January 30, 2012

Dynport Vaccine Company issued results from a phase II trial of their recombinant botulinum vaccine candidate, rBV A/B. This double-blind, placebo-controlled trial enrolled 440 healthy adults who received two dosing schedules of the vaccine or placebo administered intramuscularly at 0, 28 and 182 days or 0, 56 and 182 days. Safety and immunogenicity were evaluated for an observation period of 18 months. The vaccine was safe and well-tolerated, and elicited a strong immune response. The maximum immune responses to both botulism neurotoxin serotypes were observed 28 days after the third dose. More than 94% of the subjects maintained detectable neutralizing antibody concentrations for at least one year after the third dose. The 0, 28 and 182-day schedule produced the strongest immune response and was selected for in an upcoming phase III trial.

May 18, 2009

PharmAthene released positive results from a phase II trial of SparVax, a vaccine for the pre- and post-exposure of anthrax infection. This randomized, single blind, positive controlled trial enrolled 226 healthy subjects in the US. The subjects were randomized to receive either three doses of BioThrax (standard of care) 0.5 mL on Days 0, 14, and 28 or SparVax 50 Micrograms on Days 0, 28 and 56 or SparVax 100 Micrograms on Days 0, 28 and 56, both followed by an antigenic challenge dose. At day 182, subjects receiving SparVax were re-randomized to a challenge at the original dose, on either day 182 or 364. The antigenic challenge dose (50 or 100 Microgram) each subject received was the same as the dose assigned to that subject during the initial vaccination schedule. Both vaccines were immunogenic following the 3-dose series with response rates of approximately 90%. There were no significant differences in either TNA or ELISA geometric mean titers (GMT) between the vaccine groups 14 days after the third vaccination. A strong immunologic memory response was observed at both 6 months and 12 months and no differences were seen between SparVax groups in terms of response rate or GMT. Treatment was safe and well tolerated and generally comparable between the treatment arms. However, there were more reports of injection site pain in the BioThrax group as compared to the SparVax groups at both dose levels.

March 2, 2009

Optimer reported positive results from a phase III trial of Prulifloxacin for the treatment of bacterial gastroenteritis (travelers diarrhea). This randomized, double-blind, placebo-controlled study, dubbed OPT-099-002, enrolled 373 adult subjects who were traveling through India, Guatemala or Mexico and diagnosed with bacterial gastroenteritis. The subjects received either 600mg of oral Prulifloxacin or placebo, once daily over three days. The primary efficacy endpoint was Time to the Last Unformed Stool, defined as the time in hours from the first dose of study medication to the passage of the last unformed stool (TLUS). Prulifloxacin was statistically superior to placebo in TLUS in both the modified intent-to-treat population (n≡200) and microbiologically evaluable population (n≡173). The median TLUS for the Prulifloxacin treatment arm was 32.8 hours; (p-value of <0.0001 versus placebo). Prulifloxacin was generally well tolerated and had a similar safety profile compared to placebo.

November 3, 2008

Novartis reported positive results from two clinical trials of canakinumab for the treatment of Cryopyrin-Associated Periodic Syndromes (CAPS) and Systemic Juvenile Idiopathic Arthritis (SJIA). The CAPS study was a six month trial in subjects aged nine to 74 years old and was divided into three parts. In the first part, lasting two months, 35 subjects received a single dose of canakinumab by subcutaneous injection. All but one subject showed a rapid and long-lasting clinical and biochemical response. Part two was a randomized six-month, double-blind, placebo-controlled withdrawal design study in 31 subjects who maintained their response. The subjects were treated with canakinumab or placebo every two months. This portion of the study included the primary endpoint, a comparison between the number of subjects treated with canakinumab who experienced disease outbreaks versus those on placebo. Results showed that no subjects in the canakinumab group experienced a disease flare compared to 81% in the placebo group (p<0.001). Markers of inflammation (C-reactive protein and serum amyloid A) were normalized in patients treated with canakinumab, but increased significantly for those on placebo. Part three of the study is underway in subjects who had relapsed. The SJIA study was an open label, repeat dose trial in 26 pediatric subjects with active SJIA. Results showed substantial clinical improvement (measured by the pediatric ACR50 scale) was reached within 15 days. In addition, four subjects achieved complete remission of the disease. Based on the results Novartis plans to continue with the development of canakinumab

October 6, 2008

Crucell and Sanofi Pasteur reported positive preliminary results from a phase II trial of CL-184, a vaccine for the treatment of rabies. This randomized, single-blind, controlled study enrolled 140 healthy subjects in the US. The subjects received CL-184, a currently marketed human rabies immune globulin (HRIG) or placebo plus rabies vaccine. The neutralizing activity against the rabies virus was comparable to that provided by the HRIG. By day 14, all subjects administered the monoclonal antibodies combination together with the rabies vaccine reached the level of 0.5 IU/ml, a neutralizing activity levels thought to provide protection. The development of the immune response to the rabies vaccine was comparable in the monoclonal antibody combination and the human rabies immune globulin groups. The vaccine was well tolerated; there were no serious adverse events reported and most adverse events were mild or moderate in severity.

December 3, 2007

NovaBay released positive results from a phase I trial of AgaNase for the treatment of bacterial and viral infections. This double-blind placebo controlled study enrolled ninety-six subjects. The subjects received repeated applications of AgaNase, using various concentrations (0.1% and 0.3%) and regimens, applied by spray or swab to the anterior nares. Treatment was well tolerated, with any adverse events local, mild, and transient. The incidence of adverse events did not increase with higher dose. There was no detectable systemic exposure of AgaNase or its major metabolite. Based on the results NovaBay plans to move forward with the development of AgaNase.

November 5, 2007

Life Cycle Pharma issued positive interim results from a phase II trial of LCP-Tacro, an immunosuppressant for the prevention of organ transplant rejection in kidney transplant recipients. This three sequence, open-label, multi-center, prospective, conversion study has enrolled 10 stable kidney transplant recipients to date. Once enrolled, the subjects received Prograf, an approved therapy, for seven days. Following a 24-hour pharmacokinetic study on Day seven to determine pharmacokinetics for Prograf, all subjects were converted to once daily LCP-Tacro for seven days with no dose changes allowed. On Day fourteen and Day twenty-one, a 24-hour LCP-Tacro pharmacokinetic study was to be performed. On Day twenty-two, subjects were to be converted back to their original twice daily dose of Prograf for a safety follow-up period of thirty days ending with a safety assessment on Day fifty-two. Interim data showed LCP-Tacro demonstrated a superior profile when compared to Prograf, including better pharmacokinetics, once-a-day tablet formulation and higher bioavailability. Based on the results, the company remains on track to continue with the enrollment of subjects and plans to initiate phase III trials in 2008.

October 15, 2007

Emergent BioSolutions issued positive preliminary results from a phase II trial of their oral typhoid vaccine. This randomized, blinded, placebo-controlled, single-dose trial enrolled 151 pediatric subjects between the ages of 5 and 14 years in Vietnam. The vaccine was immunogenic, with an overall immune response rate of greater than 50%. In addition, the vaccine induced significantly higher antibody concentrations in the vaccine group compared to the placebo control group. Treatment was well tolerated with no reported serious adverse events and an adverse events profile similar to placebo. Based on the results Emergent BioSolutions plans to move forward with the development of their oral typhoid vaccine.

April 30, 2007

Iomai announced positive results from a phase II trial of the TIM ETEC vaccine patch for the treatment of traveler's diarrhea. This dose-ranging trial enrolled 400 subjects who received four different doses of the vaccine (7.5 micrograms, 22.5 micrograms, 37.5 micrograms and 50 micrograms) or placebo. IgG antibody levels were checked at 21 and 42 days. Results revealed that the patch elicited an immune response in 95% of the subjects tested, at all dose levels. Iomai plans to initiate phase III trials later in 2007.

November 20, 2006

Osiris announced positive results from a phase II trial of Prochymal for the treatment of Graft vs. Host disease (GVHD). This trial enrolled 32 subjects who were divided into two groups. In addition to standard of care, subjects received two infusions of Prochymal, a low dose (2 million cells per kilogram) or a high dose (8 million cells per kilogram) three days apart, at the onset of moderate to severe (grades II-IV) GVHD. Treatment was well tolerated at both dose levels and through repeated dosing. No serious adverse events were reported. Complete response was seen in 74% of the subjects and the overall response rate was 94%. In the sub-population of subjects with gastrointestinal GVHD, 67% had a complete response and the overall response rate was 89%. In the sub-population of the subjects with the dermatological form of GVHD, 85% had a complete response and the overall response rate was 100%. Osiris is currently evaluating Prochymal in a phase III trial for this indication.

July 25, 2005

Human Genome Sciences reported positive results of a phase I study of ABthrax, for the prevention of Anthrax infections. Primary safety endpoints were met, with no serious or dose-limiting adverse events reported. Overall adverse events were generally mild and transient, with no statistical difference in the rate of events between ABthrax and placebo. Pharmacokinetic data indicated good bioavailability, and a mean elimination half-life of 15-19 days. Biological activity was dose-dependent, and serum concentrations achieved levels predicted to be protective against anthrax infection. This randomized, single-blind, placebo-controlled, dose- escalation study enrolled 105 healthy subjects, who received one of three doses of ABthrax via intramuscular injection (0.3 mg/kg, 1.0 mg/kg and 3.0 mg/kg), one of five doses of the drug via intravenous infusion (1.0 mg/kg, 3.0 mg/kg, 10.0 mg/kg, 20.0 mg/kg and 40.0 mg/kg), or placebo.

October 18, 2004

Immtech International has reported positive results of a phase I dose-escalation trial of DB289, for the treatment of malaria. Study data met their primary pharmacokinetic endpoint, with all dosing regimens tested reaching plasma levels considered to be within the therapeutic range for the treatment of malaria. This open-label, multi-ethnic study randomized 75 healthy volunteers to receive one of three doses of the drug (200 mg, 400 mg or 600 mg) either once or twice daily for three days. Dosing information from these results has been incorporated into Immtech’s upcoming phase II standard-care-combination study in Thailand.

July 12, 2004

Isotechnika announced successful completion of a single ascending dose trial of their immunosuppressive drug ISA247. Trial data demonstrated that the trans- isomer of ISA247 may have a preferable safety and efficacy profile, compared to other therapies. The trial was designed to compare the safety, tolerability and pharmacokinetics of a pure-trans- formulation of ISA247 with the original mixed cis-/trans- formulation and currently approved therapy (cyclosporine) in healthy volunteers. Trans-ISA247 was found to be 1.5 times more potent than the mixed formulation, 5 times more potent than cyclosporine, demonstrate an improved nephrotoxicity profile over both, and had a more predictable correlation between plasma drug concentration and immunosuppressive effect profile. Isotechnika announced that trans-ISA247 would be used in all future trials.

October 20, 2003

Schering-Plough reported positive results from a case series investigating Noxafil (posaconazole oral suspension), an antifungal agent for the treatment of coccidioidomycosis. Results showed that all subjects initially received benefit from the drug, and five were long-term successes during the follow-up period. Data showed that Noxafil produced rapid and significant clinical improvements, often with initial responses within one month. Noxafil appeared to be well tolerated and most reported adverse events were mild to moderate. The multicenter study enrolled six subjects and was designed to test the efficacy and safety of Noxafil, after standard antifungal therapies have failed. Results were presented at the 41st annual meeting of the Infectious Diseases Society of America in San Diego.

September 29, 2003

Schering-Plough reported positive results from 20 separate trials with Noxafil (posaconazole suspension), an oral anti-fungal agent for the treatment of life-threatening fungal infections. Results from two open-label phase III studies demonstrated that 71% of subjects (17 of 24) with zygomycosis achieved successful clinical outcomes when given salvage therapy with Noxafil. Results from a dose-ranging pharmacokinetic study enrolling 98 subjects showed successful clinical outcomes in 77% of subjects with febrile neutropenia and 43 % of subjects with invasive fungal infections refractory to standard therapy. Data showed that optimal exposure was attained when Noxafil 800 mg/d was given as a 200 mg dose four times daily, followed by a 400 mg dose twice daily as treatment progressed. Results were presented September 2003 at the 43rd Annual Interscience Conference on Antimicrobial Agents and Chemotherapy.

September 2, 2003

Immtech reported positive results from the first stage in a phase IIa trial investigating DB289 for the treatment of malaria. Results showed that on average the malaria parasite was eliminated from the blood in approximately 95% of the subjects in less than 3 days. Subjects tested remained completely parasite free 28 days after treatment. In addition, data also indicated that the fever associated with malaria was on average eliminated within 16 hours for the same group of subjects. Data demonstrated that DB289 was safe to administer and was efficacious in the subjects tested. Treatment was well tolerated and no adverse effects were reported with DB289. The trial is being conducted at a hospital clinic in Thailand.

August 4, 2003

AEterna reported positive results from a phase III trial investigating Impavido (miltefosine), an alkyphospholipid for the treatment of cutaneous Leishmaniasis. Results showed that subjects taking Impavido had a 220% better cure rate compared with those in the placebo group. Data showed an average cure rate of 70% after treatment. Cure from the disease was assessed six months after the end of treatment; all skin lesions had to be healed at that time and no new skin lesions were allowed to appear. The treatment was well tolerated with the side effects of Impavido being limited to short episodes of vomiting or diarrhea. The randomized, double blind, placebo-controlled study enrolled 133 subjects in Colombia and Guatemala. Leishmaniasis is a tropical disease caused by the Leishmania parasite.

June 9, 2003

Fujisawa Healthcare reported positive results from a post-marketing trial investigating Prograf in combination with Mycophenolate Mofetil (MMF) in pancreas-kidney transplant patients. Results show that subjects receiving induction therapy reported significantly higher kidney survival rate, (92.0%, vs. 81.6%) than those receiving no induction therapy. There was no difference in the pancreas survival rate between the two groups. The 3 year, randomized, multicenter study enrolled 174 subjects and was conducted to assess the effect of antibody induction in SPK transplantation patients receiving maintenance immunosuppression with Prograf, MMF and a steroid taper. Half the subjects received induction therapy and half did not. Results were presented at the 2003 American Transplant Congress.

December 16, 2002

SuperGen reported positive results from more than 12 trials investigating Nipent for the treatment of multiple cancers. A phase II trial using Nipent in combination with Rituximab for the treatment of chronic lymphocytic leukemia (CLL) demonstrated that 33% of subjects achieved an objective response. Stable disease was observed in 54% of subjects with a median response of 9.8 months. A phase I trial using Nipent for the treatment of steroid refractory acute graft-versus-host disease enrolled 23 subjects with a variety of hematological malignancies. Results showed 11 subjects achieved complete responses and 6 achieved partial or mixed responses. A long term follow-up study of Nipent in 180 subjects with hairy cell leukemia demonstrated a response rate of 98%. Data showed that 83% of subjects achieved a complete response and 15% achieved a partial response.

November 18, 2002

Immtech International reported positive preliminary results from a phase IIa trial investigating DB289, a dicationic compound for the treatment of Trypanosomiasis (African sleeping sickness). Results showed that DB289 was well tolerated and cured approximately 95% of the subjects treated, although 5% of the data was not confirmed due to political unrest in the region. Trial participants were requested to attend a follow-up exam after three months; all the subjects who returned were shown to be parasite free. The trial is part of a program conducted in sub-Sahara Africa in collaboration with UNC-Chapel Hill.

April 15, 2002

Study results suggest that Novartis' Certican (everolimus) significantly reduces the incidence of acute rejection episodes and the potential for graft vasculopathy in heart transplant recipients, compared to add-on therapy with azathioprine. The 12-month, double-blind, international trial included 634 heart transplant subjects who were receiving the standard regimen of Neoral (cyclosporine) and steroids. Subjects were randomized to also receive Certican 1.5mg/day, Certican 3.0mg/day or azathioprine 1-3mg/kg/day. The acute rejection rate was 30.6% in subjects receiving Certican 1.5mg/day and 21.3% in those receiving Certican 3.0mg/day, compared to 45.8% with azathioprine 1-3mg/kg/day. Additionally, allograft vasculopathy was lower in the Certican 1.5mg and 3.0mg groups (35.7% and 30.4%), compared to subjects receiving azathioprine (52.8%).

February 11, 2002

Positive late-stage trial results were reported for Sequella's Transdermal Patch for Active Tuberculosis (TB), a diagnostic that appears to distinguish between active infectious TB, latent TB and prior TB vaccination. In the trial, the Transdermal Patch demonstrated a sensitivity of 88%, an efficacy of 93% and a specificity of 100%. Additionally, 43 out of 49 subjects with active TB had a positive reaction to the TB patch, and there were no false positives in the control population. By comparison, all control and active TB subjects had positive Mantoux skin test results.

February 4, 2002

An application for a new indication of Wyeth-Ayerst's Rapamune (sirolimus) was not recommended for approval by the Immunosuppressive Drugs Subcommittee of the FDA Antiviral Drugs Advisory Committee. The immunosuppressant agent is already approved for the prophylaxis of organ rejection in patients receiving kidney transplants. It is currently recommended that Rapamune be used in a regimen concomitantly with cyclosporine and corticosteroids. The committee's decision pertained to a new Rapamune maintenance regimen that would allow physicians to eliminate cyclosporine two to four months after transplantation. The company plans to meet with the FDA to discuss the review.

January 21, 2002

Positive results were reported from a phase Ia trial of XTL-002, a fully human high-affinity monoclonal antibody for the treatment of hepatitis C virus (HCV) infections. The single-center trial was designed to evaluate a single dose of XTL-002 in chronic HCV subjects. Subjects were divided into five groups, with each group receiving 0.25, 1.0, 2.5, 10 or 40 mg of XTL-002 in a single intravenous infusion. Results showed a significant reduction of HCV viral RNA, ranging from 2 to 100 fold, in eight out of 15 subjects. XTL-002 is being developed by XTL Biopharmaceuticals.

January 7, 2002

Phase I/II trial results indicate that Novirio Pharmaceuticals' LdT produced a reduction of hepatitis B virus replication at all dose levels, and a median reduction of 3.63 log10 among subjects taking the highest dose evaluated to date (400 mg once-daily for four weeks). The randomized 30-subject trial was designed to evaluate the safety and short-term antiviral activity of LdT in adults with chronic hepatitis B, as well as assess optimal dosing levels for future trials. Results suggested greater than 1,000-fold reductions in serum virus load for subjects receiving daily LdT doses of 100 mg or more. Based on the increased antiviral activity observed with increasing LdT doses, along with positive safety results, the company is adding an additional subject cohort to the trial to evaluate higher dose levels.

December 17, 2001

Phase II trial results show that SuperGen's Nipent (pentostatin) produced a 60% overall response rate in subjects with refractory chronic graft versus host disease (GvHD). The trial consisted of 10 evaluable subjects who had failed at least one prior immunosuppressive regimen. Subjects received Nipent every two weeks for three months, and at the end of this period those who had achieved either stable disease or who had shown improvement were weaned off other medications. Five subjects experienced a complete response and one had a partial response, producing an overall response rate of 60%.

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