled $107.5 million compared to $112.6 million as of December 31, 2017. We currently anticipate that, based on our current operating plan and without taking into account the transaction with BioCryst Pharmaceuticals, Inc. ("BioCryst"), our existing cash, cash equivalents and investments will fund our operations into the third quarter of 2019.

Corporate Updates:

On January 22, 2018, BioCryst and Idera jointly announced the signing of a definitive merger agreement to create a company focused on the development and commercialization of medicines to serve patients suffering from rare diseases. The combined company will be renamed upon closing, and will be led by Vincent Milano, the current chief executive officer of Idera. Jon Stonehouse, the current chief executive officer of BioCryst, will serve as a member of the Board of Directors. The transaction is subject to approval by the stockholders of both companies, as well as the satisfaction of customary closing conditions. The transaction is expected to be completed by the end of the third quarter of 2018.

On May 9, 2018 Endocyte, Inc. (Nasdaq:ECYT), a biopharmaceutical company developing targeted therapeutics for personalized cancer treatment, reported financial results for the first quarter ending Mar. 31, 2018 and provided an operational update (Press release, Endocyte, MAY 9, 2018, View Source [SID1234526314]).

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"We made important progress during the first quarter in establishing the design of our phase 3 VISION trial of 177Lu-PSMA-617, securing clinical supply of no-carrier-added Lutetium, and raising sufficient capital to fund the company through expected completion of the trial," said Mike Sherman, president and CEO of Endocyte. "We continue to expect the first patient visit in the VISION trial in the second quarter and are working to advance EC17/CAR T-cell therapy, our folate-targeted CAM-based therapy, for which we expect to have an IND submitted in the fourth quarter of 2018."

Mr. Sherman continued, "In addition, we are encouraged by the updated 30 patient data from the ongoing phase 2 trial at Peter MacCallum Cancer Centre in Melbourne, Australia, published today in The Lancet Oncology. We anticipate an update at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting in June with early data on the additional 20 patients dosed in the expansion phase of that trial. Enrollment also continues in the phase 2 TheraP trial in Australia comparing 177Lu-PSMA-617 to cabazitaxel in 200 patients."

First Quarter and Recent Highlights

Finalized the design for the phase 3 VISION trial evaluating 177Lu-PSMA-617 in patients with metastatic castration-resistant prostate cancer (mCRPC) following a successful End-of-Phase 2 meeting with the U.S. Food and Drug Administration.

Announced an agreement with ITM Isotopen Technologien München AG to supply no-carrier-added Lutetium (177Lu) to support the phase 3 VISION trial.

Presented data on the chimeric antigen receptor T-cell (CAR T) adaptor molecule (CAM) platform at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2018 confirming the anti-tumor activity of Endocyte’s folate-targeted EC17/CAR T-cell therapy.

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Completed an underwritten registered public offering of 20,535,714 shares of its common stock, including full exercise of the underwriters’ option to purchase additional shares of common stock, at a public offering price of $4.20 per share. Endocyte received aggregate net proceeds from the offering of approximately $80.9 million.

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Hired additional experienced clinical trial professionals to ensure strong execution and support the success of its clinical programs.

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Elected Patrick Machado, J.D., co-founder and former chief business and financial officer of Medivation, and Dawn Svoronos, former president of Merck’s Europe/Canada region, to serve on the Board of Directors, bringing significant commercial leadership and understanding of the prostate cancer market to the Board.

Expected 2018 Milestones

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First patient visit for phase 3 VISION trial of 177Lu-PSMA-617 in mCRPC (2Q 2018).

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50-patient response rate data readout of investigator-initiated trial of 177Lu-PSMA-617 in mCRPC patients at Peter MacCallum Cancer Centre in Melbourne, Australia, to be presented at the Annual Meeting of the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) (June 2018).

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Publications on additional ongoing investigator-initiated clinical trials of 177Lu-PSMA-617 in prostate cancer patients (2018).

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IND for phase 1 trial of EC17/CAR T-cell therapy in patients with osteosarcoma (4Q 2018).

First Quarter 2018 Financial Results

Endocyte reported a net loss of $8.6 million, or $0.16 per basic and diluted share, for the first quarter of 2018, compared to a net loss of $11.5 million, or $0.27 per basic and diluted share for the same period in 2017.

Research and development expenses were $5.3 million for the first quarter of 2018, compared to $8.0 million for the same period in 2017. The decrease was primarily attributable to a strategic portfolio review announced in June 2017 which led to a reduction in workforce and the discontinuation of certain research and development activities, including: a decrease of $1.4 million in expenses related to pre-clinical work and general research, including the development of EC2629; a decrease of $0.8 million in EC1169 trial expenses; a decrease of $0.6 million in EC1456 trial expenses; a decrease of $0.5 million in compensation expense as a result of employee terminations since March 31, 2017, and a decrease of $0.4 million in manufacturing expense for EC1169 and EC1456. These decreases were partially offset by: an increase of $0.8 million in expenses related to development of PSMA-617; and an increase of $0.2 million related to our CAR T-cell therapy program.

General and administrative expenses were $3.8 million for the first quarter of 2018, which were consistent with the $3.7 million of expenses for the same period in 2017.

Cash, cash equivalents and investments were $173.1 million at Mar. 31, 2018, compared to $127.6 million at Mar. 31, 2017, and $97.5 million at Dec. 31, 2017. Cash, cash equivalents and investments of $173.1 million at Mar. 31, 2018 included $80.9 million of net proceeds from our public offering of 20,535,714 shares of our common stock that closed in March 2018.

Financial Expectations

The company anticipates its cash, cash equivalents and investments balance at the end of 2018 to exceed $130 million. Based on current operational assumptions, Endocyte has sufficient cash to fund its activities through the expected end of the VISION trial and potential proof of concept of its EC17/CAR T-cell therapy.

Conference Call

Endocyte management will host a conference call today at 8:30 a.m. EDT.

U.S. and Canadian participants:(877) 845-0711

International:(760) 298-5081

A live, listen-only webcast of the conference call may be accessed by visiting the Investors & News section of the Endocyte website, www.endocyte.com.

The webcast will be recorded and available on the company’s website for 90 days following the call.

Website Information

Endocyte routinely posts important information for investors on its website, www.endocyte.com, in the "Investors & News" section. Endocyte uses this website as a means of disclosing material information in compliance with its disclosure obligations under Regulation FD. Accordingly, investors should monitor the "Investors & News" section of

Endocyte’s website, in addition to following its press releases, SEC filings, public conference calls, presentations and webcasts. The information contained on, or that may be accessed through, Endocyte’s website is not incorporated by reference into, and is not a part of, this document.