New wave of local companies tackles Lou Gehrig's disease

Patrizia Fanara and David Fineman of KineMed.

Hoping to find clues for unlocking potentially lucrative cures to diseases such as Alzheimer’s, drug development companies are starting with a more obscure but equally devastating condition: Lou Gehrig’s disease.

Five Bay Area companies and at least four local research institutions are working on programs in Lou Gehrig’s, or amyotrophic lateral sclerosis., which over three to five years gradually immobilizes patients’ bodies while their minds remain sharp.
What’s more, they’re finding success.
Each of the companies is pursuing different ALS bets, indicating how little is really known about the disease. But their goals are the same: In the short term, expand the options for the 30,000 ALS patients in the United states beyond the single drug that exists today. Longer term, they hope to use what they learn to target more prevalent diseases such as Alzheimer’s.

Neuraltus moving Lou Gehrig's disease drug into Phase III

Neuraltus Pharmaceuticals Inc. will take its experimental Lou Gehrig’s disease drug into Phase III next year.

Although the drug didn’t hit its pre-defined goals in a mid-stage clinical trial, the company said Tuesday, an analysis of the results indicated that the drug stopped the disease, also known as amyotrophic lateral sclerosis, from progressing.
The news is significant for patients with the rare and fatal muscle-crippling disease because there is only one drug approved for ALS and it extends patients’ lives only by weeks.
It also is a victory for the small, privately held Palo Alto company, which is developing the drug in a disease that is poorly understood and where much-larger Biogen Idec (NASDAQ: BIIB) also has an ALS program in Phase III. It also could set up the company to be sold to a larger biotech or pharmaceutical company or strike a collaboration deal.
Yet, perhaps most of all, the decision to move forward with the drug, called NP-001, demonstrates the power of patients to fight for a drug they believe will alter the course of a disease that can kill patients within three to five years of diagnosis. A core group of ALS patients and their caregivers fought to fill Neuraltus’ Phase II trial.

Andrew Gengos out, John Walker in as Neuraltus CEO

Andrew Gengos.

Andrew Gengos, who led Neuraltus Pharmaceuticals Inc. into a Phase II trial of its experimental treatment for Lou Gehrig's disease, has left the company. Veteran Bay Area biotech executive John Walker, Neuraltus' chairman, took over Aug. 15 as interim CEO, according to a company telephone message.

Kickstarter film project, ALS patients face life-and-death deadlines

Ben Harris, with son Rawdan and wife Rebecca.

Time is running out for Jason Harris and his brother, Ben. Only 14 days remain in Jason Harris' Kickstarter effort to fund a documentary film around Lou Gehrig's disease patients taking science and the search for potential treatments into their own hands. The story centers on Ben Harris, who wasinstrumental in efforts to fill a mid-stage trial of an experimental treatment from Palo Alto's Neuraltus Pharmaceuticals Inc. Ben also was involved in a patient-led "biohacking" movement to find the active ingredient of Neuraltus' drug, called NP-001, and to treat themselves. It is more than a story about Lou Gehrig's disease, also known as amyotrophic lateral sclerosis or ALS. It is a story about the growing movement by patients to take matters into their own hands as they and their caregivers, doctors and drug companies become increasingly frustrated with what they see as the sloth-like process of getting life-saving or disease-altering drugs approved. It also is a story that gets to the heart of a major drug-development question: When is a small extension of a patient's life -- even just a few months -- not a regulatory or business model issue but a personal or ethical issue?

Onyx Pharmaceuticals CEO Tony Coles headlines SF Business Times Biotech Forum — June 14

Onyx Pharmaceuticals CEO Tony Coles will headline the San Francisco Business Times' annual Biotech Forum.
The event begins with registration at 7:30 a.m. Thursday, June 14, at the Hilton San Francisco Union Square, 333 O'Farrell St.
Coles will be interviewed on stage by San Francisco Business Times biotech reporter Ron Leuty before joining a panel of Bay Area biotech industry experts. In addition to Coles, the panel will feature:
• Gideon Bollag, senior vice president for research, Plexxikon;
• Lissa Goldenstein, president and CEO, Auxogyn;
• Craig Muir, partner, Third Rock Ventures; and
• Glenn Oclassen, president and CEO, Transcept Pharmaceuticals.
Additionally, the event will feature presentations from three up-and-coming Bay Area life sciences companies -- Avalanche Biotechnologies, Organ-i and Neuraltus Pharmaceuticals -- about their businesses and the big scientific problems they are trying to solve.
Christie Smith, principal and West region life sciences leader at Deloitte Consulting LLP, will give opening remarks.

To order tickets for the event, call or email Jacquie Bischoff at 415-288-4972 or jbischoff@bizjournals.com. Tickets cost $70 per person or $750 for a table of 10.

ALS drug developer Neuraltus raises $9.6M toward $11M goal

Neuraltus Pharmaceuticals Inc., the Palo Alto developer of an experimental treatment for Lou Gehrig’s disease, has raised $9.6 million toward an $11.1 million offering, the company said Tuesday in a Securities and Exchange Commission filing.
Seven investors have ponied up for the round in the form of equity, option/warrants and preferred stock with warrants.

Patent fight puts ALS drug at risk

A patent fight risks slowing development of an experimental treatment for Lou Gehrig’s disease from Neuraltus Pharmaceuticals Inc.
Swiss drug company Nuvo Research AG claims in a lawsuit filed last month in U.S. District Court for Northern California that a former consultant and University of California, San Francisco , professor, Dr. Michael McGrath, tapped research tied to its drug as the backbone of a treatment now in a mid-stage clinical trial led by Palo Alto’s Neuraltus.
The suit is significant because Neuraltus’ drug is one of a handful of potential treatments that have caught the attention of patients with the neurodegenerative disease, also known as amyotrophic lateral sclerosis, or ALS.
The case also underscores how biotech companies need solid intellectual property protection.

Changing the course of disease, one tweet or 'like' at a time

"New media” or “social media” is helping recruit to clinical trials and maybe — potentially — even reshaping the way trials are done. But take a look at the companies making this happen: Apple Inc. of Cupertino, Twitter Inc. of San Francisco, WordPress developer Automattic Inc. of San Francisco, YouTube of San Bruno.
These Bay Area technologies are connecting people — whether they are fomenting revolution in the Middle East or trying to revolutionize treatment of a deadly disease — are largely due to Bay Area innovators and entrepreneurs.

Patients tap the Net to enlist in fight against ALS

Ben Harris is taking on Lou Gehrig’s disease, one Internet message board at a time.
Harris and a band of fellow patients with the fatal muscle-wasting disease are actively recruiting others into a mid-stage trial of a new drug for amyotrophic lateral sclerosis, or ALS, by Neuraltus Pharmaceuticals Inc.
The movement shows the growing role that the Internet, particularly social media, is playing in filling key clinical trials, especially those focused on rare diseases like ALS. That has implications for cash-hungry companies as well: The faster Neuraltus can enroll patients in its Phase II study, for example, the faster it can discover whether the drug actually does what leaders of the Palo Alto company believe it will do — slow or stop ALS from gradually tightening its suffocating grip.

Neuraltus ALS drug wins fast track, orphan status

An experimental treatment for Lou Gehrig’s disease won fast track and orphan drug designations from the Food and Drug Administration, potentially speeding the drug’s development by Neuraltus Pharmaceuticals Inc.
NP-001, the Palo Alto company’s treatment for amyotrophic lateral sclerosis, or ALS, received those designations last week, Neuraltus CEO Andrew Gengos told the San Francisco Business Times on Tuesday.

Neuraltus ALS drug results 'encouraging'

An experimental drug from Neuraltus Pharmaceuticals Inc. was effective against a biomarker of amyotrophic lateral sclerosis, or Lou Gehrig’s disease, in an early-stage study.
Although the clinical study by the privately held, Palo Alto-based company was only Phase I, involved just 32 people and assessed the drug’s effect on the biomarker only 24 hours after dosing, the result is significant because Neuraltus’ drug is believed to be the first to provide a statistically significantly improvement in blood levels of the biomarker.
The finding also could garner more attention for Neuraltus — particularly from investors — as it tries to raise money to undertake a more extensive Phase II study.

Neuraltus pinning hopes on Lou Gehrig's drug

A Thai drug’s boost could help an early-stage Palo Alto drug developer change the lives of patients with deadly ALS.

Neuraltus Pharmaceuticals Inc. hopes results from a Phase I trial announced in mid-November will kick its drug into a mid-stage trial early next year of 90 patients or more with amyotrophic lateral sclerosis, better known as Lou Gehrig’s disease.