==AnaptysBio (ANAB) : promising results for rheumatoid arthritis treatment

• The biotech company is testing a drug called rosnilimab in patients with rheumatoid arthritis and ulcerative colitis. Both are inflammatory conditions.

In the study called Renoir, patients showed statistically significant improvements across numerous measures. Importantly, after 12 weeks of treatment, 36% to 47% of patients showed at least a 50% improvement in symptoms of rheumatoid arthritis. Nearly seven in 10 patients — 69% — reached a low-disease activity score on another measure after 14 weeks.

These surpass historical benchmarks, Wedbush analyst Martin Fan said in a client report. He also noted the company is now planning to have the results of a study of rosnilimab in patients with ulcerative colitis in the fourth quarter of 2025 vs. earlier forecasts for the first quarter of 2026.

AnaptysBio Mets Primary Goal

The primary goal of the study was to show low levels of C-reactive protein after 12 weeks. C-reactive protein in the blood indicates there's inflammation somewhere in the body. Patients who received rosnilimab showed statistically significant improvement in C-reactive protein levels vs. the placebo group.

After 14 weeks, patients showed the best-ever improvements on scales called ACR20, ACR50 and ACR70. This means their symptoms improved by 20%, 50% and 70%. In the first group, 79% to 82% of overall patients showed improvement. More than half — 59% to 67% — reached at least a 50% symptom improvement. And 41% to 48% of patients had at least a 70% improvement in symptoms.

Notably, 68% to 71% of patients reached a low-disease activity score on another scale measuring symptoms.

Akero Therapeutics (AKRO) : drug shows it reverses scarring in liver disease patients

• The experimental drug efruxifermin showed it can help patients with a type of liver disease to reverse scarring of the organ without worsening the condition in a keenly awaited mid-stage trial.
• This drug is being studied in patients with severe scarring or cirrhosis due to a type of fatty liver disease known as metabolic dysfunction-associated steatohepatitis (MASH).

Ticker:  AKRO

 

 

 

 

 

 

 

 

Akero Therapeutics reports preliminary topline results showing statistically significant reversal of compensated cirrhosis due to MASH-by both completer and ITT analyses-at Week 96 in phase 2b symmetry study 

• Co released preliminary topline week 96 results from SYMMETRY, a Phase 2b study evaluating the efficacy and safety of its lead product candidate efruxifermin (EFX) in patients with biopsy-confirmed compensated cirrhosis (F4), Child-Pugh Class A, due to metabolic dysfunction-associated steatohepatitis (MASH). Among patients with baseline and week 96 biopsies, 39% of patients treated with 50mg EFX experienced reversal of cirrhosis with no worsening of MASH, compared to 15% for placebo. In the Intent to Treat (ITT) population, with all missing week 96 biopsies treated as failures, 29% of patients in the 50mg EFX group experienced reversal of cirrhosis with no worsening of MASH, compared to approximately 12% in the placebo group.
• With more than a doubling of effect size from weeks 36 to 96 in the 50mg group (from 10% to 24%), the SYMMETRY study underscores the benefit of longer EFX treatment for patients with compensated cirrhosis (F4).
• In a subgroup of patients with baseline and week 96 biopsies who were not taking GLP-1 at baseline, 45% in the 50mg EFX group experienced reversal of cirrhosis with no worsening of MASH compared to 17% for placebo, suggesting that the observed reversal of cirrhosis was not attributable to GLP-1 therapy.

==Sana Biotechnology (SANA) : Clinical Results from Type 1 Diabetes Study

 

Sana Biotechnology Announces Positive Clinical Results from Type 1 Diabetes Study of Islet Cell Transplantation Without Immunosuppression

• First-in-Human Study Provides Evidence that Sana’s Hypoimmune (HIP) Technology Enables Transplanted Islet Cells to Avoid Immune Rejection and Produce Insulin Without Immunosuppression

• Results Demonstrate HIP-Engineered Primary Pancreatic Islet Cells Avoid Immune Detection, Function, and Persist after Intramuscular Transplantation in First Treated Patient with Type 1 Diabetes

• Function and Persistence of Pancreatic Islets Were Detectable by Production of Consistent Levels of Circulating C-Peptide, a Marker of Insulin Production, and Increased C-Peptide Levels with a Mixed Meal Tolerance Test (MMTT)

• MRI Shows Signals Consistent with Graft Survival 28 Days after Transplantation

About the Uppsala University Hospital Investigator-Sponsored Study of UP421 in Type 1 Diabetes

The investigator-sponsored study of UP421 is supported by a grant from The Leona M. and Harry B. Helmsley Charitable Trust. The study tests the hypothesis whether HIP-engineered insulin-producing pancreatic cells can be transplanted safely and help to regain insulin production in individuals with type 1 diabetes without need of simultaneous treatment with immunosuppressive medicines. To do this, UP421 is engineered using Sana’s HIP platform at Oslo University Hospital. The study involves intramuscular surgical transplantation of primary, or donor-derived, HIP-engineered islet cells into the forearm of patients with type 1 diabetes.

==Candel Therapeutics (CADL) : Prostate cancer therapy shows promise

 

On Wednesday, Candel Therapeutics, Inc. (NASDAQ:CADL) announced results from a phase 3 trial of CAN-2409 viral immunotherapy in intermediate-to-high-risk, localized prostate cancer patients.

The trial met its primary endpoint and demonstrated statistically significant improvement in disease-free survival in patients who received CAN-2409 plus the prodrug (valacyclovir) combined with the standard of care compared to the standard of care alone.

CAN-2409, when administered with valacyclovir, is designed to induce immunogenic cell death of tumor cells by exposing them to tumor antigens in the context of an activated tumor microenvironment.

===Travere Therapeutics (TVTX) kidney-disease treatment has mixed results in trial

Travere Therapeutics Inc. released data from a late-stage study of Filspari in IgA nephropathy, a rare kidney disease. In a head-to-head study versus irbesartan, Filspari fell short of producing a statistically significant difference by one measure of kidney function, although patients on the treatment had some of the slowest annual rates of kidney function decline seen in IgA nephropathy clinical trials, Travere said in a release. 

 

Travere Therapeutics Announces Confirmatory Data from the Phase 3 PROTECT Study of FILSPARI Demonstrating Long-Term Kidney Function Preservation in IgA Nephropathy; Narrowly Missing eGFR Total Slope Endpoint versus Active Control, Irbesartan 

SAN DIEGO, Sept. 21, 2023 (GLOBE NEWSWIRE) -- Travere Therapeutics, Inc. (Nasdaq: TVTX) today announced topline two-year confirmatory secondary endpoint results from the Company's pivotal head-to-head Phase 3 PROTECT Study of FILSPARI (sparsentan) in IgA nephropathy (IgAN) versus irbesartan. FILSPARI demonstrated long-term kidney function preservation and achieved a clinically meaningful difference in estimated glomerular filtration rate (eGFR) total and chronic slope versus irbesartan, narrowly missing statistical significance in eGFR total slope while achieving statistical significance in eGFR chronic slope for purposes of regulatory review in the EU. FILSPARI is currently available under accelerated approval in the U.S. The Company will engage with regulators and expects to submit a supplemental New Drug Application (sNDA) in 1H 2024 for full approval in the U.S.

Ligand Pharma (LGND) announces that its partner Travere Therapeutics (TVTX) released topline, two-year confirmatory secondary endpoint results from its pivotal, head-to-head Phase 3 PROTECT Study of FILSPARI in IgA nephropathy versus irbesartan 

• FILSPARI demonstrated long-term kidney function preservation and achieved a clinically meaningful difference in estimated glomerular filtration rate total and chronic slope versus irbesartan, narrowly missing statistical significance in eGFR total slope while achieving statistical significance in eGFR chronic slope for purposes of regulatory review in the EU. FILSPARI is currently available under accelerated approval in the U.S. Travere will engage with regulators and expects to submit a supplemental New Drug Application in 1H 2024 for full approval in the U.S.
  
• Under Ligand's license agreement with Travere for FILSPARI, Ligand is entitled to receive net royalties of 9% on global net product sales of FILSPARI.

-=Cassava Sciences (SAVA) : Alzheimer's drug trial results

 

Cassava Sciences announces results of an interim analysis from an open-label study of simufilam, its lead drug candidate for the treatment of Alzheimer's disease

• Patients' cognition improved 1.6 Points on ADAS-Cog11.
• Patients' behavior improved 1.3 Points on NPI.
• Improvements maintained at 6 months.
• Results support advancing simufilam into Phase 3 clinical program.

-=Vir Biotechnology (VIR) : hepatitis B drug trial results

 

Vir Biotechnology reports initial data from ongoing Phase 1 trial of VIR-3434 for chronic hepatitis B Virus infection demonstrates significant and rapid reduction in hepatitis B surface antigen

Data from the first blinded cohort of eight patients, two of whom received placebo and six of whom received a single dose of 6 mg of VIR-3434, showed that six of eight patients achieved a mean reduction of 1.3 log10 IU/mL in serum hepatitis B virus surface antigen (HBsAg) by day eight, the day when nadir was achieved in most patients.

A Phase 2 trial combining VIR-3434 with Vir's HBV-targeting siRNA, VIR-2218, is expected to commence in the second half of this year.

-=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."

=Biogen (BIIB) reported earnings on Tue 22 Oct 2019 (b/o)

• Earnings + experimental Alzheimer's therapy -- the same drug was considered a failure in March 2019

Biogen plans to pursue regulatory approval for aducanumab for early Alzheimer's disease -- Company plans to submit Biologics License Application in early 2020

The co and Eisai (ESALY) announced that, after consulting with the U.S. Food and Drug Administration (FDA), Biogen plans to pursue regulatory approval for aducanumab, an investigational treatment for early Alzheimer's disease (AD). The Phase 3 EMERGE Study met its primary endpoint showing a significant reduction in clinical decline, and Biogen believes that results from a subset of patients in the Phase 3 ENGAGE Study who received sufficient exposure to high dose aducanumab support the findings from EMERGE. Patients who received aducanumab experienced significant benefits on measures of cognition and function such as memory, orientation, and language.

• Patients also experienced benefits on activities of daily living including conducting personal finances, performing household chores such as cleaning, shopping, and doing laundry, and independently traveling out of the home. If approved, aducanumab would become the first therapy to reduce the clinical decline of Alzheimer's disease and would also be the first therapy to demonstrate that removing amyloid beta resulted in better clinical outcomes.
• Based on discussions with the FDA, the Company plans to file a Biologics License Application (BLA) in early 2020 and will continue dialogue with regulatory authorities in international markets including Europe and Japan. The BLA submission will include data from the Phase 1/1b studies as well as the complete set of data from the Phase 3 studies.

Biogen beats by $0.95, beats on revs

Reports Q3 (Sep) earnings of $9.17 per share, excluding non-recurring items, $0.95 better than the S&P Capital IQ Consensus of $8.22; revenues rose 4.7% year/year to $3.6 bln vs the $3.53 bln S&P Capital IQ Consensus.

"SPINRAZA continued on a strong trajectory, particularly outside the U.S., and we are preparing for the expected launch of VUMERITY, which we believe will be an important addition to our market-leading multiple sclerosis portfolio. In addition to the recent news on aducanumab, we made strong progress in our pipeline as we initiated new clinical programs targeting Parkinson's disease and brain contusion, and we look forward to nine important data readouts by the end of next year. We continue to believe that our core focus on neuroscience will lead to new innovative treatments for patients and will maximize long-term returns for our shareholders."

Recent Events

• In October 2019 the U.S. Food and Drug Administration (FDA) issued a tentative approval for VUMERITYTM (diroximel fumarate), a novel oral fumarate with a distinct chemical structure, for the treatment of relapsing forms of MS.
• In August 2019 Biogen discontinued the Phase 2b study of BG00011 for IPF due to safety concerns.

In the third quarter of 2019 Biogen recognized a GAAP-only impairment charge of approximately $216 million and a GAAP-only gain of $61 million on fair value remeasurement of contingent consideration, both related to the discontinuation of BG00011.

Shares are currently +39% after the company announced positive results from an Alzheimer's study and disclosed plans to pursue regulatory approval following discussions with the FDA. 

-=Seattle Genetics (SGEN) soars on positive trial of breast cancer treatment

• The biotech company added a drug called tucatinib to a regimen using Roche's Herceptin and a generic medicine known as capecitabine. Using all three drugs improved outcomes in patients with breast cancer, including those whose cancer spread to the brain.
• Patients who received the regimen were 46% less likely to worsen than those who took just Herceptin and capecitabine. The tucatinib regimen also improved overall survival by 34% compared with the combination of Herceptin and capecitabine alone.
• Notably, the three-drug regimen showed promise in patients whose cancer had spread to the brain. Adding tucatinib to Herceptin and capecitabine reduced the risk of disease progression or death by 52% in these patients vs. those on the two-drug breast cancer treatment.

Seattle Genetics Inc. announced positive results in a trial of a treatment for breast cancer.
Bothell, Wash.-based Seattle Genetics said the trial of tucatinib in locally advanced or metastatic HER2-positive breast cancer met its primary endpoint of progression-free survival. Patients were treated with tucatinib in combination with trastuzumab and capecitabine to trastuzumab and capecitabine alone. The trial also met its secondary endpoints.

"Based on these findings, we plan to unblind the trial and offer tucatinib to patients on the control arm," Chief Medical Officer Roger Dansey said in a statement. The company is also planning to submit a New Drug Application to the FDA in the first quarter of 2020, he said. HER2-positive breast cancer is an aggressive form of the disease that affects 15% to 20% of cases worldwide. The trial is expected to enroll about 460 patients in North America, Europe and Asia. Leerink analysts said the trial is another win for Seattle Genetics and a "near best-case scenario."

=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.

Akebia Therapeutics (AKBA) : positive top-line results from two pivotal Phase 3 studies

Akebia Therapeutics (AKBA) : Positive Top-Line Results from Two Pivotal Phase 3 Studies of Vadadustat in Japanese Patients with Anemia Due to Chronic Kidney Disease 

• Co announced positive top-line results from two phase 3 active-controlled pivotal studies evaluating vadadustat, an investigational oral hypoxia-inducible factor prolyl hydroxylase inhibitor (HIF-PHI), in Japanese subjects with anemia due to chronic kidney disease (CKD).
• These studies were conducted by Akebia's development and commercialization collaboration partner in Japan, Mitsubishi Tanabe Pharma Corporation (MTPC).
• Each study, one in non-dialysis dependent subjects and one in hemodialysis-dependent subjects, met its primary endpoint.
• In addition, results from two phase 3 single-arm studies conducted by MTPC in peritoneal dialysis subjects and hemodialysis subjects further support vadadustat's potential in these indications.
• MTPC expects to submit a Japanese New Drug Application in 2019.

** charts before  **

 

TG Therapeutics (TGTX) : positive results from marginal zone lymphoma (MZL) trial

TG Therapeutics, Inc., a biopharmaceutical company, focuses on the acquisition, development, and commercialization of novel treatments for B-cell malignancies and autoimmune diseases in the United States.

• Sector: Healthcare
• Industry: Biotechnology
• Full Time Employees: 104
• TG Therapeutics, Inc. was incorporated in 1993 and is headquartered in New York, New York.
• http://www.tgtherapeutics.com

 

 

This week TG Therapeutics reported positive data from one cohort of a trial evaluating its lymphoma drug umbralisib.

Marginal zone lymphoma (MZL) is typically diagnosed in elderly patients. It's a slow-glowing type of non-Hodgkin lymphoma, and there are 7,500 new patients diagnosed in the U.S. with MZL annually. In the first-line setting, Rituxan is typically used. However, there remains a need for additional treatment options for relapsing patients.

TG Therapeutics reported that it met its primary endpoint of an overall response rate of between 40% to 50% in 69 MZL patients enrolled in its study. The study was designed to support a possible accelerated Food and Drug Administration (FDA) approval, and if the full primary data anticipated later this year remains solid, the company plans to file for FDA approval before year-end. By comparison, Imbruvica won accelerated approval in this indication following a midstage study in which it achieved a 46% overall response rate.

Management also updated investors on the timing for results from a cohort evaluating umbralisib in follicular lymphoma (FL) and small lymphocytic lymphoma (SLL). That cohort is fully enrolled, and results are expected later this year. Similar to the MZL cohort, the company's targeting a 40% to 50% overall response rate. If it hits that target, then TG Therapeutics hopes it can win an accelerated OK there, too.

Additionally, the company's still awaiting maturing progression-free survival data from its phase 3 trial evaluating ublituximab and umbralisib as combination therapy in chronic lymphocytic leukemia. Management expects to have results available from that study later this year or early in 2020.

Lymphoma is a multibillion dollar per year commercial opportunity. Although there have been advances in recent years, there's still an opportunity to address patients who fail to respond or see their disease progress following initial treatment. The umbrasilib data in MZL is encouraging because management says the overall response rate can't weaken between now and the primary data readout, suggesting it's only a matter of time before the company can present this drug to regulators.

It remains to be seen, though, if the company can deliver in FL and SLL or in CLL. There's also no guarantee that the FDA will approve umbralisib in MZL prior to another trial, so there's still some risk here to consider. For this reason, only risk-tolerant investors should be considering TG Therapeutics for their portfolios.

-=Horizon Pharma (HZNP) : thyroid eye disease treatment succeeds in a Phase 3 study

The company announced positive results in a late-stage trial of a treatment for active thyroid eye disease, or TED. The company said the phase 3 trial of teprotumumab met its primary endpoint of improving proptosis, or bulging of the eye compared with placebo, with 82.9% of patients showing improvement compared with 9.5% of placebo patients. The trial also succeeded in meeting secondary endpoints and achieving a consistent safety profile with the phase 2 study.

The company is expecting to submit a biologics license application to the U.S. Food and Drug Administration in mid-2019. TED is a progressive automimmune disease with a limited window for treatment without surgical intervention. As it progresses, it can lead to other eye issues and even blindness. Horizon shares have gained 49.9% in the last 12 months, while the S&P 500 (SPX) has gained 2.9%.

Horizon has high hopes for teprotumumab. In a November interview, CEO Walbert told Investor's Business Daily he expects the thyroid eye disease treatment to bring in a peak $750 million annually in the U.S. alone. The company's other key drug, a gout treatment, is expected to hit that same mark globally.

MacroGenics (MGNX) announced positive results from SOPHIA

MacroGenics announced positive results from SOPHIA, a Phase 3 clinical study of margetuximab in HER2-positive metastatic breast cancer patients

• The SOPHIA clinical trial met the primary endpoint of prolongation of progression-free survival (PFS) in patients treated with the combination of margetuximab plus chemotherapy compared to trastuzumab plus chemotherapy. Patients in the margetuximab arm experienced a 24% risk reduction in PFS compared to patients in the trastuzumab arm (HR=0.76, p=0.033). Notably, approximately 85% of patients in the study were carriers of the CD16A (Fc?RIIIa) 158F allele, which has been previously associated with diminished clinical response to HERCEPTIN and other antibodies.
• MacroGenics anticipates submitting a Biologics License Application (BLA) to the U.S. Food and Drug Administration in the second half of 2019.

A gene mutation that makes an excess of the HER-2 protein is present in about 15% of primary invasive breast cancers, according to UpToDate, an evidence-based medical resource used by clinicians. This particular trial showed longer progression-free survival in patients treated with a combination of margetuximab and chemotherapy compared to those on trastuzumab (brand name Herceptin) and chemotherapy, a standard-of-care treatment. The company also said approximately 85% of patients in the study were carriers of the CD16A 158F allele, a gene variation has been associated with a reduced clinical response to Herceptin and other antibodies. MacroGenics said it plans to submit a biologics license application to the U.S. Food and Drug Administration in the second half of 2019.

** charts before announcement ** 

 

=Eli Lilly's (LLY) diabetes drug shows promise in mid-stage trial

• Peer: NVO

Oct 4 (Reuters) - Eli Lilly and Co said on Thursday data from mid-stage trial of its experimental diabetes drug showed clinically meaningful blood sugar reduction and weight loss in people with type 2 diabetes.

Data from the six-month study showed that the drug significantly reduced blood sugar levels by up to 2.4 percent and an average weight reduction of up to 12.7 percent, Lilly said in a statement.

The company said it intends to complete its late-stage study for the drug in late 2021, and is also evaluating the drug for treating obesity, among other conditions.

About 30 million adults in the United States have diabetes, with 90 to 95 percent of them suffering from type 2 diabetes, according to Lilly.

Lilly's wide portfolio of diabetes drugs, including Humalog and Trulicity, contributed at least 38 percent to its total sales of $6.36 billion in the last quarter. 

***

Eli Lilly investigational dual GIP and GLP-1 receptor agonist shows significant reduction in HbA1c and body weight in people with type 2 diabetes  

• At 26 weeks, the primary analysis showed a robust dose response compared to placebo throughout the entire dose range of GIP/GLP-1 RA included in the study.
• The analysis of participants while on treatment comparing GIP/GLP-1 RA to dulaglutide and placebo showed significant improvements across endpoints.
• HbA1c reduction: All GIP/GLP-1 RA doses and dulaglutide showed significant blood sugar improvement (mean absolute reduction) from baseline [GIP/GLP-1 RA: -1.6% (5 mg), -2.0% (10 mg), and -2.4% (15 mg); dulaglutide -1.1% (1.5 mg)] compared to placebo (0.1%).
• The safety and efficacy of Lilly's GIP/GLP-1 RA are being studied further in a large phase 3 clinical program that will be referred to as the SURPASS program. Phase 3 studies for type 2 diabetes are expected to begin no later than early 2019 and complete in late 2021. Lilly is evaluating next steps in the study of GIP/GLP-1 RA for obesity and other conditions.

Eli Lilly announces that empagliflozin met the primary efficacy endpoint for all doses investigated (2.5, 10 and 25 mg) in the Empagliflozin as Adjunctive to inSulin thErapy Phase III program in adults with type 1 diabetes 

In EASE-2, placebo-corrected mean change from baseline in A1C at week 26 was -0.54 percent and -0.53 percent for empagliflozin 10 and 25 mg, respectively. In EASE-3, placebo-corrected mean change from baseline in A1C at week 26 was -0.28 percent, -0.45 percent and -0.52 percent for empagliflozin 2.5 mg, 10 mg and 25 mg, respectively. In addition to reduction in A1C, empagliflozin treatment was effective on secondary endpoints, showing reductions in weight, decreases in blood pressure, and decreases in total daily insulin dose. In addition, data from continuous glucose monitoring in the EASE program indicates that patients treated with empagliflozin had improved glycemic variability and spent more time in range, although the data for the 2.5 mg dose are limited.

• Based on the totality of the EASE data, partner Boehringer Ingelheim has initiated regulatory discussions for empagliflozin as adjunct to insulin for adults with type 1 diabetes.

=Akcea Therapeutics (AKCA) & Ionis Pharmaceuticals (IONS)

Akcea Therapeutics & Ionis Pharmaceuticals (IONS) announce 'positive' topline results from a Phase 2 clinical study of AKCEA-APO(a)-LRx in patients with established cardiovascular disease and elevated levels of lipoprotein 

Results from the study show:

• Statistically significant dose-dependent reductions of Lp(a) compared to placebo at all dose levels, including low monthly doses of AKCEA-APO(a)-LRx.
• Most patients in the active group achieved Lp(a) reductions below the established threshold of risk for CVD events.
• Treatment emergent adverse events were balanced between the active and placebo groups.
• Most common adverse event was injection site reactions (ISRs). ISRs were mostly mild and occurred in a minority of patients.
• No patient in the study experienced a confirmed platelet level below 100,000/mm3. The incidence of platelet levels below normal (140,000/mm3) was comparable between the active (10.5%) and placebo (14.9%) groups.
• Approximately 90% of patients completed treatment and the rate of treatment discontinuation was comparable between the active and placebo groups.

=Amarin (AMRN) : positive trial results in cholesterol treatment Vascepa

• Company said its cholesterol treatment met its primary endpoint of risk reduction. The company said Vascepa LDL-C treatment was also well tolerated.

Amarin announces topline results from Vascepa cardiovascular outcomes trial -- REDUCE-IT met its primary endpoint demonstrating an approximately 25% relative risk reduction

The co announced topline results from the Vascepa cardiovascular (CV) outcomes trial, REDUCE-IT, a global study of 8,179 statin-treated adults with elevated CV risk. REDUCE-IT met its primary endpoint demonstrating an approximately 25% relative risk reduction, to a high degree of statistical significance, in major adverse CV events (MACE) in the intent-to-treat patient population with use of Vascepa 4 grams/day as compared to placebo. Key topline results include:

• Efficacy: Approximately 25% relative risk reduction, demonstrated to a high degree of statistical significance, in the primary endpoint composite of the first occurrence of MACE, including cardiovascular death, nonfatal myocardial infarction (MI), nonfatal stroke, coronary revascularization, or unstable angina requiring hospitalization. This result was supported by robust demonstrations of efficacy across multiple secondary endpoints.
• Safety: Vascepa was well tolerated with a safety profile consistent with clinical experience associated with omega-3 fatty acids and current FDA-approved labeling. The proportions of patients experiencing adverse events and serious adverse events in REDUCE-IT were similar between the active and placebo treatment groups. Median follow-up time in REDUCE-IT was 4.9 years.

As previously described, given the successful topline results of REDUCE-IT, Amarin is in the process of increasing the number of company sales representatives promoting Vascepa to over 400 people in the United States. This will provide a greater concentration of coverage in current sales territories and provide new coverage where Amarin currently does not have sales representatives. In addition to sales force expansion in the United States, Amarin plans to work with its international partners to support regulatory efforts outside the United States based on REDUCE-IT results. As previously described in the months leading up to REDUCE-IT results, Amarin increased its Vascepa inventory levels in preparation for positive results.

=Viking Therapeutics (VKTX) : liver drug succeeds in mid-stage study

Viking Therapeutics announces 'positive' top-line results from a 12-week Phase 2 study of VK2809 
The study successfully achieved its primary endpoint, with patients receiving VK2809 demonstrating statistically significant reductions in LDL-C compared with placebo. In addition, the trial's secondary endpoint was achieved, with VK2809-treated patients experiencing statistically significant reductions in liver fat content compared with placebo. An abstract describing the results has been submitted for consideration for presentation at The Liver Meeting 2018, the annual meeting of the American Association for the Study of Liver Diseases (AASLD), scheduled for November 9-13, 2018 in San Francisco, CA. No serious adverse events (SAEs) were reported among patients receiving VK2809 or placebo.

=Inovio Pharma (INO) : positive HIV vaccine results

Inovio Pharma announces that its HIV vaccine, PENNVAX-GP, maintained durable and robust immune responses at month 12, a full six months after the last dose in a Phase 1 clinical study(4.73 )

More comprehensive immune analyses demonstrated that PENNVAX-GP (plus IL-12) generated HIV-specific CD4+ T cell and binding antibody response rates close to 100% when delivered with either CELLECTRA intramuscular or intradermal devices.

• For instance, 96% (26 of 27) of participants receiving PENNVAX-GP and IL-12 via the IM route demonstrated a CD4+ T cell response while the same percentage (96% or 27 of 28) of participants receiving the vaccine formulation via ID administration also displayed anti-HIV CD4+ T cell responses -- even though those vaccinated via intradermal administration received 1/5th the total dose compared to those vaccinated via the intramuscular device.
• The new data from subjects followed for a full one year of the study showed that the immune responses were maintained in most subjects at month 12 (or six months after the last dose) as evidenced by the durability of activated T cells as well as the magnitude of responder rates. Notably, the percentage of patients who had CD8+ T cell responses immediately after the last dose stayed the same or even increased slightly over the 6 month follow up period, clearly demonstrating durable vaccine-generated memory responses.