Fulcrum Therapeutics (FULC) : Topline Results from Phase 3 REACH Clinical Trial of Losmapimod in Facioscapulohumeral Muscular Dystrophy (FSHD)

― Losmapimod failed to show an improvement in relative surface area (RSA), a measure of reachable workspace (RWS), versus placebo at week 48 

― Fulcrum to suspend future losmapimod development 

― Robust cash position enables Fulcrum to continue its mission to develop therapies addressing diseases of high unmet need including pociredir for the treatment of sickle cell disease (SCD)

 

 

 

 

 

 

 

Fulcrum Therapeutics is a biopharmaceutical company that focuses on developing small molecule therapies to treat rare diseases. The company was founded in Cambridge, Massachusetts in July 2016 by Third Rock Ventures.
It went public on July 17, 2019.

About the REACH Trial
REACH (NCT05397470) is a Phase 3 multi-center, randomized, double-blind, placebo-controlled trial designed to evaluate the efficacy and safety of losmapimod for the treatment of FSHD. The trial enrolled 260 patients who were randomized 1:1 to receive either losmapimod, administered orally as a 15 mg tablet twice a day, or placebo over a 48-week treatment period. The primary endpoint was the absolute change from baseline in Reachable Workspace (RWS). Secondary endpoint measurements included Muscle Fat Infiltration (MFI) as measured by MRI, shoulder abductor strength as measured by hand-held dynamometry, Patient Global Impression of Change (PGIC), and the Neuro QoL Upper Extremity.

About FSHD
FSHD is a serious, rare, progressive and debilitating disease for which there are no approved treatments. It is characterized by fat infiltration of skeletal muscle leading to muscular atrophy involving primarily the face, scapula and shoulders, upper arms, and abdomen. Impact on patients includes relentless and accumulating muscle and functional loss impacting their ability to perform activities of daily living, loss of upper limb function, loss of mobility and independence, and chronic pain. FSHD is one of the most common forms of muscular dystrophy and has an estimated patient population of 30,000 in the United States alone.

==Acadia Pharmaceuticals (ACAD) : schizophrenia drug study falls short

 

Pharmaceuticals announces top-line results from the Phase 3 ADVANCE-2 trial evaluating pimavanserin for the treatment of negative symptoms of schizophrenia 

• Pimavanserin did not demonstrate a statistically significant improvement over placebo on the study's primary endpoint, the change from baseline to week 26 on the Negative Symptom Assessment-16 (NSA-16) total score (-11.8 vs. -11.1; p=0.4825; effect size=0.07). The safety and tolerability profile of pimavanserin was consistent with previous clinical trials, showing a low rate of adverse events.
• "We are disappointed the trial did not meet its primary endpoint given the significant unmet need in patients with negative symptoms of schizophrenia," said Steve Davis, Acadia's Chief Executive Officer. "We will continue to analyze these data with our scientific advisors, but we do not intend to conduct any further clinical trials with pimavanserin. We want to thank the patients, their families, and the investigators for their contributions in this important study."

==Akero's (AKRO) drug for fatty liver disease misses main trial goal

 

Ticker:  AKRO

Oct 10 (Reuters) - Akero Therapeutics said on Tuesday its drug to treat a type of fatty liver disease showed improvement in patients in a mid-stage trial, but the data showed that the results were not statistically significant. 

Akero Therapeutics reports encouraging 36-week analysis of 96-week phase 2b symmetry study, with a trend on fibrosis improvement and statistically significant results for nash resolution, markers of liver injury and fibrosis, insulin sensitization and lipoproteins 

• A trend was observed for the primary endpoint of fibrosis improvement at 36 weeks, with 22% and 24% of the 28mg and 50mg EFX-treated groups, respectively, experiencing at least a one-stage improvement in liver fibrosis and no worsening of NASH, compared with 14% for placebo. In addition, 4% of patients in each of the EFX-treated groups experienced a three- or two-stage fibrosis improvement without worsening of NASH -- from compensated cirrhosis (F4) to F1 or F2, compared with 0% for placebo.
• Statistically significant rates of NASH resolution in 63% and 60% of patients at week 36 were observed for the 28mg and 50mg EFX-treated groups, respectively, compared with 26% for placebo, representing the highest response rates reported to date for NASH resolution in this patient population. Statistically significant improvements were also observed for both EFX groups in non-invasive markers of liver injury and fibrosis, insulin sensitization and lipoproteins.

Akero Therapeutics to Present Results from Phase 2b SYMMETRY Study Investigating Efruxifermin in Patients with Compensated Cirrhosis Due to NASH

SOUTH SAN FRANCISCO, Calif., Oct. 09, 2023 (GLOBE NEWSWIRE) -- Akero Therapeutics, Inc. (Nasdaq: AKRO), a clinical-stage company developing transformational treatments for patients with serious metabolic disease marked by high unmet medical need, will hold an investor conference on Tuesday, October 10 at 8:00 a.m. ET to share topline week 36 results from its SYMMETRY study, a double-blind, placebo-controlled Phase 2b study evaluating the efficacy of efruxifermin (EFX) in patients with compensated cirrhosis due to non-alcoholic steatohepatitis (NASH). The topline results will include histology and changes from baseline in non-invasive measures of fibrosis, glycemic control, and lipoproteins as well as safety and tolerability.

=-=Vir Biotechnology (VIR) : failed seasonal influenza study

 

Vir Biotechnology announces topline data from phase 2 peninsula trial evaluating vir-2482 for the prevention of seasonal influenza 

• Co announced that the Phase 2 PENINSULA (PrevEntioN of IllNesS DUe to InfLuenza A) trial evaluating VIR-2482 for the prevention of symptomatic influenza A illness did not meet primary or secondary efficacy endpoints. In participants who received the highest dose of VIR-2482 (1,200 mg), a non-statistically significant reduction of approximately 16% in influenza A protocol-defined illness was observed. Participants who received the highest dose showed an approximately 57% reduction in symptomatic influenza A illness, when defined according to CDC influenza-like-illness criteria, which was one of two secondary endpoints. VIR-2482 was generally well tolerated and no safety signals were identified.
• PENINSULA (NCT05567783) is the first Phase 2 outpatient trial to evaluate the role of a monoclonal antibody in the prevention of influenza A illness. The dose-ranging, proof-of-concept trial enrolled approximately 3,000 men and women ages 18 to 64 without risk factors for serious complications from an influenza infection who did not receive an influenza vaccination for the flu season. The primary efficacy endpoint was the proportion of trial participants with protocol-defined influenza-like-illness1 with PCR-confirmed influenza A infection compared to placebo. Secondary endpoints included the proportion of participants with CDC-defined influenza-like-illness2 with PCR-confirmed influenza A infection and the proportion of participants with WHO-defined influenza-like-illness3 with PCR-confirmed influenza A infection.

Tricida (TCDA): Kidney Disease Drug Fails Trial

In a Phase 3 trial, the drug—called veverimer—did not meet its primary endpoint, according to the press release. That is another way of saying it failed to delay the occurrence of renal death, end-stage renal disease, and other factors.

Shares of Tricida plummeted to $0.63, hitting an all-time low following the news. It is also the stock’s largest percent decrease on record.

Investors began to doubt the drug would be approved when the Food and Drug Administration rejected the company’s new drug application in mid-August 2020, requesting additional information on its clinical benefit. Tricida submitted an appeal, but it was denied early last year.

There are currently no therapies approved by the FDA to slow progression of kidney disease through the treatment of chronic metabolic acidosis in patients with CKD, or chronic kidney disease, Tricida said.

Athira (ATHA): Alzheimer's treatment did not meet endpoint in mid-stage trial

• The results indicated Athira's lead drug, ATH-1017, also called fosgonimeton, didn't have a statistically significant impact on working memory processing speed or cognition when compared with a placebo after 26 weeks.
 

 

 

 

Mark Litton, Athira's CEO, says the company is in a "fortuitous situation" despite the disappointing results.

Ionis Pharmaceuticals (IONS) : Biogen-partnered ALS drug trial fails

 

Biogen (BIIB) and Ionis Pharmaceuticals' (IONS) approach to Lou Gehrig's disease flopped over the weekend, leading IONS stock to collapse to a five-year low Monday.

The duo tested their drug, tofersen, in patients with amyotrophic lateral sclerosis, also known as ALS. Patients with this disease lose muscle control due to dysfunction in the nerve cells of the brain and spinal cord.

Tofersen-treated patients didn't improve enough to deem the study a success, and IONS stock tumbled. SVB Leerink analyst Mani Foroohar says the data release marks "another misstep" for one of Ionis' partnered programs in central nervous system disorders. Previously, Roche (RHHBY) discontinued a study of Ionis' Huntington's disease treatment.

IONS Stock Dives On Misstep

Patients showed improvement in function — as measured by a scale for ALS assessment — but the results weren't strong enough to be statistically significant.

Biomarker data also suggested the drug was, in fact, engaging the mutated gene responsible for patients' disease. Further, patients who received tofersen showed greater declines in a marker of neurodegeneration, known as neurofilament, than those who received the placebo.

But the drug also carried some side effects. Serious adverse events occurred in almost 6% of patients who received tofersen vs. none in the placebo group, Needham analyst Joseph Stringer said in a report. Those included inflammation in the spinal cord, an infection called meningitis, a disease of the lumbar spinal nerve root and other nervous system disorders.

Cara Therapeutics (CARA) : Korsuva fails for the treatment in atopic dermatitis (AD) patients

  

 

 

 

 

 

 

 

Cara Therapeutics announces topline results from its KARE Phase 2 dose-ranging clinical trial of Oral KORSUVA for the treatment of moderate-to-severe pruritus in mild-to-severe atopic dermatitis patients

Study did not meet Primary Endpoint of worst-itch NRS change from baseline at week 12 or Secondary Endpoint of 4-point responder analysis in the ITT patient population.

Study achieved Primary Endpoint of worst-itch NRS change and Secondary Endpoint of 4-point responder analysis in pre-specified analyses of mild-to-moderate AD patients (64% of ITT patient population).

Statistically significant improvement in 4-point responder analysis in mild-to-moderate (BSA <10%) AD patients with 32% of KORSUVA-treated patients achieving a > 4-point reduction vs. 19% in placebo group (p=0.03).

KORSUVA was well tolerated at all dose levels.

Arena Pharma (ARNA) reported earnings on Mon 9 Nov 20 (a/h)

  ** charts after earnings **

 

 

 

 

 

 

 

 

Arena Pharm beats by $0.17, misses on revs, provides clinical program update

• Reports Q3 (Sep) loss of $(1.69) per share, $0.17 better than the S&P Capital IQ Consensus of ($1.86); revenues fell 98.5% year/year to $0.02 mln vs the $1.43 mln S&P Capital IQConsensus.

Program Updates:

• Etrasimod atopic dermatitis (AD) ADVISE Phase 2b trial delivered topline results; moving forward into a Phase 3 registrational program
• Etrasimod ELEVATE UC 52 Phase 3 trial in ulcerative colitis (UC) ongoing and on track to complete enrollment by year-end 2020, with the 52-week treatment period ending by year-end2021 and data expected shortly thereafter; ELEVATE UC 12 Phase 3 trial was initiated, continues to make strong progress with data expected in the same timeframe as UC 52; we continue to monitor the impact of the COVID-19 resurgence on study enrollment for both UC52 and UC 12
• Etrasimod GLADIATOR UC Phase 2 planning; initiation expected in Q1 2021 dependent onCOVID-19 impact
• Etrasimod CULTIVATE Phase 2/3 trial in Crohn's disease (CD) ongoing; confirming topline dose-ranging data in 2021; continue to suspend overall program guidance based on COVID-19impact
• Etrasimod eosinophilic esophagitis (EoE) Phase 2b VOYAGE trial with initiation expected in 2020but dependent on COVID-19 impact
• Etrasimod alopecia areata (AA) Phase 2 initiated; topline data expected H2 2021
• Olorinab CAPTIVATE Phase 2b trial in abdominal pain associated with irritable bowel syndrome(IBS-C, IBS-D) enrollment completed; topline data expected Q1 2021
• APD418 in acute heart failure (AHF) with Fast Track designation; completed the first-in-human Phase 1 trial; APD418 was generally well tolerated; Phase 2 planning ongoing

Arena Pharm announces top-line results from Phase 2b ADVISE trial evaluating etrasimod for the treatment of moderate-to-severe atopic dermatitis; primary endpoint not met

• Arena Pharmaceuticals today announced top-line results from the Phase 2b ADVISE clinical trial evaluating etrasimod, a highly selective, once-daily, oral sphingosine 1-phosphate (S1P)receptor modulator, for the treatment of moderate-to-severe atopic dermatitis (AD).
• In the ADVISE trial, the participants were representative of a moderate patient population(82.9% baseline vIGA 3). In the primary analysis, nearly one-third of participants in the 2 mgetrasimod group achieved clear or almost clear skin, as defined by the validated Investigator Global Assessment (vIGA) -- the FDA endpoint for Phase 3 registration. Importantly, the vIGA improvement was statistically significant vs. placebo at 12 weeks. Across the Eczema Area and Severity Index (EASI), EASI-75 and peak change in pruritis, etrasimod 2 mg demonstrated early and statistically significant effect at week 4.
• Etrasimod did not meet the Ph 2b primary endpoint of EASI change from baseline at week 12 as compared to placebo.
• Overall, the 12-week study showed no plateau of effect, and the safety profile was consistent with previous trials of etrasimod including low first dose heart rate effect with no titration, and no serious adverse events across the groups.
• Between weeks 4-8, the trial was impacted by unwarranted dose interruption (not related to drug safety) in 19% of the etrasimod 2 mg group. Adjusting for this dose interruption --a post-hoc Completer Analysis with participants receiving full therapeutic exposure -- etrasimod2 mg showed statistically significant effect on the EASI score compared to placebo (weeks 4and 12), EASI-75 at week 4, vIGA at week 12, and pruritis through week 8.

-=Onconova Therapeutics (ONTX) : IV rigosertib did not meet primary endpoint

IV rigosertib did not meet primary endpoint of significantly improved survival versus best supportive care in higher-risk myelodysplastic syndromes (HR-MDS)

Onconova will focus on promising pipeline opportunities including with its CDK4/6 + ARK5 inhibitor ON 123300

NEWTOWN, Pa., Aug. 24, 2020 (GLOBE NEWSWIRE) -- Onconova Therapeutics, Inc. (NASDAQ: ONTX) a biopharmaceutical company focused on discovering and developing novel products to treat cancer, today announced that INSPIRE, the company’s pivotal Phase 3 study assessing the efficacy and safety of IV rigosertib in higher-risk MDS (HR-MDS) patients, did not meet its primary endpoint of improved survival.

-=Sage Therapeutics (SAGE) : Depression drug unexpectedly fails

• Reports top-line results from pivotal Phase 3 MOUNTAIN Study evaluating the effect of SAGE-217 on depressive symptoms in adults with major depressive disorder; study did not meet primary endpoint at Day 15.

Sage Therapeutics (SAGE) collapsed Thursday after the biotech company's depression treatment failed to pass muster in a Phase 3 study. In early action on today's stock market, Sage stock plunged near 60%, and stood at roughly 60 a share.

This is the first time Sage's depression treatment, SAGE-217, missed its key goal in a test for major depressive disorder, SVB Leerink analyst Marc Goodman said in a note to clients. He has an underperform rating on Sage stock.

"The stock in premarket trading is down in the $60 range that we had previously outlined ($55-$90) as the worst case scenario," he said.

Depression Treatment Misses Its Mark

Patients took a daily pill of SAGE-217 as a depression treatment. The drug showed a statistically significant improvement on the symptoms of depression compared to a placebo on days three, eight and 12. Researchers used a scale called the Hamilton Rating Scale for Depression.

But on day 15, the depression treatment showed an average reduction of 12.6 points on that scale, compared to an average reduction of 11.2 points for the placebo. The results were not statistically significant.

In an analysis following the study, the biotech company noted two key issues. It appeared 9% of patients weren't compliant in taking a daily dose of the depression treatment. In addition, the study enrolled patients with milder symptoms than previous studies.

Sage suggested if these patients were excluded, the study would have hit its mark.

Treatment Well Tolerated

Sage noted the depression treatment was generally well tolerated. Overall adverse events in the 14-day treatment window and during 28 days of follow-up were similar between SAGE-217 and the placebo.

Chief Executive Jess Jonas reiterated his hopes for the depression treatment, noting SAGE-217 displays "good activity on most measures."

"We understand the drug development is an iterative process," he said in a written statement. "In the study, we've gathered new data on SAGE-217, data we believe support our hypothesis that SAGE-217 has a unique profile with the potential for rapid and robust onset with durable effect."

Pfizer (PFE) and GlycoMimetics (GLYC) reports Top-Line Phase 3 results for Rivipansel; study did not meet its primary or key secondary endpoints

** charts after announcement **

 

Co announced today that the Phase 3 Rivipansel (GMI-1070): Evaluating Safety, Efficacy and Time to Discharge (RESET) pivotal study did not meet its primary or key secondary efficacy endpoints. 

The objective of the trial was to evaluate the efficacy and safety of rivipansel in patients aged six and older with sickle cell disease (SCD) who were hospitalized for a vaso-occlusive crisis (VOC) and required treatment with intravenous (IV) opioids. The primary endpoint was time to readiness-for-discharge and the key secondary efficacy endpoints were time-to-discharge, cumulative IV opioid consumption, and time to discontinuation of IV opioids.

Detailed analyses of the RESET study, including additional data on efficacy and safety endpoints, which are not available at this time, will be submitted for presentation at a future scientific meeting.

Detailed analyses of the RESET study, including additional data on efficacy and safety endpoints, which are not available at this time, will be submitted for presentation at a future scientific meeting.

** charts before  announcement **

 

=Puma Biotech. (PBYI) reported earnings on Thur 9 May 2019 (a/h)

Puma Biotech. beats by $0.41, beats on revs

• Reports Q1 (Mar) earnings of $0.21 per share, $0.41 better than the single analyst estimate of ($0.20); revenues rose 49.0% year/year to $99.1 mln vs the $67.25 mln S&P Capital IQ Consensus.
• Commentary: "During 2019, we anticipate the following key milestones for Puma: presenting data from the Phase III trial of neratinib in third-line metastatic breast cancer patients in the second quarter of 2019; filing a new drug application for neratinib based on the results of the Phase III trial in third-line metastatic breast cancer in the summer of 2019; meeting with the FDA to discuss the clinical development and regulatory strategy for the SUMMIT trial in the summer of 2019; receiving regulatory decisions for the extended adjuvant HER2-positive early stage breast cancer indication in other countries in the second half of 2019; reporting additional data from the Phase II CONTROL trial in the second quarter of 2019; and reporting Phase II data from the SUMMIT basket trial in patients with HER2 mutations in the second half of 2019."

Puma Biotech investors dump the stock after sales breast cancer drug NERLYNX fall 25% sequentially

Shares of Puma Biotech (PBYI) -36% are getting crushed after the company reported disappointing sales of its breast cancer drug NERLYNX, its first commercial product on the market.

Net NERLYNX revenue in the first quarter of $45.6 mln grew 27% yr/yr but fell 25% qtr/qtr in the seventh quarter since the drug's launch in 3Q17. New drugs don't experience seasonality like other industries that see a sequential decline from the fourth quarter to the first quarter, so a sequential decline is worrisome for investors.

The company said that there was an increase in patients discontinuing use of the drug due to its side effects. A key concern for the drug even before approval was diarrhea.

Management said that it continues to educate physicians and patients about the importance of antidiarrheal medicine along with the NERLYNX as some physicians don't prescribe antidiarrheal medicine and some patients don't fill the prescription.

The stock got crushed on disappointing 3Q18 NETLYNX sales in November as well.

Cantor Fitzgerald and Citigroup downgraded the stock this morning.

Puma has a ~$700 mln market value with the stock testing late 2018 lows down ~36% premarket.

=Biogen (BIIB) halts research on Alzheimer's drug

• Biogen is halting two global phase-three trials testing the once-promising Alzheimer’s drug aducanumab, delivering a late-stage blow to researchers searching for therapies for the incurable degenerative disease.
• In 2018, pharmaceutical companies including Eli Lilly, AstraZeneca, Pfizer and Merck halted research or development into possible Alzheimer’s and dementia therapies due to disappointing results.

March 21 (Reuters) - Biogen and partner Eisai Co Ltd are ending two late-stage trials testing an Alzheimer's drug, they said Thursday, marking the latest setback for an industry keen to develop treatments for the memory-robbing disease.

Shares in Biogen slid 25 percent to $81.60 in premarket trading.

The decision to discontinue the trials testing drug aducanumab was made after an independent data monitoring committee reported the drug was unlikely to be successful, the companies said. The recommendation was not based on safety concerns, they added.

After dozens of experimental Alzheimer's drugs have failed in the recent past, there is a desperate need for a treatment that works.

The disease is the most common form of dementia that affects nearly 50 million people worldwide and is expected to rise to more than 131 million by 2050, according to Alzheimer's Disease International.

Biogen said it would continue to develop other treatments for Alzheimer's.

"This disappointing news confirms the complexity of treating Alzheimer's disease and the need to further advance knowledge in neuroscience," Biogen Chief Executive Officer Michel Vounatsos said.

"We will continue advancing our pipeline of potential therapies in Alzheimer's disease." The companies, however, for now will also discontinue a mid-stage study and a long-term extension study of aducanumab.

====

Several drugmakers have recently come up short in efforts to find an effective treatment for Alzheimer’s. Among the drugmakers still pursuing a beta amyloid-targeting compound is Roche Holding AG. Like Biogen, the Swiss company has persisted despite setbacks. After its candidate, gantenerumab, failed an earlier test four years ago, Roche decided to ratchet up the dose and try again with a pair of big late-stage trials. Results from the studies aren’t expected for about another two years.

Roche mostly abandoned a second beta amyloid compound earlier this year, halting two large studies after concluding they would fail. Roche’s partner on the second compound, Alzheimer’s specialist AC Immune SA, at one point fell more than 12 percent in premarket trading in New York.

Immunogen (IMGN) : ovarian cancer drug fails in a late-stage study

 

daily

monthly

March 1 (Reuters) - U.S. drug developer ImmunoGen Inc said on Friday its experimental ovarian cancer drug failed to meet the main goal of a late-stage study. When compared with chemotherapy, the drug, mirvetuximab soravtansine, did not significantly prolong the duration that ovarian cancer patients survived without the disease progressing.

The company said that based on the efficacy signals, overall response rate and survival, it will conduct additional analysis to evaluation the potential benefit of mirvetuximab soravtansine for FRα-positive platinum-resistant ovarian cancer. "ImmunoGen is in a strong financial position with approximately $295 million in cash on our balance sheet, and we will continue to advance our portfolio of next-generation ADCs, which includes three additional development candidates targeting a range of tumor types in both hematologic malignancies and solid tumors," said Chief Executive Mark Enyedy. The stock has tumbled 57% over the past 12 months through Thursday.

-=Novavax (NVAX) respiratory infection vaccine fails in late-stage trial

Novavax, Inc. also develops NanoFlu vaccine, which is in Phase I/II clinical trial for treating seasonal influenza in older adults; and Ebola virus glycoprotein vaccine that is in Phase I clinical trial. Its preclinical programs include Zika virus; combination respiratory vaccine candidate to protect against RSV and seasonal influenza, as well as other infectious disease vaccine candidates. Novavax, Inc. was

http://www.novavax.com
Sector: Healthcare
Industry: Biotechnology
Full Time Employees: 362
Founded in 1987
Headquartered in Gaithersburg, Maryland.




Feb 28 (Reuters) - Novavax Inc said on Thursday its vaccine to prevent RSV infection, a leading cause of a respiratory disease in infants, failed to meet the main goal of a late stage study.

The vaccine was not able to lower respiratory tract infection caused by RSV, or respiratory syncytial virus, the company said.

ResVax is a vaccine administered to mothers during pregnancy intended to protect infants against RSV disease.

"While this study did not meet the pre-specified success criterion for the primary clinical endpoint of this trial, the data indicate that ResVax protects infants from some of the most serious consequences of RSV," Chief Executive Officer Stanley Erck said in a statement.

The company said it planned to meet with U.S. and European health regulators to review the data.

G1 Therapeutics (GTHX) : placebo beats drug in cancer response rate test

G1 Therapeutics’ cancer drug trilaciclib has failed to beat placebo against multiple efficacy measures in phase 2. Trilaciclib performed well against its neutropenia-related primary endpoints but fell short in terms of response rate, prompting investors to send G1's stock down 26%.

   

death cross

North Carolina-based G1 designed the phase 2 to assess whether short-acting intravenous CDK4/6 inhibitor trilaciclib could reduce the occurrence and duration of Grade 4 neutropenia—a condition defined by abnormally low levels of a type of white blood cell—in patients with second or third-line small cell lung cancer (SCLC). The trial hit its primary endpoints by showing that trilaciclib, when added to chemotherapy drug topotecan, is more effective than placebo at controlling neutropenia.

That primary endpoint success enabled G1 to paint the results as positive. However, investors looked past the neutropenia data and zeroed in on a line deep in G1’s statement about rates of objective responses and clinical benefit seen in the trial, as well as the progression-free survival.

Of the 26 patients in the placebo arm, six responded, resulting in an objective response rate (ORR) of 23.1%. In the trilaciclib arm, four of the 30 patients responded, giving an ORR of 13.3%. The clinical benefit rate and progression-free survival were almost identical across the two arms, coming in at around 60% and 4.2 months, respectively.

G1 thinks trilaciclib may improve overall survival (OS) by preserving immune system function during chemotherapy. But G1 is yet to generate the mature OS data needed to validate that idea, and trilaciclib’s performance against other measures of efficacy has been mixed. A metastatic triple-negative breast cancer trial recently linked use of trilaciclib to improved ORR, but an earlier study in first-line SCLC found it was numerically worse than placebo against the objective response yardstick.

Despite that, G1 Chief Medical Officer Raj Malik thinks the biotech has emerged from the midphase program with data to support the advance of trilaciclib.

“We now have four randomized phase 2 trials showing trilaciclib’s multi-lineage myelopreservation benefits. We plan to meet with U.S. and European regulatory authorities in 2019 to discuss the totality of trilaciclib data and pathways to approval,” Malik said in a statement.

=Progenics (PGNX) falls on missed study endpoint

Progenics Pharmaceuticals Inc. (PGNX) shares fell in the extended session Wednesday after the cancer drug company said one of its imaging agents was not sensitive enough to detect prostate cancer in a clinical study. Progenics shares fell 19% after hours, following a 1.6% decline to close the regular session at $7.30. The company said a late-stage clinical trial found its so-called "1404" imaging agent needed to positively detect prostate cancer with a 60% or more sensitivity but did so with a 47% to 51% range. The imaging agent, however, was able to detect the absence of prostate cancer correctly with a specificity of 71% to 75% compared with the study target of 60% or more.

=Merrimack Pharma (MACK) : trial of pancreatic cancer treatment fails to meet main goals

Merrimack Pharma announces top-line results from the CARRIE study -- did not meet primary or secondary endpoints  

The co announced top-line results from the CARRIE study, a randomized Phase 2 trial evaluating the addition of MM-141 (istiratumab) to standard-of-care treatment in patients with previously untreated metastatic pancreatic cancer and high serum levels of free Insulin-like Growth Factor-1 (IGF-1). The study did not meet its primary or secondary efficacy endpoints in patients who received MM-141 in combination with nab-paclitaxel and gemcitabine, compared to nab-paclitaxel and gemcitabine alone. These results were consistent in all subgroups analyzed. Based on these results, Merrimack will not devote additional resources to the development of MM-141.

• Merrimack plans to present the complete data from this Phase 2 study at an upcoming medical oncology meeting.

The company said it would now focus on developing other programs in its pipeline, which includes a drug for breast cancer and lung cancer.

=Celldex Therapeutics (CLDX) : failed phase 2b trial of treatment of triple-negative breast cancer

Celldex Therapeutics reports that the randomized Phase 2b METRIC Study of glembatumumab vedotin compared to Xeloda in patients with metastatic triple-negative breast cancers that overexpress gpNMB failed to meet its primary endpoint

The randomized, Phase 2b METRIC Study of glembatumumab vedotin compared to Xeloda (capecitabine) in patients with metastatic triple-negative breast cancers that overexpress gpNMB failed to meet its primary endpoint, progression-free survival (PFS) as assessed by an independent, central reading of patient scans (Hazard ratio = 0.95; median PFS: glembatumumab vedotin 2.9 months vs. Xeloda 2.8 months; p=0.76).

• There was no significant advantage for glembatumumab vedotin in key secondary endpoints, including overall response rate, duration of response and overall survival. The glembatumumab vedotin safety profile was consistent with prior experience.

Celldex believes its pipeline prioritization and organizational restructuring efforts will extend financial resources beyond the guidance issued in the Company's year-end 2017 earnings press release and associated filings. The Company plans to provide revised guidance in its first quarter 2018 financial results in early May.