HD66: Search for a cure or a killer?

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HD66: Search for a cure or a killer? Page 12

by Babs Carryer


  “Imagine the bad press this could generate,” Matt interrupts.

  “He’s right; we’d be overwhelmed by requests,” Gigi says shaking her head. “How would we pay for that?”

  “We couldn’t get our investors or the board to approve this, anyway,” Matt concludes. “No fucking way.”

  “I can’t stand this conversation,” Errol says and slams the door behind him.

  “Brie, I’m sorry,” Jim says quietly. “You don’t know how sorry I really am.” He looks at me long and hard.

  I blink back my tears. They have no idea. They think I am just a bleeding heart trying to save people that I don’t know. I’m trying to save someone that I do know. Maybe even me. And none of you are helping!

  “Errol’s not really angry,” Gigi tells me with a downward smile. How condescending.

  “I know. He’s just sad.” Me too. I limp off to get a glass of water from the fridge. I know that technically Quixotic could apply for an individual case for my father, but his disease had not progressed to the point where it was immediately life threatening. That means that the FDA would likely say no. From what I just learned, the request would open up a whole can of worms for our company. We’d have to open up to every request. Like Matt said, we just can’t do that. I get the rationalization – the painful reality of a startup which has the capability to help but not the capacity. Knowing that didn’t stop me from crying all night. Arwen stays glued to my side, purring my tears away.

  Lying next to me, Neal knows that something is bothering me. He never presses if I don’t want to talk about something. “I love you,” he says and holds me close.

  “Just a bad day,” I cry into his chest.

  I am glad that I hadn’t told my parents. How ironic. I cannot help my own father even though we probably have the only thing on the planet that can help him.

  Chapter 22

  June 5, that same year

  I am ecstatic. At last. The deal is done. We signed three weeks ago. We have the money. The clinical trial will start soon. NOW IS THE TIME. I am going to tell them. All of them.

  I enter the room for our weekly on-the-same-page meeting. “The Phase III trial for HD66 will include almost 1,000 subjects and will last 12 months,” Matt announces. “NGX is paying for the whole thing. That’s fucking beautiful.”

  “What will it cost?” I ask.

  “It’s probably about $100 million,” Gigi estimates. “We are getting paid to have them keep on paying, Brie.”

  “My life as a startup,” I chuckle back to her.

  I had taken a week off to spend time with my folks. I wanted to tell my mother first. To let her know about Quixotic, the trial, and the risks. “It could be good for Dad if he actually gets the drug,” I explained. “It might be bad if he gets the alternative, the control, which is a placebo. There is no way of knowing which he will get.” I carefully warned her, “Our drug is unproven, it might never make it to market, and there are the usual risks of side effects.”

  “Brie, you sound like a television commercial,” my mother laughed. “Those commercials where the bottom of the screen has warnings…”

  “Like ‘Perpatrol may cause internal bleeding, heart attack, aches, diarrhea,’ and so on,” I joined in the fun. I really did end with the ubiquitous “Ask your doctor about it.” My father wasn’t going to be any worse off than he was right now, I reasoned.

  My mother was beyond happy. “Oh, Brie.” She wept when she dropped me off at the airport to return to Pittsburgh. She promised to talk it over with his doctor. If he agreed, then we would tell my father together.

  …….

  It was not an easy process enrolling him, however. In fact it was impossible. First I had to tell Quixotic about my dad having HD.

  “I thought something like this was going on with you. I’m so sorry.” Jim looked at me and then immediately busied himself with paying the bill for our lunch. I thought I saw a tear escape down his chin. “We’ll make it happen.” That’s all he said. He smiled at me. I couldn’t look at him. I couldn’t even manage a thank you. He had known!

  When we got back to the office, I walked down the hall to see Matt, who was shocked when I told him. “Wow, you are in luck, my dear,” he exclaimed! “The world is so small; what are the chances that you would be working for a company trying to solve a disease that you didn’t know you might have…” he broke off. “Oh, fuck, I’m an idiot. Sorry; I didn’t mean to imply…” He was clearly embarrassed, an emotion that I had not seen in him before.

  Errol was harsher. “Good that we know,” he said accusingly. “I didn’t think that we kept secrets at Quixotic. But always good to know in the end.” He wandered off down the hall.

  I was pissed. How could Errol be so insensitive? Of all people!

  Gigi was sympathetic. “Oh my God, Brie. I am sooo sorry.” She hugged me lightly. “You should think about getting tested.” The clinical response. “I assume that you haven’t done that or I would be able to see by your face…” She caught herself. “Never mind, Brie. We’re all here for you. And of course for your father.”

  “Can we just focus on my father and the trial?” I asked. “I would like to see that my dad gets into the trial.”

  “Oh dear, that may be more difficult that you anticipate,” she said quietly.

  “Oh, don’t worry. I will make sure that he fully understands the risks that he might not get our drug. I’ve already told them and they are hot to go.”

  “That’s not what I meant. I have to talk to the others. It’s not as simple as you think.” With that she ushered me out of her office. “I’ll get back to you. As soon as we know something.”

  What does she mean “not as simple?” Who cares? This is finally my chance. Nothing can stop me now. I’ve waited and waited and now I want my father in the trial. Don’t they understand what’s at stake here?

  I saw the trio later that afternoon, Matt, Jim, and Gigi, huddling over the whiteboard in the small conference room. Errol stormed in and back out in five minutes. I was not sure if they were discussing me, my father, or something else entirely.

  Errol pointed at me as he stomped down the hall, “You…” But he disappeared into his lab.

  What I learned the next day was that, because he is my father, he created a conflict of interest for Quixotic. “If he provided you with inside information during the trial that would, or could, crater objectivity,” Matt explained.

  “No worries, Matt. I resign, effective immediately.” He looked shocked. Indeed, it would break my heart to leave Quixotic, particularly at this exciting time, but I couldn’t possibly not do that for my father.

  “Matt, if he has a chance of getting access to our drug before it comes on the market, it’s a life-saving moment.” Matt looked like he would burst. “To be able to do that for my father...” The chance to save a life? Matt looked like he was going to explode. “He would do the same for me. It’s not really a choice.”

  “Fuck, Brie. Does Neal know?”

  I cringed as I admitted that I had kept all of this from Neal. “No. I couldn’t… I couldn’t tell him. Not yet. Not until… I have to get tested. Then I’ll tell him.”

  “You’ll know then, about you.” He looked at me, a cold blue stare.

  “Yes, right. I’ll know then. Definitively. Matt, I want to tell Jim, Gigi, and Errol myself about resigning. I’ll get you an official resignation letter after I tell them, OK?”

  “Suit yourself,” he shrugged.

  He was certainly not making much of an effort. I decided to tell Jim first. I invited him for a second lunch that month.

  “Resign? Has it come to this? I’m so sorry,” he lamented. “But I understand. We are fully supportive of you, Brie.” I quickly ordered dessert so that I wouldn’t start crying. “I’ll see what we can do,” he added. “It may not be necessary; let me do some checking. I’ve been looking into this myself.” I looked at him wondering if he just said what I thought he said. But he was busy with th
e bill and his wallet.

  Gigi was adamant, “I do not accept this, Brie. And remember, you work for me!” She pointed to the door of her office. I slunk out.

  Later that week Jim and Matt met with me to tell me that my leaving Quixotic wouldn’t fully solve the problem. “You see,” Jim explained gently, “you have vested options in Quixotic, which means that there is still the potential for conflict since you are technically a shareholder.”

  “But I haven’t exercised my options, and I won’t. The company can have them back.”

  “We thought you would say that. I’m checking with Stan,” Matt replied. “It’s complicated,” he added. “But not necessarily unsolvable. We’d like you to stay on, obviously. You’re an invaluable help to us all. And God knows, we need all the help we can get.”

  “For the moment,” Jim added, “we’d like for you to not make any final decision about leaving.”

  “Not until we have some definitive answers,” Matt added. “Let’s make sure we cover all options. So to speak.” He smiled grimly.

  “OK,” I acquiesced. “You know that I don’t want to leave.”

  “We know,” Jim concluded quietly. “We so very much know.”

  I was sulking in my office when Errol gave me a call. “Brie, I want you to know something.”

  “Don’t, Errol,” I responded. “It’s not necessary. Everybody is so darn sorry. I get it, Errol, I really do. There’s nothing that we can do.”

  “Never say that to me again,” he said and hung up. Darn him. Everyone is against me. All I want to do is to help my father. Why is it so difficult?

  Gigi came to see me that afternoon. “We are not accepting your resignation,” she said curtly. “This is not a viable option.” She too smiled at the pun. “These conflicts happen, and we will figure it out. Conflicts are only bad if you don’t disclose them. Don’t make any plans that can’t be changed,” she stated as she swept out the door. The air had a wake like Errol’s Scoot.

  I will not give up. There has to be a way.

  …….

  Six months later, December 5

  “The honeymoon is over! NGX realizes that they didn’t get everything that they wanted,” Matt announces at our weekly meeting, slapping the table forcefully.

  “They realize how much they left on the table,” Stan informed us as we look at the table.

  “They will so fucking get over it,” Matt stated confidently.

  Stan, however, looked worried, “They figured it out. Hate to say it…”

  “Then don’t,” Matt snapped.

  Jim was concerned too. “Big companies can do big things if they are crossed.”

  I was counting on NGX and the clinical trial for the drug to get approval. For my father. The last resort. Now even that might be taken away from me. By them.

  The next few months I hardly saw Stan. I noticed a lot of big books on his desk. Like he was back in law school, he appeared to be reading a lot.

  As Jim had predicted we had kept all assets outside of HD66 from NGX by forming a new company, Quixotic Labs. We put $10 million from our upfront money into the new company so it was a well-funded startup with valuable intellectual property. We had other drugs in the pipeline. Gigi kept talking about Parkinson’s and glancing at Errol. Maybe he was getting closer? The Russians were pressuring us about Parkinson’s too. I sometimes got the impression that no one cared about HD anymore.

  I was relying on HD66 making it through the trial that NGX was sponsoring. I figured that NGX would want complete control over everything associated with Quixotic. I had thought the deal structure was very clever once I understood it. “To stop us from continuing our other programs with similar drugs based on the core components of HD66, NGX would have to buy Quixotic in her entirety,” I told Neal.

  “That’s clearly a dangerous strategy,” he countered.

  “The question is, what will NGX do now that they know?”

  “What does Jim say?” Neal asked. Neal really liked Jim. He never said that he didn’t like the others but I sensed hesitation in him when I talked about Gigi and Matt.

  “He agrees that NGX will have to buy the whole company. ‘It’s an insurance policy,’ he told me.”

  “You mean that NGX would have to buy the whole company or else you can do deals with other companies for drugs like the one you have for Parkinson’s?” Neal is smart. As an entrepreneur he got it right away. “So, you could just keep on selling off assets, one drug at a time?” He sounded smug.

  “Yes, I think that’s what they had in mind when we crafted the deal.”

  “Wow, a lot of money could be had. NGX would have to pay more if they wanted all of the assets. More than the $300 million specified in the existing contract. That’s huge, Brie.”

  “Yep, a lot of money.”

  “No wonder I love you” he told me kissing me and squeezing me tightly. “Come on, let’s go to bed…”

  In the middle of night, while petting Arwen, I recalled how silent Errol was about NGX. After his big explosions early on, and his blatant satisfaction at the favorable terms of the deal, he seemed to move on, ignoring anything to do with NGX. Unusual for Errol to be so reserved.

  …….

  The next week, December 10

  I get an email from Shala asking me to stop by the lab. She says that she wants my opinion on something. Something that they have discovered in the lab.

  “Hey Errol, I announce as he sweeps by me in the hall looking preoccupied.

  “Hey, beautiful? What’s up?”

  “Oh, I just want to stop by the university lab and see what you are up to.”

  He peers at me over his glasses and frowns. “Of course, stop by anytime. You’ll be very interested in something that we’ve done. I haven’t told anyone else. It’s different than other discoveries. Something very unusual. At first I thought it was a disaster, but it’s fascinating. You’ll see. I’m sure the gang would love to see you. Shala in particular looooves you.” With a swoop of his hand, like he is sweeping long hair back from his face, he imitates Shala, “That lovely Brie friend. She is now my good friend too, Dr. Errol.”

  I laugh. “Is it something that we could commercialize?” I ask.

  “Hmmm, maybe,” Errol replies thoughtfully. “You can bet that someone would pay a lot for this one,” he finished. “Anyway, glad you are coming,” he adds with a twinkle in his eye. “I have a little something extra, for you.”

  I’m sure that I look surprised. “For me?”

  “Something very special and only for you,” he says with a chuckle. “You will understand why when I tell you. But… not yet!” and he swishes out my door, shirttails flapping.

  He’s been teasing me for weeks about this. What does he have that is so special? Is it something for my father? I’ve almost given up.

  …….

  2 months later, March 1

  The trial is over. The relationship with NGX has gone from bad to worse. In couple terms they are not speaking to us. This morning the bubble burst. Without warning, NGX stopped the trial. Stopped. Dead in the water. Now we’ll never get HD66 to market.

  We always knew that there were three potential outcomes of the trial. The one that we all expected, that our investors wanted to happen, is that the clinical endpoint was met, meaning that the drug worked. In this scenario, because HD is an orphan disease, we could go immediately to the FDA for fast-track approval. I had been counting on that. A second scenario would be an outright acquisition of Quixotic by NGX. A third option, the one Matt told us was the most probable, is that the data from the trial would be good, but not good enough to go straight to the FDA. In that case, NGX would have to extend the trial to include a Phase IIIB. That would add another 12-24 months to our plans. That would be OK with us because “moving forward is still moving forward,” Matt told us. The fourth scenario is one we never considered or discussed, at least not out loud. That would be a failure to meet our clinical end point. In other words, if patients show
ed no improvement, or there were serious adverse side effects. Then we’d have a problem – a big one. Our drug would not make it to market – ever. And my dad would die a horrible, inevitable death.

  The pathological optimism that you have to have in a startup precludes the ability to consider the fourth option. Plus, we genuinely didn’t think that could happen. We were so careful in Phase II A and B. We studied the data from those trials to make sure that there was nothing hidden, nothing that we didn’t know about. We were confident going into Phase III. Who wouldn’t be? After all, NGX clearly believed that we had something, $300 million worth of something.

  So, their press release this morning came as a surprise. “Holy fucking shit,” I heard Matt scream from down the hall. It was the middle of the morning, 10:30 a.m. Gigi and Jim poked their heads out of their offices. So did at least a half a dozen other Quixoticers.

  I follow Gigi and Jim into Matt’s office. “NGX has cancelled the trial,” he informs us in a strange monotone. Like the quiet before the storm. He proceeds to read from his laptop:

  “NeuroGenex, Inc. has halted development of an experimental drug to treat the devastating neurodegenerative disorder known as Huntington’s Disease, saying Wednesday that it proved ineffective in a late-stage clinical trial.

  The decision deals a blow to an estimated 30,000 Americans who suffer from Huntington’s, a fatal degenerative condition for which there is no cure.

  ‘There were a lot of people wishing for a positive outcome, who were hanging onto this as their hope,’ said Martin Nextburg of Orono Maine, who was diagnosed with Huntington’s in 2008 and who had been featured on a national Public Broadcasting Station special about the disease.

  “It was also a setback for Cambridge-based NeuroGenex and its chief executive, Martin Stronghold, who had bet more than $300 million on the high-risk Huntington’s program earlier this year when he licensed HD66 — the drug — from Quixotic, an early-stage company based in Pittsburgh, PA. Shares of NeuroGenex, which trades under the symbol, NGX, fell nearly 5 in post-market trading Wednesday evening, losing $1.89.

 

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