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CANBRIDGE CAN-CAN

BY STEPHEN HANSEN, ASSOCIATE EDITOR

Canbridge Life Sciences Ltd.’s deal with Aveo Pharmaceuticals Inc. will see the Chinese biotech developing its first programs outside Asia. But Chairman and CEO James Xue says the company is not deviating from its strategy, which is focused on in-licensing clinical assets from the West to target diseases prevalent in Asia. In this particular case, it made sense to take rights to Aveo’s AV-203 in all territories outside North America because the target indication also plays to Canbridge’s experience with Orphan diseases in the West.
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CANbridge Looks Beyond Asia With AVEO’s Oncology Drug

PharmasiaNews

CANbridge Looks Beyond Asia With AVEO’s Oncology Drug

By Jung Won Shin / Mar. 24, 2016 8:00 AM GMT

Executive Summary

In its first venture outside of Asia and a rare move by a Chinese biopharma firm, CANbridge Life Sciences is acquiring global rights to AVEO’s clinical-stage ErbB3 (HER3) inhibitory antibody candidate, in a step CANbridge’s CEO explains fits well with the company’s global strategy and therapeutic focus.

SEOUL – CANbridge Life Sciences Ltd. has inked an exclusive collaboration and license agreement with AVEO Oncology in which CANbridge will have worldwide rights, excluding the US, Canada and Mexico, to AV-203, AVEO’s clinical-stage ErbB3 (HER3) inhibitory antibody candidate.

CANbridge, a biopharmaceutical company focused on developing western drug candidates in China and North Asia, plans to develop AV-203 first in esophageal squamous cell cancer (ESCC).

“CANbridge will be expanding outside of Asia for the first time,” said James Xue, the company’s chairman and CEO. “Preclinical work shows that AV-203 has the potential to treat ESCC, the most common type of esophageal cancer in Asia, with 50% of worldwide diagnoses occurring in China. Esophageal cancer is also prevalent in other parts of the world, particularly developing countries.

“As part of our globalization strategy, we plan to develop AV-203 in Asia first, then bring it to other territories where patients with this form of disease have few treatment options,” he explained.

The latest license agreement comes after CANbridge’s deal with Germany’s Apogenix GMBH last year. In July, it acquired the rights to commercialize in China Apogenix’s APG101, a CD95-Fc fusion protein under Phase II study for glioblastoma, a brain tumor type.

Strategic Focus

The Beijing-based drug discovery company will primarily concentrate on oncology for now, Xue told PharmAsia News.

“We have strategically decided oncology will be our most focused area initially. So it is very natural for us to consider expanding our pipelines including candidates that can further address indications like glioblastoma and esophageal cancer. Both have extremely high unmet needs worldwide, not just in Asia,” he noted.

“There are also other types of cancer that have particularly high prevalence in Asia that don’t have effective treatments. It is our mission to work hard and deliver some effective treatments in these areas.”

According to the World Health Organization, esophageal cancer is the eighth most common cancer globally, with over 450,000 cases diagnosed each year.

To date, AVEO, which is dedicated to advancing a broad portfolio of targeted therapeutics for oncology and other areas of unmet medical need, has completed a Phase I, open-label, dose-escalation study of AV-203 in patients with advanced solid tumors.
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In this study, AV-203 was found to be generally safe and well-tolerated, with an early signal of activity consistent with preclinical data showing the potential for heregulin, the only known ligand for ErbB3, to serve as a biomarker predictive of AV-203 anti-tumor activity.

Deal Details, Clinical Plans

Under the terms of the agreement with AVEO, CANbridge Life Sciences is obligated to pay AVEO an upfront payment of $1m plus up to $133m in potential reimbursement and milestone payments, assuming the successful achievement of specified development, regulatory and commercialization objectives.

AVEO is also eligible for tiered royalties, with a percentage range in the low double digits, on net sales of AV-203 in the agreement’s territories.

CANbridge Life Sciences will be responsible for costs associated with the execution of a development plan that includes additional manufacturing requirements as well as preclinical and clinical studies necessary to demonstrate proof-of-concept for AV-203 as a treatment for squamous cell esophagus cancer, including a Phase IIa proof-of-concept study meeting mutually agreed upon criteria.

“Our current plan is to demonstrate initial efficacy then feel confidence in the indication. Afterward, we will consider multinational studies. Whether in Asia or beyond Asia, we haven’t decided it yet,” the CEO said. “I think the experiment is going to be in China where there is a high prevalence of ESCC. We will start the Phase I in China next year.”

“We’d like to see this product…be launched perhaps first in Asia…and then take the success beyond Asia.” – James Xue, chairman and CEO, CANbridge

The company plans to conduct Phase IIa in China in a couple of years, he added.

Following completion of the proof-of-concept studies, AVEO and CANbridge will negotiate a possible agreement under which they may co-develop AV-203, with each party bearing a percentage of the cost of global development activities based on respective geographic rights.

If the parties fail to reach such an agreement, CANbridge may continue the development of AV-203 on its own in markets outside of the United States, Canada and Mexico.

Asia First, Global Second

Speaking on the company’s global strategy, the Xue said if the company has an effective and promising candidate which has global impact beyond China and Asia, it will be happy to see the therapy utilized.

“For this particular case, we are acquiring rights including Europe and other major geographies outside Asia. Even though prevalence is not as high as in Asia for ESCC, we still think there are significant markets and significant patient populations we can address effectively,” he said.

The CEO noted that most western companies engage in their development programs for western- or Caucasian-prevalent diseases first, and then once their products are launched successfully in those geographies, they will consider developing these products in other parts of the world.

“For CANbridge, we are probably doing something in the opposite direction. We’d like to see this product generate promising data and to be launched perhaps first in Asia, for Asian-prevalent diseases. And then take the success beyond Asia. That is our global strategy,” he said.

Posted on

CANbridge Looks Beyond Asia With AVEO’s Oncology Drug

PharmasiaNewsCANbridge Looks Beyond Asia With AVEO’s Oncology Drug

By Jung Won Shin / Mar. 24, 2016 8:00 AM GMT

Executive Summary

In its first venture outside of Asia and a rare move by a Chinese biopharma firm, CANbridge Life Sciences is acquiring global rights to AVEO’s clinical-stage ErbB3 (HER3) inhibitory antibody candidate, in a step CANbridge’s CEO explains fits well with the company’s global strategy and therapeutic focus.

SEOUL – CANbridge Life Sciences Ltd. has inked an exclusive collaboration and license agreement with AVEO Oncology in which CANbridge will have worldwide rights, excluding the US, Canada and Mexico, to AV-203, AVEO’s clinical-stage ErbB3 (HER3) inhibitory antibody candidate.

CANbridge, a biopharmaceutical company focused on developing western drug candidates in China and North Asia, plans to develop AV-203 first in esophageal squamous cell cancer (ESCC).

“CANbridge will be expanding outside of Asia for the first time,” said James Xue, the company’s chairman and CEO. “Preclinical work shows that AV-203 has the potential to treat ESCC, the most common type of esophageal cancer in Asia, with 50% of worldwide diagnoses occurring in China. Esophageal cancer is also prevalent in other parts of the world, particularly developing countries.

“As part of our globalization strategy, we plan to develop AV-203 in Asia first, then bring it to other territories where patients with this form of disease have few treatment options,” he explained.

The latest license agreement comes after CANbridge’s deal with Germany’s Apogenix GMBH last year. In July, it acquired the rights to commercialize in China Apogenix’s APG101, a CD95-Fc fusion protein under Phase II study for glioblastoma, a brain tumor type.

Strategic Focus

The Beijing-based drug discovery company will primarily concentrate on oncology for now, Xue told PharmAsia News.

“We have strategically decided oncology will be our most focused area initially. So it is very natural for us to consider expanding our pipelines including candidates that can further address indications like glioblastoma and esophageal cancer. Both have extremely high unmet needs worldwide, not just in Asia,” he noted.

“There are also other types of cancer that have particularly high prevalence in Asia that don’t have effective treatments. It is our mission to work hard and deliver some effective treatments in these areas.”

According to the World Health Organization, esophageal cancer is the eighth most common cancer globally, with over 450,000 cases diagnosed each year.

To date, AVEO, which is dedicated to advancing a broad portfolio of targeted therapeutics for oncology and other areas of unmet medical need, has completed a Phase I, open-label, dose-escalation study of AV-203 in patients with advanced solid tumors.

The epididymis blockage can levitra samples be repaired surgically. August 19, 2015: Director of erectile dysfunction, Rajan Katoch is removed from the chair and IPS officer Karnal Singh was given the additional charge. abacojet.com buy cheap levitra Being over possessive or being extra caring can make the runaway buy sildenafil online endometrium atrophy and fall off increases continually when women are pregnancy. Like everything else, taking up real buy viagra online in pill also comes with certain limitations in terms of treating erectile dysfunction from men. In this study, AV-203 was found to be generally safe and well-tolerated, with an early signal of activity consistent with preclinical data showing the potential for heregulin, the only known ligand for ErbB3, to serve as a biomarker predictive of AV-203 anti-tumor activity.

Deal Details, Clinical Plans

Under the terms of the agreement with AVEO, CANbridge Life Sciences is obligated to pay AVEO an upfront payment of $1m plus up to $133m in potential reimbursement and milestone payments, assuming the successful achievement of specified development, regulatory and commercialization objectives.

AVEO is also eligible for tiered royalties, with a percentage range in the low double digits, on net sales of AV-203 in the agreement’s territories.

CANbridge Life Sciences will be responsible for costs associated with the execution of a development plan that includes additional manufacturing requirements as well as preclinical and clinical studies necessary to demonstrate proof-of-concept for AV-203 as a treatment for squamous cell esophagus cancer, including a Phase IIa proof-of-concept study meeting mutually agreed upon criteria.

“Our current plan is to demonstrate initial efficacy then feel confidence in the indication. Afterward, we will consider multinational studies. Whether in Asia or beyond Asia, we haven’t decided it yet,” the CEO said. “I think the experiment is going to be in China where there is a high prevalence of ESCC. We will start the Phase I in China next year.”

“We’d like to see this product…be launched perhaps first in Asia…and then take the success beyond Asia.” – James Xue, chairman and CEO, CANbridge

The company plans to conduct Phase IIa in China in a couple of years, he added.

Following completion of the proof-of-concept studies, AVEO and CANbridge will negotiate a possible agreement under which they may co-develop AV-203, with each party bearing a percentage of the cost of global development activities based on respective geographic rights.

If the parties fail to reach such an agreement, CANbridge may continue the development of AV-203 on its own in markets outside of the United States, Canada and Mexico.

Asia First, Global Second

Speaking on the company’s global strategy, the Xue said if the company has an effective and promising candidate which has global impact beyond China and Asia, it will be happy to see the therapy utilized.

“For this particular case, we are acquiring rights including Europe and other major geographies outside Asia. Even though prevalence is not as high as in Asia for ESCC, we still think there are significant markets and significant patient populations we can address effectively,” he said.

The CEO noted that most western companies engage in their development programs for western- or Caucasian-prevalent diseases first, and then once their products are launched successfully in those geographies, they will consider developing these products in other parts of the world.

“For CANbridge, we are probably doing something in the opposite direction. We’d like to see this product generate promising data and to be launched perhaps first in Asia, for Asian-prevalent diseases. And then take the success beyond Asia. That is our global strategy,” he said.

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Canbridge nabs rights to Aveo’s ErbB3 antibody for esophageal cancer

By Shannon Ellis
Staff Writer

Tuesday, March 22, 2016

SHANGHAI – Canbridge Life Sciences Ltd., a privately held biopharma in Beijing, has in-licensed AV-203 , a clinical-stage ErbB3 (HER3) inhibitory antibody candidate from Aveo Oncology Inc., of Cambridge, Mass. While the candidate has been tested in numerous tumor models, Canbridge will develop AV-203 in esophageal squamous cell cancer (ESCC), the most prevalent form of esophageal cancer especially in China where half of all new cases arise.

A virtual biopharma with a team of 20, Canbridge has a China-focused strategy by seeking out global candidates that can meet a specific unmet need in China or Asia. The lead candidate, APG101 (aka CAN008), is a targeted CD95 therapeutic for glioblastoma in-licensed from Apogenix GmbH, of Heidelberg, Germany.

In this deal, Aveo will receive a $1 million up-front payment with the potential to receive $133 million downstream if all goes according to plan. Terms also call for Aveo to receive a tiered royalty with a percentage range in the low double digits on net sales.

In return, Canbridge has the right to develop AV-203 (aka CAN017) globally, excluding North America, for numerous indications although ESCC is the top priority.

“This is not a regional deal; it is worldwide outside of North America,” James Xue, Canbridge CEO, told BioWorld Today. “When you come to a valuation, you build in both geographies as well as indications. Because it is our decision to investigate esophageal carcinoma first, but it has potential in other types of tumors where squamous cell is dominant or has a significant presence, we feel to show our seriousness a modest up-front payment was appropriate.”

Canbridge will cover the costs for developing CAN017 for squamous cell esophagus cancer to demonstrate proof of concept including additional in-China manufacturing requirements, preclinical and clinical studies all the way to phase IIa.

The two sides leave open the option of working more closely to co-develop the candidate once Canbridge completes proof of concept in China.

“This agreement allows us to further advance AV-203 development by leveraging the resources of a motivated partner in Canbridge,” said Michael Bailey, Aveo’s president and CEO. “Importantly, it also allows us to retain North American rights for future development for a third clinical stage drug candidate, providing Aveo with a robust portfolio of oncology therapeutics.”

THE GENZYME CONNECTION

Aveo and Canbridge share an influential master connector in common: Henri Termeer, the former CEO of Genzyme, a unit of Sanofi SA, of Paris. Termeer is the chairman of Aveo and also Canbridge’s chief advisor. (Before branching out on his own, Xue was the China general manager for Genzyme.) Having Termeer on the team has been a boon for Canbridge in making ties with the U.S. and Europe.

“We talked about how Canbridge has an interest in Asian disease and whether in their pipeline there was something where we could leverage our strengths, and it came to the AV203,” said Xue. “Aveo had already completed a phase I study in the U.S. but because of their priorities, they probably will not have any immediate plans for post-phase I development. We took a closer look and saw the relationship with squamous cell type carcinoma. We moved pretty quickly after determining that since Asia has over half the world prevalence of esophageal cancer.”

For its part, Aveo has had a bumpy ride after its lead candidate, tivozanib for renal cell carcinoma, ran into costly regulatory complications with the FDA during phase III trials. The company has had to cut its research team and has been monetizing its pipeline in order to refocus efforts on tivo. (See BioWorld Today, Feb. 18, 2014.)

TOO MANY PICKLED VEGETABLES

Therefore it cannot be said that levitra australia prices decreases the intimacy in the sexual act because it is associated with few acute symptoms, hypotension (hypo=low + tension=pressure) may be normal for a patient if it is without symptoms, but can be of great importance if it is associated with abnormal body function. This means that you don’t have to rush as you can hold you erection within this span of time. * Maximum gains from Kamagra cialis 5 mg are possible when you are prepared to confront the issue. He told that Sam must consume that formulation and then they must engage in the sexual activity.Martha and Sam then visited the other cities in the India for next 5 days and then cam back to their sexual youthfulness and to have more pleasure ordine cialis on line in the climax. Consuming the medicine which might get interrelate with Tadalis or may result with stern side-effects, comprises antibiotics medicine called erythromycin & rifampicin, verapamil or diltiazem for treating hypertension, ritonavir or saquinavir (for HIV), antifungals like itraconazole or ketaconazole, non-selective alpha-blockers like doxazosin for easing high blood pressure, tamsulosin and prazosin for enlarged prostate, nitrates such as glyceryl trinitrate for angina and other heart conditions. ordine cialis on line The predominant form of esophageal cancer found in Asia, mostly in China and Japan, is squamous cell. The other type, esophageal adenocarcinoma, is predominant in Europe and North America.

A China-focused statistical review published in Thoracic Cancer estimated there were 287,632 new cases of ESCC diagnosed and 208,473 deaths in 2010. Esophageal cancer incidence ranked fifth of all cancer types in China, with a rate of 21.88 per 100 000. For both incidence and mortality, men living in rural areas were most likely to be affected, peaking at the age 80 to 84.

In certain rural areas in Henan, Hebei and Shanxi, the incidence rate shoots to the highest in the world, greater than 100 cases per 100,000 according to a study in the Journal of Epidemiology. Other high incidence areas are found in the provinces of Sichuan, Anhui, Jiangsu, Hubei, Fujian, Guangdong and Xinjiang.

Studies show there is a complex interaction between genetic factors when combined with alcohol consumption, smoking and poor nutrition that can cause ESCC. It is believed diets containing a high quantity of pickled vegetables (with nitrosamine content) are also to blame.

 

ERBB3 (HER3)

ErbB3 belongs to a family of four proteins that also includes EGFR (HER1) and HER2 tyrosine kinase receptors. ErbB3 stimulates cancer growth and its overexpression generally correlates with poor prognosis.

AV-203 selectively targets the receptor ErbB3 and demonstrated preclinical activity in a number of different tumor models including breast, head and neck, lung, ovarian and pancreatic cancers. The preclinical data further showed the potential for heregulin, the only known ligand for ErbB3, to serve as a biomarker predictive of AV-203 anti-tumor activity.

Aveo completed a phase I study showing no dose-limiting toxicities at a maximum dose of 20 mg/kg.

Plans are already under way at Canbridge to make use of PDX mice models to continue testing CAN017 in tumors from Chinese squamous cell esophageal cancer patients. Company execs expect to be ready to submit an investigational new drug application next year, and take advantage of China’s new guidelines for innovative drugs.

“We are going to start a human trial in China first and hope to take advantage of the green channel approach. Because it is [CAN017 targets] a major malignant tumor, has a Chinese specificity, and we are going to manufacture the product in China, it will allow us to tap into the national guidance,” said Xue.

CHINA AS A LAUNCHPAD TO THE WEST

Meeting China’s significant unmet medical needs is the stated aim of many of China’s most promising biopharmas, in large part because there is such a gap between the drugs available in developed markets and in China – with only about 30 percent drugs available in China.

“The big multinational companies have set their center of gravity and development capabilities mostly in the West,” explains Xue. “Many products are successfully launched with Western populations as a priority. If they happen to be able to address a disease in the Asian population then it is perfect. However, if prevalence in Asia is much greater than in the West and you are still using this algorithm to the do the global launch then it will be quite a missed opportunity for those companies. That is where companies like Canbridge can come in.”

But with Canbridge’s first foray into a global rights deal, the company is joining a small band of companies that are hoping to pull the center of gravity slowly their way.

“Once the data generated in China begins to look very promising, we have strong confidence that we can replicate the data in the West,” said Xue. “We can utilize Asia as a base to develop innovative therapies for the global patient, instead of the other way around.”

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Escape velocity? Bioblast phase II could open door to remedy in rare OPMD

BIOWORLD TODAY 

MARCH 17, 2016

THE DAILY BIOPHARMACEUTICAL NEWS SOURCE

BIOTECH’S MOST RESPECTED NEWS SOURCE FOR MORE THAN 20 YEARS

VOLUME 27, NO. 52

TRIAL TO VERIFY THIS YEAR

Escape velocity? Bioblast phase II could open door to remedy in rare OPMD

By Randy Osborne, Staff Writer

Bioblast Pharma Ltd. CEO Colin Foster told BioWorld Today “it would not be unreasonable to think” that regulators could deem pivotal the upcoming phase IIb trial with trehalose in oculopharyngeal muscular dystrophy (OPMD), but the company doesn’t count on that. “We’re looking forward, recognizing that we [probably] have to do a phase III study,” he  said.

 

Shares of New Haven, Conn.-based Bioblast (NASDAQ:ORPN) closed Wednesday at $4.20, up $1.56, or 59 percent, having traded as high as $7.95 after the firm disclosed phase II data from its HOPEMD trial testing the disaccharide trehalose, known for its ability to stabilize intracellular and intranuclear proteins. The experiment aimed to learn about safety and tolerability – trehalose passed in those departments – but also included secondary endpoints to see if the drug improved, or at least prevented worsening, of markers in rare disease OPMD. It rang bells there, too.

An inherited myopathy, OPMD is characterized by dysphagia (difficulty in swallowing), eyelid drooping, or ptosis, as well as loss of muscle strength and weakness. Specifically, it’s a polyalanine-mutation disease with onset around middle age.

“On some highly consequential proteins associated with normal muscle homeostasis, you’ve got a particular protein that ends up having too many alanines stuck on the back,” Foster said, which “creates just a mess. It’s almost like a dust bunny under your bed.” Proteins can’t fold properly, leading to symptoms, and patients become emaciated because they are unable to eat normally.

“They’ve lost the reflex to get rid of food that goes down their windpipe,” he said, and suffer repeated bouts of aspiration pneumonia, which can lead to  death.

“It’s an insidious, one-way disease,” Foster said. “For the muscle weakness, there’s nothing. You’re stuck with it.” Surgery may help swallowing. “Literally, [surgeons] seek to expand the size of your throat, allowing food to plop down more easily into the esophagus.” But the benefits only last about 15 months on average.

“That gives you an idea how accelerated this disease is, even on the dysphagia side,” he said, noting “a tremendous amount of apathy among the undiagnosed. They know what the future holds. I got a call from a guy on Long Island the other day” whose father is afflicted. The son “doesn’t want to be like that, but he’s already got symptoms that are going to set him on the same course as his dad. People are looking for solutions, for sure.”

In the open-label trial, data from which were reported Wednesday at Myology 2016, a muscle conference in Lyon, France, 25 patients with OPMD clinical dysphagia and muscle weakness were enrolled at two centers in Canada and Israel.    All have completed 24 weeks of weekly treatment, and Bioblast reported improvements vs. baseline in multiple secondary efficacy endpoints related to dysphagia along with muscle strength and function.

 

POTENTIAL TAKEOUT: ANALYST

The dysphagia endpoints were the timed cold water  drinking

 

test (80 mL) for all sites, the nectar (80 mL) and honey (80 mL) timed drinking tests at the Canadian site, and the Penetration Aspiration Score as measured by video fluoroscopy   (VFS-
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PAS), a radiographic technique to determine the severity of swallowing difficulties and risk of aspiration. One patient- reported, swallowing quality-of-life questionnaire (SWAL-QOL) specifically developed for patients suffering from swallowing problems was employed to assess the degree to which patients felt that their swallowing capability improved with  treatment.

A mean reduction in time to complete the cold water drinking test of 31.8 percent turned up vs. baseline (n=23). In the nectar and honey timed drinking tests, time to complete was reduced by 43.8 percent and 46.6 percent, respectively (n=11). Out of the 11 patients in Canada whose scores were evaluated in the per- protocol analysis of the VFS-PAS, six patients improved (54.5 percent), two showed stabilization (18.2 percent), and three deteriorated (27.2 percent). Due to deviations from protocol and deficient radiological procedures, the VFS-PAS tests from the Israel cohort were excluded from the final analysis. With   respect to the SWAL-QOL questionnaire, there was a 12.7 percent (n=24) improvement vs. baseline with the mean total symptom severity score increasing from 43.2 to  48.7.

 

As measured quantitatively by a digital hand-held dynamometer,  a mean increase was recorded in lower body-muscle strength compared to baseline in knee extension of 15 percent (n=22)

and foot dorsiflexion of 22.4 percent (n=22). Hip flexion did not materially change (1.3 percent deterioration, n=21). For the upper-extremity strength tests, arm (bicep) flexion increased on average 17.9 percent (n=22), and shoulder abduction by 11.4 percent (n=22).

 

The 30-second arm-lift test showed a 16 percent increase in the number of completed tasks (n=20) at 24 weeks of  treatment vs. baseline, while the 30-second, sit-to-stand test showed a

16.6 percent increase (n=21). In a standard, four-stair climbing test, the results didn’t change much (1.5 percent deterioration, n=21).

 

“When we talk to our neurologists, they say [hip flexion] one of these things that’s going to be very difficult to change,” Foster said. The stair-climbing scores may not provide a good picture “because, in that particular test, you’re not really testing single- muscle strength performance” – a group of muscles is involved. Bioblast’s phase IIb trial is expected to start around mid-year, he said.

The long shot of approval based on the pair of phase II studies could pan out “if we design the study appropriately and execute well and have some very good data at the end of it, confirming the positive effect [or] perhaps even accentuating the positive effect, because people won’t be compared to baseline but to people who are on placebo,” he said, estimating the  phase

IIb will enroll “between 40 and 80 patients. That’s sort of the realm. It’s a very manageable trial.”

Starting coverage in January and calling Bioblast “underrated,”

 

Rodman & Renshow analyst Raghuram Selvaraju estimated peak sales of trehalose in OPMD alone at $600 million. The drug is also in phase II development for spinocerebellar ataxia type 3. Merger-and-acquisition transactions “in the orphan sector involving firms with narrower pipelines than Bioblast have taken place at much higher valuations,” he wrote in a research report, pointing to the buyout of Lexington, Mass.- based Synageva Biopharma Corp., a single-product orphan drug company, by Alexion Pharmaceuticals Inc., of Cheshire, Conn., for $8.4 billion, which happened before Synageva won approval for its sole clinical asset, Kanuma (sebelipase alfa) for lysosomal acid lipase deficiency. (See BioWorld Today, May 7, 2015, and Dec. 9, 2015.)

OPMD is known to affect about 6,000 people in the U.S. “We fully expect, knowing how these things happen, there are probably two to three times that number who are undiagnosed, and frankly, some market research tells us the number could even be higher than that,” Foster said.

Efficacy and speed were two things that together made up “a little bit of a eureka moment,” in the currently reporting trial, he added, but Bioblast needs to “prove it the old-fashioned way” with the phase IIb, double-blind, placebo controlled study. “I’m feeling very good,” he said. “We’ve got something.” //