Assembly Biosciences reports chronic hepatitis data, shares jump

Assembly Biosciences announces presentation of ABI-H0731, ABI-H2158 data

Assembly Biosciences (ASMB) announced that data on its lead HBV core inhibitor candidates, ABI-H0731 and ABI-H2158 for the treatment of chronic HBV will be featured in a late-breaking poster session during the American Association for the Study of Liver Diseases Annual Meeting.

Shares jump on hepatitis data, Stockwinners

A hepatitis B infection can result in either an acute infection or a chronic infection. When a person is first infected with the hepatitis B virus, it is called an “acute infection” (or a new infection). Most healthy adults that are infected do not have any symptoms and are able to get rid of the virus without any problems. Some adults are unable to get rid of the virus after six months and they are diagnosed as having a “chronic infection.”

Title: Continued Therapy with ABI-H0731+Nrtl Results in Sequential Reduction/Loss of HBV DNA, HBV RNA, HBeAg, HBcrAg and HBsAg in HBeAg-Positive Patients.

Abstract Summary: Final results from Phase 2a are reported for HBeAg+ patients with chronic HBV infection treated with 731+Nrtl for 24 weeks.

In Study 202, greater mean log10 declines in HBV DNA and RNA were achieved with 731+Nrtl versus entecavir alone.

In Study 201, the proportion of patients on 731+Nrtl versus Nrtl alone achieving DNA target not detected was 69% vs 0%, and the proportion of patients achieving RNA less than35 U/mL whose RNA was greater than or equal to35 U/mL at baseline was 52% vs 0% respectively.

In Study 211, there are 64 HBeAg+ patients currently on extended treatment beyond 24 weeks. Among the 27 HBeAg+ patients receiving 731+Nrtl in Study 201, 41% have now achieved DNA TND along with RNA less than35 U/mL and HBeAg less than1 IU/mL.

At their last time point, Study 202 patients now in Study 211 have demonstrated mean DNA and RNA declines of 6.1 and 3.0 logs, respectively, with observed mean log changes of greater than or equal to 0.6 for HBeAg, greater than 0.8 log for HBcrAg and greater than or equal to 0.4 log for HBsAg.

731 continues to exhibit a favorable safety and tolerability profile in patients treated for up to 1 year, with only mild/moderate adverse events and lab abnormalities, and only a single discontinuation due to a Grade 1 rash.

The combination of 731+NrtI results in faster and deeper declines in HBV DNA and RNA than NrtI alone, as well as subsequent declines in the surrogate markers of cccDNA predictive of cccDNA pool depletion, and HBsAg.

A Visual Guide to Hepatitis, Stockwinners

The emergent data supports the continued development of 731.

Abstract data are as of the time of submission; the poster is expected to include updated safety and efficacy results. Title: The Second-Generation Hepatitis B Virus Core Inhibitor ABI-H2158 is Associated with Potent Antiviral Activity in a 14-Day Monotherapy Study in HBeAg-positive Patients with Chronic Hepatitis B.

Abstract Summary: The Phase 1b study is enrolling sequential cohorts of 9 patients and each cohort will be randomized to receive 2158 or placebo QD for 14 days in a blinded manner.

Dosing in the 1st cohort has been completed. In patients receiving 2158, mean declines from Baseline to Day 15 in HBV DNA and RNA levels were 2.3 log10 and 2.1 log10 IU/mL respectively.

No serious AEs, dose limiting toxicities or premature discontinuations were reported.

Three patients reported a total of 5 mild, drug-related AEs that recovered without intervention; dizziness, fatigue, rash, headache and upper abdominal pain.

Treatment emergent laboratory abnormalities were infrequent, mild and transient, with no ALT elevations Grade greater than or equal to 1 in severity.

Day 14 plasma 2158 Cmax and AUC0-24hr were 3,390 ng/mL and 46,100 hr*ng/mL, respectively.

Results from the initial 100 mg low dose of ABI-H2158 cohort demonstrated potent antiviral activity, a favourable safety profile when administered for 14 days, and support once daily dosing in CHB patients.

Sarepta reports positive results for muscular dystrophy Type 2E treatment

Sarepta announces results from gene therapy trial for beta sarcoglycanopathy

Sarepta Therapeutics (SPRT) announced the nine-month functional results from three Limb-girdle muscular dystrophy Type 2E ,LGMD2E, clinical trial participants who received SRP-9003. SRP-9003 is an investigational gene therapy intended to transduce skeletal and cardiac muscle with a gene that codes for the full-length, native beta-sarcoglycan protein, the lack of which is the sole cause of LGMD2E.

Sarepta drops after U.K. trial halt,
Sarepta rises on muscular dystrophy data, Stockwinners

In Cohort 1 of the SRP-9003 study, three participants ages 4-13 were treated with an infusion of SRP-9003 at a dose of 5x1013vg/kg.

Improvements in functional outcomes were observed at day 270 for all three participants.

“We have now observed consistent functional improvements, in addition to high levels of expression of the missing protein of interest and strong results in related biomarkers, in both of our first cohorts for Duchenne muscular dystrophy and LGMD2E. We intend to test one higher dose of SRP-9003 in LGMD2E participants, select our clinical dose and then advance our SRP-9003 program, along with our other five LGMD programs, as rapidly as possible,” said Doug Ingram, Sarepta’s president and chief executive officer.

“With the results of our first LGMD2E cohort, Sarepta continues to build its gene therapy engine, an enduring model created to design, develop and bring to the medical and patient community transformative therapies for those living with, and too often dying from, rare genetic disease.”

No new safety signals were observed and the safety profile seen to date supports the ability to dose escalate in the next cohort of the study. As previously disclosed, two participants in the study had elevated liver enzymes, one of which was designated a serious adverse event (SAE), as the participant had associated transient increase in bilirubin. Both events occurred when the participants were tapered off oral steroids and, in both instances, elevated liver enzymes returned to baseline and symptoms resolved following supplemental steroid treatment.

SPRT is up $5.52 to $86.86.

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AstraZeneca and Merck report prostate cancer data

AstraZeneca, Merck presents results from Phase 3 PROfound trial of LYNPARZA

AstraZeneca (AZN) and Merck (MRK) presented detailed results from the Phase 3 PROfound trial in 387 men with metastatic castration-resistant prostate cancer who have a mutation in their homologous recombination repair genes and whose disease had progressed on prior treatment with new hormonal agent treatments e.g. abiraterone or enzalutamide.

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AstraZeneca and Merck report prostate cancer, Stockwinners

The trial was designed to analyze men with mCRPC harboring HRR-mutated genes in two cohorts: the primary endpoint was in those with mutations in BRCA1/2 or ATM genes and then, if LYNPARZA showed clinical benefit, a formal analysis was performed of the overall trial population of men with HRRm genes.

Merck presents results from Phase 3 KEYNOTE-426 study, Stockwinners
AstraZeneca and Merck report prostate cancer , Stockwinners

Results showed a statistically-significant and clinically-meaningful improvement with LYNPARZA in the primary endpoint of radiographic progression-free survival in BRCA1/2 or ATM-mutated tumors reducing the risk of disease progression or death by a median of 7.4 months versus 3.6 months for those receiving abiraterone or enzalutamide.

LYNPARZA reduced the risk of disease progression or death by 66% for these men. The trial also met the key secondary endpoint of rPFS in the overall HRRm population, where LYNPARZA reduced the risk of disease progression or death by 51% and improved rPFS to a median of 5.8 months vs. 3.5 months for those receiving abiraterone or enzalutamide.

In the key secondary endpoint of time to pain progression, median TTPP was not reached with LYNPARZA and was 9.92 months with abiraterone and enzalutamide in patients with BRCA1/2 or ATM mutations.

Results also showed a trend at this interim analysis time point for improvement in overall survival, another key secondary endpoint. LYNPARZA extended OS to a median of 18.5 months versus 15.1 months for abiraterone or enzalutamide in men with BRCA1/2 or ATM-mutated tumors, of which 81% started on abiraterone or enzalutamide and, following confirmed disease progression, then switched to LYNPARZA.

At this interim analysis, the OS endpoint did not meet statistical significance. In an exploratory analysis, a similar trend in OS was observed at this interim analysis in the HRRm population with a median of 17.5 months for men treated with LYNPARZA vs. 14.3 months for those receiving abiraterone or enzalutamide.

The trial showed a confirmed overall response rate a key secondary endpoint of 33.3% for LYNPARZA vs. 2.3% for abiraterone or enzalutamide in patients with BRCA1/2 or ATM mutations.

In an exploratory analysis of patients in the overall HRRm population, confirmed ORR was 21.7 % for LYNPARZA vs. 4.5% for patients receiving abiraterone or enzalutamide. The safety and tolerability profile of LYNPARZA in the PROfound trial was in line with that observed in prior clinical trials.

The most common adverse events greater than or equal to20% for LYNPARZA compared to abiraterone or enzalutamide were anemia, nausea, fatigue and asthenia, decreased appetite, and diarrhea. Grade 3 or above AEs were anemia, fatigue and asthenia, vomiting, dyspnea, urinary tract infection, nausea, decreased appetite, diarrhea, and back pain. AEs led to discontinuation of treatment in 16% of patients on LYNPARZA vs. 9% on abiraterone and enzalutamide.

AstraZeneca and Merck are also exploring additional trials in prostate cancer, including the ongoing Phase 3 PROpel trial, evaluating LYNPARZA as a first-line therapy in mCRPC for patients with or without HRR mutations, in combination with abiraterone acetate.

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Celgene sells its Otezla to Amgen for $13.4 billion

Amgen to acquire Otezla from Celgene for $13.4B in cash

Amgen (AMGN) announced that it has entered into an agreement with Celgene (CELG) in connection with its previously announced merger with Bristol-Myers (BMY) to acquire worldwide rights to Otezla, “the only oral, non-biologic treatment for psoriasis and psoriatic arthritis,” and certain related assets and liabilities, for $13.4B in cash, or approximately $11.2B, net of the present value of $2.2B in anticipated future cash tax benefits.

Bristol Meyers Comments on Celgene purchase, Stockwinners
Celgene sells Otezla to pave the way for its merger with Bristol-Meyers, Stockwinners

Otezla (apremilast) is a prescription medicine approved for the treatment of patients with moderate to severe plaque psoriasis for whom phototherapy or systemic therapy is appropriate. Otezla is a prescription medicine approved for the treatment of adult patients with active psoriatic arthritis. Otezla is a prescription medicine approved for the treatment of adult patients with oral ulcers associated with Behçet’s Disease.

Amgen goes shopping, Stockwinners

Amgen believes that the acquisition of Otezla offers many benefits including: A strong strategic fit with Amgen’s long-standing expertise in psoriasis and inflammation; A differentiated, oral therapy complementary to Amgen’s existing inflammation franchise of innovative biologics and biosimilar products; At least low double-digit Otezla sales growth, on average, over the next five years; Acceleration of Amgen’s near- and long-term revenue growth; Immediate non-GAAP EPS accretion; Intellectual Property exclusivity through at least 2028 in the U.S.; Worldwide rights which fit well with Amgen’s international presence and global expansion objectives; Support of increased R&D investment in 2020 to advance Amgen’s innovative pipeline of first-in-class molecules; No interruption in deployment of Amgen’s capital allocation priorities. Sales of Otezla in 2018 were $1.6B driven by strong volume growth.

Bristol-Myers treatment for colorectal cancer approved, Stockwinners
Bristol-Myers is in the process of buying Celgene, Stockwinners

The closing of the acquisition is contingent on Bristol-Myers entering into a consent decree with the Federal Trade Commission in connection with the pending Celgene merger, the closing of the pending merger with Celgene and the satisfaction of other customary closing conditions.

The transaction is expected to close by the end of 2019.

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Cambrex sold for $2.4 billion

Cambrex to be acquired by Permira Funds for $60.00 per share in cash, or $2.4B

Cambrex sold for $2.4 billion, Stockwinners

Cambrex (CBM) announced that it has signed a definitive agreement to be acquired by an affiliate of the Permira funds in a transaction valued at approximately $2.4B, including Cambrex’s net debt.

Under the terms of the merger agreement, Cambrex shareholders will receive $60.00 in cash for each share of Cambrex common stock, which represents a 47.1% premium to the August 6 closing stock price and a 37.3% premium to the 60-day volume weighted average closing price leading up to this announcement.

Completion of the transaction is subject to customary closing conditions, including receipt of approval by Cambrex’s shareholders and customary regulatory approvals. Closing is expected to occur during the fourth quarter.

Permira goes shopping, Stockwinners

The transaction will be financed through a combination of debt and equity financing.

Cambrex Corporation provides various products and services for the development and commercialization of new and generic therapeutics worldwide. Its products comprise active pharmaceutical ingredients and pharmaceutical intermediates that are used in the production of prescription and over-the-counter drug products, as well as finished dosage forms.

The company serves generic drug companies; and companies that discover and commercialize small molecule human therapeutics. The company sells its products directly, as well as through independent agents. 

Cambrex announced that it will not hold a second quarter 2019 earnings conference call and will not update previously provided financial guidance given the pending acquisition.

The Permira investment team advises the Permira Funds. The investment team identifies long-term macro trends to back, across five key sectors including healthcare. Healthcare is one of the World’s largest industries, spanning hundreds of sub-sectors (e.g., from Biotechnology to Heavy Medical Equipment, from Hospitals to Veterinary medicine). It has the potential to create significant value for its customers through improving the human experience but its costs are also potentially limitless. The sector’s fundamental trends and complexity along with its scale generate attractive investment opportunities.

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Pancreatic cancer data sends shares of Tyme Technologies higher

Tyme Technologies presents updated data from TYME-88-Panc Phase II study

Tyme Techs. shares jump of data, Stockwinners

Tyme Technologies (TYME) announced that its multicenter open-label Phase II TYME-88-Panc study evaluating SM-88 as an oral monotherapy in patients with advanced pancreatic cancer continues to demonstrate encouraging results and a well-tolerated safety profile.

The data from the TYME-88-Panc study were presented at the European Society of Medical Oncology 21st World Congress on Gastrointestinal Cancer.

The Annual meeting is held in Barcelona this year, Stockwinners

Updated results from the ongoing multicenter open-label Phase II TYME-88-Panc study involved 49 heavily pretreated patients with radiographically progressive metastatic pancreatic cancer who had significant disease related morbidity before receiving TYME’s investigational agent SM-88.

More than 80% of patients had received at least two prior lines of therapy. Of the 49 patients, 38 patients were evaluable for efficacy, as defined in the protocol.

Pancreatic cancer, Stockwinners

TYME-88-Panc is a two-part study in which Part 1 was intended to determine optimal dosing and assess if early clinical benefit supported further development of SM-88 in pancreatic cancer.

This study is being performed under a TYME IND with input from the FDA prior to study initiation. In this study, based on information available as of April 25, 2019, the median overall survival of evaluable patients was 6.4 months.

Certain efficacy indicators correlate A RECIST clinical benefit rate of stable disease or better was achieved by 44% of patients with available imaging. Notably, patients achieving stable disease or better demonstrated a statistically significant improvement in survival with a 92% reduction in risk of death.

The CBR was durable with majority of these patients remaining in stable disease or better at more than 7 months after receiving treatment with SM-88.

The measurement of CTCs is emerging as an important prognostic indicator in patients with pancreatic cancer. This is now the second TYME study in cancer patients showing that SM-88 reduces CTCs.

In a previous study of patients with prostate cancer, SM-88 treatment was also associated with a reduction in CTC count. In the TYME-88-Panc study, a median reduction of 63% in CTC burden was observed in evaluable patients. Importantly, patients with available results reaching an 80% reduction or greater in CTCs demonstrated a 60% decrease in risk of death.

In addition to these findings from the TYME-88-Panc study, data were also presented on subgroup analyses. TYME identified several screening criteria that were associated with rapidly declining prognostic factors defined as greater than 2 lines of prior therapy; age greater than 75 years old; albumin less than 3.5 g/dl. Patients with no indicators of poor prognosis had a better trend in survival.

TYME identified key sub-groups of patients who performed better. Patients with 1 or 2 prior lines of therapy had a better trend in survival. Female patients had a statistically significant trend toward better survival. These encouraging findings warrant further clinical evaluation of these subgroups. As of April 25, 2019, the study reported that SM-88 was well tolerated with only 4.0% of patients who experienced serious adverse events deemed at least possibly related to SM-88. One patient with reported SAEs continued on treatment.

The TYME-88-Panc research results are from an investigational study. SM-88 is not approved for the treatment of patients with any disease condition.

TYME is up 30 cents to $1.52.

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Allergan sold for $63 billion

AbbVie to acquire Allergan in cash, stock deal valued around $63B

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Allergan sold for $63 billion, Stockwinners

AbbVie (ABBV) and Allergan (AGN) announced that the companies have entered into a definitive transaction agreement under which AbbVie will acquire Allergan in a cash and stock transaction for a transaction equity value of approximately $63B, based on the closing price of AbbVie’s common stock of $78.45 on June 24.

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Abbvie to pay $63B to buy Allergan, Stockwinners

Upon completion of the transaction, AbbVie will continue to be incorporated in Delaware as AbbVie Inc. and have its principal executive offices in North Chicago, IL.

AbbVie will continue to be led by Richard Gonzalez as chairman and CEO.

Two members of Allergan’s board, including chairman and CEO, Brent Saunders, will join AbbVie’s board upon completion of the transaction.

Under the terms of the Transaction Agreement, Allergan Shareholders will receive 0.8660 AbbVie Shares and $120.30 in cash for each Allergan Share that they hold, for a total consideration of $188.24 per Allergan Share.

The transaction represents a 45% premium to the closing price of Allergan’s Shares on June 24.

AbbVie anticipates that the Acquisition will provide annual pre-tax synergies and other cost reductions of at least $2B in year three while leaving investments in key growth franchises untouched.

Botox is one of Allergan’s leading products, Stockwinners

The synergies and other cost reductions will be a result of optimizing the research and early stage portfolio, and reducing overlapping R&D resources, driving efficiencies in SG&A, including sales and marketing and central support function costs, and eliminating redundancies in manufacturing and supply chain, and leveraging procurement spend.

The synergies estimate excludes any potential revenue synergies.

AbbVie is expected to generate significant annual operating cash flow, which will support a debt reduction target of $15B to $18B before the end of 2021, while also enabling a continued commitment to Baa2/BBB or better credit rating and continued dividend growth.

It is expected that, immediately after the closing of the Acquisition, AbbVie Shareholders will own approximately 83% of AbbVie on a fully diluted basis and the Allergan Shareholders will own approximately 17% of AbbVie on a fully diluted basis.

PIPER COMMENTS

Piper Jaffray analyst Christopher Raymond said his first reaction to the deal could be summed up with the phrase “two turkeys don’t make an eagle,” but he is “willing to listen” despite his skepticism about the transaction.

Though he cannot say he is “excited at the prospect of AbbVie entering the field of medical aesthetics,” EPS accretion of 10% in year one and over 20% at peak, and the potential for meaningful deleveraging and cost cutting, has his attention, Raymond said. He keeps a Neutral rating on AbbVie based on his initial reaction to the deal announcement.

Humira is AbbVie’s blockbuster drug, Stockwinners

Wells Fargo

Wells Fargo analyst David #Maris said he views the deal as a good alternative for Allergan versus the current share price, but he is not convinced its a better long term alternative given the eventual biosimilar threat to Abbvie’s blockbuster drug Humira.

With that said, Maris tells investors that “deals at such premiums are rarely killed because of a bad strategic fit or longer -term value outlook in the absence of other bidders.” Though he would not completely rule out an activist investor disrupting the deal, he thinks it is unlikely given there has been a strategic review of the company for some time. Maris, who said he thinks the deal could go through, keeps an Outperform rating on Allergan shares.

Leerink 

SVB Leerink analyst Marc Goodman is not surprise that one of the large pharma companies has made a bid on Allergan (AGN) given the multi-year stock weakness.

Juvederm is another one of Allergan’s top selling products. Stockwinners

However, he believes a $188 price is “too low,” as he “can’t believe that Allergan is not being taken out at least at $200,” which “begs the question” whether this was a process or is AbbVie (ABBV) “opportunistically pursuing a wounded stock.” If it is the latter, Goodman believes this bid could initiate others to pursue Allergan as well. He has an Outperform rating on Allegan’s shares.

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Array BioPharma sold for $11.4 billion

Pfizer to acquire Array BioPharma for $48.00 per share in cash, or $11.4B

Array BioPharma sold for $11.4 billion, Stockwinners

Pfizer (PFE) and Array BioPharma (ARRY) announced that they have entered into a definitive merger agreement under which Pfizer will acquire Array, a commercial stage biopharmaceutical company focused on the discovery, development and commercialization of targeted small molecule medicines to treat cancer and other diseases of high unmet need.

Array BioPharma Inc., a biopharmaceutical company, focuses on the discovery, development, and commercialization of small molecule drugs to treat patients with cancer and other diseases. It provides BRAFTOVITM (encorafenib) capsules in combination with MEKTOVI (binimetinib) tablets for the treatment of patients with unresectable or metastatic melanoma with a BRAF mutation. The company’s lead clinical programs include encorafenib and binimetinib that are investigated in approximately 30 clinical trials for various solid tumor indications, including a Phase III trial in BRAF-mutant colorectal cancer. Its product pipeline also includes ipatasertib, an AKT inhibitor that is in Phase III trial to treat prostate or breast cancers; selumetinib, a MEK inhibitor for cancer; larotrectinib, a PanTrk inhibitor that is in a Phase II/registration clinical trial for cancer; tucatinib, a HER2 inhibitor for breast cancer, which is in Phase II/registration trial; and ARRY-797 that is in Phase III clinical trial for Lamin A/C-related dilated cardiomyopathy. 

Pfizer has agreed to acquire Array for $48 per share in cash, for a total enterprise value of approximately $11.4B.

The Boards of Directors of both companies have approved the merger. Upon the close of the transaction,

Array’s employees will join Pfizer and continue to be located in Cambridge, Massachusetts and Morrisville, North Carolina, as well as Boulder, Colorado, which becomes part of Pfizer’s Oncology Research & Development network in addition to La Jolla, California and Pearl River, New York.

Pfizer expects to finance the majority of the transaction with debt and the balance with existing cash.

The transaction is expected to be dilutive to Pfizer’s Adjusted Diluted EPS by 4c-5c in 2019, 4c-5c in 2020, neutral in 2021, and accretive beginning in 2022, with additional accretion and growth anticipated thereafter.

Pfizer will provide any appropriate updates to its current 2019 guidance in conjunction with its third quarter 2019 earnings release.

Under the terms of the merger agreement, a subsidiary of Pfizer will commence a cash tender offer to purchase all outstanding shares of Array common stock for $48 per share in cash for a total enterprise value of approximately $11.4B.

The closing of the tender offer is subject to customary closing conditions, including regulatory approvals and the tender of a majority of the outstanding shares of Array common stock.

The merger agreement contemplates that Pfizer will acquire any shares of Array that are not tendered into the offer through a second-step merger, which will be completed promptly following the closing of the tender offer.

Pfizer expects to complete the acquisition in the second half of 2019.

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Bempegaldesleukin data should send Nektar shares higher

Nektar presents biomarker, clinical data from PIVOT-02 Phase 2 study

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Nektar presents biomarker, clinical data from PIVOT-02 Phase 2 study, Stockwinners

Nektar Therapeutics (NKTR) announced that biomarker and clinical data from PIVOT-02 was presented at the 2019 American Society of Clinical Oncology Meeting in Chicago, Illinois.

#Bempegaldesleukin is an investigational, CD122-preferential IL-2 pathway agonist designed to provide sustained signaling through the IL-2 beta-gamma receptor.

Bempegaldesleukin is under investigation in clinical trial NCT03729245 (A Study of NKTR-214 in Combination With Nivolumab Compared With the Investigator’s Choice of a Tyrosine Kinase Inhibitor (TKI) Therapy (Either Sunitinib or Cabozantinib Monotherapy) for Advanced Metastatic Renal Cell Carcinoma (RCC)).

PIVOT-02 is an ongoing Phase 2 study evaluating bempeg in combination with nivolumab in solid tumors.

Exploratory biomarker analyses of PIVOT-02 baseline tumor biopsies identified immune signatures that potentially enrich for response in patients with 1L metastatic melanoma and not 1L metastatic urothelial carcinoma.

Notable response rates were seen in both 1L metastatic melanoma and 1L metastatic urothelial cancer patients, regardless of PD-L1 status or unfavorable tumor microenvironments.

At a median time of follow-up of 12.7 months, confirmed objective response rate was 53% in efficacy-evaluable patients, with 34% of patients achieving confirmed complete responses. 42% of patients achieved a maximum reduction of 100% in target lesions. DCR, also known as disease control rate was 74%.

Median time to response was 2 months. Median duration of response was not reached. At the 12.7 month median follow-up, data were too immature to calculate median progression-free survival. 80% of patients with responses have ongoing responses. Amongst the 35 patients with known pre-treatment PD-L1 status, ORR in PD-L1 negative patients was 6/14 and in PD-L1 positive patients was 13/21.

One of three patients with unknown PD-L1 baseline status experienced a CR.

A total of 6/41 of patients experienced a Grade 3 or higher TRAE with 4/41 patients discontinuing treatment due to a TRAE. A total of 41 patients have been treated at the RP2D with 3 patients discontinuing prior to 1st scan due to an unrelated treatment-emergent adverse event and patient decision.

A Phase 3 trial evaluating bempeg in combination with nivolumab versus nivolumab in first-line advanced melanoma patients is currently recruiting patients.

A Phase 2 pivotal trial evaluating bempeg in combination with nivolumab in first-line metastatic urothelial cancer is currently recruiting patients.

 Piper Jaffray

Piper Jaffray analyst Tyler Van Buren reiterates an Overweight rating and $100 price target on Nektar, and believes shares should be up significantly on Monday following updated data from the bempegaldesleukin + nivo PIVOT-02 melanoma cohort.

The increase in complete response rate from 24% to 34% at just beyond 12 months is more than the analyst and investors were anticipating and “the waterfall plot is like nothing [he has] ever seen in solid tumors.”

Ultimately, Van Buren believes the high quality of responses and the ability to maintain patients on therapy is contributing to robust durability, which increases his confidence in the ultimate outcome of the Phase III trial which will have a final mPFS evaluation around Q3 of 2020.

NKTR closed at $31.32.

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Cara Therapeutics shares jump on KALM-1 data

Cara Therapeutics announces topline data from KALM-1 Phase 3 trial

Cara reports positive top-line data from CR845, Stockwinners
Cara reports positive results from the KALM-1 pivotal Phase 3 trial of Korsuva Injection in hemodialysis patients , Stockwinners

Cara Therapeutics (CARA) announced topline data from the KALM-1 pivotal Phase 3 trial of Korsuva Injection in hemodialysis patients with moderate-to-severe chronic kidney disease-associated pruritus, or CKD-aP.

Chronic kidney disease-associated pruritus ( #CKD-aP ) is a distressing, often overlooked condition in patients with CKD and end-stage renal disease. It affects ~40% of patients with end-stage renal disease and has been associated with poor quality of life, poor sleep, depression, and mortality.  Despite being an annoyance, CKD-associated pruritus (CKD-aP) can adversely affect the quality of life (QOL) and medical outcomes. 

The proportion of patients on 0.5 mcg/kg of Korsuva Injection achieving a three-point or greater improvement from baseline in the weekly mean of the daily 24 hour Worst Itching Intensity Numeric Rating Scale, or WI-NRS, score at week 12 was 51% vs. 28% for patients on placebo.

The proportion of patients on 0.5 mcg/kg of Korsuva Injection achieving a four-point or greater improvement from baseline in the weekly mean of the daily 24 hour WI-NRS score at week 12 was 39% vs. 18% for patients on placebo.

Patients on #Korsuva Injection experienced a 43% improvement in the average total Skindex-10 score at week 12 vs. patients on placebo.

Patients on Korsuva Injection experienced a 35% improvement in the average total 5-D Itch score at week 12 vs. patients on placebo.

Korsuva was generally well-tolerated with a safety profile consistent with that seen in earlier Korsuva clinical trials.

Overall, the incidence of adverse events, or AEs, and serious AEs were similar across both Korsuva and placebo groups.

The most common treatment emergent AEs reported in greater than 5% of patients were diarrhea, dizziness, nasopharyngitis and vomiting.

CARA is up 12.5% to $20.21 per share.

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Mustang Bio soars on its gene therapy data

NEJM reports ‘medical breakthrough’ in Mustang Bio cell and gene therapy

Mustang Bio (MBIO) announced that the New England Journal of Medicine has published data from St. Jude Children’s Research Hospital, the nation’s “leading hospital” for understanding, treating and curing childhood cancer and other life-threatening diseases.

Mustang Bio soars on its gene therapy data, Stockwinners

The data comes from a Phase 1/2 clinical trial of a lentiviral gene therapy for the treatment of newly diagnosed infants under two years old with XSCID, also referred to as SCID-X1 and commonly known as “bubble boy disease.”

Under a licensing agreement with St. Jude, Mustang will develop the lentiviral gene therapy for commercial use as MB-107.

The multi-center Phase 1/2 clinical trial is evaluating the safety and efficacy of a lentiviral vector to transfer a normal copy of the IL2RG gene to bone marrow stem cells in newly diagnosed infants under the age of two with XSCID, preceded by low exposure-targeted busulfan conditioning.

A total of 10 infants have received the therapy to date in this clinical trial. Among the data highlights, bone marrow harvest, busulfan conditioning and cell infusion were well tolerated.

In seven of the eight cases, normalization of naive T-cell and natural killer cell numbers occurred within three to four months after treatment, accompanied by vector marking in T, B, NK and myeloid cells and marrow progenitors.

All patients cleared previous infections and are growing normally. Seven of the eight infants treated have developed normal IgM levels to date.

Most patients were discharged from the hospital within one month.

Data Highlights:

  • Bone marrow harvest, busulfan conditioning and cell infusion were well tolerated.
  • In seven of the eight cases, normalization of CD3+, CD4+ and CD4+ naïve T-cell and natural killer (“NK”) cell numbers occurred within three to four months after treatment, accompanied by vector marking in T, B, NK and myeloid cells and marrow progenitors.
    • The eighth infant had insufficient T cells initially, but normalization of T cells occurred following an unconditioned boost of gene-corrected cells, and the patient is progressing favorably.
  • All patients cleared previous infections and are growing normally.
  • Seven of the eight infants treated have developed normal IgM levels to date.
    • Four of these seven infants have discontinued monthly infusions of intravenous immunoglobulin (IVIG) therapy to date.
    • Three of those four infants that discontinued monthly IVIG infusions have responded to vaccines to date.

MBIO closed at $2.66, it last traded at $8.91.

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Intercept falls on NASH study

Intercept falls after releasing ‘supportive data’ from Phase 3 NASH study

Intercept Pharmaceuticals (ICPT) overnight announced additional “supportive data” from its Phase 3 Regenerate study of obeticholic acid in patients with liver fibrosis due to nonalcoholic steatohepatitis.

stockwinners.com ICPT

Intercept falls after releasing ‘supportive data’ from Phase 3 NASH study, Stockwinners

Nonalcoholic steatohepatitis (NASH) is liver inflammation and damage caused by a buildup of fat in the liver. It is part of a group of conditions called nonalcoholic fatty liver disease. You may be told you have a “fatty liver.”

The new data based on additional analyses show that obeticholic acid “demonstrated robust efficacy across a range of additional histologic and biochemical parameters,” Intercept said in a statement.

The data are being presented today at the International Liver Congress in Vienna, Austria.

The primary efficacy analysis assessed efficacy at 18 months in 931 patients with stage 2 or 3 liver fibrosis due to NASH.

Patients with biopsy proven NASH with fibrosis were randomized 1:1:1 to receive placebo, OCA 10 mg or OCA 25 mg once daily.

A repeat biopsy was conducted after 18 months for histologic endpoint assessment.

Overall, study discontinuations in the primary efficacy analysis population were balanced across treatment groups.

As previously reported, in the primary efficacy analysis, once-daily OCA 25 mg met the primary endpoint of fibrosis improvement with no worsening of NASH in 23.1% of patients compared to 11.9% of placebo patients at the planned 18-month interim analysis, the company said.

In the primary efficacy analysis, a numerically greater proportion of patients in both OCA treatment groups compared to placebo achieved the primary endpoint of NASH resolution with no worsening of liver fibrosis; however, this did not reach statistical significance.

As agreed with the FDA, in order for the primary objective to be met, the study was required to achieve one of the two primary endpoints, it added.

Additional supportive efficacy analyses were conducted using the per protocol population.

Approximately three-fold more patients in the OCA 25 mg group achieved an improvement of fibrosis by greater than or equal to 2 stages compared to placebo, Intercept reported.

ICPT is down 12%, or $14.14, to $106.54.

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Sorrento Therapeutics reports positive Parkinson’s disease study, shares rise

Sorrento Therapeutics announces top line results from Parkinson’s disease study


Sorrento Therapeutics announces top line results from Parkinson’s disease study, Stockwinners

Sorrento Therapeutics (SRNE) announced top line results in a discovery study aimed at exploring potential benefits of resiniferatoxin in controlling neuro-inflammatory processes associated with Parkinson’s disease in a rodent model.

Many clinical and pathological studies have shown that the disease extends beyond the substantia nigra and involves non-dopaminergic neurotransmitter systems that mediate both motor and non-motor symptoms.

Several therapeutic strategies have been proposed to treat such symptoms. However, despite the significant morbidity associated with these symptoms, research into and drug development for these problems remain scarce.

Transient receptor potential vanilloid 1, or TRPV1, is involved in pain perception and is highly expressed in sensory neurons. TRPV1 is also present in the brain where it may play a role in modulating neuronal function, controlling motor behavior and regulating neuroinflammation.

TRPV1 receptors are considered an important modulator of basal ganglia functions, and pharmacological changes to cells expressing those receptors might lead to protective therapies in Parkinson-induced motor symptoms.

Intrathecal administration of RTX at 7 days after AAV-A53T resulted in improved motor function analyzed by fine motor kinematic analysis when compared to control mice administered with vehicle for RTX.

No significant adverse effects after RTX infusion were observed. However, no significant effects in dopamine and its metabolites levels in striatum were observed.

The findings demonstrated activity of intrathecal administration of RTX at the early intervention paradigm to rescue motor deficits in AAV-A53T model.

The lack of effect on dopamine levels suggest that behavioral recovery is driven by mechanisms other than direct protection of nigrostriatal dopaminergic neurons, including modulation of neuroinflammation and/or analgesic effect of RTX after IT delivery.

A provisional patent has been filed for non-dopaminergic therapeutic approach with resiniferatoxin for symptomatic relief of motor symptoms associated with Parkinson’s disease.

The full results of the study will be released in publication in a scientific journal and presented at an upcoming neurosciences conference later this year.

Enabling preclinical work will continue with the goal of filing an IND by the end of the year. If results are confirmed, human clinical trials to start soon after. announces top line results from Parkinson’s disease study

Sorrento Therapeutics announced top line results in a discovery study aimed at exploring potential benefits of resiniferatoxin in controlling neuro-inflammatory processes associated with Parkinson’s disease in a rodent model.

Many clinical and pathological studies have shown that the disease extends beyond the substantia nigra and involves non-dopaminergic neurotransmitter systems that mediate both motor and non-motor symptoms.

Several therapeutic strategies have been proposed to treat such symptoms. However, despite the significant morbidity associated with these symptoms, research into and drug development for these problems remain scarce.

Transient receptor potential vanilloid 1, or TRPV1, is involved in pain perception and is highly expressed in sensory neurons.

TRPV1 is also present in the brain where it may play a role in modulating neuronal function, controlling motor behavior and regulating neuroinflammation. TRPV1 receptors are considered an important modulator of basal ganglia functions, and pharmacological changes to cells expressing those receptors might lead to protective therapies in Parkinson-induced motor symptoms.

Intrathecal administration of RTX at 7 days after AAV-A53T resulted in improved motor function analyzed by fine motor kinematic analysis when compared to control mice administered with vehicle for RTX.

No significant adverse effects after RTX infusion were observed. However, no significant effects in dopamine and its metabolites levels in striatum were observed.

The findings demonstrated activity of intrathecal administration of RTX at the early intervention paradigm to rescue motor deficits in AAV-A53T model.

The lack of effect on dopamine levels suggest that behavioral recovery is driven by mechanisms other than direct protection of nigrostriatal dopaminergic neurons, including modulation of neuroinflammation and/or analgesic effect of RTX after IT delivery.

A provisional patent has been filed for non-dopaminergic therapeutic approach with resiniferatoxin for symptomatic relief of motor symptoms associated with Parkinson’s disease. The full results of the study will be released in publication in a scientific journal and presented at an upcoming neurosciences conference later this year.

Enabling preclinical work will continue with the goal of filing an IND by the end of the year. If results are confirmed, human clinical trials to start soon after.

SRNE closed at $4.09.

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Zogenix tumbles on FDA action

Zogenix gets refusal to file letter from FDA on Fintepla NDA


Zogenix gets refusal to file letter from FDA on Fintepla NDA , Stockwinners

Zogenix (ZGNX) announced that it received a Refusal to File, RTF, letter from the U.S. Food and Drug Administration regarding its New Drug Application for FINTEPLA, for the treatment of seizures associated with Dravet syndrome.

Dravet syndrome is a rare, catastrophic, lifelong form of epilepsy that begins in the first year of life with frequent and/or prolonged seizures. Previously known as Severe Myoclonic Epilepsy of Infancy (SMEI), it affects 1:15,700 individuals, 80% of whom have a mutation in their SCN1A gene.

Upon its preliminary review, the FDA determined that the NDA, submitted on February 5, 2019, was not sufficiently complete to permit a substantive review.

In the letter, the FDA cited two reasons for the RTF decision: first, certain non-clinical studies were not submitted to allow assessment of the chronic administration of fenfluramine; and, second, the application contained an incorrect version of a clinical dataset, which prevented the completion of the review process that is necessary to support the filing of the NDA.

The FDA has not requested or recommended additional clinical efficacy or safety studies.

The company will seek immediate guidance, including a Type A meeting with the FDA, to clarify and respond to the issues identified in the RTF letter.

“We remain highly confident in FINTEPLA’s clinical profile demonstrated in the Phase 3 program in Dravet syndrome and are committed to advancing the product candidate as a potential new treatment option for this and other rare and often catastrophic epileptic encephalopathies,” said Stephen J. Farr, Ph.D., President and CEO of Zogenix.

“We are fully committed to working with the FDA as quickly as possible to address the open issues and clarify the path to successfully re-filing our application.”

Zogenix’s Marketing Authorization Application, MAA, for FINTEPLA for the treatment of seizures associated with Dravet syndrome was previously accepted for review by the European Medicines Agency, EMA, and the Company anticipates an approvability decision could be reached by the EMA in the first quarter of 2020.

Piper Jaffray

Piper Jaffray analyst Danielle Brill lowered her price target on Zogenix to $68 after it received a refusal to file letter from FDA on its Fintepla new drug application.

The analyst notes that while the issues mentioned by the FDA sound like a simple fix, if the agency requires the company to conduct new toxicity studies, the re-submission could be delayed another 12-15 months.

In spite of the “discouraging news”, longer term, Brill still keeps her Overweight rating on Zogenix and believes that “the company should be able to leverage existing ICH data with fenfluramine” based on their reported prior FDA interactions, modeling a launch in Q2 of 2020.

ZGNX closed at $51.85, it last traded at $35.00.

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Orthofix reports positive results of its artificial cervical disc

Orthofix announces full two-year outcomes from IDE study of M6 cervical disc

Orthofix reports positive results of its artificial cervical disc, Stockwinners

Orthofix Medical (OFIX) announced the full two-year outcomes from its U.S. Investigational Device Exemption study of the M6-C artificial cervical disc.

Currently, the most common form of surgery for treating cervical degenerative disc disease is an anterior cervical discectomy and fusion (ACDF). More than 200,000 cervical procedures are performed each year to relieve compression on the spinal cord or nerve roots. Spinal fusion surgery creates a solid union between two or more vertebrae to help strengthen the spine and alleviate chronic neck pain. There are several types of spinal fusion surgery, as well as varied instrumentation used to secure the fusion.

The data demonstrates that patients treated with the M6-C artificial cervical disc had significant improvements in neck and arm pain, function and quality of life scores.

Additionally, these patients had a significant difference in the reduction of pain and opioid medications use when compared to anterior cervical discectomy and fusion patients.

At 24 months, patients in the ACDF group who were still using pain medications had a seven times higher rate of opioid use than those in the M6-C disc group.

A prospective, non-randomized, concurrently controlled clinical trial, the M6-C IDE study was conducted at 23 sites in the United States with an average patient age of 44 years.

The study evaluated the safety and effectiveness of the M6-C artificial cervical disc compared to ACDF for the treatment of single level symptomatic cervical radiculopathy with or without cord compression.

The overall success rate for the protocol-specified primary endpoint for the M6-C disc patients was 86.8 percent at 24 months and 79.3 percent in the control group.

This data statistically demonstrates that cervical disc replacement with the M6-C disc is not inferior to treatment with ACDF. Secondary outcomes at 24 months include: Patients who received the M6-C disc demonstrated statistically significant improvement in the Neck Disability Index as measured at week six and months three, six, 12 and 24.

Meaningful clinical improvement was seen in the following pain scores: 91.2 percent of patients who received the M6-C disc reported an improvement in neck pain compared to 77.9 percent in patients who underwent the ACDF procedure.

90.5 percent of the M6-C patients reported improvement in arm pain scores compared to 79.9 percent in ACDF patients. Prior to surgery, 80.6 percent of the M6-C disc patients and 85.7 percent of the ACDF patients were taking some type of pain medication for the treatment of their cervical spine condition. At 24 months, the rate of M6-C patients who were still taking some type of pain medication dropped to 14.0 percent compared to 38.2 percent of the ACDF patients.

Of these, there was a seven times higher rate of opioid use with the ACDF patients than with patients who received the M6-C disc.

There was a statistically significant difference in the average mean surgery time – 74.5 minutes for patients receiving the M6-C disc versus 120.2 minutes for those patients having the ACDF procedure.

In addition, there was a statistically significant difference in the mean length of hospital stay – 0.61 days for the M6-C patients versus 1.10 days for ACDF patients. Subsequent surgery at the treated level was needed in 4.8 percent of the ACDF patients compared to 1.9 percent of the M6-C disc patients.

There were no device migrations reported in the study. Overall patients receiving the M6-C disc reported a 92-percent satisfaction rate with the surgery, and 93 percent said they would have the surgery again.

There were 3.8 percent serious adverse events related to the device or procedure in the M6-C disc group versus 6.1 percent in the ACDF group.

The M6-C disc received FDA approval in February 2019 based on the results of this study.

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