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RNAi Executives Provide Some New Details on Financing, Drug Pipelines at BIO-CEO Meeting


This year’s BIO-CEO conference included a well-attended RNAi panel discussion, featuring the heads of several RNAi therapeutics firms, entitled: “What’s All the Fuss about RNAi?” Although it remains uncertain whether the participants’ presentations — which, for the most part, amounted to corporate overviews well-suited to an audience unfamiliar with RNAi — helped answer this question, they did provide a few new details about each company’s ongoing activities.

Those participating in the RNAi focus session included Acuity Pharmaceuticals CEO Dale Pfost; CytRx CEO Steven Kriegsman; Intradigm CEO John Spears; Nucleonics CEO Robert Towarnicki; and Sirna Therapeutics CEO Howard Robin. (Alnylam Pharmaceuticals CEO John Maraganore was scheduled to participate, but a company spokeswoman told RNAi News that a last-minute situation prevented his attendance.)

Following is a roundup of new information provided by the executives at the conference.


Pfost told RNAi News on the sidelines of the BIO-CEO event that the company has just gotten a commitment of $7 million from an investor under a Series B financing round. He said that with the commitment, the company expects to raise a total of $15 million in the financing, coming in at the high end of a range he provided RNAi News last week (see RNAi News, 2/20/2004).

Pfost said that the $15 million would be sufficient to take Acuity through phase II testing of its preclinical age-related macular degeneration drug Cand5, which is slated to enter the clinic at the end of 2004 or in early 2005.

Pfost noted that Acuity expects to develop Cand5 alone through phase II, at which point it will consider various opportunities, including partnerships and outlicensing deals.


Kriegsman noted during his presentation that CytRx’s amyotrophic lateral sclerosis program is proceeding apace, with a pre-IND meeting with the US Food and Drug Administration slated for the third quarter. The ALS program is targeting the superoxide dismutase (SOD-1) gene, which has been associated with autosomal dominant familial ALS.

“We plan to file our IND before the end of the year,” Kriegsman said, adding that “we have every indication that we’ll be in humans early next year. We’ve talked to the regulators and … gotten an indication that we’ll only have to treat 10 to 12 patients in a phase I trial.”

If this turns out to be the case, and CytRx can enlist the support of ALS advocacy groups, he said that “we could have a drug on the market in 2006 and [then] do a phase IV trial.”

Kriegsman said that CytRx plans to develop and commercialize its ALS drug alone, but is planning to partner on its obesity and type II diabetes projects later on. While the company has been approached by potential partners, he said, CytRx doesn’t plan to seriously entertain collaboration discussions for another six to nine months.

As for the company’s other interests, Kriegsman told RNAi News that CytRx is considering starting RNAi drug development programs in Huntington’s disease and Parkinson’s disease.


Spears said that Intradigm has selected the VEGF pathway as the first target for its RNAi therapeutics development program. This target, he said, allows the company to pursue ocular, neovascular, and oncologic indications.

The company, he said, plans to file an IND in an undisclosed indication within the next 12 months. Spears told RNAi News that the clinical candidate would target VEGF, but the company is waiting for some preclinical cancer data to come in before deciding on a particular indication in which to file the IND. Those data are expected to be ready in the “next couple months,” he said.

He also noted during his presentation that Intradigm is experimenting with a systemically delivered RNAi drug for AMD.


Towarnicki provided an update on Nucleonics’ development of hepatitis B and C treatments using the company’s gene silencing technology, called expressed-interfering RNA.

Recently, Catherine Pachuk, Nucleonics co-founder and vice president of biological research, told RNAi News that the company’s technology allows for the expression of dsRNA against multiple genomic sequences (see RNAi News, 2/20/2004).

Towarnicki said during his presentation at the RNAi session that the company is in the final stages of selecting the 18 siRNAs “that we’re going to incorporate into our final construct. That construct will go into a transgenic mouse model … [using] a clinically safe vector system licensed from Wyeth.”

He added that the system “has been utilized in seven DNA vaccine trials, it has been fully scaled for GMP manufacture, [and] we have formulated an IV bolus for high-efficiency transfection of the hepatocytes.” Towarnicki noted that Nucleonics has already had discussions with the FDA’s vaccines group about its hepatitis B and C candidates.

Nucleonics has also been talking with several large pharmaceutical firms about possible proof-of-concept-style collaborations, Towarnicki told RNAi News last week. He characterized the possible arrangements as ones in which Nucleonics would use its technology to knock down a validated target provided by its partner in order to demonstrate the ability of the eiRNA.

At the conference, Towarnicki noted that Nucleonics already has “one small collaboration with a large pharma. We’re not at liberty to disclose [the partnership’s target] per their requirements, but we’re working in the area of inflammatory disease.”


Robin said at the BIO-CEO meeting that Sirna is planning to select a hepatitis C siRNA-based drug candidate this year, and probably will file an IND for it in 2005.

He also noted that Sirna is doing work with the Cleveland Clinic in the area of oncology, the company’s third disease area of interest after hepatitis and AMD. This effort is focusing on breast cancer, he said, noting that this indication is not necessarily the one Sirna plans to pursue, but that the company needed a modality for its proof-of-principle experiments.

Getting in on the Game

In addition to the corporate news coming out of the BIO-CEO focus session, Kenneth Kosik, professor of neurology and neuroscience at Harvard Medical School, provided an overview of RNAi and of his efforts to develop a treatment for Alzheimer’s disease using the technology.

Kosik is using RNAi to knock down beta-secretase (BACE), an enzyme associated with the build up beta-amyloid plaques in the brain that is the hallmark of Alzheimer’s disease. While his work is currently an entirely academic effort, he told RNAi News that he now is looking into striking an industry partnership.

Based on the reaction to his presentation, it appears that Kosik won’t have much trouble finding potential collaborators. After the focus session concluded, Robin and Sirna COO Nassim Usman both requested Kosik get in contact with their company, as did Intradigm’s CSO Martin Woodle and Kriegsman.

Of these suitors, Sirna seems like it might be a natural fit for Kosik. The researcher said he is collaborating with University of Iowa researcher Beverly Davidson on his neurodegenerative disease research. Bharat Chowrira, Sirna’s vice president of legal affairs, told RNAi News after the Kosik presentation that Sirna recently hired Davidson as a full-time consultant on the company’s research into Huntington’s disease, as well as BACE and Alzheimer’s disease.


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