On November 7, 2019 CytomX Therapeutics, Inc. (Nasdaq: CTMX), a clinical-stage oncology-focused biopharmaceutical company pioneering a novel class of investigational antibody therapeutics based on its Probody therapeutic technology platform, reported third quarter 2019 financial results (Press release, CytomX Therapeutics, NOV 7, 2019, View Source/news-releases/news-release-details/cytomx-therapeutics-announces-third-quarter-2019-financial" target="_blank" title="View Source/news-releases/news-release-details/cytomx-therapeutics-announces-third-quarter-2019-financial" rel="nofollow">View Source [SID1234550635]).

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As of September 30, 2019, CytomX had cash, cash equivalents and short-term investments of $325.7 million.

"The CytomX clinical pipeline made excellent progress in Q3 as we continued to advance multiple Probody therapeutic programs across the portfolio," said Sean McCarthy, D.Phil., president, chief executive officer and chairman of CytomX Therapeutics. "The initiation of a Phase 2 trial of our anti-PD-L1 Probody CX-072 in combination with ipilimumab in patients with relapsed refractory melanoma marks our ongoing evolution into a product-focused company seeking to realize the full potential of our novel technology platform. Our first in class Probody Drug Conjugate programs, CX-2009 and CX-2029, also continued to move forward and our pipeline overall is positioned for significant data updates in 2020."

Business Highlights and Recent Developments

CX-072 Anti-PD-L1 Probody Therapeutic Clinical Program

In October, CytomX announced the initiation of the PROCLAIM (Probody Clinical Assessment In Man) CX-072-002 Phase 2 study evaluating the efficacy and tolerability of the anti-PD-L1 Probody CX-072, in combination with the anti-CTLA-4 antibody, ipilimumab, in patients with relapsed or refractory melanoma. The study utilizes a Simon Two-Stage design with approximately 40 patients being enrolled into Stage 1. CytomX anticipates initial data from Stage 1 in 2020. Additional information on this trial is available at ClinicalTrials.gov using the identifier NCT03993379.
The Company also announced in October updated clinical data from the Phase 1 PROCLAIM-CX-072-001 dose-finding study of CX-072 in combination with ipilimumab. With enrollment complete, 27 evaluable patients had received ipilimumab (3, 6 or 10 mg/kg) combined with CX-072 (0.3, 1, 3 or 10 mg/kg), with the study achieving a disease control rate (stable disease or better) of 37%. 5 patients achieved confirmed objective responses by RECIST v1.1, including one complete response, for an overall response rate (ORR) of 19% in this heavily pretreated patient population. The median duration of response was 14.6 months (1.9 – 21.2 months) with 4 of the 5 responders still on treatment as of the latest data snapshot. Of the 27 patients treated across all doses, Grade 3/4 treatment related adverse events (TRAEs) were reported in 9 (33%) patients. Grade 3/4 immune-related adverse events (irAEs) were reported in 3 (15%) patients.
Enrollment within the monotherapy cohorts of the PROCLAIM-CX-072-001 study is complete with evaluation of the activity and tolerability of CX-072 monotherapy continuing with ongoing treatment in select cohorts. Additional information on this trial is available at ClinicalTrials.gov using the identifier NCT03013491.
CX-2009 Anti-CD166 Probody Drug Conjugate Clinical Program

CytomX anticipates announcing next steps for the PROCLAIM-CX-2009 clinical program by the end of 2019.
BMS-986249 Anti-CTLA-4 Probody Therapeutic Clinical Program

Bristol-Myers Squibb (BMS) is conducting a Phase 1/2a dose escalation clinical study evaluating BMS-986249 alone and in combination with OPDIVO (nivolumab) in advanced solid tumors.
BMS is preparing to initiate the Phase 2 portion of this clinical trial, upon which CytomX is entitled to a $10 million milestone payment. Additional information on this trial is available at ClinicalTrials.gov using the Identifier NCT03369223.
CX-2029 Anti-CD71 Probody Drug Conjugate Clinical Program

CytomX continued enrollment of patients in the PROCLAIM-CX-2029 Phase 1/2 study, which is partnered with AbbVie, evaluating CX-2029 as monotherapy in patients with solid tumors or lymphomas. Additional information on this trial is available at ClinicalTrials.gov using the Identifier NCT003543813.
Additional Corporate Highlights

In October, the Company announced the appointment of Amy C. Peterson, M.D., as executive vice president and chief development officer. In this new role, Dr. Peterson will have oversight of a multi-disciplinary team focused on advancing all aspects of CytomX’s clinical development activities.
In July, the Company announced that its partner AbbVie selected a second target under the companies’ 2016 Discovery Collaboration and Licensing Agreement to discover and develop Probody drug conjugates. The target selection triggered a $10 million payment to CytomX from AbbVie.
Third Quarter 2019 Financial Results
Cash, cash equivalents and short-term investments totaled $325.7 million as of September 30, 2019, compared to $436.1 million as of December 31, 2018.

Revenue was $10.7 million for the three months ended September 30, 2019, compared to $12.5 million for the three months ended September 30, 2018. The decrease in revenue of $1.8 million for the three months ended September 30, 2019 compared to the corresponding period in 2018 was primarily due to a $1.7 million decrease in revenue recognition under the CD71 Agreement with AbbVie due to ongoing dose escalation.

Research and development expenses increased $0.4 million during the three months ended September 30, 2019 compared to the corresponding period in 2018. The increase was attributable to an increase of $1.3 million in personnel-related expenses primarily due to an increase in headcount; an increase of $0.7 million in consulting expenses resulting from increased clinical trial activities and an increase of $0.8 million in the allocation of information technology and facilities related expenses driven partly from an increase in headcount; which amounts were offset by a decrease of $2.4 million in laboratory contracts and services and laboratory supplies as a result of timing of manufacturing activities as well as reduced costs relating to CX-188 which is not presently being advanced.

General and administrative expenses increased by $0.3 million during the three months ended September 30, 2019 compared to the corresponding period in 2018. The increase was attributable to an increase of $0.4 million in consulting expenses primarily related to IT, software implementation and finance services; an increase of $0.5 million in dues and subscriptions primarily related to software and other IT services; an increase of $0.2 million in building maintenance charges; which amounts were partially offset by a decrease of $0.8 million of information technology and facilities-related expenses allocated to the general and administrative functions.

Teleconference Scheduled Today at 5:00 p.m. ET
Conference Call/Webcast Information

CytomX management will host a conference call today at 5:00 p.m. ET. Interested parties may access the live audio webcast of the teleconference through the "Investor & News" section of CytomX’s website at View Source or by dialing 1-877-809-6037 (U.S. and Canada) or 1-615-247-0221 (International) and using the passcode 9449326. An archive of the webcast will be available on the CytomX website from November 7, 2019, until November 14, 2019.

On November 7, 2019 Corcept Therapeutics Incorporated (NASDAQ: CORT), a commercial-stage company engaged in the discovery and development of drugs to treat severe metabolic, oncologic and psychiatric disorders by modulating the effects of the stress hormone cortisol, reported its results for the quarter ended September 30, 2019 (Press release, Corcept Therapeutics, NOV 7, 2019, View Source [SID1234550634]).

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Financial Highlights

Revenue of $81.5 million, a 26 percent increase from third quarter 2018

GAAP diluted net income of $0.22 per share, compared to $0.14 per share in third quarter 2018

Non-GAAP diluted net income of $0.31 per share, compared to $0.22 per share in third quarter 2018

Cash and investments of $266.9 million, compared to $225.7 million in second quarter 2019

2019 revenue guidance narrowed to $300 – $315 million
Corcept reported quarterly revenue of $81.5 million in the third quarter, compared to $64.4 million in the third quarter of 2018. Third quarter GAAP net income was $26.3 million, compared to $17.7 million in the same period last year. Excluding non-cash expenses related to stock-based compensation and the utilization of deferred tax assets, together with related income tax effects, non-GAAP net income in the third quarter was $37.8 million, compared to $27.9 million in the third quarter of 2018. A reconciliation of GAAP to non-GAAP net income is included below.
The company narrowed 2019 revenue guidance to $300 – $315 million. Guidance had previously been $285 – $315 million.
Third quarter operating expenses were $48.5 million, compared to $41.5 million in the third quarter of 2018, primarily due to increased spending to recruit and compensate additional personnel and discover and develop new selective cortisol modulators, as well as increased legal expense. Cash and investments were $266.9 million at September 30, 2019, an increase of $41.2 million from June 30, 2019.
"Our Cushing’s syndrome business had an excellent quarter," said Joseph K. Belanoff, MD, Corcept’s Chief Executive Officer. "We expect the number of patients receiving Korlym and physicians prescribing the medication to continue to increase. To reach more doctors, we are expanding our sales force. We expect the clinical specialists we are hiring now to begin contributing to our results next year.
"I am also pleased to announce an important advance in our program to treat serious metabolic disorders. In a double-blind, placebo-controlled trial in healthy subjects, our selective cortisol modulator miricorilant significantly reduced the weight gain caused by the commonly prescribed antipsychotic medication olanzapine (Eli Lily’s drug, Zyprexa). We have already initiated one of two planned Phase 2 trials to further test miricorilant’s activity in this indication."
Cushing’s Syndrome

European sites begin dosing patients in Phase 3 trial ("GRACE") of relacorilant to treat patients with Cushing’s syndrome

Double-blind, placebo-controlled, Phase 3 trial of relacorilant in patients whose Cushing’s syndrome is caused by adrenal adenomas to start in the first quarter of next year
"As of today, 42 of 62 planned sites are recruiting patients for GRACE," said Andreas Grauer, MD, Corcept’s Chief Medical Officer. "We expect to open an additional 13 sites by the end of the year. The activation pace of ex-US sites, which we expect will provide the majority of enrollments, has refined our estimate of the trial’s completion date. Our plan is to submit our NDA in the fourth quarter of 2021.
We spent substantial time in Europe in the past quarter helping clinical site activation and speaking to investigators. Most important, our investigators are highly enthusiastic about GRACE1, because of relacorilant’s positive Phase 2 efficacy and side effect profile."

Patients in relacorilant’s Phase 2 trial exhibited meaningful improvements in glucose control and hypertension – two of Cushing’s syndrome’s most common and pernicious symptoms. The trial also met a wide range of secondary endpoints, including weight loss, liver function, coagulopathy, insulin resistance, cognitive function, mood and quality of life. These results were achieved without relacorilant causing Korlym’s significant off-target effects – vaginal bleeding, endometrial thickening and low potassium2.
In addition to GRACE, Corcept plans to start a Phase 3, double-blind, placebo-controlled trial of relacorilant in patients whose Cushing’s syndrome is caused by an adrenal adenoma – a population that has not been rigorously studied. Patients with adrenal Cushing’s syndrome typically experience a slower onset of symptoms, but their ultimate health outcomes are poor. Corcept expects to enroll 130 patients at sites in the United States and Europe in the study. Most of the planned investigators and sites are also participating in GRACE.
1For more about GRACE, go to cushingresearch.com.
2For more data, see our poster from the 2019 American Association of Clinical Endocrinologists’ 28th Annual Congress, available at the Investors/Events tab of our website.
Metabolic Disease

Positive top-line results from double-blind, placebo-controlled, Phase 1b trial of miricorilant to reduce antipsychotic-induced weight gain

Recruiting underway in double-blind, placebo-controlled, Phase 2 trial of miricorilant to reverse recent antipsychotic-induced weight gain
"Our program to develop miricorilant as a treatment for metabolic disorders is off to an excellent start," said Dr. Grauer. "Antipsychotic medications such as olanzapine are essential to the health of millions of patients, but the weight gain and other metabolic side effects they cause are life-threatening and often lead patients to discontinue treatment. At the first dose level tested in our Phase 1b trial, healthy subjects given olanzapine plus miricorilant gained less weight than subjects receiving olanzapine plus placebo (see Figure 1). In addition, markers of liver damage that often rise temporarily at the start of olanzapine therapy increased less sharply in subjects receiving miricorilant, suggesting that miricorilant may have protective effects in the liver (see Figure 2). Five subjects in the olanzapine alone group were unable to complete the study due to elevated liver enzymes, while one patient in the miricorilant group experienced this problem.
avgbodyweightlivera01.jpg
The Phase 1b trial’s first part enrolled 66 healthy subjects, each of whom received olanzapine (10 mg) and either miricorilant (600 mg) or placebo daily. The trial’s duration was two weeks. The second part of the trial, which is planned to start in December, will test a higher dose of miricorilant (900 mg) in 30 healthy subjects. The study’s full results will be presented at a scientific meeting in 2020.

"These preliminary results are especially encouraging given the short duration of treatment and the low dose of miricorilant. They are consistent with the effects we had previously seen in animal studies. Our plan is to confirm these findings and explore the full breadth of miricorilant’s activity," said Dr. Grauer.
In addition to the second part of its Phase 1b trial, Corcept plans to conduct two double-blind, placebo-controlled Phase 2 trials of miricorilant for the treatment of patients with antipsychotic-induced weight gain. The first trial, which is underway, will test miricorilant’s activity in reversing recent weight gain. It is expected to enroll 100 patients with schizophrenia at 20 sites in the United States. Patients will continue to receive their established antipsychotic medication and will have either miricorilant or placebo added to their regimen for 12 weeks. A second Phase 2 trial is planned to start next year. It will enroll patients with long-standing weight gain. A third Phase 2 trial, testing miricorilant’s activity in preventing antipsychotic-induced weight gain, is under consideration.
Next year, Corcept also plans to start a double-blind, placebo-controlled, Phase 2 trial of miricorilant as a treatment for patients with non-alcoholic steatohepatitis (NASH), a serious liver disorder that afflicts millions of people.
Solid Tumor

European Commission designates relacorilant orphan drug for treatment of pancreatic cancer

Phase 3 trial of relacorilant plus nab-paclitaxel in patients with metastatic pancreatic cancer to start upon completion of consultations with the U.S. Food and Drug Administration (FDA)
"We are pleased the European Commission (EC) has joined the FDA in designating relacorilant an orphan drug for the treatment of pancreatic cancer," said Dr. Grauer. "The EC based its decision on the European Medicines Agency’s finding that relacorilant has the potential to significantly benefit patients.
"We presented the clinical data reviewed by the EMA at last year’s ASCO (Free ASCO Whitepaper) meeting and it was indeed promising," said Dr. Grauer. "Seven of 25 patients with metastatic pancreatic cancer treated with relacorilant plus nab-paclitaxel (Celgene’s drug, Abraxane) achieved durable disease control, meaning their tumors either shrank or ceased growing for 16 weeks or longer. Tumor response in two patients lasted more than 50 weeks3. All of these patients’ tumors had progressed during multiple lines of prior therapy, including treatments with nab-paclitaxel or another taxane. That any of them responded is remarkable. We have sought FDA guidance as to the optimum development path in pancreatic cancer and plan to start a Phase 3 trial promptly upon the conclusion of our discussions."
Corcept’s 180-patient, placebo-controlled Phase 2 trial of relacorilant plus nab-paclitaxel in ovarian cancer continues to enroll patients at sites in the United States and the European Union. Dosing also continues in the company’s Phase 1/2 study of exicorilant plus enzalutamide in patients with castration-resistant prostate cancer.
3For more data, see our ASCO (Free ASCO Whitepaper) poster at the Investors/Past Events tab of our website.
Conference Call
We will hold a conference call on November 7, 2019, at 5:00 p.m. Eastern Time (2:00 p.m. Pacific Time). To participate, dial 1-877-260-1479 from the United States or 1-334-323-0522 internationally approximately ten minutes before the start of the call (passcode 8532239). A replay will be available through November 21, 2019 at 1-888-203-1112 in the United States and 1-719-457-0820 internationally (passcode 8532239).
Hypercortisolism
Hypercortisolism, often referred to as Cushing’s syndrome, is caused by excessive activity of the hormone cortisol. Endogenous Cushing’s syndrome is an orphan disease that most often affects adults aged 20-50. In the United States, an estimated 20,000 patients have Cushing’s syndrome, with about 3,000 new patients diagnosed each year. Symptoms vary, but most patients experience one or more of the following manifestations: high blood sugar, diabetes, high blood pressure, upper-body obesity, rounded face, increased fat around the neck, thinning arms and legs, severe fatigue and weak muscles. Irritability, anxiety, cognitive disturbances and depression are also common. Hypercortisolism can affect every organ system in the body and can be lethal if not treated effectively.

On November 7, 2019 Sophiris Bio Inc. (NASDAQ: SPHS), a biopharmaceutical company studying topsalysin (PRX302), a first-in-class, pore-forming protein, in late-stage clinical trials for the treatment of patients with urological diseases, reported financial results for the third quarter 2019 and provided an overview of recent corporate highlights (Press release, Sophiris Bio, NOV 7, 2019, View Source [SID1234550633]).

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"We took a significant step forward with the agreement of a Phase 3 trial design with the FDA for our localized prostate cancer program, a step that advances discussions around potential strategic agreements necessary to help fund the development of topsalysin", said Randall E. Woods, president and CEO of Sophiris. "The design agreed to with the FDA aligns with the design previously approved by the EMA which will allow us to complete a global Phase 3 program for the potential approval of topsalyin for treating patients with intermediate risk localized prostate cancer in both the US and Europe."

Recent Corporate Highlights:

On August 30, 2019, the Company announced the closing of a registered offering in which the Company raised net proceeds of $3.6 million. This support from a new investor provides the company additional financial resources to execute on a funding strategy for the Phase 3 localized prostate cancer study. The company estimates that the cash and cash equivalents currently on hand will fund the Company through at least March 31, 2020.

On October 21, 2019, the Company announced that following an End of Phase 2/ Pre-Phase 3 meeting with the United States Food and Drug Administration there is agreement regarding the design of a single Phase 3 clinical trial to evaluate the potential of topsalysin as a targeted focal therapy to treat patients with intermediate risk localized prostate cancer. The Phase 3 study design agreed upon with the FDA is consistent with the design previously agreed upon with the EMA. In addition, the FDA has indicated that in order to receive approval, we will need to evaluate all patients that progress to alternative treatments for an additional 12 months, for a total of 24 months of data, post the administration of the study drug. The Company believes that a single Phase 3 trial, if successful, will provide the clinical data necessary for approval in both the United States and Europe.

From November 10-13, 2019, Sophiris CEO Randall E. Woods will attend the 25th Annual International Partnering Conference Bio-Europe 2019.

Financial Results:

At September 30, 2019, the Company had cash, cash equivalents and securities available-for-sale of $6.3 million and working capital of $3.1 million. The Company expects that its cash and cash equivalents and securities available-for-sale will be sufficient to fund its operations and debt service through March 2020, assuming no new clinical trials are initiated and the Company continues operating as a going concern. The Company will require significant funding to advance topsalysin in clinical development and to continue its operations. As of September 30, 2019, the outstanding principal balance of the Company’s term loan was $5.6 million. The Company began making principal payments on its term loan in April 2019.

For the three months ended September 30, 2019

The Company reported a net loss of $1.0 million or ($0.03) per share for the three months ended September 30, 2019, compared to net loss of $2.9 million or ($0.10) per share for the three months ended September 30, 2018.

Research and development expenses

Research and development expenses were $0.7 million for the three months ended September 30, 2019, compared to $1.8 million for the three months ended September 30, 2018. The decrease in research and development costs is primarily attributable to decreases in the costs associated with manufacturing activities for topsalysin and, to a lesser extent, a decrease in clinical costs associated with the Company’s completed Phase 2b clinical trial of topsalysin for localized prostate cancer.

General and administrative expenses

General and administrative expenses were $1.4 million for the three months ended September 30, 2019, compared to $1.2 million for the three months ended September 30, 2018. Included as a component of general and administrative expense for the three months ended September 30, 2019 was $0.4 million of offering costs which were allocated to the common share purchase warrants issued in its August 2019 financing. These offering costs were allocated to general and administrative expense as the common share purchase warrants were classified as liabilities. General and administrative expenses included non-cash stock-based compensation expense of $0.1 million for the three months ended September 30, 2019 as compared to $0.2 million for the three months ended September 30, 2018.

Gain on revaluation of the warrant liability

Gain on revaluation of the warrant liability was $1.3 million for the three months ended September 30, 2019, compared to $0.2 million for the three months ended September 30, 2018. As the Company’s warrants may require the Company to pay the warrant holders cash under certain provisions of the warrants, the Company accounts for the warrants as a liability, and the Company is required to calculate the fair value of these warrants each reporting date. Certain inputs utilized in the Company’s Black-Scholes fair value calculation may fluctuate in future periods based upon factors which are outside of the Company’s control. A significant change in one or more of these inputs used in the calculation of the fair value may cause a significant change to the fair value of the Company’s warrant liability, which could also result in a material non-cash gain or loss being reported in the Company’s consolidated statement of operations and comprehensive loss.

For the nine months ended September 30, 2019

The Company reported a net loss of $5.5 million or ($0.18) per share for the nine months ended September 30, 2019 compared to a net loss of $12.3 million or ($0.41) per share for the nine months ended September 30, 2018.

Research and development expenses

Research and development expenses were $3.4 million for the nine months ended September 30, 2019 compared to $8.7 million for the nine months ended September 30, 2018. The decrease in research and development costs was primarily attributable to decreases in the costs associated with manufacturing activities for topsalysin, and to a lesser extent, a decrease in clinical costs associated with the Company’s completed Phase 2b clinical trial of topsalysin for localized prostate cancer.

General and administrative expenses

General and administrative expenses were $3.9 million for the nine months ended September 30, 2019 compared to $3.5 million for the nine months ended September 30, 2018. Included as a component of general and administrative expense for the nine months ended September 30, 2019 was $0.4 million of offering costs which were allocated to the common share purchase warrants issued in its August 2019 financing. These offering costs were allocated to general and administrative expense as the common share purchase warrants were classified as liabilities. General and administrative expenses included non-cash stock-based compensation expense of $0.4 million for the nine months ended September 30, 2019 as compared to $0.5 million for the nine months ended September 30, 2018.

Gain on revaluation of the warrant liability

Gain on revaluation of the warrant liability was $2.1 million for the nine months ended September 30, 2019 as compared to $0.1 million for the nine months ended September 30, 2018.

On November 7, 2019 Corcept Therapeutics Incorporated (NASDAQ: CORT), a commercial-stage company engaged in the discovery and development of drugs to treat severe metabolic, oncologic and psychiatric disorders by modulating the effects of the stress hormone cortisol, reported its results for the quarter ended September 30, 2019 (Press release, Corcept Therapeutics, NOV 7, 2019, https://ir.corcept.com/news-releases/news-release-details/corcept-therapeutics-announces-third-quarter-2019-financial [SID1234550631]).

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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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Financial Highlights

Revenue of $81.5 million, a 26 percent increase from third quarter 2018
GAAP diluted net income of $0.22 per share, compared to $0.14 per share in third quarter 2018
Non-GAAP diluted net income of $0.31 per share, compared to $0.22 per share in third quarter 2018
Cash and investments of $266.9 million, compared to $225.7 million in second quarter 2019
2019 revenue guidance narrowed to $300 – $315 million
Corcept reported quarterly revenue of $81.5 million in the third quarter, compared to $64.4 million in the third quarter of 2018. Third quarter GAAP net income was $26.3 million, compared to $17.7 million in the same period last year. Excluding non-cash expenses related to stock-based compensation and the utilization of deferred tax assets, together with related income tax effects, non-GAAP net income in the third quarter was $37.8 million, compared to $27.9 million in the third quarter of 2018. A reconciliation of GAAP to non-GAAP net income is included below.

The company narrowed 2019 revenue guidance to $300 – $315 million. Guidance had previously been
$285 – $315 million.

Third quarter operating expenses were $48.5 million, compared to $41.5 million in the third quarter of 2018, primarily due to increased spending to recruit and compensate additional personnel and discover and develop new selective cortisol modulators, as well as increased legal expense. Cash and investments were $266.9 million at September 30, 2019, an increase of $41.2 million from June 30, 2019.

"Our Cushing’s syndrome business had an excellent quarter," said Joseph K. Belanoff, MD, Corcept’s Chief Executive Officer. "We expect the number of patients receiving Korlym and physicians prescribing the medication to continue to increase. To reach more doctors, we are expanding our sales force. We expect the clinical specialists we are hiring now to begin contributing to our results next year.

"I am also pleased to announce an important advance in our program to treat serious metabolic disorders. In a double-blind, placebo-controlled trial in healthy subjects, our selective cortisol modulator miricorilant significantly reduced the weight gain caused by the commonly prescribed antipsychotic medication olanzapine (Eli Lily’s drug, Zyprexa). We have already initiated one of two planned Phase 2 trials to further test miricorilant’s activity in this indication."

Cushing’s Syndrome

European sites begin dosing patients in Phase 3 trial ("GRACE") of relacorilant to treat patients with Cushing’s syndrome
Double-blind, placebo-controlled, Phase 3 trial of relacorilant in patients whose Cushing’s syndrome is caused by adrenal adenomas to start in the first quarter of next year
"As of today, 42 of 62 planned sites are recruiting patients for GRACE," said Andreas Grauer, MD, Corcept’s Chief Medical Officer. "We expect to open an additional 13 sites by the end of the year. The activation pace of ex-US sites, which we expect will provide the majority of enrollments, has refined our estimate of the trial’s completion date. Our plan is to submit our NDA in the fourth quarter of 2021.

We spent substantial time in Europe in the past quarter helping clinical site activation and speaking to investigators. Most important, our investigators are highly enthusiastic about GRACE1, because of relacorilant’s positive Phase 2 efficacy and side effect profile."

Patients in relacorilant’s Phase 2 trial exhibited meaningful improvements in glucose control and hypertension – two of Cushing’s syndrome’s most common and pernicious symptoms. The trial also met a wide range of secondary endpoints, including weight loss, liver function, coagulopathy, insulin resistance, cognitive function, mood and quality of life. These results were achieved without relacorilant causing Korlym’s significant off-target effects – vaginal bleeding, endometrial thickening and low potassium.2

In addition to GRACE, Corcept plans to start a Phase 3, double-blind, placebo-controlled trial of relacorilant in patients whose Cushing’s syndrome is caused by an adrenal adenoma – a population that has not been rigorously studied. Patients with adrenal Cushing’s syndrome typically experience a slower onset of symptoms, but their ultimate health outcomes are poor. Corcept expects to enroll 130 patients at sites in the United States and Europe in the study. Most of the planned investigators and sites are also participating in GRACE.

Metabolic Disease

Positive top-line results from double-blind, placebo-controlled, Phase 1b trial of miricorilant to reduce antipsychotic-induced weight gain
Recruiting underway in double-blind, placebo-controlled, Phase 2 trial of miricorilant to reverse recent antipsychotic-induced weight gain
"Our program to develop miricorilant as a treatment for metabolic disorders is off to an excellent start," said Dr. Grauer. "Antipsychotic medications such as olanzapine are essential to the health of millions of patients, but the weight gain and other metabolic side effects they cause are life-threatening and often lead patients to discontinue treatment. At the first dose level tested in our Phase 1b trial, healthy subjects given olanzapine plus miricorilant gained less weight than subjects receiving olanzapine plus placebo (see Figure 1). In addition, markers of liver damage that often rise temporarily at the start of olanzapine therapy increased less sharply in subjects receiving miricorilant, suggesting that miricorilant may have protective effects in the liver (see Figure 2). Five subjects in the olanzapine alone group were unable to complete the study due to elevated liver enzymes, while one patient in the miricorilant group experienced this problem."

A photo accompanying this announcement is available at View Source

The Phase 1b trial’s first part enrolled 66 healthy subjects, each of whom received olanzapine (10 mg) and either miricorilant (600 mg) or placebo daily. The trial’s duration was two weeks. The second part of the trial, which is planned to start in December, will test a higher dose of miricorilant (900 mg) in 30 healthy subjects. The study’s full results will be presented at a scientific meeting in 2020.

"These preliminary results are especially encouraging given the short duration of treatment and the low dose of miricorilant. They are consistent with the effects we had previously seen in animal studies. Our plan is to confirm these findings and explore the full breadth of miricorilant’s activity," said Dr. Grauer.

In addition to the second part of its Phase 1b trial, Corcept plans to conduct two double-blind, placebo-controlled Phase 2 trials of miricorilant for the treatment of patients with antipsychotic-induced weight gain. The first trial, which is underway, will test miricorilant’s activity in reversing recent weight gain. It is expected to enroll 100 patients with schizophrenia at 20 sites in the United States. Patients will continue to receive their established antipsychotic medication and will have either miricorilant or placebo added to their regimen for 12 weeks. A second Phase 2 trial is planned to start next year. It will enroll patients with long-standing weight gain. A third Phase 2 trial, testing miricorilant’s activity in preventing antipsychotic-induced weight gain, is under consideration.

Next year, Corcept also plans to start a double-blind, placebo-controlled, Phase 2 trial of miricorilant as a treatment for patients with non-alcoholic steatohepatitis (NASH), a serious liver disorder that afflicts millions of people.

Solid Tumors

European Commission designates relacorilant orphan drug for treatment of pancreatic cancer
Phase 3 trial of relacorilant plus nab-paclitaxel in patients with metastatic pancreatic cancer to start upon completion of consultations with the U.S. Food and Drug Administration (FDA)
"We are pleased the European Commission (EC) has joined the FDA in designating relacorilant an orphan drug for the treatment of pancreatic cancer," said Dr. Grauer. "The EC based its decision on the European Medicines Agency’s finding that relacorilant has the potential to significantly benefit patients.

"We presented the clinical data reviewed by the EMA at last year’s ASCO (Free ASCO Whitepaper) meeting and it was indeed promising," said Dr. Grauer. "Seven of 25 patients with metastatic pancreatic cancer treated with relacorilant plus nab-paclitaxel (Celgene’s drug, Abraxane) achieved durable disease control, meaning their tumors either shrank or ceased growing for 16 weeks or longer. Tumor response in two patients lasted more than 50 weeks.3 All of these patients’ tumors had progressed during multiple lines of prior therapy, including treatments with nab-paclitaxel or another taxane. That any of them responded is remarkable. We have sought FDA guidance as to the optimum development path in pancreatic cancer and plan to start a Phase 3 trial promptly upon the conclusion of our discussions."

Corcept’s 180-patient, placebo-controlled Phase 2 trial of relacorilant plus nab-paclitaxel in ovarian cancer continues to enroll patients at sites in the United States and the European Union. Dosing also continues in the company’s Phase 1/2 study of exicorilant plus enzalutamide in patients with castration-resistant prostate cancer.

Conference Call

We will hold a conference call on November 7, 2019, at 5:00 p.m. Eastern Time (2:00 p.m. Pacific Time). To participate, dial 1-877-260-1479 from the United States or 1-334-323-0522 internationally approximately ten minutes before the start of the call (passcode 8532239). A replay will be available through November 21, 2019 at 1-888-203-1112 in the United States and 1-719-457-0820 internationally (passcode 8532239).

Hypercortisolism

Hypercortisolism, often referred to as Cushing’s syndrome, is caused by excessive activity of the hormone cortisol. Endogenous Cushing’s syndrome is an orphan disease that most often affects adults aged 20-50. In the United States, an estimated 20,000 patients have Cushing’s syndrome, with about 3,000 new patients diagnosed each year. Symptoms vary, but most patients experience one or more of the following manifestations: high blood sugar, diabetes, high blood pressure, upper-body obesity, rounded face, increased fat around the neck, thinning arms and legs, severe fatigue and weak muscles. Irritability, anxiety, cognitive disturbances and depression are also common. Hypercortisolism can affect every organ system in the body and can be lethal if not treated effectively.

On November 7, 2019 MEI Pharma, Inc. (NASDAQ: MEIP), a late-stage pharmaceutical company focused on advancing new therapies for cancer, reported results for its first quarter ended September 30, 2019 (Press release, MEI Pharma, NOV 7, 2019, View Source [SID1234550625]).

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"During this past quarter, our primary focus remained on advancing the clinical development of ME-401, as well as voruciclib, while continuing to explore additional clinical collaborations and partnering opportunities to effectively leverage the potential of our drug candidates for patients," said Daniel P. Gold, Ph.D., president and chief executive officer of MEI Pharma. "As reported at the MEI Investor and Analyst event in October, the ME-401 intermittent dosing regimen continues to demonstrate high activity with a well-tolerated safety profile, supporting the Phase 2 TIDAL study in patients with relapsed or refractory follicular lymphoma. We are also focused on the expansion of the investigation of the ME-401 intermittent dosing regimen across a variety of B-cell malignancies in combination with other therapies, like Rituxan or zanubrutinib, an investigational BTK inhibitor as per our clinical collaboration agreement with BeiGene."

Recent Highlights

In October 2019, at MEI’s Investor and Analyst Event, the company reported updated data from the ongoing Phase 1b study of ME-401, an investigational selective oral inhibitor of phosphatidylinositol 3-kinase (PI3K) delta. The data demonstrate:
Overall response rates of 78% in relapsed or refractory (r/r) follicular lymphoma (FL) and 89% in r/r chronic lymphocytic leukemia or small lymphocytic lymphoma (CLL/SLL).
Rates of Grade 3 adverse events of special interest related to ME-401 exposure were observed in <10% of patients dosed on an intermittent schedule (IS).
Median duration of response not yet reached in patients with FL or CLL/SLL on the IS regimen. Median follow-up for FL and CLL/SLL patients is 9.2 months (range 3.4-20.7 months) and 7.4 months (range 2.6-14.7 months), respectively.
In July 2019, Tamar Howson, M.S., MBA, a highly experienced business development executive with over 30 years of service in the pharmaceutical and biotechnology industry, joined the Board of Directors.
First Quarter Fiscal Year 2020 Financial Results

As of September 30, 2019, MEI had $65.9 million in cash, cash equivalents and short-term investments, with no outstanding debt.
For the quarter ended September 30, 2019, cash used in operations was $14.1 million, compared to $12.8 million for 2018.
Research and development expenses were $9.0 million for the quarter ended September 30, 2019, compared to $6.1 million for 2018. The increase was primarily related to increased development costs associated with ME-401 and increased headcount and professional services to support our development activities.
General and administrative expenses were $4.1 million for the quarter ended September 30, 2019, compared to $3.4 million for 2018. The increase primarily relates to increased headcount and increased professional services expenses to support our activities.
Revenues were $1.2 million for the quarter ended September 30, 2019, resulting from the recognition of fees allocated to research and development activities related to the Helsinn and Kyowa Kirin License Agreements. This compares with revenues of $0.5 million for the quarter ended September 30, 2018, resulting from the recognition of fees allocated to research and development activities related to the Helsinn License Agreement.
Net loss was $3.0 million, or $0.04 per share, for the quarter ended September 30, 2019, compared to net loss of $14.5 million, or $0.21 per share for 2018. The net loss decreased primarily as a result of a non-cash gain related to changes in the fair value of warrants issued in connection with the May 2018 financing. The company had 73,634,927 shares of common stock outstanding as of September 30, 2019, compared with 71,115,444 shares as of September 30, 2018.
The adjusted net loss for the quarter ended September 30, 2019, excluding a non-cash gain related to changes in the fair value of the warrants (a non-GAAP measure), was $12.3 million, compared to an adjusted net loss of $9.6 million for 2018.