On November 7, 2017 Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, reported its consolidated financial results for the third quarter 2017, and highlighted recent progress in advancing its pipeline (Press release, Alnylam, NOV 7, 2017, View Source [SID1234521669]).

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"In our view, 2017 has proven to be a remarkable year for RNAi therapeutics, for Alnylam, and, most importantly, for the patients that we serve. With patisiran, our recent APOLLO Phase 3 study results demonstrate what we believe to be the transformative potential for RNAi therapeutics as a new class of innovative medicines. With these data, we expect to submit our first regulatory filings in the coming months, and are planning for the possibility of having regulatory approval for patisiran in mid-2018," said John Maraganore, Ph.D., Chief Executive Officer of Alnylam. "We have also made significant progress across our other programs, including the initiation of our ENVISION Phase 3 program for givosiran in acute hepatic porphyrias and, with our partners at The Medicines Company, the ORION Phase 3 program in hypercholesterolemia. Additionally, we aim to resume dosing in all fitusiran studies, including the ATLAS Phase 3 program, as soon as possible. These milestones position Alnylam with multiple late-stage clinical assets, while we concurrently transition into a fully integrated commercial company with the goal of delivering innovative medicines to patients around the world."

Third Quarter 2017 and Recent Significant Corporate Highlights

Advanced patisiran, an investigational RNAi therapeutic in development for the treatment of patients with hereditary ATTR (hATTR) amyloidosis, with positive results from the APOLLO Phase 3 study (N=225).
Patisiran met its primary endpoint (p = 9.26 x 10-24) with a 34.0 point mean difference relative to placebo and a negative 6.0 point mean change (improvement) relative to baseline in the modified neuropathy impairment score (mNIS+7) at 18 months, as well as all secondary endpoints (p less than 0.001), including a 21.1 point mean difference relative to placebo and a negative 6.7 point mean change (improvement) relative to baseline in the NORFOLK Quality of Life-Diabetic Neuropathy score (NORFOLK QOL-DN) (p = 1.10 x 10-10), at 18 months.
Patisiran also demonstrated a favorable safety and tolerability profile relative to placebo. The most commonly reported adverse events (AEs) for patisiran were generally mild to moderate and included peripheral edema (29.7 percent) and infusion-related reactions (IRRs) (18.9 percent), and the frequency of deaths and serious adverse events (SAEs) was similar in the patisiran and placebo groups. No deaths were considered drug-related.
Specifically, the Company believes that these data support a potentially "best-in-class" product profile, with significant benefit relative to placebo, negative mean and median values (improvement) for mNIS+7 and QOL measures relative to baseline, and encouraging safety and tolerability.
In addition, patisiran achieved significant effects in the study’s cardiac subpopulation, including on disease biomarker, echocardiographic, and functional parameters.
The Company believes the totality of the APOLLO data are consistent with a clinically meaningful impact for patisiran on hATTR amyloidosis, and plans to submit an NDA for patisiran by the end of 2017 and a Marketing Authorisation Application (MAA) shortly thereafter.
Advanced ALN-TTRsc02, a subcutaneously administered investigational RNAi therapeutic in development for the treatment of ATTR amyloidosis.
Presented updated Phase 1 data showing up to 95% transthyretin (TTR) knockdown with a single 50 mg dose, with durability supportive of a once quarterly and, possibly, bi-annual subcutaneous dose regimen.
Reaffirmed guidance to initiate a Phase 3 program for ALN-TTRsc02 in 2018.
Advanced givosiran, an investigational RNAi therapeutic in development for the treatment of acute hepatic porphyrias (AHPs), with initiation of the ENVISION Phase 3 study.
The Company reached alignment with the U.S. Food and Drug Administration (FDA) on the design of ENVISION, including an interim analysis based on reduction of urinary aminolevulinic acid (ALA), a biomarker that the FDA considers to be reasonably likely to predict clinical benefit.
The Company has also reached alignment on the ENVISION Phase 3 study design with the European Medicines Agency (EMA).
The Company is guiding that it expects interim analysis results in mid-2018 and, pending FDA review of the program at the time of interim analysis and assuming positive results, it expects to submit an NDA at or around year-end 2018.
Advanced fitusiran, an investigational RNAi therapeutic in development for the treatment of hemophilia A and B with or without inhibitors, with new positive data from the Phase 2 open-label extension (OLE) study presented at the International Society on Thrombosis and Haemostasis 2017 Congress.
Results from the Phase 1 study were published in The New England Journal of Medicine in a paper titled, "Targeting of Antithrombin in Hemophilia A or B with RNAi Therapy."
Announced the initiation of the ATLAS Phase 3 program, a global, multicenter clinical program designed to evaluate the safety and efficacy of fitusiran in patients with hemophilia A and B with or without inhibitors.
The Company temporarily suspended dosing in all ongoing studies of fitusiran following the observation of a fatal thrombotic SAE that occurred in a patient with hemophilia A without inhibitors who was receiving fitusiran in the Phase 2 OLE study. Alnylam and fitusiran study investigators have aligned on a risk management plan for further advancement of fitusiran and are now conferring with global regulators with the goal of resuming dosing as soon as possible, potentially by the end of 2017.
Alnylam and The Medicines Company announced initiation of the ORION-11 Phase 3 study of inclisiran, an investigational RNAi therapeutic targeting PCSK9 in development for the treatment of hypercholesterolemia, in patients with atherosclerotic cardiovascular disease (ASCVD).
The companies announced new positive data from the ORION-1 Phase 2 study of inclisiran at the European Society of Cardiology Congress 2017.
Advanced cemdisiran (formerly known as ALN-CC5), a subcutaneously administered investigational RNAi therapeutic targeting complement component C5 for the treatment of complement-mediated diseases, with the initiation of a Phase 2 clinical study in patients with atypical hemolytic-uremic syndrome (aHUS).
Advanced lumasiran (formerly known as ALN-GO1), an investigational RNAi therapeutic in development for the treatment of primary hyperoxaluria type 1 (PH1), with new positive data from the Phase 1/2 study presented at the American Society of Nephrology Kidney Week 2017 Annual Meeting.
Alnylam announced a licensing agreement with Vir Biotechnology for the development and commercialization of RNAi therapeutics for infectious diseases, including hepatitis B.
Advanced RNAi platform technology with new pre-clinical data on Alnylam’s next generation "Enhanced Stabilization Chemistry Plus" (ESC+) GalNAc-siRNA conjugate platform presented at the 13th Annual Meeting of the Oligonucleotide Therapeutics Society.
Upcoming Events

Alnylam announces today that Alnylam scientists and collaborators will present new results from multiple pipeline programs at the 59th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting, being held December 9 – 12, 2017 in Atlanta, Georgia. Presentations include:
Explore: A Prospective, Multinational History Study of Patients with Acute Hepatic Porphyrias (AHP) with Recurrent Attacks
Session: 102. Regulation of Iron Metabolism: Poster II
Date/Time: Sunday, December 10, 6:00 – 8:00 p.m. ET
Perioperative Management in Patients with Hemophilia Receiving Fitusiran, an Investigational RNAi Therapeutic Targeting Antithrombin for the Treatment of Hemophilia
Session: 322. Disorders of Coagulation or Fibrinolysis: Poster II
Date/Time: Sunday, December 10, 6:00 – 8:00 p.m. ET
In Silico Modeling of the Impact of Antithrombin Lowering on Thrombin Generation in Rare Bleeding Disorders
Session: 321. Blood Coagulation and Fibrinolytic Factors: Poster III
Date/Time: Monday, December 11, 6:00 – 8:00 p.m. ET
Alnylam plans to file its first NDA for patisiran with the FDA by the end of 2017, followed by an MAA in the EU in early 2018.
Alnylam plans to meet with global regulatory authorities with the goal of reaching agreement on a risk mitigation plan and resumption of dosing in fitusiran clinical studies.
Financial results for the quarter ended September 30, 2017

"Alnylam’s strong balance sheet and overall financial position allow us to build our commercial capabilities in preparation for anticipated product launches in the U.S. and Western Europe during 2018, assuming regulatory approvals in the U.S. and EU," said Manmeet Soni, Chief Financial Officer of Alnylam. "Additionally, we continue to invest in our broad pipeline of investigational RNAi therapeutics, advancing our four late-stage programs as well as the early-stage pipeline programs."

Cash and Investments
At September 30, 2017, Alnylam had cash, cash equivalents and fixed income marketable securities, and restricted investments of $1.15 billion, as compared to $1.09 billion at December 31, 2016.

GAAP and Non-GAAP Net Loss
The net loss according to accounting principles generally accepted in the U.S. (GAAP) for the third quarter of 2017 was $122.9 million, or $1.34 per share on both a basic and diluted basis, as compared to a net loss of $104.1 million, or $1.21 per share on both a basic and diluted basis, for the same period in the previous year.

The non-GAAP net loss for the third quarter of 2017 was $97.0 million, or $1.06 per share on both a basic and diluted basis, as compared to a non-GAAP net loss of $88.5 million, or $1.03 per share on both a basic and diluted basis for the same period in the previous year.

The non-GAAP net loss excludes stock-based compensation expense. See "Use of Non-GAAP Financial Measures" below for a description of non-GAAP financial measures and a reconciliation between GAAP and non-GAAP net loss appearing later in this press release.

Revenues
Revenues were $17.1 million in the third quarter of 2017, as compared to $13.7 million in the third quarter of 2016. Revenues for the third quarter of 2017 included $14.6 million from the Company’s alliance with Sanofi Genzyme, $2.3 million from the Company’s alliance with The Medicines Company and $0.2 million from other sources.

GAAP and Non-GAAP Research and Development Expenses
GAAP research and development (R&D) expenses were $95.3 million in the third quarter of 2017 as compared to $97.9 million in the third quarter of 2016.

Non-GAAP R&D expenses were $80.2 million in the third quarter of 2017 as compared to $88.6 million in the third quarter of 2016. Non-GAAP R&D expenses exclude stock-based compensation expense. A reconciliation between GAAP and non-GAAP R&D expenses appears later in this press release.

GAAP and Non-GAAP General and Administrative Expenses
GAAP general and administrative (G&A) expenses were $47.6 million in the third quarter of 2017 as compared to $22.4 million in the third quarter of 2016.

Non-GAAP G&A expenses were $36.8 million in the third quarter of 2017 as compared to $16.2 million in the third quarter of 2016. Non-GAAP G&A expenses exclude stock-based compensation expense. A reconciliation between GAAP and non-GAAP G&A expenses appears later in this press release.

Financial Guidance
Alnylam remains on track to end 2017 with greater than $1.0 billion in cash, cash equivalents and fixed income marketable securities including $150.0 million in restricted investments.

Conference Call Information
Management will provide an update on the Company and discuss third quarter 2017 results as well as expectations for the future via conference call on Tuesday, November 7, 2017 at 4:30 p.m. ET. To access the call, please dial 877-312-7507 (domestic) or 631-813-4828 (international) five minutes prior to the start time and refer to conference ID 2432127. A replay of the call will be available beginning at 7:30 p.m. ET on the day of the call. To access the replay, please dial 855-859-2056 (domestic) or 404-537-3406 (international), and refer to conference ID 2432127.

Alnylam – Sanofi Genzyme Alliance
In January 2014, Alnylam and Sanofi Genzyme, the specialty care global business unit of Sanofi, formed an alliance to accelerate the advancement of RNAi therapeutics as a potential new class of innovative medicines for patients around the world with rare genetic diseases. The alliance enables Sanofi Genzyme to expand its rare disease pipeline with Alnylam’s novel RNAi technology and provides access to Alnylam’s R&D engine, while Alnylam benefits from Sanofi Genzyme’s proven global capabilities to advance late-stage development and, upon commercialization, accelerate market access for these promising genetic medicine products.

In the case of patisiran, Alnylam will advance the product in the United States, Canada and Western Europe, while Sanofi Genzyme will advance the product in the rest of the world. In November 2016, Sanofi Genzyme elected to co-develop (through Sanofi R&D) and co-commercialize fitusiran in the United States, Canada and Western Europe, in addition to commercializing fitusiran in its rest of world territories. Sanofi Genzyme has the right to opt in to develop and commercialize lumasiran in territories outside of the United States, Canada and Western Europe and could elect to exercise its one right to a global license for lumasiran. In the case of ALN-TTRsc02, Sanofi Genzyme has the right to opt into the program with co-development/co-commercialization rights.

About RNAi
RNAi (RNA interference) is a natural cellular process of gene silencing that represents one of the most promising and rapidly advancing frontiers in biology and drug development today. Its discovery has been heralded as "a major scientific breakthrough that happens once every decade or so," and was recognized with the award of the 2006 Nobel Prize for Physiology or Medicine. By harnessing the natural biological process of RNAi occurring in our cells, a major new class of medicines, known as RNAi therapeutics, is on the horizon. Small interfering RNA (siRNA), the molecules that mediate RNAi and comprise Alnylam’s RNAi therapeutic platform, function upstream of today’s medicines by potently silencing messenger RNA (mRNA) – the genetic precursors – that encode for disease-causing proteins, thus preventing them from being made. This is a revolutionary approach with the potential to transform the care of patients with genetic and other diseases.

About LNP Technology
Alnylam has licenses to Arbutus LNP intellectual property for use in RNAi therapeutic products using LNP technology.

On November 7, 2017 Bellicum Pharmaceuticals, Inc. (NASDAQ:BLCM), a leader in developing novel, controllable cellular immunotherapies for cancers and orphan inherited blood disorders, reported financial results for the third quarter ended September 30, 2017, and provided an update on recent developments (Press release, Bellicum Pharmaceuticals, NOV 7, 2017, View Source;p=RssLanding&cat=news&id=2315120 [SID1234521670]).

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"We made good progress advancing our pipeline in the third quarter. Enrollment in our clinical program for BPX-501 remains on track and we progressed our plans for future trials in adult AML and a pediatric orphan blood disorder," said Rick Fair, Bellicum’s President & Chief Executive Officer. "On BPX-601, we modified our Phase 1 trial to accelerate evaluation of our first clinical GoCAR-T candidate, and we look forward to reporting preliminary results next year. Finally, we continued to advance several exciting preclinical programs, leveraging our dual-switch controllable cell therapy platform."

PROGRAM HIGHLIGHTS AND CURRENT UPDATES

BPX-501
Adjunct T-cell therapy incorporating the CaspaCIDe safety switch, administered after a haploidentical hematopoietic stem cell transplant (haplo-HSCT), to improve outcomes and reduce mortality

Bellicum Continues to Advance its BPX-501 Program
Enrollment in the EU BP-004 clinical trial remains on track to be complete by the end of 2017. Bellicum has also initiated C-004, an observational trial in pediatric patients receiving transplants from matched unrelated donors (MUD) without BPX-501. The outcomes of both trials could form the basis for filings of European Marketing Authorization Applications for BPX-501 and rimiducid. A BPX-501 abstract, highlighting data on immune reconstitution from the EU BP-004 clinical trial, has been accepted for an oral presentation at the upcoming 59th Annual Meeting of the American Society of Hematology (ASH) (Free ASH Whitepaper) in December.
Company Prepares for Additional BPX-501 Trials in U.S.
Planning is ongoing for two additional trials of BPX-501 to expand the eligible patient population and support potential U.S. registration. These trials are being developed in adult patients with acute myeloid leukemia (AML) and in a distinct orphan inherited blood disorder patient population.
BPX-601
Novel GoCAR-T product candidate designed with the proprietary iMC activation switch to improve efficacy

Phase 1 BPX-601 Clinical Trial Progressing
Enrollment and treatment is currently ongoing in the Company’s Phase 1 trial in nonresectable pancreatic cancer patients who test positive for prostate stem cell antigen (PSCA). Based on data from the initial three treated patients, the Company—with support from the principal investigator and the FDA—amended the study protocol to allow activation of the iMC switch seven days following the administration of BPX-601 versus the previous 30-day schedule. This will enable an earlier evaluation of the first clinical experience with GoCAR-T, the Company’s platform to enhance and control CAR-T cell activation, proliferation, and survival.
BPX-701
High affinity T-cell receptor (TCR) product candidate designed with the CaspaCIDe safety switch

Phase 1 BPX-701 Clinical Trial Progressing
Enrollment and treatment is currently ongoing in the Company’s Phase 1 clinical trial in patients with refractory or relapsed AML and myelodysplastic syndromes (MDS) who test positive for preferentially-expressed antigen in melanoma (PRAME).
CD19 Program
Bellicum is working with academic collaborators to evaluate the benefit of CaspaCIDe in managing serious toxicities associated with CD19 CAR-T cells

In collaboration with Ospedale Pediatrico Bambino Gesù (OPBG), a leading European pediatric research center and hospital, a Phase 1 clinical trial of a CaspaCIDe-enabled CD19 CAR-T is expected to begin in the fourth quarter.
Preclinical Programs

At ASH (Free ASH Whitepaper) 2017, the Company will present preclinical results of its controllable CAR-T technology in three poster presentations, including a study evaluating Bellicum’s dual-switch CAR-T cells targeting CD123. The data will further support the potential of the Company’s technology platform to control cells in vivo and improve the benefit / risk profile of adoptive cell therapies.
Third Quarter and Nine Months Ended September 30, 2017 Financial Results

Bellicum reported a net loss of $23.4 million for the third quarter of 2017 and $69.9 million for the nine months ended September 30, 2017, compared to a net loss of $17.7 million and $49.3 million for the comparable periods in 2016. The results included non-cash, stock-based compensation charges of $3.7 million and $10.2 million for the third quarter and nine months ended September 30, 2017, and $3.1 million and $9.2 million for the comparable periods in 2016.

As of September 30, 2017, cash and investments totaled $118.6 million. Bellicum expects that it will end 2017 with approximately $90 to $95 million in cash, cash equivalents and investments, and continues to expect that current cash resources will be sufficient to meet operating requirements through 2018.

Research and development expenses were $18.1 million and $51.4 million for the three and nine months ended September 30, 2017, respectively, compared to $13.3 million and $36.2 million during the comparable periods in 2016. The higher expenses in the 2017 periods were primarily due to an increase in clinical development and manufacturing costs due to increased enrollment in clinical trials, principally BP-004, and increased personnel expenses, overhead charges and manufacturing facility start-up costs.

General and administrative expenses were $4.6 million and $16.0 million for the three and nine months ended September 30, 2017, respectively, compared to $4.3 million and $12.7 million during the comparable periods in 2016. The higher expenses in the 2017 periods were primarily due to the Company’s overall growth, including an increase in personnel related costs, principally due to hiring additional employees and severance costs, higher facility costs and increased legal, accounting and travel expenses.

About BPX-501
BPX-501 is an adjunct T-cell therapy administered after allogeneic HSCT, comprising genetically modified donor T cells incorporating Bellicum’s CaspaCIDe safety switch. It is designed to provide a safety net to eliminate alloreactive BPX-501 T cells (via administration of activator agent rimiducid) should uncontrollable GvHD occur. This could enable physicians to more safely perform allogeneic stem cell transplants by administering BPX-501 engineered T cells to speed immune reconstitution, provide control over viral infections and enhance Graft-versus-leukemia effect, without unacceptable GvHD risk. The ongoing BP-004 clinical study of BPX-501 is being conducted at transplant centers in the U.S. and Europe.

On November 7, 2017 ChemoCentryx, Inc., (Nasdaq:CCXI), a biopharmaceutical company developing new medications targeted at inflammatory and autoimmune diseases and cancer, reported financial results for the third quarter ended September 30, 2017 (Press release, ChemoCentryx, NOV 7, 2017, View Source [SID1234521673]).

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"Our pursuit of new and better medicines for people with orphan diseases has been relentless," said Thomas J. Schall, Ph.D., President and Chief Executive Officer of ChemoCentryx. "Dedicated to creating value for patients and shareholders alike, we at CCXI started with basic science in the discovery of novel molecules that selectively inhibit chemoattractant receptors, which are the molecular guidance systems of destructive inflammatory cells involved in a wide range of diseases and conditions. Now we have advanced two of those novel molecules, avacopan and CCX140, well into late-stage clinical trials. In doing so, we move closer to the next phase of value creation – the potential commercialization of our targeted medicines to help those suffering from serious renal diseases."

Recent Highlights

ChemoCentryx’s Phase III ADVOCATE trial of avacopan for the treatment of ANCA-associated vasculitis has surpassed 30 percent of its target patient enrollment with more than 185 sites activated. The trial will test the safety and efficacy of avacopan following 12 months of treatment and will include approximately 300 patients. In addition to testing the effect of avacopan on improving active vasculitis, the ADVOCATE trial will also test the effect of avacopan on preventing a recurrence of vasculitis.

ChemoCentryx recently received orphan designation in Switzerland from SwissMedic for avacopan for the treatment of two forms of ANCA-vasculitis: microscopic polyangiitis and granulomatosis with polyangiitis (formerly known as Wegener’s granulomatosis). This designation is in addition to the previously received orphan designations from the U.S. Food and Drug Administration (FDA) and European Medicines Agency (EMA) for avacopan to treat ANCA-vasculitis.

ChemoCentryx recently launched a registration-supporting clinical trial to study avacopan in a second indication, C3 Glomerulopathy (C3G), a rare disorder that often affects the young, requiring dialysis and often kidney transplant. Sites have been activated for the trial and patient enrollment has begun. Earlier this year ChemoCentryx announced that it had received both EMA orphan medicinal product designation and FDA orphan drug designation for avacopan in the treatment of C3G.

ChemoCentryx is launching a third registration-supporting trial, involving its CCR2 inhibitor, CCX140, to treat the debilitating kidney disorder known as Focal Segmental Glomerulosclerosis (FSGS), for which there is no approved treatment option. The Company plans to launch a trial in the fourth quarter of 2017.
Third Quarter 2017 Financial Results

Pro forma cash, cash equivalents, investments and remaining upfront commitments totaled $154.8 million at September 30, 2017.

Revenue was $9.0 million for the third quarter, compared to $4.1 million for the same period in 2016. The increase in revenue from 2016 to 2017 were due to: (i) amortization of the upfront license fee commitments from Vifor pursuant to the avacopan and CCX140 agreements; as well as (ii) collaboration revenue for development services under the CCX140 Agreement in 2017. These increases were partially offset by lower grant revenue from the FDA to support the clinical development of avacopan for the treatment of patients with ANCA vasculitis.

Research and development expenses were $12.3 million for the third quarter, compared to $8.4 million for the same period in 2016. The increase in research and development expenses from 2016 to 2017 was primarily attributable to the initiation and patient enrollment of the avacopan Phase III ADVOCATE trial in patients with ANCA vasculitis and start-up expenses for the Phase II clinical trial of avacopan for the treatment of C3G. These increases were partially offset by lower costs associated with the completion of the avacopan CLEAR and CLASSIC Phase II clinical trials for the treatment of ANCA vasculitis and enrollment completion of the CCX872 Phase I trial in patients with advanced pancreatic cancer in 2016.

General and administrative expenses were $3.6 million for the third quarter, compared to $3.2 million for the same period in 2016. The increase from 2016 to 2017 was primarily due to accounting related fees associated with preparing to meet the requirements pursuant to the Sarbanes-Oxley Act of 2002.

Net losses for the third quarter were $6.6 million, compared to $7.1 million for the same period in 2016.

Total shares outstanding at September 30, 2017 were approximately 48.8 million shares.

The Company expects to utilize cash and cash equivalents in the range of $50 million and $55 million in 2017, of which $39.0 million has been used for the nine months ended September 30, 2017.

Conference Call and Webcast

The Company will host a conference call and webcast today, November 7, 2017 at 5:00 p.m. Eastern Time / 2:00 p.m. Pacific Time. To participate by telephone, please dial 877-303-8028 (Domestic) or 760-536-5167 (International). The conference ID number is 7796287. A live and archived audio webcast can be accessed through the Investors section of the Company’s website at www.ChemoCentryx.com. The archived webcast will remain available on the Company’s website for fourteen (14) days following the conference call.

On November 7, 2017 CytomX Therapeutics, Inc. (NASDAQ: CTMX), a biopharmaceutical company developing investigational Probody therapeutics for the treatment of cancer, reported third quarter 2017 financial results (Press release, CytomX Therapeutics, NOV 7, 2017, View Source;p=RssLanding&cat=news&id=2315188 [SID1234521674]).

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As of September 30, 2017, CytomX had cash, cash equivalents and short-term investments of $331.3 million. Based upon its current operating plan, the Company expects its existing capital resources will be sufficient to fund operations into 2020.

"We have continued to enjoy a highly productive year with excellent progress in advancing our deep pipeline of potentially transformative Probody therapeutics and the addition of another major partner in Amgen," said Sean McCarthy, D.Phil., president and chief executive officer of CytomX Therapeutics. "I am delighted with the team’s strong execution across all areas of the organization, and we continue to be well positioned for initial data read outs in 2018 from our lead, wholly owned programs, CX-072 and CX-2009. We also expect that Probody therapeutics targeting CTLA-4, PD-1, and CD71 will enter the clinic next year, demonstrating the considerable momentum behind our pipeline."

Q3’17 BUSINESS HIGHLIGHTS AND RECENT DEVELOPMENTS

PROCLAIM-CX-072 (PD-L1 Probody Therapeutic) Clinical Program

Patient enrollment continued in the monotherapy dose escalation arm (Part A1) of the study evaluating CX-072 in patients with advanced unresectable solid tumors or lymphomas. Enrollment is expected to be complete by year-end.
Patient enrollment has now been initiated in all other dose escalation arms of the study:
Monotherapy expansion in patients with PD-L1-positive tumors at multiple dose levels (Part A2);
Concomitant schedule for CX-072 plus ipilimumab in patients with advanced unresectable solid tumors or lymphomas (Part B1);
Phased schedule for CX-072 plus ipilimumab in patients with advanced unresectable solid tumors or lymphomas (Part B2); and
Combination of CX-072 plus vemurafinib in patients with V600E BRAF-positive melanoma (Part C)
During the first half of 2018, an expansion cohort of the study at the recommended Phase 2 dose is expected to begin enrolling patients to evaluate CX-072 as monotherapy in a tumor type with known sensitivity to PD-L1 and/or PD-1 inhibitors (Part D).
PROCLAIM-CX-2009 (CD166 Probody Drug Conjugate) Clinical Program

CX-2009 is a first-in-class Probody drug conjugate (PDC) that targets CD166, an antigen that is broadly and highly expressed in many types of cancers, but has been considered undruggable given that it is also expressed in normal tissue.
Patient enrollment continues in the PROCLAIM-CX-2009 study, a Phase 1/2 clinical trial evaluating CX-2009 as monotherapy in a subset of CD166-positive cancers.
The study was initiated at a dose of 0.25 mg/kg, has advanced through several patient cohorts and is currently enrolling at a dose of 2 mg/kg.
Expansion cohorts at the recommended Phase 2 dose in one or more CD166 positive tumor types are expected to initiate in 2018.
CX-2029 (CD71 Probody Drug Conjugate) Preclinical Program

CytomX, in collaboration with AbbVie, is advancing CX-2029, a CD71-directed PDC, through Investigational New Drug (IND) application-enabling studies and expects to file an IND application in the first half of 2018.
A $15 million milestone payment ($14 million net of associated license fees) was received from AbbVie in conjunction with meeting certain criteria allowing the initiation of GLP toxicology studies by CytomX.
CX-188 (PD-1 Probody Therapeutic) Preclinical Program

CytomX is advancing CX-188, a PD-1-directed Probody therapeutic, through IND application-enabling studies and expects to file an IND application in the second half of 2018.
Amgen Partnership

During the quarter, Amgen and CytomX entered into a strategic collaboration in immuno-oncology in the field of Probody T-cell engaging bispecific antibodies including the co-development of a CytomX Probody T-cell engaging bispecific against the Epidermal Growth Factor Receptor (EGFR), a highly validated oncology target expressed on multiple human cancer types.
Under the terms of the agreement, Amgen and CytomX will co-develop a Probody T-cell engaging bispecific against EGFR-CD3 with CytomX leading early development. Amgen will lead later development and commercialization with global late-stage development costs shared between the two companies.
Amgen made an upfront payment of $40 million and purchased $20 million of CytomX common stock.
CytomX is eligible to receive up to $455 million in development, regulatory and commercial milestones for the EGFR program.
Amgen will lead global commercial activities with CytomX able to opt into a profit share in the U.S. and receive tiered, double-digit royalties on net product sales outside of the U.S.
Amgen also received exclusive worldwide rights to develop and commercialize up to three undisclosed targets. Should Amgen ultimately pursue all of these targets, CytomX will be eligible to receive up to $950 million in additional upfront and milestone payments and high single-digit to mid-double digit royalty payments on any resulting products.
CytomX also received the rights from Amgen to an undisclosed preclinical T-cell engaging bispecific program; Amgen is eligible to receive milestones and royalty payments on any resulting products from this CytomX program.
Bristol-Myers Squibb (BMS) Partnership

BMS continues to advance its CTLA-4-directed Probody therapeutic, which is expected to enter the clinic in early 2018.
In addition, CytomX and Bristol-Myers Squibb are evaluating a Probody version of Bristol-Myers Squibb’s CTLA-4 nonfucosylated (CLTA-4-NF) version of ipilimumab as part of the current collaboration.
Third Quarter Financial Results
Cash, cash equivalents and investments totaled $331.3 million as of September 30, 2017, compared to $181.9 million as of December 31, 2016. The increase reflects a $200 million upfront payment received from BMS in connection with the expansion of the existing collaboration.

Revenue was $24.1 million for the three months ended September 30, 2017, compared to $3.5 million for the three months ended September 30, 2016. The increase was primarily attributable to the recognition of $14.0 million, net of the associated license fees, from the milestone payment received from AbbVie as a result of the Company achieving certain milestones required to be met to begin GLP toxicology studies under the CD71 Agreement and an increase of $6.3 million related to the recognition of the an upfront payment received from BMS in connection with the expansion of our collaboration pursuant to an amendment of our Collaboration and License Agreement entered into in March 2017.

Research and development expenses were $28.9 million for the three months ended September 30, 2017, compared to $13.3 million for the corresponding period in 2016. The increase was primarily attributable to $10.7 million of in-process research and development expense recognized and a $1.2 million sublicense fee payable to UCSB as a result of the Amgen agreement, an increase of $1.7 million in pharmacology studies and clinical trial expenses resulting from the advancement of CX-072, CX- 2009 and CX-2029 in 2017, an increase of $0.9 million in allocations resulting from increases in facilities-related expenses, an increase of $0.4 million in consulting and contracted services and an increase of $0.4 million in personnel-related expense resulting from an increase in headcount.

General and administrative expenses were $6.2 million for the three months ended September 30, 2017, compared to $5 million for the corresponding period in 2016. The increase was primarily attributable to an increase of $0.3 million in personnel-related expenses due to an increase in headcount, an increase of $0.3 million in legal expenses and an increase of $0.4 million in consulting and public relations expenses.

On November 7, 2017 Ignyta, Inc. (Nasdaq: RXDX), a biotechnology company focused on precision medicine in oncology, reported company highlights and financial results for the third quarter ended September 30, 2017 (Press release, Ignyta, NOV 7, 2017, View Source [SID1234521678]). The company is issuing this press release in lieu of conducting a conference call.

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"In addition, during the quarter we continued to advance our pipeline of precision medicines targeted at the molecular and immunological drivers of cancer, and we strengthened our balance sheet through an equity offering that provides us with additional resources to continue developing meaningful new cancer therapies for patients."
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"We are pleased with the continued development and regulatory progress of our lead product candidate, entrectinib—an investigational, CNS-active, potent, and selective tyrosine kinase inhibitor being developed for tumors that harbor TRK or ROS1 fusions—as we approach the expected submission of two NDAs and a PMA in 2018," said Jonathan Lim, M.D., Chairman and CEO of Ignyta. "In addition, during the quarter we continued to advance our pipeline of precision medicines targeted at the molecular and immunological drivers of cancer, and we strengthened our balance sheet through an equity offering that provides us with additional resources to continue developing meaningful new cancer therapies for patients."

Company Highlights

Entrectinib

Regulatory Updates: Orphan Drug Designation and PRIME

In July 2017, we announced that FDA granted orphan drug designation to entrectinib for "treatment of NTRK fusion-positive solid tumors."

In October 2017, we announced that the European Medicines Agency (EMA) granted Priority Medicines (PRIME) designation for entrectinib in the treatment of NTRK fusion-positive, locally advanced or metastatic solid tumors in adult and pediatric patients who have either progressed following prior therapies or who have no acceptable standard therapy. Entrectinib is the only TRK inhibitor to have been granted PRIME designation, which is analogous to the Breakthrough Therapy Designation from the U.S. FDA that entrectinib received earlier in 2017.

Development and Clinical Data Updates Towards Dual TRK and ROS1 NDA Submissions

In September 2017, we announced completion of enrollment of the efficacy data sets for both the TRK tissue-agnostic (i.e., fusion-positive solid tumor) cohort and the ROS1 NSCLC cohort to support dual NDA submissions in 2018.

In October 2017, at the WCLC, we announced updated interim results from our clinical trials, including the STARTRK-2 trial, of entrectinib. In this interim analysis (based upon an enrollment cut-off of December 31, 2016 and data cut-off of September 13, 2017):

Entrectinib demonstrated a 78% confirmed objective response rate (ORR; by Investigator; 95% CI: 60.0, 90.7) and a 69% confirmed ORR (by Blinded Independent Central Review, or BICR; 95% CI: 50.0, 83.9) in 32 patients with locally advanced or metastatic non-small cell lung cancer (NSCLC) that harbored ROS1 fusions;
Entrectinib demonstrated compelling durability in these patients, with a median duration of response (mDOR) of 28.6 months (by BICR; 95% CI: 6.8, 34.8; median follow-up of 12.9 months) and a median progression-free survival (mPFS) of 29.6 months (by BICR; 95% CI: 7.7, 36.6; median follow-up of 8.5 months); and
Of the patients evaluated, 11 had CNS metastases at baseline as assessed by investigator, and 83% (5 out of 6; by BICR) of the patients with BICR-confirmed measurable CNS metastases at presentation had confirmed intracranial RECIST responses to treatment with entrectinib.
Entrectinib remained well tolerated, with more than 200 patients treated at the recommended Phase 2 dose, with mostly Grade 1-2 reversible treatment-related adverse events. The program is tracking towards dual NDA submissions in TRK and ROS1 in 2018, if supported by clinical data, with an anticipated U.S. commercial launch in both indications in 2019.

RXDX-105

In September 2017, at the ESMO (Free ESMO Whitepaper) conference, we announced new Phase 1b clinical data on RXDX-105—an investigational, VEGFR-sparing, potent RET inhibitor—in which a preliminary ORR of 75 percent was observed in patients with non-KIF5B-RET fusions, with six of eight patients achieving a confirmed partial response. In contrast, those with KIF5B-RET fusions (14 patients) did not demonstrate RECIST responses. RXDX-105 continued to be well tolerated, with the most common treatment-related adverse events being Grade 1 or 2 and reversible with dose modifications. The most common Grade 3 treatment-related adverse events (> 5%) were rash (10%), hypophosphatemia (7%) and elevated ALT (7%).

RXDX-106

In October 2017, at the AACR (Free AACR Whitepaper) Tumor Immunology and Immunotherapy meeting, we presented new data highlighting the immuno-oncological efficacy of RXDX-106—a novel immunomodulatory agent with potent anti-tumor activity, alone and in combination with checkpoint inhibitors, that in preclinical models has demonstrated immunomodulatory effects in the tumor microenvironment (TME) through TYRO3, AXL, and MER (TAM) receptor tyrosine kinase (RTK) inhibition. The data presented demonstrated immune-mediated, single-agent anti-tumor activity of RXDX-106 in multiple tumor models. The anti-tumor effect was further enhanced by combination therapy with immune checkpoint inhibitors, potentially by reversing immunosuppression of innate immunity in the TME. The data also suggested that RXDX-106 has a novel mechanism of enhancing overall immune function by activating both innate and adaptive immunity, as observed by treatment-mediated changes in relevant cytokine levels and immune cell biomarkers, and regulating cross-talk between immune and cancer cells. These promising early findings support further development of RXDX-106 to potentially treat a wide variety of cancers.

Financing Transaction

In October 2017, the company raised aggregate gross proceeds of $160.0 million after issuing 10.0 million shares of its common stock in an underwritten public offering at a price to the public of $16.00 per share.

Third Quarter 2017 Financial Results

For the third quarter of 2017, net loss was $28.6 million, or $0.51 per share, compared with $23.3 million, or $0.56 per share, for the third quarter of 2016.

Ignyta did not record any revenue for the third quarter of 2017 or for the third quarter of 2016.

Research and development expenses for the third quarter of 2017 were $21.7 million, compared with $16.6 million for the third quarter of 2016. This increase was due to an increase in external clinical development costs and the chemistry, manufacturing and control costs associated with entrectinib and our other product candidates, and increased facilities costs of $1.1 million due to the expansion of our leased facilities space.

General and administrative expenses for the third quarter of 2017 were $6.5 million, compared with $6.1 million for the third quarter of 2016. This increase was due to an increase in our facilities costs, as described above, and an increase in outside services expenses, due to an increase in pre-launch commercial activities, which was partially offset by a reduction in depreciation expense.

At September 30, 2017, we had cash, cash equivalents and available-for-sale securities totaling $144.8 million—which does not include the $150 million of net proceeds from the October financing—and current and long-term debt of $32.0 million. At December 31, 2016, we had cash, cash equivalents and available-for-sale securities totaling $133.0 million and current and long-term debt of $32.0 million.